What changed in this filing

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Item 7. Management’s Discussion and Analysis

2026-03-05 compared with 2025-03-13 · 746 added, 576 removed, 1304 unchanged (50% of the section changed)

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In addition, you should read the “Risk Factors” and “Information Regarding Forward-Looking Statements” sections of this Annual Report on Form 10-K for a discussion of important factors that could cause actual results to differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

For a full discussion and analysis of financial condition and results of operations for the year ended December 31, 2023, including a year-over-year comparison to the year ended December 31, 2022, please read the “Management's Discussion and Analysis of Financial Condition and Results of Operations” section of our Annual Report on Form 10-K for the year ended December 31, 2023, which we filed with the SEC on February 27, 2024.

For a full discussion and analysis of financial condition and results of operations for the year ended December 31, 2024, including a year-over-year comparison to the year ended December 31, 2023, please read the “Management's Discussion and Analysis of Financial Condition and Results of Operations” section of our Annual Report on Form 10-K for the year ended December 31, 2024, which we filed with the SEC on March 13, 2025.

We are a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.

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• ABBV-RGX-314:

We are developing ABBV-RGX-314 (surabgene lomparvovec) in collaboration with AbbVie as a potential one-time treatment for chronic retinal conditions that cause total or partial vision loss, including wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).

We are developing ABBV-RGX-314 (surabgene lomparvovec, sura-vec) in collaboration with AbbVie as a potential one-time treatment for chronic retinal conditions that cause total or partial vision loss, including wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).

ABBV-RGX-314 is currently being evaluated in multiple clinical trials, including two pivotal trials (ATMOSPHERE and ASCENT), one Phase II bridging study, one long-term follow-up study and a fellow eye sub-study in patients with wet AMD, all utilizing subretinal delivery.

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Within the Phase II study in DR, we are also evaluating ABBV-RGX-314 in diabetic macular edema (DME).

Additionally, we are planning a Phase IIb/III program in DR and expect to dose the first patient in a two-part Phase IIb/III study (NAAVIGATE) in the second quarter of 2026.

ABBV-RGX-314 uses the NAV ® AAV8 vector to deliver a gene encoding a therapeutic antibody fragment to inhibit vascular endothelial growth factor (VEGF).

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Subretinal Delivery

Enrollment continues to be on track in the ATMOSPHERE ® and ASCENT pivotal trials for the treatment of patients with wet AMD using subretinal delivery.

Enrollment in the ATMOSPHERE ® and ASCENT ® pivotal trials for the treatment of patients with wet AMD using subretinal delivery was completed in October 2025.

These trials are expected to support global regulatory submissions with the U.S.

Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Topline data from these trials are expected to be shared in 2026.

Topline data from these trials are expected to be shared in the fourth quarter of 2026 in partnership with AbbVie.

Suprachoroidal Delivery

The AAVIATE ® trial is a multi-center, open label, randomized, controlled, dose-escalation Phase II trial to evaluate the efficacy, safety and tolerability of suprachoroidal delivery of ABBV-RGX-314 for the treatment of wet AMD.

As of July 29, 2024, ABBV-RGX-314 at dose level 3 with short course prophylactic steroid eye drops continues to be well tolerated with no drug-related serious adverse events (SAEs) and no cases of intraocular inflammation, endophthalmitis, vasculitis, retinal artery occlusion, choroidal effusion or hypotony.

Mild episcleritis occurred in three patients, all resolved and completed treatment with topical steroids.

There were no cases of elevated intraocular pressure.

Based on this favorable safety profile, the Phase II AAVIATE trial is initiating enrollment in a new cohort to evaluate ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye).

Patients in this cohort will also receive short course prophylactic steroid eye drops.

Based on the favorable safety profile observed as of July 29, 2024, the Phase II AAVIATE trial enrolled a cohort to evaluate ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye).

Patients in this cohort received short course prophylactic steroid eye drops.

The ALTITUDE ® trial is a multi-center, open label, randomized, controlled, dose-escalation Phase II trial to evaluate the efficacy, safety and tolerability of ABBV-RGX-314 using suprachoroidal delivery for the treatment of DR.

Based on positive interim results from this trial, we, with partner AbbVie, conducted an End-of-Phase II meeting with the FDA in the fourth quarter of 2024.

In January 2025, we and AbbVie announced we will plan a Phase III program for ABBV-RGX-314 in DR.

The program is expected to support global regulatory submissions.

The ALTITUDE trial is now enrolling a new cohort of patients with center-involved DME.

In November 2023, we announced data showing ABBV-RGX-314 was well tolerated at dose levels 1 and 2 and positive signals of efficacy, including 20.8% of patients exhibiting > 2-step Diabetic Retinopathy Severity Scale (DRSS) improvement without additional DR treatment at one year.

In August 2025, we and AbbVie executed an amendment to our collaboration agreement and announced plans to initiate a pivotal program consisting of a Phase IIb/III trial (NAAVIGATE) as well as a second Phase III trial.

NAAVIGATE is a Phase IIb/III multicenter, randomized, masked, sham-controlled study to evaluate the safety and efficacy of sura-vec in subjects with non-proliferative DR (NPDR) without center-involved diabetic macular edema (CI-DME).

The primary endpoint is > 2-step improvement on the diabetic retinopathy severity scale (DRSS) at one year.

Following an interim analysis, REGENXBIO and AbbVie will initiate a Phase III expansion, which will include two Phase III trials, including a U.S.

trial and a parallel global trial, led by AbbVie.

We expect to dose the first patient in NAAVIGATE in the second quarter of 2026.

The ALTITUDE trial includes a new cohort of patients with center-involved DME evaluating ABBV-RGX-314 at dose level 4.

Enrollment completed in this cohort in June 2025.

DME is a vision-threatening complication of DR;

an estimated 34 million people globally have DME.

Patients will receive a one-time, in-office injection of ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops.

Patients received a one-time, in-office injection of ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops.

We are developing RGX-202 as an investigational AAV therapeutic for the treatment of Duchenne muscular dystrophy (Duchenne), using the NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal domain as well as a muscle-specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne.

Other differentiating elements of RGX-202 include the proactive immune suppression regimen and in-house, state-of-the-art manufacturing that has demonstrated leading purity levels in Duchenne (>80% full capsids).

AFFINITY DUCHENNE ® is a multicenter, open-label Phase I/II/III trial to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous dose of RGX-202 in patients with Duchenne aged one and older.

The initiation of the pivotal study as well as positive safety and efficacy data from the Phase I/II portion of the study, including the first functional data, were announced in November 2024.

In March 2025, we announced that the pivotal study was nearly 50% enrolled, and we expect to complete enrollment in the study in 2025, share top line data in the first half of 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026.

We expect to share additional Phase I/II biomarker data at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, including the first biomarker data from the cohort of patients aged 1-3.

The company expects to share additional efficacy and safety data, including additional functional data, in the first half of 2025.

The initiation of the pivotal study was designed to enroll approximately 30 patients in the U.S.

In October 2025, we announced that enrollment in the AFFINITY DUCHENNE pivotal trial had completed and that we continue to enroll participants in the planned confirmatory trial.

We expect to share topline data in the early second quarter of 2026 and request a pre-Biologics License Application (BLA) meeting with the FDA in mid-2026.

We began manufacturing the first batches of RGX-202 intended for commercial supply at our Manufacturing Innovation Center and completed the Process Performance Qualification (PPQ) campaign in the fourth quarter of 2025.

Additional regulatory interactions with the FDA and the EMA are planned for 1H 2026.

We are also recruiting patients in the AFFINITY BEYOND ® trial, an observational screening study.

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We are developing RGX-121 (clemidsogene lanparvovec) in collaboration with Nippon Shinyaku in the United States and certain countries in Asia as an investigational one-time AAV therapeutic for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, using the NAV AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase enzyme.

In the pivotal phase of the Phase I/II/III CAMPSIITE ® trial, RGX-121 achieved its primary endpoint, a reduction in cerebrospinal fluid Heparan sulfate levels of D2S6, a biomarker indicative of brain disease activity, with statistical significance.

In September 2024, we announced positive data from the pivotal dose level of RGX-121 demonstrating long-term systemic effect.

We plan to use levels of cerebrospinal fluid Heparan sulfate D2S6 as a surrogate endpoint reasonably likely to predict clinical benefit for accelerated approval.

A BLA for RGX-121 seeking accelerated approval was submitted to the FDA in March 2025, which we believe is likely to be eligible for priority review.

We expect potential approval of RGX-121 in the second half of 2025.

Potential approval of the BLA for RGX-121 could result in receipt of a Rare Pediatric Disease Priority Review Voucher in 2025, assuming the statutory criteria are met.

A BLA for RGX-121 seeking accelerated approval was submitted to the FDA in March 2025.

The FDA subsequently granted priority review of the BLA and successfully completed mid-cycle meeting, Pre-license inspection (PLI) and Bioresearch monitoring information (BIMO) inspections.

The PLI and BIMO inspections were completed with no observations.

In August 2025, we announced that the FDA review timeline had been extended following submission of 12-month clinical data for all patients in the pivotal study of RGX-121 (n=13) in response to an FDA information request.

The Prescription Drug User Fee Act (PDUFA) goal date was extended from November 9, 2025 to February 8, 2026.

The longer-term data submitted to the FDA were presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in September 2025.

These results showed that in the pivotal phase of the CAMPSIITE trial (n=13), participants through one year sustained an 82% median reduction of cerebrospinal fluid (CSF) levels of HS D2S6.

These longer-term data were consistent with previously reported topline pivotal results from the CAMPSIITE trial.

In January 2026, we announced that the FDA placed the RGX-121 program on clinical hold in relation to a serious adverse event in a patient treated in the Phase I/II trial of RGX-111.

The FDA cited the similarities in products, study populations, and shared risk between the clinical studies.

In February 2026, we announced that the FDA issued a Complete Response Letter (CRL) for the RGX-121 BLA.

The FDA stated in the CRL that it had agreed to the study protocol in principle and outlined several reasons for not approving the gene therapy, including uncertainty regarding the study eligibility criteria to adequately define a population with neuronopathic disease (vs.

attenuated disease), the comparability of the natural history external control to the study population, and the appropriateness of CSF HS D2S6 as a surrogate endpoint reasonably likely to predict clinical benefit.

The CRL lists several potential paths forward, including a new study, treating additional patients and conducting longer-term follow up, and using an untreated control arm.

Throughout active discussions during the BLA process, we believed we had addressed the points raised in the CRL through the submission of additional data and responses to numerous information requests.

The FDA did not agree the data set provided substantial evidence of effectiveness to support approval of RGX-121 for the treatment of MPS II.

We plan to request a Type A meeting with the FDA.

As of March 2026, we plan to work with the FDA to address the clinical hold and CRL, and discuss potential paths forward for the program, Potential approval of the BLA for RGX-121 could result in receipt of a Rare Pediatric Disease Priority Review Voucher (PRV), assuming the statutory criteria are met.

If approved, RGX-121 would be the first approved gene therapy and one-time treatment for MPS II.

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In November 2023, future development of RGX-111 was halted as a result of a strategic pipeline prioritization and corporate restructuring.

Prior to that announcement, RGX-111 was demonstrated to be well tolerated and indicated encouraging biomarker and neurodevelopmental results in a Phase I/II study.

Efforts to continue development of RGX-111 are set to be reinitiated following our announcement in January 2025 of a strategic partnership with Nippon Shinyaku.

Strategic Pipeline Prioritization and Restructuring

In November 2023, we implemented a strategic pipeline prioritization and corporate restructuring designed to prioritize the development of ABBV-RGX-314, RGX-202 and RGX-121, and to seek strategic alternatives for our other clinical stage product candidates:

(i) RGX-111 for the treatment of MPS I, (ii) RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, and (iii) RGX-381 for the treatment of the ocular manifestations of CLN2 disease.

The restructuring plan included a reduction in workforce and other planned operating expenses, primarily in rare neurodegenerative disease development, early research and other general and administrative areas.

We implemented a reduction in workforce of approximately 15%, which was substantially completed in the fourth quarter of 2023.

For additional information regarding the corporate restructuring, please refer to Note 14, “Restructuring” to the accompanying audited consolidated financial statements.

Collaboration and License Agreement with AbbVie

Prior to that announcement, RGX-111 demonstrated to be well tolerated and indicated encouraging biomarker and neurodevelopmental results in a Phase I/II study.

Efforts to continue development of RGX-111 as part of the strategic partnership with Nippon Shinyaku are ongoing.

In January 2026, we announced that the FDA placed the RGX-111 program on clinical hold following preliminary analysis of a single case of neoplasm (intraventricular CNS tumor) in a participant treated in the Phase I/II study.

The case was identified during a routine brain MRI of an asymptomatic five-year-old participant who received intracisternal RGX-111 four years prior.

Preliminary genetic analysis of the resected tumor detected an AAV vector genome integration event associated with overexpression of a proto-oncogene (PLAG1), which is known to be susceptible to chromosomal rearrangements.

Final analysis of the resected tumor was conducted by an independent third-party lab, and, as previously reported, detected an AAV vector genome integration event associated with overexpression of a PLAG1.

Clonal integration of AAV vector elements into the PLAG1 gene was detected in the tumor tissue.

Analyses supported classification as a PLAG1‑family neuroepithelial tumor and are consistent with the hypothesis that AAV vector integration at the PLAG1 site contributed to tumor formation.

Of note, this participant had a background of factors that could have contributed to risk of oncogenic transformation.

This child underwent unsuccessful stem cell transplant at 4 months of age, with loss of donor chimerism, and he received chemotherapeutics that may have contributed to DNA damage.

The report concludes, based on formal neuropsychologic testing and developmental pediatrician assessment, that the patient’s neurocognitive development is above average, which indicates mitigation of MPS I disease, and the patient continues to do well.

We anticipate the analysis will be published in a peer-reviewed journal this year.

AbbVie Collaboration for ABBV-RGX-314

In September 2021, we entered into a collaboration and license agreement with AbbVie Global Enterprises Ltd.

(AbbVie), a subsidiary of AbbVie Inc., to jointly develop and commercialize ABBV-RGX-314 (the AbbVie Collaboration Agreement).

(AbbVie), a subsidiary of AbbVie Inc., to jointly develop and commercialize ABBV-RGX-314 (as amended, the AbbVie Collaboration Agreement).

Pursuant to the AbbVie Collaboration Agreement, both we and AbbVie are active participants in the development of ABBV-RGX-314 and development expenses are shared between the parties in accordance with the agreement.

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For additional information regarding the AbbVie Collaboration Agreement, please refer to Note 10, “License and Collaboration Agreements—AbbVie Collaboration and License Agreement” to the accompanying audited consolidated financial statements.

Collaboration and License Agreement with Nippon Shinyaku

In August 2025, we and AbbVie entered into an amendment to the AbbVie Collaboration Agreement which modified the development plan and milestone payment structure for the ABBV-RGX-314 DR program.

Under the amendment, we will conduct the first registration enabling trial for DR suprachoroidal (SCS) treatment as a combined Phase IIb/III trial (NAAVIGATE) which will be performed in two parts (Part 1 and Part 2), and AbbVie will conduct the second registration enabling trial as a separate, standalone Phase III trial.

In lieu of the $200.0 million milestone due to us under the original AbbVie Collaboration Agreement upon first patient dosed in the first registration enabling trial for DR SCS treatment, AbbVie will pay us $100.0 million upon first patient dosed in the NAAVIGATE trial and an additional $100.0 million upon first patient dosed in the subsequent Phase III trial.

Also pursuant to the amendment, AbbVie will lead a new Phase III randomized controlled study (ACHIEVE) to assess the injection burden, adverse events, change in disease activity, and long-term preservation of visual acuity of ABBV-RGX-314 in adult participants with neovascular AMD.

We will be responsible for our development expenses to conduct Part 1 of the NAAVIGATE trial and the parties will share the development expenses related to Part 2 of the NAAVIGATE trial and the subsequent Phase III trial for DR in accordance with the existing terms of the AbbVie Collaboration Agreement.

AbbVie will be responsible for all development expenses related to the ACHIEVE study.

Nippon Shinyaku Collaboration for RGX-121 and RGX-111

In January 2025, we entered into a collaboration and license agreement with Nippon Shinyaku Co., Ltd.

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We are responsible for the manufacturing of RGX-121 and RGX-111 for clinical development and commercial supply, and manufacturing expenses will be allocated between the parties in accordance with the terms of the Nippon Shinyaku Collaboration Agreement.

Nippon Shinyaku will be responsible, at its sole cost, for the commercialization of RGX-121 and RGX-111 in the licensed territories.

Under the terms of the Nippon Shinyaku Collaboration Agreement, we will receive an up-front payment of $110.0 million from Nippon Shinyaku following the effective date of the agreement in March 2025 and we are eligible to receive up to $700.0 million from Nippon Shinyaku upon the achievement of specified development and sales-based milestones.

Nippon Shinyaku is responsible, at its sole cost, for the commercialization of RGX-121 and RGX-111 in the licensed territories.

Under the terms of the Nippon Shinyaku Collaboration Agreement, we received an up-front payment of $110.0 million from Nippon Shinyaku following the effective date of the agreement in March 2025 and are eligible to receive up to $700.0 million from Nippon Shinyaku upon the achievement of specified development and sales-based milestones.

We are also eligible to receive double-digit royalties on net sales of RGX-121 and RGX-111 by Nippon Shinyaku, subject to specified offsets and reductions.

We also retain all rights to, and any proceeds related to the sale of, any priority review vouchers that may be issued upon the potential approvals of RGX-121 and RGX-111.

We retain all rights to, and any proceeds related to the sale of, any priority review vouchers that may be issued upon the potential approvals of RGX-121 and RGX-111.

We recognized $84.7 million of revenue under the Nippon Shinyaku Collaboration Agreement during the year ended December 31, 2025.

For additional information regarding the Nippon Shinyaku Collaboration Agreement, please refer to Note 10, “License and Collaboration Agreements—Nippon Shinyaku Collaboration and License Agreement” to the accompanying audited consolidated financial statements.

Overview of Our NAV Technology Platform

In May 2025, we entered into a loan agreement with entities managed by Healthcare Royalty Management, LLC (collectively and with other affiliated entities, HCR).

Pursuant to the terms of the loan agreement, future royalties, sales-based milestone payments and certain development milestone payments earned under the Nippon Shinyaku Collaboration Agreement, along with consideration earned under various other NAV Technology Platform license agreements, shall be used to repay principal and interest owed to HCR.

For additional information regarding the May 2025 loan agreement with HCR, please refer to Note 7, “Royalty Monetization Liabilities—2025 Royalty Bond” to the accompanying audited consolidated financial statements.

NAV Technology Licensing Platform

In addition to our internal product development efforts, we also selectively license the NAV Technology Platform and other intellectual property rights to other leading biotechnology and pharmaceutical companies, which we refer to as NAV Technology Licensees.

As of December 31, 2024, our NAV Technology Platform was being applied in one commercial product, Zolgensma ® , and the preclinical and clinical development of a number of other licensed products.

Licensing the NAV Technology Platform allows us to maintain our internal product development focus on our core disease indications and therapeutic areas while still expanding the NAV gene therapy pipeline, developing a greater breadth of treatments for patients, providing additional technological and potential clinical proof-of-concept for our NAV Technology Platform and creating potential additional revenue opportunities.

As of December 31, 2025, our NAV Technology Platform was being applied in two commercial products, Zolgensma ® and Itvisma ® , and the preclinical and clinical development of various other licensed products.

Financial Overview

Our revenues to date consist primarily of license and royalty revenue resulting from the licensing of our NAV Technology Platform and other intellectual property rights.

Our revenues to date have been primarily generated from the licensing of our NAV Technology Platform and other intellectual property rights to NAV Technology Licensees and collaborators.

We have not generated any revenues from commercial sales of our own products.

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License agreements generally have a term at least equal to the life of the underlying patents, but are terminable at the option of the licensee.

Consideration from licensees under our license agreements may include:

(i) up-front and annual fees, (ii) milestone payments based on the achievement of certain development and sales-based milestones, (iii) sublicense fees, (iv) royalties on sales of licensed products, (v) fees for services related to the development of licensed products and (vi) other consideration payable upon optional goods and services purchased by licensees.

Future license and royalty revenues are dependent on the successful development and commercialization of licensed products, which is uncertain, and revenues may fluctuate significantly from period to period.

Additionally, we may never receive consideration in our license agreements that is contemplated on option fees, development and sales-based milestone payments, royalties on sales of licensed products or sublicense fees, given the contingent nature of these payments.

Our revenues are concentrated among a low number of licensees and licenses are terminable at the option of the licensee.

The termination of our licenses by licensees may materially impact the amount of revenue we recognize in future periods.

Consideration payable to us under our license and collaboration agreements may include:

(i) up-front and annual fees, (ii) milestone payments based on the achievement of certain development and sales-based milestones, (iii) sublicense fees, (iv) royalties on sales of licensed products, (v) fees for services related to the development and manufacturing of licensed products and (vi) other consideration payable upon optional goods and services purchased by licensees and collaborators.

Future revenues under our license and collaboration arrangements are dependent on the successful development and commercialization of licensed products, which is uncertain, and revenues may fluctuate significantly from period to period.

Additionally, we may never receive consideration under our license or collaboration agreements that is contemplated on optional goods and services, development and sales-based milestones, royalties on sales of licensed products or sublicense fees, given the contingent nature of these payments.

Our revenues are concentrated among a low number of licensees and collaborators and the arrangements are terminable at the option of the counterparty.

The termination of our license and collaborations arrangements may materially impact the amount of revenue we recognize in future periods.

Please refer to Note 16, “Segment and Geographical Information” to the accompanying audited consolidated financial statements for a description of segment and geographical information regarding our revenues.

Zolgensma Royalties

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma, which is marketed by Novartis Gene Therapies, Inc.

(formerly AveXis, Inc.) (Novartis Gene Therapies), a wholly owned subsidiary of Novartis AG (Novartis), for the treatment of spinal muscular atrophy (SMA).

Zolgensma is a licensed product under our license agreement with Novartis Gene Therapies for the development and commercialization of treatments for SMA using the NAV Technology Platform.

Zolgensma and Itvisma Royalties

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma and Itvisma, which are marketed by Novartis Gene Therapies, Inc.

(Novartis Gene Therapies), a wholly owned subsidiary of Novartis AG (Novartis), for the treatment of spinal muscular atrophy (SMA).

Zolgensma and Itvisma are licensed products under our license agreement with Novartis Gene Therapies for the development and commercialization of treatments for SMA using the NAV Technology Platform.

Operating Expenses

Our operating expenses consist primarily of cost of revenues, research and development expenses and general and administrative expenses.

Our operating expenses consist primarily of cost of license and royalty revenues, research and development expenses and general and administrative expenses.

Personnel costs including salaries, wages, benefits, bonuses and stock-based compensation expense, comprise a significant component of research and development and general and administrative expenses.

We allocate indirect expenses associated with our facilities, information technology costs, depreciation and other overhead costs between research and development and general and administrative categories based on employee headcount and the nature of work performed by each employee or using other reasonable allocation methodologies.

Cost of Revenues

Our cost of revenues consists primarily of upstream fees due to our licensors as a result of revenue generated from the licensing of our NAV Technology Platform and other intellectual property rights, including sublicense fees and royalties on net sales of licensed products.

Cost of License and Royalty Revenues

Our cost of license and royalty revenues consists primarily of upstream fees due to our licensors as a result of revenue generated from the licensing of our NAV Technology Platform and other intellectual property rights, including sublicense fees and royalties on net sales of licensed products.

Sublicense fees are based on a percentage of license fees received by us from licensees and are recognized in the period that the underlying license revenue is recognized.

Royalties are based on a percentage of net sales of licensed products by licensees and are recognized in the period that the underlying sales occur.

Future costs of revenues are uncertain due to the nature of our license agreements and significant fluctuations in cost of revenues may occur from period to period.

Future costs of revenues are uncertain due to the nature of our license agreements and significant fluctuations in cost of license and royalty revenues may occur from period to period.

Research and Development Expense

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Total research and development

* Certain amounts reported in prior years have been reclassified to conform to the current year's presentation.

Direct expenses related to the development of ABBV-RGX-314 for the years ended December 31, 2024, 2023 and 2022 include net cost reimbursement from AbbVie under our eye care collaboration of $78.3 million, $74.2 million and $19.3 million, respectively, which were recorded as a reduction of research and development expenses.

Net cost reimbursement from AbbVie includes reimbursement of personnel and overhead costs attributable to the development of ABBV-RGX-314, the underlying costs of which are reported as unallocated expenses in the table above.

Direct expenses related to the development of ABBV-RGX-314 for the years ended December 31, 2025 and 2024 include net cost reimbursement from AbbVie under our eye care collaboration of $60.2 million and $78.3 million, respectively, which were recorded as a reduction of research and development expenses.

In addition to reimbursement of direct development expenses, net cost reimbursement from AbbVie includes reimbursement of personnel and overhead costs attributable to the development of ABBV-RGX-314, the underlying costs of which are reported as unallocated expenses in the table above.

We typically utilize our employee and infrastructure resources across our development programs.

As a result, we generally do not allocate personnel and other internal costs, such as facilities and other overhead costs, to specific product candidates or development programs.

Platform and early research reported in the table above includes direct costs not identifiable with a specific lead product candidate, including costs associated with our research and development platform used across programs, process and analytical development, early research and development for prospective product candidates and new technologies, and other costs in support of research and development activities.

Direct expenses related to the development of product candidates for which we have discontinued internal development are included in other product candidates in the table above.

We expect to continue to incur minor development expenses associated with long-term follow up studies for certain discontinued product candidates.

Platform and early research reported in the table above includes direct costs not identifiable with a specific lead product candidate, including costs associated with our research and development platform used across programs, manufacturing support, process and analytical development, early research and development for prospective product candidates and new technologies, and other costs in support of research and development activities.

General and Administrative Expense

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We expect that our general and administrative expenses will increase as we continue to develop, and potentially commercialize, our product candidates.

Specifically, we expect general and administrative costs associated with the potential commercialization of our product candidates to increase in future periods as we and our commercial partners prepare for and carry out product launch efforts, in particular for the potential commercialization of our RGX-202 and ABBV-RGX-314 product candidates.

Other Income (Expense)

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Investment Income

Investment income consists of interest income earned and gains and losses realized from our cash and cash equivalents, marketable securities and non-marketable equity securities.

Investment income consists of interest income earned and gains and losses realized from our cash equivalents, marketable securities and non-marketable equity securities.

Cash equivalents are comprised of money market mutual funds and highly liquid debt securities with original maturities of 90 days or less at acquisition.

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Interest Expense

Interest expense consists primarily of interest imputed on the liability related to the sale of future Zolgensma royalties to entities managed by Healthcare Royalty Management, LLC (collectively, HCR).

Interest expense is recognized using the effective interest method, based on our estimate of total royalty payments expected to be received by HCR under the royalty purchase agreement.

For further information regarding the royalty purchase agreement with HCR, please refer to Note 7, “Liability Related to Sale of Future Royalties” to the accompanying audited consolidated financial statements.

Interest expense is primarily associated with our royalty monetization liabilities, including our December 2020 royalty purchase agreement (2020 Royalty Purchase Agreement) and May 2025 loan agreement (2025 Royalty Bond) with HCR.

For further information regarding our royalty monetization liabilities and associated interest expense, please refer to Note 7, “Royalty Monetization Liabilities” to the accompanying audited consolidated financial statements.

Critical Accounting Policies and Estimates

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ASC 606 requires entities to recognize revenue when control of the promised goods or services is transferred to customers at an amount that reflects the consideration to which the entity expects to be entitled to in exchange for those goods or services.

The following five steps are performed to determine the appropriate revenue recognition for arrangements within the scope

The following five steps are performed to determine the appropriate revenue recognition for arrangements within the scope of ASC 606:

(i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract and (v) recognize revenue when (or as) the entity satisfies the performance obligations.

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In determining the appropriate interest rates for significant financing components, we evaluate the credit profile of the customer and prevailing market interest rates and select an interest rate in which we believe would be charged to the customer in a separate financing arrangement over a similar financing term.

License and Royalty Revenue

We license our NAV Technology Platform and other intellectual property rights to other biotechnology and pharmaceutical companies.

We license our NAV Technology Platform and other intellectual property rights to other biotechnology and pharmaceutical companies, including collaborators for the joint development and commercialization of our product candidates.

The terms of the licenses vary, and licenses may be exclusive or non-exclusive and may be sublicensable by the licensee.

Licenses may grant intellectual property rights for purposes of internal and preclinical research and development only, or may include the rights, or options to obtain future rights, to commercialize drug therapies for specific diseases using our NAV Technology Platform and other licensed rights.

Licenses may grant intellectual property rights for purposes of internal and preclinical research and development only, or may include the rights, or options to obtain future rights, to commercialize drug therapies for specific diseases using the NAV Technology Platform and other licensed rights.

License agreements generally have a term at least equal to the life of the underlying patents, but are terminable at the option of the licensee.

Consideration payable to us under our license agreements may include:

Our license agreements are accounted for as contracts with customers within the scope of ASC 606, with the exception of transactions for which the counterparty is determined not to be a customer.

At the inception of each license agreement, we determine the contract term for purposes of applying the requirements of ASC 606.

Consideration payable to us under our license and collaboration agreements may include:

(i) up-front and annual fees, (ii) milestone payments based on the achievement of certain development and sales-based milestones, (iii) sublicense fees, (iv) royalties on sales of licensed products, (v) fees for services related to the development and manufacturing of licensed products and (vi) other consideration payable upon optional goods and services purchased by licensees and collaborators.

We evaluate our agreements with collaboration partners to determine whether they are within the scope of ASC 808, Collaborative Arrangements (ASC 808).

For collaboration arrangements within the scope of ASC 808 that contain multiple elements, we identify the various transactions with the counterparty and determine if any unit of account is more reflective of a transaction with a customer and therefore should be accounted for within the scope of ASC 606.

For transactions that are accounted for pursuant to ASC 606, we apply the five-step model as described in our revenue recognition policies.

For transactions that are accounted for pursuant to ASC 808, an appropriate method of recognition and presentation is determined and consistently applied in accordance with our accounting policies for collaborative arrangements.

Our license and collaboration agreements are accounted for as contracts with customers within the scope of ASC 606, with the exception of transactions for which the counterparty is determined not to be a customer.

At the inception of each agreement, we determine the contract term for purposes of applying the requirements of ASC 606.

Licenses are generally terminable at the option of the licensee with advance notice to us.

For each license granted, including licenses granted upon the exercise of license options, we evaluate these termination rights to determine whether a substantive termination penalty would be incurred by the licensee upon termination.

For each license granted, we evaluate these termination rights to determine whether a substantive termination penalty would be incurred by the licensee upon termination.

If the licensee incurs a substantive termination penalty upon termination, the contract term for revenue recognition purposes is generally equal to the stated term of the license, which is the life of the underlying licensed patents.

Alternatively, if the licensee does not incur a substantive termination penalty upon termination, the contract term for revenue recognition purposes may be shorter than the stated term of the license, in which case the termination rights may be accounted for as contract renewal options.

The determination of whether a substantive termination penalty is associated with the termination rights requires significant judgment.

In making this determination, we consider, among other things, the nature of the intellectual property rights that would be returned to us upon termination, including the exclusivity of the licensed rights and the stage of development of the licensed products, the payment terms, including the amount and timing of non-refundable or guaranteed payments, and the business purpose of the termination rights granted to the licensee.

Generally, the most significant judgment in determining whether a substantive termination penalty exists relates to the amount of any up-front or guaranteed non-refundable payments relative to the amount of annual payments that may be avoided by the licensee upon termination of the license.

We consider all of the facts and circumstances relevant to each license when making this determination.

Performance obligations under our license agreements may include (i) the delivery of intellectual property licenses, (ii) options granted to licensees to acquire additional licenses, to the extent the options represent material rights to the licensee, and (iii) research and development services to be performed by us related to licensed products.

License agreements may provide licensees with contract renewal options or options to acquire additional licenses, goods or other services.

Options are evaluated at the inception of the license agreement to determine whether they provide material rights to the licensee.

In making this determination, we consider whether the options are priced at an incremental discount to the standalone selling price for the underlying licenses, goods or services, in which case the option is considered to be a material right to the licensee and is accounted for as a separate performance obligation under the current license agreement.

At the inception of each license agreement which contains performance obligations for research and development services, we evaluate whether the license is distinct from the research and development services, which requires judgment.

In making this determination, we consider, among other things, the stage of development of the licensed products and whether the research and development services will significantly impact further development of the licensed products.

determined that the license is not distinct from the research and development services, the license is combined with the research and development services into a single performance obligation.

We evaluate the transaction price of our license agreements at the inception of each agreement and at each reporting date.

The transaction price includes the fixed consideration payable to us during the contract term, as well as any variable consideration to the extent that it is probable that a significant reversal of revenue will not occur in the future.

Fixed consideration under the license agreements may include up-front and annual fees payable during the contract term and fees for development services related to licensed products.

Variable consideration under the license agreements may include development and sales-based milestone payments, sublicense fees and royalties on sales of licensed products.

Consideration contingent upon the exercise of options by a licensee is excluded from the transaction price and not accounted for as part of the license agreement until the option is exercised.

The transaction price for each license agreement is allocated to the underlying performance obligations based on their relative standalone selling prices and recognized as revenue when (or as) the performance obligations are satisfied.

Consideration allocated to performance obligations for the delivery of an intellectual property license is recognized as revenue in full upon the delivery of the license to the licensee.

Consideration allocated to performance obligations for license options is recognized as revenue in full upon the earlier of the option exercise or expiration.

The exercise of a license option by a licensee is accounted for as a new license for revenue recognition purposes.

Consideration allocated to performance obligations for research and development services is recognized as revenue as the services are performed by us.

Up-front and annual licenses fees payable to us over the contract term of each license are included in the transaction price, and the portion of this consideration allocated to the performance obligation for the delivery of the intellectual property license is recognized as revenue in full upon the delivery of the license to the licensee.

If annual license fees are payable to us in periods beyond 12 months from the delivery of the license, a significant financing component is deemed to exist which provides a financing benefit to the licensee.

If a significant financing component is identified, we adjust the transaction price for the license to include only the present value of the annual license fees payable to us over the contract term.

The discounted portion of the license fees is recognized as interest income from licensing over the financing period of the license.

Performance obligations under our license and collaboration agreements may include (i) the delivery of intellectual property licenses, (ii) development and manufacturing services to be performed by us related to licensed products and (iii) options granted to purchase additional goods and services, to the extent the options convey material rights.

At the inception of each license agreement which contains performance obligations for development, manufacturing or other services, we evaluate whether the license is distinct from the services, which requires judgment.

In making this determination, we consider, among other things, the stage of development of the licensed products and whether the services will significantly impact further development of the licensed products.

If it is determined that the license is not distinct from the services, the license is combined with the services into a single performance obligation.

Agreements may provide licensees and collaborators with options to purchase additional goods or other services, including options to purchase commercial supply of licensed products.

Options are evaluated at the inception of the agreement to determine whether they provide material rights to the customer.

In making this determination, we consider whether the options are priced at an incremental discount to the standalone selling price of the underlying goods or services, in which case the option is considered to be a material right.

Material rights are accounted for as separate performance obligations under the current arrangement.

We evaluate the transaction price of our license and collaboration agreements at contract inception and at each reporting date.

The transaction price includes the fixed consideration payable to us over the contract term, as well as any variable consideration to the extent that it is probable that a significant reversal of revenue will not occur in the future.

Fixed consideration under the agreements may include up-front and annual fees payable to us over the contract term and fixed fees for development, manufacturing and other services.

Variable consideration under the agreements may include development and sales-based milestone payments, payments for development, manufacturing and other services, sublicense fees and royalties on sales of licensed products.

Consideration contingent upon the exercise of options by the customer is excluded from the transaction price and not accounted for as part of the arrangement until the option is exercised.

The transaction price of our license and collaboration arrangements is allocated to the underlying performance obligations based on their relative standalone selling prices and recognized as revenue when (or as) the performance obligations are satisfied.

Variable consideration payable based on services performed is allocated directly to the performance obligation for such services.

Consideration allocated to performance obligations for the delivery of intellectual property licenses is recognized as license and royalty revenue in full upon the delivery of the license.

Consideration allocated to performance obligations for development, manufacturing and other services is recognized as service revenue as we perform the services.

Consideration allocated to performance obligations for material rights to purchase additional goods and services is recognized as revenue upon the satisfaction of the performance obligations underlying the optional goods and services purchased by the customer.

Service revenue is recognized using a measure of progress that

best reflects the pattern of satisfaction of the performance obligations.

At each reporting date, we re-evaluate the measure of progress and adjust service revenue on a cumulative catch-up basis to reflect our best estimate of the services performed to date versus the total services to be performed under the arrangement.

Development milestone payments are payable to us upon the achievement of specified development milestones.

At the inception of each license agreement that contains development milestone payments, we evaluate whether the milestones are considered probable of achievement and estimate the amount to be included in the transaction price using the most likely amount method.

If it is probable that a significant revenue reversal will not occur in the future, milestone payments are included in the transaction price and recognized as revenue upon the delivery of the license.

At the inception of each license agreement that contains development milestone payments, we evaluate whether the milestones are probable of achievement and estimate the amount to be included in the transaction price using the most likely amount method.

If it is probable that a significant revenue reversal will not occur in the future, milestone payments are included in the transaction price.

Milestone payments contingent on the achievement of development milestones that are not within our control or the control of the licensee, such as regulatory approvals, are not considered probable of being achieved and are excluded from the transaction price until the milestone is achieved.

At each reporting date, we re-evaluate the probability of achievement of each outstanding development milestone and, if necessary, adjust the transaction price for any milestones for which the probability of achievement has changed due to current facts and circumstances.

Any such adjustments are recorded on a cumulative catch-up basis and recognized as revenue in the period of the adjustment.

Royalties on sales of licensed products, sales-based milestone payments, including milestones payable upon first commercial sales of licensed products, and sublicense fees based on the receipt of certain fees by licensees from any sublicensees are excluded from the transaction price of each license and recognized as revenue in the period that the related sales or sublicenses occur, provided that the associated license has been delivered to the licensee.

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma, which is a licensed product under our license agreement with Novartis Gene Therapies for the development and commercialization of treatments for SMA.

We recognize royalty revenue from net sales of Zolgensma in the period in which the underlying products are sold by Novartis Gene Therapies, which in certain cases may require us to estimate royalty revenue for periods of net sales which have not yet been reported to us.

The increase to the transaction price as a result of any such adjustments is then allocated to the underlying performance obligations in a manner similar to the allocation of the initial transaction price and, to the extent the performance obligations are satisfied, recognized as revenue on a cumulative catch-up basis in the period of the adjustment.

Royalties on sales of licensed products, sales-based milestone payments, including milestones payable upon first commercial sales of licensed products, and sublicense fees based on the receipt of certain fees by licensees from any sublicensees are excluded from the transaction price of each license and recognized as license and royalty revenue in the period that the related sales or sublicenses occur, provided that the associated license has been delivered to the licensee.

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma and Itvisma, which are licensed products under our license agreement with Novartis Gene Therapies for the development and commercialization of treatments for SMA.

We recognize royalty revenue from net sales of Zolgensma and Itvisma in the period in which the underlying products are sold by Novartis Gene Therapies, which in certain cases may require us to estimate royalty revenue for periods of net sales which have not yet been reported to us.

Estimated royalties are reconciled to actual amounts reported in subsequent periods, and any differences are recognized as an adjustment to royalty revenue in the period the royalties are reported.

We receive payments from licensees based on the billing schedules established in each license agreement.

Amounts recognized as revenue which have not yet been received from licensees, including unbilled royalties, are recorded as accounts receivable when our rights to the consideration are conditional solely upon the passage of time.

Amounts recognized as revenue which have not yet been received from licensees are recorded as contract assets when our rights to the consideration are not unconditional.

Contract assets are recorded as other current assets on the consolidated balance sheets.

If a licensee elects to terminate a license prior to the end of the license term, the licensed intellectual property is returned to us and any consideration recorded as accounts receivable or contract assets which is not contractually payable by the licensee is charged off as a reduction of license revenue in the period of the termination.

Amounts received by us prior to the delivery of underlying performance obligations are deferred and recognized as

revenue upon the satisfaction of the performance obligations.

We receive payments from licensees and collaborators based on the billing schedules established in the associated agreements.

Amounts recognized as revenue which have not yet been received from the customer are recorded as accounts receivable when our rights to the consideration are conditional solely upon the passage of time.

Amounts recognized as revenue which have not yet been received from customers are recorded as contract assets when our rights to the consideration are not unconditional.

Contract assets are recorded as other current assets on the consolidated balance sheets if the consideration is expected to be realized within 12 months from the reporting date, or as other assets if the consideration is expected to be realized in periods beyond 12 months from the reporting date.

Amounts received by us prior to the delivery of underlying performance obligations are deferred and recognized as revenue upon the satisfaction of the performance obligations.

Deferred revenue which is not expected to be recognized within 12 months from the reporting date is recorded as non-current on the consolidated balance sheets.

Collaborative Arrangements

We evaluate our agreements with collaboration partners to determine whether they are within the scope of ASC 808, Collaborative Arrangements (ASC 808).

We evaluate our agreements with collaboration partners to determine whether they are within the scope of ASC 808.

Such arrangements are within the scope of ASC 808 if they involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities.

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For transactions that are accounted for pursuant to ASC 808, an appropriate method of recognition and presentation is determined and consistently applied.

For transactions that are accounted for pursuant to ASC 606, we apply the five-step model as described in our revenue recognition policies.

For transactions that are accounted for pursuant to ASC 808, an appropriate method of recognition and presentation is determined and consistently applied.

For additional information regarding our collaborative arrangements, including our collaborations with AbbVie and Nippon Shinyaku, refer to Note 10, “License and Collaboration Agreements” to the accompanying audited consolidated financial statements.

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We estimate the fair value of our stock option awards using the Black-Scholes option-pricing model, which requires the input of subjective assumptions, including (i) the fair value of the underlying common stock, (ii) the expected stock price volatility, (iii) the expected term of the award, (iv) the risk-free interest rate and (v) expected dividends.

The fair value of our common stock, as used as an input to determine the fair value of our stock option awards, is based on the closing price of our common stock on the date of the

The fair value of our common stock, as used as an input to determine the fair value of our stock option awards, is based on the closing price of our common stock on the date of the grant.

We estimate expected stock price volatility based on the historical volatility of our common stock over a period of time commensurate with the expected term of our stock option awards.

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We estimate the fair value of our restricted stock units based on the closing price of our common stock on the date of the grant.

Interest Expense on Liability Related to Sale of Future Royalties

We recorded a liability for the net proceeds received from the sale of our Zolgensma royalty payments to HCR.

The liability is amortized over the estimated life of the arrangement using the effective interest method.

The total amount of royalty payments received by HCR under the agreement, less the net proceeds we received from the sale, is recorded as interest expense over the life of the arrangement.

We estimate the effective interest rate based on our estimate of total future royalty payments to be received by HCR under the agreement.

We reassess these estimates at each reporting date and adjust the effective interest rate and amortization of the liability on a prospective basis as necessary.

Royalty Monetization Liabilities

Net proceeds received under our royalty monetization agreements with HCR are recorded as liabilities and accounted for as debt.

The liabilities are amortized over the estimated life of the arrangements using the effective interest method.

For arrangements in which there is no stated interest rate, the total amount of royalty and other payments paid to HCR under the arrangement, less the net proceeds we received from HCR, is recorded as interest expense over the life of the arrangement.

We estimate the effective interest rates of our royalty monetization liabilities based on our estimate of total payments to be paid to HCR under the arrangement.

We reassess these estimates at each reporting date and adjust the effective interest rate and amortization of the liabilities on a prospective basis as necessary.

We account for income taxes in accordance with ASC 740, Income Taxes, which provides for deferred taxes using an asset and liability approach.

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As of December 31, 2025, we had federal net operating loss (NOL) carryforwards of $754.4 million, U.S.

state NOL carryforwards of $408.9 million and federal and state research and development tax credit carryforwards of $87.2 million (net of unrecognized tax benefits of $0.1 million) which may be available to offset future income tax liabilities.

state NOL carryforwards of $394.9 million and federal and state research and development tax credit carryforwards of $95.1 million which may be available to offset future income tax liabilities.

Our federal NOL carryforwards and a portion of our state NOL carryforwards as of December 31, 2025 may be carried forward indefinitely.

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License and royalty revenue

Service revenue

Total revenues

Operating Expenses

Cost of revenues

Cost of license and royalty revenues

Research and development

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Impairment of long-lived assets

Other operating expenses (income)

Other operating expenses

Total operating expenses

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Total other income (expense)

Loss before income taxes

Income Tax Benefit

Comparison of the Years Ended December 31, 2025 and 2024

License and Royalty Revenue.

License and royalty revenue decreased by $6.9 million, from $90.2 million for the year ended December 31, 2023 to $83.3 million for the year ended December 31, 2024.

The decrease was primarily attributable to non-recurring development milestone revenue recognized in 2023, and Zolgensma royalty revenues, which decreased from $85.3 million in 2023 to $81.5 million in 2024.

As reported by Novartis, sales of Zolgensma in 2024 were $1.21 billion, consistent with 2023, and the product continues to treat mainly incident patients in established markets.

The decrease in Zolgensma royalties was primarily attributable to fluctuations in the effective royalty rate under the license agreement with Novartis.

License and royalty revenue increased by $74.3 million, from $82.0 million for the year ended December 31, 2024 to $156.3 million for the year ended December 31, 2025.

The increase was primarily attributable to $72.9 million of up-front license revenue recognized under our collaboration with Nippon Shinyaku in 2025, as well as an increase in royalty revenues for Zolgensma and Itvisma.

Total royalty revenues for Zolgensma and Itvisma increased by $1.7 million, from $81.5 million in 2024 to $83.2 million in 2025.

Novartis reported combined Zolgensma and Itvisma sales of $1.23 billion in 2025, as compared to $1.21 billion in 2024.

As reported by Novartis, the increase reflects continued strong demand for Zolgensma in the incident SMA population, and was partially driven by the approval and launch of Itvisma in the fourth quarter of 2025.

Zolgensma and Itvisma royalty revenues for 2025 were $82.5 million and $0.6 million, respectively.

Itvisma royalties for 2025 reflect a product launch in the late fourth quarter of 2025.

Service Revenue.

Service revenue increased by $12.8 million, from $1.4 million for the year ended December 31, 2024 to $14.2 million for the year ended December 31, 2025.

The increase was primarily attributable to $11.8 million of service revenue recognized under our collaboration with Nippon Shinyaku in 2025, largely driven by the performance of RGX-121 development and manufacturing services.

Cost of License and Royalty Revenues.

Cost of license and royalty revenues decreased by $13.3 million, from $33.6 million for the year ended December 31, 2024 to $20.3 million for the year ended December 31, 2025.

The decrease was largely driven by a reduction in upstream Zolgensma royalties payable to licensors for net sales in certain territories outside the United States.

Research and Development Expense.

Research and development expenses decreased by $23.7 million, from $232.3 million for the year ended December 31, 2023 to $208.5 million for the year ended December 31, 2024.

The decrease was primarily attributable to the following:

• a decrease of $10.5 million in personnel-related costs for research and development personnel, including a $2.6 million decrease in stock-based compensation expense, primarily driven by the reduction in workforce associated with our corporate restructuring in the fourth quarter of 2023;

• a decrease of $10.3 million in manufacturing expenses and other costs of clinical supply for our lead product candidates, largely driven by ABBV-RGX-314 and RGX-121 clinical supply costs;

• a decrease of $6.2 million in preclinical activities and other early-stage research and development;

• a decrease of $6.1 million in costs for laboratories and facilities used by research and development personnel, including a $1.1 million decrease in depreciation expense allocated to research and development functions, primarily driven by a decrease in laboratory supplies and consumables.

The decrease in research and development expenses was partially offset by an increase of $10.5 million in costs associated with clinical trial activities, largely driven by clinical trial expenses for ABBV-RGX-314 and RGX-202.

Research and development expenses increased by $19.8 million, from $208.5 million for the year ended December 31, 2024 to $228.3 million for the year ended December 31, 2025.

The increase was primarily attributable to the following:

• an increase of $8.1 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates, largely driven by manufacturing costs for ABBV-RGX-314, RGX-202 and RGX-121;

• an increase of $5.8 million in personnel-related costs due to increased headcount of development personnel, net of a $2.0 million decrease in stock-based compensation expense;

• an increase of $5.7 million in costs associated with clinical trials and regulatory activities, largely driven by clinical trial expenses for RGX-202 pivotal trials.

General and Administrative Expense.

General and administrative expenses decreased by $11.9 million, from $88.5 million for the year ended December 31, 2023 to $76.6 million for the year ended December 31, 2024.

The decrease was primarily attributable to professional services and consulting fees, including legal and other corporate advisory services, and other corporate overhead expenses.

General and administrative expenses increased by $6.2 million, from $76.6 million for the year ended December 31, 2024 to $82.9 million for the year ended December 31, 2025.

The increase was largely driven by professional services, consulting and other corporate advisory services.

Credit Losses (Recoveries).

We recognized credit recoveries of $5.0 million during the year ended December 31, 2024 related to the full collection of amounts due under our settlement agreement with Abeona Therapeutics Inc.

We recognized credit recoveries of $5.0 million during the year ended December 31, 2024 upon the full collection of amounts due under our settlement agreement with Abeona Therapeutics Inc.

(Abeona), for which we had previously recorded an allowance for credit losses.

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Investment Income.

Investment income increased by $7.4 million, from $11.3 million for the year ended December 31, 2023 to $18.7 million for the year ended December 31, 2024.

The increase was largely attributable to the achievement of milestones associated with the acquisition of our non-marketable equity securities of Corlieve Therapeutics SAS (Corlieve) by uniQure N.V.

(uniQure) in July 2021.

The Company recognized realized gains of $6.6 million and $2.2 million upon the achievement of such milestones during 2024 and 2023, respectively.

The remaining increase was primarily attributable to higher yields on investments in cash equivalents and marketable debt securities.

Investment income decreased by $6.5 million, from $18.7 million for the year ended December 31, 2024 to $12.2 million for the year ended December 31, 2025.

The decrease was primarily attributable to the achievement of milestones associated with the July 2021 acquisition of our non-marketable equity securities of Corlieve Therapeutics SAS (Corlieve) by uniQure N.V.

We recognized realized gains of $6.6 million upon the achievement of such milestones during 2024.

No such milestones were achieved during 2025 and no gains were realized during the period.

Interest Expense.

Interest expense increased by $32.3 million, from $12.7 million for the year ended December 31, 2024 to $45.0 million for the year ended December 31, 2025.

The increase was primarily attributable to interest expense under our royalty monetization liabilities, driven largely by an increase in forecasted Zolgensma and Itvisma royalties expected to be paid to HCR under the 2020 Royalty Purchase Agreement and interest expense incurred to date under the 2025 Royalty Bond issued in May 2025.

Liquidity and Capital Resources

Sources of Liquidity

As of December 31, 2024, we had cash, cash equivalents and marketable securities of $244.9 million, which were primarily derived from the sale of our common stock and pre-funded warrants and license fees received under the AbbVie Collaboration Agreement, as described below.

We expect that our cash, cash equivalents and marketable securities as of December 31, 2024, along with the up-front payment of $110.0 million expected to be received under the Nippon Shinyaku Collaboration Agreement, will enable us to fund our operating expenses and capital expenditure requirements, and are sufficient to meet our financial commitments and obligations, for at least the next 12 months from the date of this report, based on our current business plan.

Our recent sources of liquidity include the following events and transactions:

• In March 2024, we completed a public offering of 4,565,260 shares of our common stock at a price of $23.00 per share and 1,521,740 pre-funded warrants to purchase shares of our common stock at a price of $22.9999 per pre-funded warrant, which equaled the public offering price per share of the common stock less the $0.0001 exercise price of each pre-funded warrant.

The aggregate net proceeds received from the offering were $131.1 million, net of underwriting discounts and commissions and offering expenses.

• In September 2021, we entered into the AbbVie Collaboration Agreement for the development and commercialization of ABBV-RGX-314.

Pursuant to the AbbVie Collaboration Agreement, we received an up-front fee of $370.0 million from AbbVie upon the effective date of the agreement in November 2021, and we are eligible to receive up to $1.38 billion from AbbVie upon the achievement of specified development and sales-based milestones.

Additionally, the parties will share equally in the net profits and net losses associated with the commercialization of ABBV-RGX-314 in the United States, and we are eligible to receive tiered royalties on net sales by AbbVie of ABBV-RGX-314 outside the United States.

As of December 31, 2025, we had cash, cash equivalents and marketable securities of $240.9 million, which were primarily derived from the royalty monetization in May 2025, the up-front payment received under the Nippon Shinyaku Collaboration Agreement in March 2025 and the sale of our common stock and pre-funded warrants in March 2024, each as described below:

• In May 2025, we entered into a loan agreement with HCR pursuant to which HCR will provide us with an aggregate limited recourse loan of up to $250.0 million (the 2025 Royalty Bond).

The 2025 Royalty Bond is disbursable to us in three tranches, with $150.0 million funded on the closing date in May 2025, $50.0 million available to be funded if sales of a specified product exceed a specified sales threshold prior to December 31, 2026, and $50.0 million available to be funded if both parties exercise an option in 2027.

Proceeds received from the initial funding tranche of the 2025 Royalty Bond in May 2025, net of discounts and transaction costs, were $144.5 million.

The 2025 Royalty Bond matures in 2035, subject to potential extension, and bears interest at a rate of 9.75% plus the 3-month secured overnight financing rate as administered by the Federal Reserve Bank of New York (SOFR), with a minimum interest rate of 14.0%.

Prior to the maturity date, interest and principal under the 2025 Royalty Bond will be paid quarterly to HCR solely using proceeds

received, net of upstream obligations to licensors, from certain specified royalties, milestone payments, license fees and other consideration payable to us under the Zolgensma and Itvisma license with Novartis Gene Therapies, the Nippon Shinyaku Collaboration Agreement and certain other NAV Technology Platform license agreements.

• In January 2025, we entered into the Nippon Shinyaku Collaboration Agreement for the development and commercialization of RGX-121 and RGX-111 in the United States and certain countries in Asia.

Pursuant the Nippon Shinyaku Collaboration Agreement, we will receive an up-front payment of $110.0 million following the effective date of the agreement in March 2025 and are eligible to receive up to $700.0 million upon the achievement of specified development and sales-based milestones.

Pursuant the Nippon Shinyaku Collaboration Agreement, we received an up-front payment of $110.0 million following the effective date of the agreement in March 2025 and are eligible to receive up to $700.0 million upon the achievement of specified development and sales-based milestones.

We are also eligible to receive double-digit royalties on net sales of RGX-121 and RGX-111 by Nippon Shinyaku, subject to specified offsets and reductions.

We intend to devote the majority of our current capital to preclinical research, clinical development, seeking regulatory approval of our product candidates and, if approved, commercialization of our product candidates, as well as additional capital expenditures needed to support these activities.

Because of the numerous risks and uncertainties associated with the development and commercialization of gene therapy product candidates, we are unable to estimate the total amount of operating expenditures and capital outlays necessary to complete the development of our product candidates.

Additionally, our estimates are based on assumptions that may prove to be wrong, and we may use our available capital resources sooner than we currently expect which could accelerate our liquidity needs.

At-the-Market Offering Programs

In September 2023, we entered into an ATM Equity Offering SM Sales Agreement with BofA Securities, Inc.

(BofA) pursuant to which we may offer and sell shares of our common stock having an aggregate offering price of up to $150.0 million from time to time through BofA, acting as our sales agent (the BofA ATM Program).

We terminated the BofA ATM Program effective in November 2024.

No shares of common stock were sold under the BofA ATM Program prior to its termination.

The aggregate net proceeds received from the offering were $131.1 million, net of underwriting discounts and commissions and offering expenses.

At-the-Market Offering Program

In December 2024, we entered into a Sales Agreement with Leerink Partners LLC (Leerink) pursuant to which we may offer and sell shares of our common stock having an aggregate offering price of up to $150.0 million from time to time through Leerink, acting as our sales agent (the Leerink ATM Program).

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We intend to use proceeds obtained from the sale of shares under the Leerink ATM Program, if any, for general corporate purposes.

Private Placement

In July 2023, we sold 257,466 shares of our common stock in a private placement transaction for which we received aggregate net proceeds of $4.9 million, net of offering expenses.

Future Liquidity and Ability to Continue as a Going Concern

We expect that our cash, cash equivalents and marketable securities of $240.9 million as of December 31, 2025 will enable us to fund our operating expenses and capital expenditure requirements, and are sufficient to meet our financial commitments and obligations into early 2027.

This estimate is based on our current operating plan, and excludes the potential effects of any future financings or material milestone payments that may be received under our licensing and collaboration arrangements.

We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our capital resources sooner than expected.

These conditions raise substantial doubt about our ability to continue as a going concern within 12 months from the issuance date of our consolidated financial statements for the year ended December 31, 2025, which accompany this Annual Report on Form 10-K.

Our ability to continue as a going concern will depend heavily on the successful development, approval and commercialization of our product candidates and our ability to raise additional capital to fund operations.

If we are unable to raise capital sufficient to meet our working capital needs in the future, we may be forced to delay expenditures, reduce the scope of our development activities or make other changes to our operating plans.

Our consolidated cash flows were as follows (in thousands):

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Net cash provided by (used in) investing activities

Net cash provided by (used in) financing activities

Net cash provided by financing activities

Net increase (decrease) in cash and cash equivalents and restricted cash

Cash Flows from Operating Activities

Our net cash used in operating activities for the year ended December 31, 2024 decreased by $45.3 million from the year ended December 31, 2023, largely as a result of lower operating expenses and increased cost reimbursement received from AbbVie under our ABBV-RGX-314 collaboration in 2024.

Our net cash used in operating activities for the year ended December 31, 2025 decreased by $49.2 million from the year ended December 31, 2024, largely as a result of the $110.0 million up-front fee received from Nippon Shinyaku in March 2025, partially offset by an increase in operating expenses in 2025.

We expect to continue to incur regular net cash outflows from operations for the foreseeable future as we continue the development and advancement of our product candidates and other research programs.

For the year ended December 31, 2025, our net cash used in operating activities of $124.0 million consisted of a net loss of $193.9 million, offset by adjustments for non-cash items of $58.1 million and favorable changes in operating assets and liabilities of $11.8 million.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $34.6 million, depreciation and amortization expense of $15.6 million, and non-cash interest expense of $12.9 million.

The changes in operating assets and liabilities include an increase in deferred revenue of $29.3 million, which was primarily attributable to the deferred portion of the $110.0 million up-front payment received under our collaboration with Nippon Shinyaku in the first quarter of 2025.

The favorable changes in operating assets and liabilities were partially offset by an increase in accounts receivable of $7.7 million, which was driven largely by reimbursable costs due from Nippon Shinyaku under our collaboration for RGX-121 and RGX-111 and royalties receivable on net sales of Zolgensma and Itvisma.

Other changes in operating working capital occurred in the normal course of business.

For the year ended December 31, 2024, our net cash used in operating activities of $173.1 million consisted of a net loss of $227.1 million, offset by adjustments for non-cash items of $48.4 million and favorable changes in operating assets and liabilities of $5.5 million.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $38.5 million and depreciation and amortization expense of $16.2 million, partially offset by realized gains on investments, credit recoveries and the accretion of discounts on marketable debt securities during the period.

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Other changes in operating working capital occurred in the normal course of business.

For the year ended December 31, 2023, our net cash used in operating activities of $218.4 million consisted of a net loss of $263.5 million and unfavorable changes in operating assets and liabilities of $10.9 million, offset by adjustments for non-cash items of $56.0 million.

The changes in operating assets and liabilities include an increase in other current assets of $10.5 million, which was driven primarily by an increase in net cost reimbursement due from AbbVie under our ABBV-RGX-314 collaboration.

Other changes in operating working capital occurred in the normal course of business.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $40.3 million and depreciation and amortization expense of $17.3 million.

Cash Flows from Investing Activities

For the year ended December 31, 2024, our net cash provided by investing activities consisted of $290.2 million in maturities of marketable debt securities and $5.8 million in proceeds received from uniQure upon the achievement of milestones associated with their acquisition of Corlieve, offset by $190.1 million used to purchase marketable debt securities and $2.4 million used to purchase property and equipment.

For the year ended December 31, 2025, our net cash used in investing activities primarily consisted of $332.6 million used to purchase marketable debt securities and $2.4 million used to purchase property and equipment, partially offset by $319.1 million in maturities of marketable debt securities.

Cash Flows from Financing Activities

For the year ended December 31, 2024, our net cash provided by financing activities primarily consisted of $131.1 million in net proceeds received from the public offering of common stock and pre-funded warrants completed in March 2024, net of underwriting discounts and commissions and other offering expenses paid during the period, and $2.7 million in proceeds received from the exercise of stock options and issuance of common stock under our employee stock purchase plan.

Our net cash provided by financing activities was partially offset by $39.9 million of Zolgensma royalties paid, net of imputed interest, under our royalty purchase agreement with HCR.

For the year ended December 31, 2023, our net cash used in financing activities primarily consisted of $42.3 million of Zolgensma royalties paid, net of imputed interest, under our royalty purchase agreement with HCR.

Our net cash used in financing activities was partially offset by $4.9 million in net proceeds received from a private placement of our common stock in July 2023 and $3.3 million in proceeds received from the exercise of stock options and issuance of common stock under our employee stock purchase plan.

For the year ended December 31, 2025, our net cash provided by financing activities primarily consisted of $144.5 million in proceeds received from the issuance of the 2025 Royalty Bond and warrants to HCR in May 2025, net of discounts and transaction costs paid during the period, and was partially offset by $28.1 million of royalties paid, net of interest, under our royalty monetization liabilities.

For the year ended December 31, 2024, our net cash provided by financing activities primarily consisted of $131.1 million in proceeds received from the public offering of common stock and pre-funded warrants completed in March 2024, net of underwriting discounts and commissions and other offering expenses paid during the period, and $2.7 million in proceeds received from the exercise of stock options and issuance of common stock under our employee stock purchase plan.

Our net cash provided by financing activities was partially offset by $39.9 million of royalties paid, net of interest, under our royalty monetization liabilities.

Additional Capital Requirements

Our material capital requirements from known contractual and other obligations primarily relate to vendor service contracts and purchase commitments, in-license agreements, operating lease agreements and our Zolgensma royalty purchase agreement with HCR.

Our material capital requirements from known contractual and other obligations primarily relate to our vendor service contracts and purchase commitments, in-license agreements, operating lease agreements and royalty monetization agreements.

In the normal course of business, we enter into services agreements with CROs, CMOs and other third parties.

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We are no longer obligated to pay sublicense fees to Penn under the license agreement, but remain obligated to pay Penn royalties on net sales of licensed products, milestone fees and reimbursement of certain patent maintenance costs in accordance with the Penn License.

As of December 31, 2024, we had $6.0 million remaining payable to Penn under the Penn Letter Agreement, in addition to other amounts payable Penn under the Penn License.

As of December 31, 2025, we had $3.0 million remaining payable to Penn under the Penn Letter Agreement, in addition to other amounts payable under the Penn License.

We have entered into a number of long-term operating leases for office, laboratory and manufacturing space in Rockville, Maryland, Washington, D.C.

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Please refer to Note 6, “Leases” to the accompanying consolidated financial statements for further information regarding our lease commitments.

Under the terms of our royalty purchase agreement with HCR, our future Zolgensma royalties, less amounts payable by us to certain licensors, will be payable to HCR up to a specified capped amount.

As of December 31, 2024, the total amount of future Zolgensma royalties to be paid to HCR under the agreement was $95.6 million.

We have no obligation to repay any amounts to HCR if total future Zolgensma royalty payments are not sufficient to repay these amounts.

Under the terms of the 2020 Royalty Purchase Agreement, our Zolgensma and Itvisma royalties, less amounts payable by us to certain licensors, are payable to HCR up to a specified capped amount.

As of December 31, 2025, the total amount of future royalties payable to HCR under the 2020 Royalty Purchase Agreement was $35.5 million.

We have no obligation to repay any amounts to HCR if total future Zolgensma and Itvisma royalty payments from Novartis are not sufficient to repay these amounts.

Upon full repayment of our obligation under the 2020 Royalty Purchase Agreement, future Zolgensma and Itvisma royalties shall be included in the royalty interest payable to HCR under the 2025 Royalty Bond.

Under the terms of the 2025 Royalty Bond, interest and principal shall be paid quarterly to HCR solely using proceeds received, net of upstream obligations to licensors, from certain specified royalties, milestone payments, license fees and other consideration payable to us under the Zolgensma and Itvisma license with Novartis Gene Therapies, the Nippon Shinyaku Collaboration Agreement and certain other NAV Technology Platform license agreements.

If the proceeds received under the specified license agreements are insufficient to pay the quarterly interest due to HCR, unpaid interest will accrue to the principal balance.

The 2025 Royalty Bond matures in May 2035, subject to potential extension, unless repaid in full at an earlier date.

Upon maturity, the outstanding principal and interest shall be due and payable to HCR.

Other than through the payment of proceeds received under the specified license agreements, the 2025 Royalty Bond may not be prepaid prior to maturity.

The 2025 Royalty Bond is collateralized by a security interest and lien on the specified royalties and license fees.

As of December 31, 2025, the principal balance outstanding under the 2025 Royalty Bond was $163.4 million.

Future Funding Requirements

We have incurred cumulative losses since our inception and had an accumulated deficit of $932.1 million as of December 31, 2024.

We have incurred cumulative losses since our inception and had an accumulated deficit of $1.13 billion as of December 31, 2025.

Our transition to recurring profitability is dependent upon achieving a level of revenues adequate to support our cost structure, which depends heavily on the successful development, approval and commercialization of our product candidates.

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We expect to continue to incur significant research and development and general and administrative expenses for the foreseeable future as we continue the development of, and seek regulatory approval for, our product candidates.

Subject to obtaining regulatory approval for our product candidates, we expect to incur significant commercialization expenses for product sales, marketing, manufacturing and distribution.

Subject to obtaining regulatory approval for our product

candidates, we expect to incur significant commercialization expenses for product sales, marketing, manufacturing and distribution.

Additionally, we expect to continue to incur capital expenditures associated with building out additional laboratory and manufacturing capacity to further support the development of our product candidates and potential commercialization efforts.

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• the scope, progress, results and costs of drug discovery, laboratory testing, preclinical development and clinical trials for our product candidates;

• delays or costs due to a clinical hold or CRL, including BLA resubmission;

• whether we receive a PRV and are able to monetize or otherwise realize any potential value associated with such a voucher;

• the value of any PRV received diminishes including any decreases due to demand for these vouchers;

• the costs associated with building out additional laboratory and manufacturing capacity;

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• revenue, if any, received from commercial sales of our products, should any of our product candidates receive marketing approval;

• revenue received from commercial sales of Zolgensma and the timing and amount of Zolgensma royalties paid to HCR under our royalty purchase agreement;

• revenue received from other commercial sales of our licensees’ and collaborators’ products, should any of their product candidates receive marketing approval, and other revenue received under our licensing agreements and collaborations;

• the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending any intellectual property-related claims;

• revenue received from commercial sales of Zolgensma and Itvisma, and the timing and amount of Zolgensma and Itvisma royalties paid to HCR under our royalty monetization agreements;

• revenue received from other commercial sales of our licensees’ and collaborators’ products, should any of their product candidates receive marketing approval, other revenue received under our licensing agreements and collaborations, and the timing and amount of any such revenues payable to HCR under our royalty monetization agreements;

• the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights including against Sarepta and defending any intellectual property-related claims;

• our current licensing agreements or collaborations remaining in effect, including the AbbVie Collaboration Agreement relating to ABBV-RGX-314 and the Nippon Shinyaku Collaboration Agreement relating to RGX-121 and RGX-111, and our ability to timely achieve any milestones set forth in such agreements or collaborations;

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Our cash equivalents and marketable securities as of December 31, 2025 consisted of money market mutual funds, U.S.

government and agency securities, certificates of deposit and corporate bonds.

government and agency securities and corporate bonds.

If market interest rates were to increase immediately and uniformly by 100 basis points, or one percentage point, from levels at December 31, 2025, we estimate that the increase would have resulted in a hypothetical decline of $0.8 million in the net fair value of our interest-sensitive securities as of December 31, 2025.

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In our management’s opinion, we have maintained effective internal control over financial reporting as of December 31, 2025, based on criteria established in the COSO 2013 framework.

The effectiveness of our internal control over financial reporting as of December 31, 2024 has been audited by PricewaterhouseCoopers LLP, our independent registered public accounting firm, as stated in their report which accompanies our audited consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K.

As a non-accelerated filer as defined in Rule 12b-2 under the Exchange Act, our independent registered public accounting firm is not required to issue an attestation report on our internal control over financial reporting and no such report is included in this Annual Report on Form 10-K.

Changes in Internal Control over Financial Reporting

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Purchase/Sale

Purchased/Sold (b)

Purchased/Sold (a)

Stephen Pakola, M.D.

Executive Vice President, Chief Medical Officer

Entities affiliated with Allan M.

Executive Vice President, Chief Legal Officer

(a) Entities affiliated with Allan M.

Fox, a director of the Company, include AMF Family Gift Trust LLC, AMF Gift Trust LLC, HBF Gift Trust LLC and Quaker Gray LLC (collectively, the Fox LLCs).

Fox maintains voting and investment control over the shares held by the Fox LLCs in his capacity as manager of the Fox LLCs.

(b) The aggregate number of shares in this column includes shares that may be forfeited or withheld to satisfy exercise price and tax withholding obligations at the time of vesting.

(a) The aggregate number of shares in this column includes shares that may be forfeited or withheld to satisfy exercise price and/or tax withholding obligations at the time of vesting.

Other than as described above, during the three months ended December 31, 2025, none of our directors or Section 16 reporting officers adopted or terminated any Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement (as such terms are defined in Item 408 of the SEC's Regulation S-K).

Amendment to Bylaws

On March 4, 2026, the Board of Directors amended and restated the Company’s bylaws (as so amended and restated, the Amended and Restated Bylaws), effective immediately.

The Amended and Restated Bylaws modify the Company’s prior bylaws to (i) revise the procedures and disclosure requirements governing stockholder nominations of director candidates, including to require compliance with Rule 14a-19 under the Exchange Act, (ii) update procedures and rules relating to stockholder meetings, (iii) make certain changes to conform to recent amendments to the Delaware General Corporation Law and (iv) make certain other ministerial and conforming changes.

The foregoing summary is not, nor is it intended to be, a complete or comprehensive summary of all of the changes reflected in Amended and Restated Bylaws.

The foregoing summary is qualified in its entirety by the full text of the Amended and Restated Bylaws, a copy of which is filed as Exhibit 3.2 hereto and incorporated herein by reference.

DISCLOSURE REGARDING FOREIGN JURISDICTIONS THAT PREVENT INSPECTIONS

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To the Board of Directors and Stockholders of REGENXBIO Inc.

Opinions on the Financial Statements and Internal Control over Financial Reporting

Opinion on the Financial Statements

We have audited the accompanying consolidated balance sheets of REGENXBIO Inc.

and its subsidiaries (the “Company”) as of December 31, 2024 and 2023, and the related consolidated statements of operations and comprehensive loss, of stockholders’ equity and of cash flows for each of the three years in the period ended December 31, 2024, including the related notes (collectively referred to as the “consolidated financial statements”).

We also have audited the Company's internal control over financial reporting as of December 31, 2024, based on criteria established in Internal Control - Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission (COSO).

In our opinion, the consolidated financial statements referred to above present fairly, in all material respects, the financial position of the Company as of December 31, 2024 and 2023, and the results of its operations and its cash flows for each of the three years in the period ended December 31, 2024 in conformity with accounting principles generally accepted in the United States of America.

Also in our opinion, the Company maintained, in all material respects, effective internal control over financial reporting as of December 31, 2024, based on criteria established in Internal Control - Integrated Framework (2013) issued by the COSO.

Basis for Opinions

The Company's management is responsible for these consolidated financial statements, for maintaining effective internal control over financial reporting, and for its assessment of the effectiveness of internal control over financial reporting, included in Management’s Report on Internal Control over Financial Reporting appearing under Item 9A.

Our responsibility is to express opinions on the Company’s consolidated financial statements and on the Company's internal control over financial reporting based on our audits.

and its subsidiaries (the "Company") as of December 31, 2025 and 2024, and the related consolidated statements of operations and comprehensive loss, of stockholders’ equity and of cash flows for the years then ended, including the related notes (collectively referred to as the "consolidated financial statements").

In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company as of December 31, 2025 and 2024, and the results of its operations and its cash flows for the years then ended in conformity with accounting principles generally accepted in the United States of America.

Substantial Doubt about the Company's Ability to Continue as a Going Concern

The accompanying consolidated financial statements have been prepared assuming that the Company will continue as a going concern.

As discussed in Note 1 to the consolidated financial statements, the Company has incurred cumulative losses from operations since inception, an accumulated deficit of $1.13 billion and cash outflows from operating activities that raise substantial doubt about its ability to continue as a going concern.

Management's plans in regard to these matters are also described in Note 1.

The consolidated financial statements do not include any adjustments that might result from the outcome of this uncertainty.

Basis for Opinion

These consolidated financial statements are the responsibility of the Company’s management.

Our responsibility is to express an opinion on the Company’s consolidated financial statements based on our audits.

We are a public accounting firm registered with the Public Company Accounting Oversight Board (United States) (PCAOB) and are required to be independent with respect to the Company in accordance with the U.S.

federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.

We conducted our audits in accordance with the standards of the PCAOB.

Those standards require that we plan and perform the audits to obtain reasonable assurance about whether the consolidated financial statements are free of material misstatement, whether due to error or fraud, and whether effective internal control over financial reporting was maintained in all material respects.

Our audits of the consolidated financial statements included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud, and performing procedures that respond to those risks.

We conducted our audits of these consolidated financial statements in accordance with the standards of the PCAOB.

The Company is not required to have, nor were we engaged to perform, an audit of its internal control over financial reporting.

As part of our audits we are required to obtain an understanding of internal control over financial reporting but not for the purpose of expressing an opinion on the effectiveness of the Company's internal control over financial reporting.

Accordingly, we express no such opinion.

Our audits included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud, and performing procedures that respond to those risks.

Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the consolidated financial statements.

Our audits also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the consolidated financial statements.

Our audit of internal control over financial reporting included obtaining an understanding of internal control over financial reporting, assessing the risk that a material weakness exists, and testing and evaluating the design and operating effectiveness of internal control based on the assessed risk.

Our audits also included performing such other procedures as we considered necessary in the circumstances.

We believe that our audits provide a reasonable basis for our opinions.

Definition and Limitations of Internal Control over Financial Reporting

A company’s internal control over financial reporting is a process designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles.

A company’s internal control over financial reporting includes those policies and procedures that (i) pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the company;

(ii) provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the company are being made only in accordance with authorizations of management and directors of the company;

and (iii) provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or disposition of the company’s assets that could have a material effect on the financial statements.

Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements.

Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.

We believe that our audits provide a reasonable basis for our opinion.

Critical Audit Matters

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The communication of critical audit matters does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates.

Revenue Recognition – Royalty Revenue

As described in Notes 2 and 10 to the consolidated financial statements, the Company recognizes royalty revenue on sales of licensed products.

The Company’s consolidated royalty revenue was $81.5 million for the year ended December 31, 2024.

The principal consideration for our determination that performing procedures relating to royalty revenue recognition is a critical audit matter is a high degree of auditor effort in performing procedures related to the Company’s revenue recognition.

Revenue Recognition – Determination of the Standalone Selling Price of the RGX-121 Intellectual Property License for the United States Under the Nippon Shinyaku Collaboration Agreement

As described in Notes 2 and 10 to the consolidated financial statements, the Company’s license agreements are accounted for as contracts with customers within the scope of ASC 606, with the exception of transactions for which the counterparty is determined not to be a customer.

In January 2025, the Company entered into a collaboration and license agreement with Nippon Shinyaku Co., Ltd.

(Nippon Shinyaku) for the development and commercialization of RGX-121, the Company’s product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II), and RGX-111, the Company's product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I) (the Nippon Shinyaku Collaboration Agreement).

Management identified the following performance obligations under the agreement:

(i) delivery of intellectual property licenses to develop and commercialize RGX-121 and RGX-111 in the United States and Asia territories, (ii) development services for RGX-121 and RGX-111 in the United States, including manufacturing of clinical supply and commercial supply prior to regulatory approval, and (iii) material rights granted to Nippon Shinyaku to purchase commercial supply for sales in licensed territories.

The selling prices of intellectual property licenses were determined based on discounted cash flow models for each of the licensed products in the respective licensed territories and were adjusted for the probability of developmental, regulatory and commercial success.

Significant assumptions and judgments were required to estimate the future cash flows, including the addressable market, sales price per unit, discount rates and probabilities of success for each of the licensed products and territories.

For the year ended December 31, 2025, the Company recognized total revenues of $84.7 million related to the Nippon Shinyaku Collaboration Agreement, of which a majority related to the RGX-121 intellectual property license for the United States.

The principal considerations for our determination that performing procedures relating to the determination of the standalone selling price of the RGX-121 intellectual property license for the United States under the Nippon Shinyaku Collaboration Agreement is a critical audit matter are (i) the significant judgment by management when determining the standalone selling price of the RGX-121 intellectual property license for the United States;

(ii) a high degree of auditor judgment, subjectivity, and effort in performing procedures and evaluating management’s significant assumptions related to the addressable market, sales price per unit, discount rate and probability of success;

and (iii) the audit effort involved the use of professionals with specialized skill and knowledge.

Addressing the matter involved performing procedures and evaluating audit evidence in connection with forming our overall opinion on the consolidated financial statements.

These procedures included testing the effectiveness of controls over the revenue recognition process.

These procedures also included, among others, (i) obtaining and inspecting third party royalty reports and the related cash settlement, where applicable, (ii) testing the completeness and accuracy of data provided by management, and (iii) evaluating the financial statement presentation and related disclosures.

These procedures included, among others (i) reading the contractual terms of the Nippon Shinyaku Collaboration Agreement;

(ii) testing management’s process for determining the standalone selling price of the RGX-121 intellectual property license for the United States;

(iii) evaluating the appropriateness of the discounted cash flow model;

and (iv) evaluating the reasonableness of the significant assumptions used by management related to the addressable market, sales price per unit, discount rate and probability of success.

Evaluating the reasonableness of management’s assumptions related to the addressable market, sales price per unit, and the probability of success involved considering (i) the consistency with external market and industry data and (ii) whether the assumptions were consistent with evidence obtained in other areas of the audit.

Professionals with specialized skill and knowledge were used to assist in evaluating (i) the appropriateness of the discounted cash flow model and (ii) the reasonableness of the discount rate assumption.

/s/ PricewaterhouseCoopers LLP

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Marketable securities

Accounts receivable (net of allowance of $ 4,587 as of December 31, 2023)

Accounts receivable

Prepaid expenses

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Operating lease liabilities

Liability related to sale of future royalties

Royalty monetization liabilities

Total current liabilities

Deferred revenue

Operating lease liabilities

Liability related to sale of future royalties

Royalty monetization liabilities

Other liabilities

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License and royalty revenue

Service revenue

Total revenues

Operating Expenses

Cost of revenues

Cost of license and royalty revenues

Research and development

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Impairment of long-lived assets

Other operating expenses (income)

Other operating expenses

Total operating expenses

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Total other income (expense)

Loss before income taxes

Income Tax Benefit

Other Comprehensive Income (Loss)

Unrealized gain (loss) on available-for-sale securities, net

Total other comprehensive income (loss)

Other Comprehensive Income

Unrealized gain on available-for-sale securities, net

Total other comprehensive income

Comprehensive loss

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stock purchase plan

Stock-based compensation expense

Unrealized loss on available-for-sale securities, net

Balances at December 31, 2022

Vesting of restricted stock units, net of tax

Exercise of stock options, net of tax

Issuance of common stock under employee

stock purchase plan

Issuance of common stock upon private placement,

net of transaction costs of $ 126

Issuance of common stock and pre-funded warrants

upon public offering, net of transaction

costs of $ 534

Exercise of pre-funded warrants

Stock-based compensation expense

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stock purchase plan

Issuance of common stock and pre-funded warrants

upon public offering, net of transaction

costs of $ 534

Issuance of warrants, net of transaction costs

Exercise of pre-funded warrants

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Provision for credit losses (recoveries)

Net amortization of premiums (accretion of discounts) on marketable debt securities

Net realized loss (gain) on investments

Net accretion of discounts on marketable debt securities

Net realized gain on investments

Impairment of long-lived assets

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net of issuance costs

Proceeds from private placement of common stock, net of issuance costs

Offering expenses related to at-the-market offering programs

Repayments under liability related to sale of future royalties, net of imputed interest

Net cash provided by (used in) financing activities

Expenses related to at-the-market offering programs

Proceeds from issuance of royalty bond and warrants, net of transaction costs

Repayments under royalty monetization liabilities, net of interest

Net cash provided by financing activities

Net increase (decrease) in cash and cash equivalents and restricted cash

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Cash paid (received) for income taxes

Cash paid for imputed interest under liability related to sale of future royalties

Cash paid for interest under royalty monetization liabilities

Supplemental disclosures of non-cash investing and financing information

Purchases of property and equipment in accounts payable and accrued expenses

and other current liabilities

Deferred equity offering costs in accounts payable and accrued expenses

and other current liabilities

Deferred equity offering costs reclassified

The accompanying notes are an integral part of these consolidated financial statements.

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The Company's investigational gene therapies use adeno-associated virus (AAV) vectors from its proprietary gene delivery platform (NAV Technology Platform).

The NAV ® Technology Platform consists of exclusive rights to a large portfolio of proprietary AAV vectors.

The NAV ® Technology Platform has consisted of exclusive rights to a large portfolio of proprietary AAV vectors.

The Company has developed a broad pipeline of gene therapy product candidates using the NAV Technology Platform as a one-time treatment to address an array of diseases.

In addition to its internal product development efforts, the Company also selectively licenses the NAV Technology Platform and other intellectual property rights to other leading biotechnology and pharmaceutical companies (NAV Technology Licensees).

As of December 31, 2024, the NAV Technology Platform was being applied by NAV Technology Licensees in one commercial product, Zolgensma ® , and in the preclinical and clinical development of a number of other licensed products.

The Company's lead product candidates include ABBV-RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR), RGX-202 for the treatment of Duchenne muscular dystrophy, RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II) and RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I).

In addition to its internal product development efforts, the Company has also selectively licensed the NAV Technology Platform and other intellectual property rights to other leading biotechnology and pharmaceutical companies (NAV Technology Licensees).

As of December 31, 2025, the NAV Technology Platform was being applied by NAV Technology Licensees in two commercial products, Zolgensma ® and Itvisma ® , and in the preclinical and clinical development of various other licensed products.

Additionally, the Company has licensed intellectual property rights to collaborators for the joint development and commercialization of certain product candidates.

The Company was formed in 2008 in the State of Delaware and is headquartered in Rockville, Maryland.

In January 2025, the Company and Nippon Shinyaku Co., Ltd.

(Nippon Shinyaku) entered into a collaboration and license agreement (the Nippon Shinyaku Collaboration Agreement) for the joint development and commercialization of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II) and RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I).

Pursuant the Nippon Shinyaku Collaboration Agreement, the Company will receive an up-front payment of $ 110.0 million following the effective date of the agreement and is eligible to receive up to $ 700.0 million upon the achievement of specified development and sales-based milestones.

The Nippon Shinyaku Collaboration Agreement became effective in March 2025.

Please refer to Note 10 for further information on the Nippon Shinyaku Collaboration Agreement.

The Company has incurred cumulative losses since inception and as of December 31, 2024, had generated an accumulated deficit of $ 932.1 million.

The Company has incurred cumulative losses since inception and as of December 31, 2025, had generated an accumulated deficit of $ 1.13 billion.

The Company's ability to transition to recurring profitability is dependent upon achieving a level of revenues adequate to support its cost structure, which depends heavily on the successful development, approval and commercialization of its product candidates.

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There is no assurance that the Company will be able to raise sufficient capital or obtain financing on favorable terms, or at all.

The Company’s ability to continue as a going concern may be impacted by the development and potential commercialization of its product candidates and its ability to raise additional capital to fund its operations.

As of December 31, 2025, the Company had cash, cash equivalents and marketable securities of $ 240.9 million, which management believes is sufficient to fund operations into early 2027.

This estimate is based on the Company's current operating plan, and excludes the potential effects of any future financings or material milestone payments that may be received under the Company's licensing and collaboration arrangements.

The Company has based this estimate on assumptions that may prove to be wrong, and it could exhaust its capital resources sooner than expected.

These conditions raise substantial doubt about the Company's ability to continue as a going concern within 12 months from the date these consolidated financial statements were issued.

The Company’s ability to continue as a going concern will depend heavily on the successful development, approval and commercialization of its product candidates and its ability to raise additional capital to fund its operations.

If the Company is unable to raise capital sufficient to meet its working capital needs in the future, it may be forced to delay expenditures, reduce the scope of its development activities or make other changes to its operating plans.

As of December 31, 2024, the Company had cash, cash equivalents and marketable securities of $ 244.9 million.

Management believes that its cash, cash equivalents and marketable securities as of December 31, 2024, along with the up-front payment of $ 110.0 million expected to be received under the Nippon Shinyaku Collaboration Agreement, is sufficient to fund operations for at least the next 12 months from the date these consolidated financial statements were issued.

Summary of Significant Accounting Policies

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dollar are included in results of operations as incurred.

During the years ended December 31, 2024, 2023 and 2022 , the Company recorded aggregate net foreign currency transaction losses of $ 0.9 million , $ 0.3 million and $ 0.7 million, respectively, which are included in other operating expenses (income) in the consolidated statements of operations and comprehensive loss.

During the years ended December 31, 2025 and 2024 , the Company recorded aggregate net foreign currency transaction losses of $ 0.1 million and $ 0.9 million, respectively, which are included in other operating expenses in the consolidated statements of operations and comprehensive loss.

Use of Estimates

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Estimates are used in the following areas, among others:

license and royalty revenue, the allowance for credit losses, accrued research and development expenses and other accrued liabilities, stock-based compensation expense, interest expense under the liability related to the sale of future royalties, income taxes and fair value measurements.

revenue recognition, the allowance for credit losses, accrued research and development expenses and other accrued liabilities, stock-based compensation expense, interest expense under royalty monetization liabilities, income taxes and fair value measurements.

Reclassifications

Certain amounts reported in prior periods have been reclassified to conform to current period financial statement presentation, including separate presentation of service revenue in the statements of operations and comprehensive loss.

As a result of the Company's collaboration and license agreement with Nippon Shinyaku Co., Ltd.

(Nippon Shinyaku) which became effective in March 2025, the Company has modified the presentation of its revenues and now presents service revenues separately from license and royalty revenues.

The modified presentation has been applied retrospectively to all prior periods presented.

The reclassifications have no effect on previously reported financial position, results of operations and cash flows.

Cash and Cash Equivalents

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Marketable debt securities with remaining maturity dates exceeding 12 months which are not intended to be sold prior to maturity for use in current operations are classified as non-current assets.

Unrealized gains and losses on available-for-sale debt securities, net of any related tax effects, are excluded from results of operations and are included in other comprehensive income (loss) and reported as a separate component of stockholders’ equity until realized.

Unrealized gains and losses on available-for-sale debt securities, net of any related tax effects, are excluded from results of operations and are included in other comprehensive income and reported as a separate component of stockholders’ equity until realized.

The Company uses the aggregate portfolio approach to release the tax effects of unrealized gains and losses on available-for-sale debt securities in accumulated other comprehensive loss.

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If a credit loss is deemed to exist, the credit loss is recognized in results of operations and an allowance for credit losses is recorded against the amortized cost basis of the security.

In determining whether a credit loss exists related to impaired available-for-sale debt securities, the Company considers, among other factors, the extent of the unrealized loss relative to the amortized cost basis, the credit rating of the issuer and any recent changes thereto, current and expected future economic conditions, and any adverse events or other changes in circumstances that have occurred which may indicate a potential credit loss.

economic conditions, and any adverse events or other changes in circumstances that have occurred which may indicate a potential credit loss.

The Company did not record an allowance for credit losses on its available-for-sale debt securities as of December 31, 2025 or 2024.

Accounts Receivable

Accounts receivable primarily consist of consideration due to the Company resulting from its license agreements with customers.

Accounts receivable include amounts invoiced to licensees as well as rights to consideration which have not yet been invoiced, including unbilled royalties, and for which payment is conditional solely upon the passage of time.

If a licensee elects to terminate a license prior to the end of the license term, the licensed intellectual property is returned to the Company and any accounts

receivable from the licensee which are not contractually payable to the Company are charged off as a reduction of license revenue in the period of the termination.

Accounts receivable consist of consideration due to the Company resulting from its agreements with customers.

Accounts receivable include amounts invoiced to customers as well as rights to consideration which have not yet been invoiced, including unbilled royalties and services, and for which payment is conditional solely upon the passage of time.

If a licensee elects to terminate a license prior to the end of the license term, the licensed intellectual property is returned to the Company and any accounts receivable from the licensee which are not contractually payable to the Company are charged off as a reduction of revenue in the period of the termination.

Accounts receivable which are not expected to be received by the Company within 12 months from the reporting date are stated net of a discount to present value and recorded as non-current assets on the consolidated balance sheets.

The present value discount is recognized as a reduction of revenue in the period in which the accounts receivable are initially recorded and is accreted as interest income from licensing over the term of the receivables.

The present value discount is recognized as a reduction of revenue in the period in which the accounts receivable balance is initially recorded and is accreted as interest income from licensing over the term of the receivables.

Accounts receivable are stated net of an allowance for credit losses, if deemed necessary based on the Company’s evaluation of collectability and potential credit losses.

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Accounts receivable balances are written off against the allowance for credit losses when the potential for collectability is considered remote.

Please refer to Note 10 for further information regarding the allowance for credit losses related to accounts receivable.

The Company did not record an allowance for credit losses on its accounts receivable as of December 31, 2025 or 2024.

Concentrations of Credit Risk and Off-balance Sheet Risk

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The following table summarizes those customers who represented at least 10% of revenues or total net accounts receivable for the periods presented:

Accounts Receivable, Net

Accounts Receivable

Years Ended December 31,

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Capitalized implementation costs for cloud-based applications and associated amortization are classified on the consolidated balance sheets and statements of operations and comprehensive loss in the same manner as the costs of the associated hosting arrangement.

As of December 31, 2024 and 2023, the Company had recorded capitalized costs, net of amounts amortized, of less than $ 0.1 million and $ 1.0 million, respectively, related to the implementation of cloud-based software applications, which were included in prepaid expenses and other assets on the consolidated balance sheets.

Amortization of capitalized implementation costs for cloud-based applications recorded for the years ended December 31, 2024, 2023 and 2022 was $ 1.0 million, $ 1.4 million and $ 1.3 million, respectively, and was included in general and administrative expenses in the consolidated statements of operations and comprehensive loss.

As of December 31, 2025 and 2024, the Company had recorded capitalized costs, net of amounts amortized, of $ 0.1 million and less than $ 0.1 million, respectively, related to the implementation of cloud-based software applications, which were included in prepaid expenses and other assets on the consolidated balance sheets.

Amortization of capitalized implementation costs for cloud-based applications recorded for the years ended December 31, 2025 and 2024 was less than $ 0.1 million and $ 1.0 million, respectively, and was included in general and administrative expenses in the consolidated statements of operations and comprehensive loss.

Impairment of Long-lived Assets

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Please refer to Note 4 for further information on the Company's fair value measurements.

Liability Related to Sale of Future Royalties

As discussed in Note 7, the Company recorded a liability for the net proceeds received from the sale of its Zolgensma royalty payments to entities managed by Healthcare Royalty Management, LLC (collectively, HCR).

The liability is accounted for as debt since the return to HCR is explicitly capped under the royalty purchase agreement, and is amortized over the estimated life of the arrangement using the effective interest method.

The total amount of royalty payments received by HCR under the agreement, less the net proceeds received by the Company, is recorded as interest expense over the life of the arrangement.

The Company estimates the effective interest rate based on its estimate of total royalty payments to be received by HCR under the agreement.

The Company reassesses these estimates at each reporting date and adjusts the effective interest rate and amortization of the liability on a prospective basis as necessary.

Due to its continuing involvement in the underlying license agreement with Novartis Gene Therapies, Inc.

(formerly AveXis, Inc.) (Novartis Gene Therapies), the Company continues to recognize royalty revenue on net sales of Zolgensma and records the royalty payments to HCR as a reduction of the liability when paid.

As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the royalty purchase agreement.

The portion of the liability related to the sale of future royalties which is expected to be amortized within 12 months of the reporting date is recorded as a current liability, with the remaining portion of the liability recorded as a non-current liability.

Pre-funded Warrants

Royalty Monetization Liabilities

As discussed in Note 7, the Company has entered into multiple royalty monetization arrangements with entities managed by Healthcare Royalty Management, LLC (collectively and with other affiliated entities, HCR).

Net proceeds received by the Company under these arrangements are recorded as liabilities and accounted for as debt since the return to HCR is explicitly capped under the agreements.

The liabilities are amortized over the estimated life of the arrangements using the effective interest method.

For arrangements in which there is no stated interest rate, the total amount of royalty and other payments paid to HCR under the arrangement, less the net proceeds received by the Company, is recorded as interest expense over the life of the arrangement.

The Company estimates the effective interest rates of its royalty monetization liabilities based on its estimate of total payments to be paid to HCR under the arrangements.

The Company reassesses these estimates at each reporting date and adjusts the effective interest rate and amortization of the liabilities on a prospective basis as necessary.

Due to its continuing involvement in the license and collaboration agreements underlying the royalty monetization arrangements with HCR, the Company continues to recognize license and royalty revenues under these license and collaboration agreements and records any payments to HCR as a reduction of the associated royalty monetization liability when paid.

As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the royalty monetization agreement.

The portion of royalty monetization liabilities representing principal that is expected to be paid within 12 months of the reporting date is recorded as a current liability, with the remaining portion of the liabilities recorded as non-current.

Warrants are accounted for based on the specific terms of the warrant agreements.

The Company's pre-funded warrants are indexed to the Company's common stock and meet the criteria to be classified as equity.

Proceeds from the issuance of pre-funded warrants are recorded within additional paid-in capital and are not subject to remeasurement.

Please refer to Note 9 for further information regarding pre-funded warrants issued by the Company.

The Company's warrants, including pre-funded warrants, are indexed to the Company's common stock and meet the criteria to be classified as equity.

Net proceeds received from the issuance of warrants are recorded within additional paid-in capital and are not subject to remeasurement.

Please refer to Note 9 for further information regarding warrants issued by the Company.

Revenue Recognition

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The following five steps

are performed to determine the appropriate revenue recognition for arrangements within the scope of ASC 606:

The following five steps are performed to determine the appropriate revenue recognition for arrangements within the scope of ASC 606:

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In determining the appropriate interest rates for significant financing components, the Company evaluates the credit profile of the customer and prevailing market interest rates and selects an interest rate in which it believes would be charged to the customer in a separate financing arrangement over a similar financing term.

License and Royalty Revenue

The Company licenses its NAV Technology Platform and other intellectual property rights to other biotechnology and pharmaceutical companies, including collaborators for the joint development and commercialization of its product candidates.

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License agreements generally have a term at least equal to the life of the underlying patents, but are terminable at the option of the licensee.

Consideration payable to the Company under its license agreements may include:

The Company’s license agreements are accounted for as contracts with customers within the scope of ASC 606, with the exception of transactions for which the counterparty is determined not to be a customer.

At the inception of each license agreement, the Company determines the contract term for purposes of applying the requirements of ASC 606.

Consideration payable to the Company under its license and collaboration agreements may include:

(i) up-front and annual fees, (ii) milestone payments based on the achievement of certain development and sales-based milestones, (iii) sublicense fees, (iv) royalties on sales of licensed products, (v) fees for services related to the development and manufacturing of licensed products and (vi) other consideration payable upon optional goods and services purchased by licensees and collaborators.

The Company evaluates its agreements with collaboration partners to determine whether they are within the scope of ASC 808, Collaborative Arrangements (ASC 808).

For collaboration arrangements within the scope of ASC 808 that contain multiple elements, the Company identifies the various transactions with the counterparty and determines if any unit of account is more reflective of a transaction with a customer and therefore should be accounted for within the scope of ASC 606.

For transactions that are accounted for pursuant to ASC 606, the Company applies the five-step model as described in its revenue recognition policies.

For transactions that are accounted for pursuant to ASC 808, an appropriate method of recognition and presentation is determined and consistently applied in accordance with the Company's accounting policies for collaborative arrangements.

The Company’s license and collaboration agreements are accounted for as contracts with customers within the scope of ASC 606, with the exception of transactions for which the counterparty is determined not to be a customer.

At the inception of each agreement, the Company determines the contract term for purposes of applying the requirements of ASC 606.

Licenses are generally terminable at the option of the licensee with advance notice to the Company.

For each license granted, including licenses granted upon the exercise of license options, the Company evaluates these termination rights to determine whether a substantive termination penalty would be incurred by the licensee upon termination.

For each license granted, the Company evaluates these termination rights to determine whether a substantive termination penalty would be incurred by the licensee upon termination.

The determination of whether a substantive termination penalty is associated with the termination rights requires significant judgment.

In making this determination, the Company considers, among other things, the nature of the intellectual property rights that would be returned to the Company upon termination, including the exclusivity of the licensed rights and the stage of development of the licensed products, the payment terms, including the amount and timing of non-refundable or guaranteed payments, and the business purpose of the termination rights granted to the licensee.

The Company considers all of the facts and circumstances relevant to each license when making this determination.

Performance obligations under the Company’s license agreements may include (i) the delivery of intellectual property licenses, (ii) options granted to licensees to acquire additional licenses, to the extent the options represent material rights to the licensee, and (iii) research and development services to be performed by the Company related to licensed products.

License agreements may provide licensees with contract renewal options or options to acquire additional licenses, goods or other services.

Options are evaluated at the inception of the license agreement to determine whether they provide material rights to the licensee.

In making this determination, the Company considers whether the options are priced at an incremental discount to the standalone selling price for the underlying licenses, goods or services, in which case the option is considered to be a material right to the licensee and is accounted for as a separate performance obligation under the current license agreement.

At the inception of each license agreement which contains

performance obligations for research and development services, the Company evaluates whether the license is distinct from the research and development services, which requires judgment.

In making this determination, the Company considers, among other things, the stage of development of the licensed products and whether the research and development services will significantly impact further development of the licensed products.

If it is determined that the license is not distinct from the research and development services, the license is combined with the research and development services into a single performance obligation.

The Company evaluates the transaction price of its license agreements at the inception of each agreement and at each reporting date.

The transaction price includes the fixed consideration payable to the Company during the contract term, as well as any variable consideration to the extent that it is probable that a significant reversal of revenue will not occur in the future.

Fixed consideration under the license agreements may include up-front and annual fees payable during the contract term and fees for development services performed by the Company.

Variable consideration under the license agreements may include development and sales-based milestone payments, sublicense fees and royalties on sales of licensed products.

Consideration contingent upon the exercise of options by a licensee is excluded from the transaction price and not accounted for as part of the license agreement until the option is exercised.

Consideration allocated to performance obligations for the delivery of an intellectual property license is recognized as revenue in full upon the delivery of the license to the licensee.

Consideration allocated to performance obligations for license options is recognized as revenue in full upon the earlier of the option exercise or expiration.

The exercise of a license option by a licensee is accounted for as a new license for revenue recognition purposes.

Consideration allocated to performance obligations for research and development services is recognized as revenue as the services are performed by the Company.

Up-front and annual licenses fees payable to the Company over the contract term of each license are included in the transaction price, and the portion of this consideration allocated to the performance obligation for the delivery of the intellectual property license is recognized as revenue in full upon the delivery of the license to the licensee.

If annual license fees are payable to the Company in periods beyond 12 months from the delivery of the license, a significant financing component is deemed to exist which provides a financing benefit to the licensee.

If a significant financing component is identified, the Company adjusts the transaction price for the license to include only the present value of the annual license fees payable to the Company over the contract term.

The discounted portion of the license fees is recognized as interest income from licensing over the financing period of the license.

Performance obligations under the Company’s license and collaboration agreements may include (i) the delivery of intellectual property licenses, (ii) development and manufacturing services to be performed by the Company related to licensed products and (iii) options granted to purchase additional goods and services, to the extent the options convey material rights.

At the inception of each license agreement which contains performance obligations for development, manufacturing or other services, the Company evaluates whether the license is distinct from the services, which requires judgment.

In making this determination, the Company considers, among other things, the stage of development of the licensed products and whether the services will significantly impact further development of the licensed products.

If it is determined that the license is not distinct from the services, the license is combined with the services into a single performance obligation.

Agreements may provide licensees and collaborators with options to purchase additional goods or other services, including options to purchase commercial supply of licensed products.

Options are evaluated at the inception of the agreement to determine whether they provide material rights to the customer.

In making this determination, the Company considers whether the options are priced at an incremental discount to the standalone selling price of the underlying goods or services, in which case the option is considered to be a material right.

Material rights are accounted for as separate performance obligations under the current arrangement.

The Company evaluates the transaction price of its license and collaboration agreements at contract inception and at each reporting date.

The transaction price includes the fixed consideration payable to the Company over the contract term, as well as any variable consideration to the extent that it is probable that a significant reversal of revenue will not occur in the future.

Fixed consideration under the agreements may include up-front and annual fees payable over the contract term and fixed fees for development, manufacturing and other services performed by the Company.

Variable consideration under the agreements may include development and sales-based milestone payments, payments for development, manufacturing and other services performed by the Company, sublicense fees and royalties on sales of licensed products.

Consideration contingent upon the exercise of options by the customer is excluded from the transaction price and not accounted for as part of the arrangement until the option is exercised.

The transaction price of the Company's license and collaboration arrangements is allocated to the underlying performance obligations based on their relative standalone selling prices and recognized as revenue when (or as) the performance obligations are satisfied.

Variable consideration payable based on services performed by the Company is allocated directly to the performance obligation for such services.

Consideration allocated to performance obligations for the delivery of intellectual property licenses is recognized as license and royalty revenue in full upon the delivery of the license.

Consideration allocated to performance obligations for development, manufacturing and other services is recognized as service revenue as the services are performed by the Company.

Service revenue is recognized using a measure of progress that best reflects the pattern of satisfaction of the performance obligations.

At each reporting date, the Company re-evaluates the measure of progress and adjusts service revenue on a cumulative catch-up basis to reflect its best estimate of the services performed to date versus the total services to be performed under the arrangement.

Development milestone payments are payable to the Company upon the achievement of specified development milestones.

At the inception of each license agreement that contains development milestone payments, the Company evaluates whether the milestones are considered probable of achievement and estimates the amount to be included in the transaction price using the most likely amount method.

At the inception of each license agreement that contains development milestone payments, the Company evaluates whether the milestones are probable of achievement and estimates the amount to be included in the transaction price using the most likely amount method.

If it is probable that a significant revenue reversal will not occur in the future, milestone payments are included in the transaction price.

Milestone payments contingent on the achievement of development milestones that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved and are excluded from the transaction price until the milestone is achieved.

At each reporting date, the Company re-evaluates the probability of achievement of each outstanding development milestone and, if necessary, adjusts the transaction price for any milestones for which the probability of achievement has changed due to current facts and circumstances.

Any such adjustments are recorded on a cumulative catch-up basis and recognized as revenue in the period of the adjustment.

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma, which is a licensed product under the Company’s license agreement with Novartis Gene Therapies, a wholly owned subsidiary of Novartis AG (Novartis), for the development and commercialization of treatments for spinal muscular atrophy (SMA).

The Company recognizes royalty revenue from net sales of Zolgensma in the period in which the underlying products are sold by Novartis Gene Therapies, which in certain cases may require the Company to estimate royalty revenue for periods of net sales which have not yet been reported to the Company.

Royalties on sales of licensed products, sales-based milestone payments, including milestones payable upon first commercial sales of licensed products, and sublicense fees based on the receipt of certain fees by licensees from any sublicensees are excluded from the transaction price of each license and recognized as license and royalty revenue in the period that the related sales or sublicenses occur, provided that the associated license has been delivered to the licensee.

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma and Itvisma, which are licensed products under the Company’s license agreement with Novartis Gene Therapies, Inc.

(Novartis Gene Therapies), a wholly owned subsidiary of Novartis AG (Novartis), for the development and commercialization of treatments for spinal muscular atrophy (SMA).

The Company recognizes royalty revenue from net sales of Zolgensma and Itvisma in the period in which the underlying products are sold by Novartis Gene Therapies, which in certain cases may require the Company to estimate royalty revenue for periods of net sales which have not yet been reported to the Company.

Estimated royalties are reconciled to actual amounts reported in subsequent periods, and any differences are recognized as an adjustment to royalty revenue in the period the royalties are reported.

The Company receives payments from licensees based on the billing schedules established in each license agreement.

Amounts recognized as revenue which have not yet been received from licensees, including unbilled royalties, are recorded as accounts receivable when the Company’s rights to the consideration are conditional solely upon the passage of time.

Amounts recognized as revenue which have not yet been received from licensees are recorded as contract assets when the Company’s rights to the

consideration are not unconditional.

The Company receives payments from licensees and collaborators based on the billing schedules established in the associated agreements.

Amounts recognized as revenue which have not yet been received from the customer are recorded as accounts receivable when the Company’s rights to the consideration are conditional solely upon the passage of time.

Amounts recognized as revenue which have not yet been received from customers are recorded as contract assets when the Company’s rights to the consideration are not unconditional.

If a licensee elects to terminate a license prior to the end of the license term, the licensed intellectual property is returned to the Company and any consideration recorded as accounts receivable or contract assets which is not contractually payable by the licensee is charged off as a reduction of license revenue in the period of the termination.

Amounts received by the Company prior to the delivery of underlying performance obligations are deferred and recognized as revenue upon the satisfaction of the performance obligations by the Company.

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Collaborative Arrangements

The Company evaluates its agreements with collaboration partners to determine whether they are within the scope of ASC 808, Collaborative Arrangements (ASC 808).

The Company evaluates its agreements with collaboration partners to determine whether they are within the scope of ASC 808.

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For transactions that are accounted for pursuant to ASC 808, an appropriate method of recognition and presentation is determined and consistently applied.

For transactions that are accounted for pursuant to ASC 606, the Company applies the five-step model as described in its revenue recognition policies.

For transactions that are accounted for pursuant to ASC 808, an appropriate method of recognition and presentation is determined and consistently applied.

For transactions accounted for as collaborative arrangements under ASC 808, payments to and from collaboration partners associated with multiple activities in a collaboration arrangement are classified based on the nature of each separate activity.

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At the end of each reporting period, the Company records a net amount due to or from collaboration partners for activities performed by the parties under the collaboration.

Cost of Revenues

Cost of revenues consists primarily of sublicense fees and royalties on net sales of licensed products as specified in the Company’s agreements with its licensors.

Cost of License and Royalty Revenues

Cost of license and royalty revenues consists primarily of sublicense fees and royalties on net sales of licensed products as specified in the Company’s agreements with its licensors.

Sublicense fees are based on a percentage of license fees received by the Company from licensees and are recognized in the period that the underlying revenue is recognized.

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Advance payments for goods or services related to research and development activities are deferred and expensed as the goods are delivered or the services are performed.

Research and development costs include salaries, wages, benefits and other personnel costs, laboratory and facilities costs, allocated overhead costs, license and milestone fees, and costs of goods and services associated with preclinical research and clinical trial activities, associated manufacturing-related activities, regulatory activities and other related services performed by third parties.

Research and development costs include salaries, wages, benefits and other personnel-related costs, laboratory and facilities costs, allocated overhead costs, license and milestone fees, and costs of goods and services associated with preclinical research and clinical trial activities, associated manufacturing-related activities, regulatory activities and other related services performed by third parties.

At the end of each reporting period, the Company compares payments made to third-party service providers to the estimated expenses incurred based on the services provided and progress toward completion of the research or development objectives.

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The Company accounts for its stock-based compensation awards in accordance with ASC 718, Compensation—Stock Compensation (ASC 718).

ASC 718 requires all stock-based awards to employees and nonemployees to be recognized as expense

based on the grant date fair value of the awards.

ASC 718 requires all stock-based awards to employees and nonemployees to be recognized as expense based on the grant date fair value of the awards.

The Company’s stock-based awards include stock options and restricted stock units granted to employees and nonemployees and shares issued to employees under its employee stock purchase plan.

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Diluted net loss per share is calculated by adjusting the weighted-average common shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury-stock method.

For purposes of computing both basic and diluted net loss per share, pre-funded

warrants are considered outstanding shares upon issuance because the underlying shares may be issued for nominal consideration and are exercisable after the original issuance date.

For purposes of computing both basic and diluted net loss per share, pre-funded warrants are considered outstanding shares upon issuance because the underlying shares may be issued for nominal consideration and are exercisable after the original issuance date.

Contingently convertible shares in which conversion is based on non-market-priced contingencies are excluded from the calculations of both basic and diluted net loss per share until the contingency has been fully met.

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The Company adopted this standard for annual reporting periods beginning January 1, 2024 and interim reporting periods beginning January 1, 2025, on a retrospective basis for all periods presented.

Please refer to Note 17 for further information regarding reportable segment information.

Recent Accounting Pronouncements Not Yet Adopted

Please refer to Note 16 for further information regarding reportable segment information, including the information required to be disclosed pursuant to this standard.

In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740):

Improvements to Income Tax Disclosures , which enhances the disclosure of an entity's effective tax rate reconciliation and requires the disclosure of income taxes paid to be disaggregated by jurisdiction.

The standard is effective for the Company beginning January 1, 2025, with early adoption permitted.

Upon the adoption of this standard, the Company will modify its disclosures for income taxes, as applicable.

The Company adopted this standard for annual reporting periods beginning January 1, 2025, on a retrospective basis for all periods presented.

Please refer to Note 13 for further information regarding income taxes, including the information required to be disclosed pursuant to this standard.

Recent Accounting Pronouncements Not Yet Adopted

In November 2024, the FASB issued ASU 2024-03, Income Statement – Reporting Comprehensive Income – Expense Disaggregation Disclosures (Subtopic 220-40):

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The new standard requires disclosures about specific types of expenses included in the expense captions presented on the face of the income statement as well as disclosures about selling expenses.

In January 2025, the FASB issued ASU 2025-01, which clarifies the effective date of ASU 2024-03 with respect to interim periods.

The standard is effective for the Company for annual periods beginning January 1, 2027 and interim periods beginning January 1, 2028, with early adoption permitted.

The standard may be applied either prospectively to financial statements issued for reporting periods after the effective date or retrospectively to any or all prior periods presented in the financial statements.

The Company is evaluating the impact of this standard on its consolidated financial statements and related disclosures.

Marketable Securities

The Company is currently in the process of evaluating the impact of this standard on its consolidated financial statements and related disclosures.

Marketable Securities and Other Investments

The following tables present a summary of the Company’s marketable securities, which consist solely of available-for-sale debt securities (in thousands):

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government and agency securities

Certificates of deposit

Corporate bonds

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As of December 31, 2025 and 2024, the balance in accumulated other comprehensive loss consisted solely of unrealized gains and losses on available-for-sale debt securities, net of reclassification adjustments for realized gains and losses and income tax effects.

Unrealized gain (loss) on available-for-sale securities, net, as presented in the consolidated statements of operations and comprehensive loss consisted of the following (in thousands):

Years Ended December 31,

Unrealized gain (loss) before reclassifications

Realized losses (gains) reclassified to investment income

Unrealized gain (loss) on available-for-sale securities, net

The Company did no t recognize any realized gains or losses on available-for-sale securities during the years ended December 31, 2025 and 2024, and no income tax effects or reclassification adjustments were recorded in accumulated other comprehensive loss during the periods.

The following tables present the fair values and unrealized losses of available-for-sale debt securities held by the Company in an unrealized loss position for less than 12 months and 12 months or greater (in thousands):

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government and agency securities

Certificates of deposit

Corporate bonds

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Corporate bonds

As of December 31, 2024 , available-for-sale debt securities held by the Company which were in an unrealized loss position consisted of 22 investment grade security positions.

As of December 31, 2025 , available-for-sale debt securities held by the Company in an unrealized loss position consisted of nine investment grade security positions.

The Company has the intent and ability to hold such securities until recovery, and based on the credit quality of the issuers and low severity of each unrealized loss position relative to its amortized cost basis, the Company did not identify any credit losses associated with its available-for-sale debt securities.

The Company did not recognize any impairment or credit losses on available-for-sale debt securities during the years ended December 31, 2025 and 2024.

Non-marketable Equity Securities

Non-marketable equity securities are measured at cost less impairment, adjusted for observable price changes for identical or similar investments of the same issuer.

The Company did no t hold any non-marketable equity securities as of December 31, 2025 and 2024.

No remeasurements or impairment losses were recorded on non-marketable equity securities during the years ended December 31, 2025 and 2024.

Prior to the acquisition of Corlieve Therapeutics SAS (Corlieve) by uniQure N.V.

(uniQure) in July 2021, the Company held non-marketable equity securities of Corlieve which were originally acquired by the Company in June 2 020 as consideration under a license and collaboration agreement with Corlieve.

As additional consideration, the Company became eligible to receive payments of up to € 37.1 million from uniQure contingent upon the achievement of various development and regulatory milestones.

During the year ended December 31, 2024, the Company received € 5.6 million in milestone payments from uniQure and recognized investment income of $ 6.6 million, respectively, related to the achievement of the milestones during the period.

No milestones were achieved or paid by uniQure during the year ended December 31, 2025.

As of December 31, 2025, there were € 29.7 million ($ 35.0 million as of December 31, 2025) in remaining milestones which have not been paid or achieved and have not been recognized in the consolidated financial statements.

Proceeds contingent upon the achievement of the remaining milestones will be recognized as investment income in the period in which any uncertainty regarding realization is substantially resolved, which may not occur until the achievement of the underlying milestones.

It is at least reasonably possible that some or all of the proceeds contingent upon these milestones will not be realized by the Company.

Fair Value Measurements

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government and agency securities

Certificates of deposit

Corporate bonds

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Money market mutual funds

government and agency securities

Total cash equivalents

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Management estimates that the carrying values of its current accounts receivable, other current assets, accounts payable, accrued expenses and other current liabilities approximate fair value due to the short-term nature of those instruments.

Accounts receivable which contain non-current portions and certain non-current payables reported as other liabilities are recorded at their present values using a discount rate that is based on prevailing market rates on the date the amounts were initially recorded.

Management does not believe there have been any significant changes in market conditions or credit quality that would cause the discount rates initially used to be materially different from those that would be used as of December 31, 2024 to determine the present value of these instruments.

Certain non-current accounts receivable and non-current payables reported as other liabilities are recorded at their present values using a discount rate that is based on prevailing market rates on the date the amounts were initially recorded.

Management does not believe there have been any significant changes in market conditions or credit quality that would cause the discount rates initially used to be materially different

from those that would be used as of December 31, 2025 to determine the present value of these instruments.

Accordingly, management estimates that the carrying values of its non-current accounts receivable and other liabilities approximate the fair value of those instruments.

Management estimates that the carrying value of the liability related to the sale of future royalties approximates fair value.

As discussed in Note 7, the carrying value of the liability related to the sale of future royalties is based on the Company’s estimate of future royalties expected to be paid by the Company over the life of the arrangement, which are considered Level 3 inputs.

Management estimates that the carrying values of its royalty monetization liabilities approximate fair value.

As discussed in Note 7, the carrying values of royalty monetization liabilities are based on the Company’s estimate of future royalties, milestones and other consideration to be paid over the life of the arrangement, which are considered Level 3 inputs, as well as any remaining repayment obligations upon maturity of the instruments.

Long-lived assets, if determined to be impaired, are measured at fair value on a nonrecurring basis using Level 3 inputs.

Please refer to Note 6 for further information on nonrecurring fair value measurements of long-lived assets during the years ended December 31, 2025 and 2024.

Non-marketable Equity Securities

Non-marketable equity securities are measured at cost less impairment, adjusted for observable price changes for identical or similar investments of the same issuer.

The Company did not hold any non-marketable equity securities as of December 31, 2024 and 2023.

No remeasurements or impairment losses were recorded on non-marketable equity securities during the years ended December 31, 2024, 2023 and 2022.

Prior to the acquisition of Corlieve Therapeutics SAS (Corlieve) by uniQure N.V.

In connection with the acquisition of Corlieve by uniQure in July 2021, the Company received proceeds of € 5.3 million ($ 6.1 million) from uniQure in exchange for its ownership interest in Corlieve, of which $ 5.6 million was received upon the closing of the acquisition and $ 0.5 million was received in August 2022 upon the expiration of a hold back period.

As additional consideration, the Company became eligible to receive payments of up to € 37.1 million from uniQure contingent upon the achievement of various development and regulatory milestones.

During the years ended December 31, 2024 and 2023, the Company received € 5.6 million and € 1.9 million, respectively, in milestone payments from uniQure and recognized investment income of $ 6.6 million and $ 2.2 million, respectively, related to the achievement of the milestones during the period.

As of December 31, 2024, there were € 29.7 million ($ 31.0 million as of December 31, 2024) in remaining milestones which have not been paid or achieved and have not been recognized in the consolidated financial statements.

It is at least reasonably possible that some or all of the proceeds contingent upon these milestones will not be realized by the Company.

Property and Equipment, Net

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Property and equipment, net

During the years ended December 31, 2024, 2023 and 2022, the Company recorded depreciation and amortization expense of $ 16.2 million , $ 17.3 million and $ 12.9 million, respectively.

During the years ended December 31, 2025 and 2024, the Company recorded depreciation and amortization expense of $ 15.6 million and $ 16.2 million, respectively.

In March 2024, the Company entered into an agreement to sublease its office facilities in New York, New York.

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The tenant improvement allowance was recorded as a reduction of the right-of-use assets for the lease and is amortized on a straight-line basis as a reduction of lease expense over the term of the lease.

Company began occupation of a portion of the facility upon the completion of its construction in 2021.

The Company began occupation of a portion of the facility upon the completion of its construction in 2021.

The remaining portion of the building, primarily associated with the manufacturing facility, was activated upon the completion of its construction in 2022.

As of December 31, 2024, the Company had recorded property and equipment at cost of $ 132.6 million related to the buildout at 9804 Medical Center Drive, of which $ 131.6 million was placed in service and $ 1.0 million has not yet been placed in service.

As of December 31, 2025, the Company had recorded right-of-use assets of $ 40.8 million and lease liabilities of $ 65.6 million related to the 9804 Medical Center Drive Lease.

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As of December 31, 2025, total undiscounted future minimum lease payments to be received by the Company over the term of the New York Sublease were $ 0.7 million.

The Company recognized sublease income of $ 0.3 million under the New York Sublease during the year ended December 31, 2024.

During the years ended December 31, 2025 and 2024, the Company recognized sublease income of $ 0.5 million and $ 0.3 million, respectively, under the New York Sublease.

The New York Sublease is classified as an operating lease and the Company was not relieved of its primary obligation under the New York Lease.

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As such, the Company recognized impairment losses of $ 2.1 million during the year ended December 31, 2024 on the long-lived asset group associated with the New York Sublease.

The impairment losses were allocated to the various assets within the long-lived asset group based on their

relative carrying values and consisted of $ 1.4 million recorded to the right-of-use assets and $ 0.7 million recorded to property and equipment.

No material impairment losses on long-lived assets were recorded during the years ended December 31, 2023 and 2022.

The impairment losses were allocated to the various assets within the long-lived asset group based on their relative carrying values and consisted of $ 1.4 million recorded to the right-of-use assets and $ 0.7 million recorded to property and equipment.

No impairment losses on long-lived assets were recorded during the year ended December 31, 2025.

As of December 31, 2025, the Company had recorded right-of-use assets of $ 0.3 million and lease liabilities of $ 1.4 million related to the New York Lease.

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As of December 31, 2025, the Company’s extension option under the DC Lease was excluded from the measurement of the right-of-use assets and lease liabilities as it was not reasonably certain of exercise.

The Company recorded the right-of-use assets and lease liabilities related to the DC Lease upon its commencement in October 2022.

As of December 31, 2025 the Company had recorded right-of-use assets of $ 2.7 million and lease liabilities of $ 2.8 million related to the DC Lease.

The Company leases additional office, laboratory and warehousing space, as well as laboratory and other equipment, under operating leases with various expiration dates through 2029 , including leases which have been executed but have not yet commenced.

The Company leases additional office, laboratory and warehousing facilities, as well as laboratory and other equipment, under operating leases with various expiration dates through 2029 , including leases which have been executed but have not yet commenced.

Operating Lease Information

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Right-of-use assets acquired through operating lease liabilities

Cash paid for amounts included in operating lease liabilities for the years ended December 31, 2024, 2023 and 2022 includes zero , $ 0.1 million and $ 1.3 million, respectively, received by the Company during the period under its tenant improvement allowances, which were deemed in-substance lease payments and included in the calculation of the lease liability.

Short-term lease expense for the years ended December 31, 2025 and 2024 was not material and is included in operating lease cost in the table above.

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The table above excludes future minimum lease payments for leases which were executed but had not yet commenced as of December 31, 2025, the total of which were not material.

Liability Related to Sale of Future Royalties

Royalty Monetization Liabilities

Royalty monetization liabilities are accounted for as debt and consist of the following (in thousands):

As of December 31,

2020 Royalty Purchase Agreement

2025 Royalty Bond

Current portion of royalty monetization liabilities

Non-current portion of royalty monetization liabilities

2020 Royalty Purchase Agreement

In December 2020, the Company entered into a royalty purchase agreement (the 2020 Royalty Purchase Agreement) with HCR.

Under the Royalty Purchase Agreement, HCR purchased the Company’s rights to a capped amount of Zolgensma royalty payments under the Company’s license agreement (the Novartis License) with Novartis Gene Therapies, including $ 4.0 million of royalty payments received by the Company in the fourth quarter of 2020 (the Pledged Royalties).

In consideration for these rights, HCR paid the Company $ 200.0 million (the Purchase Price), less $4.0 million representing the payment of the Pledged Royalties to HCR.

Beginning upon the effective date of the Royalty Purchase Agreement, Zolgensma royalty payments, up to a specified threshold, shall be paid to HCR, net of upstream royalties payable by the Company to certain licensors in accordance with existing license agreements.

Pursuant to the Royalty Purchase Agreement, the total amount of royalty payments to be received by HCR was subject to an increasing cap (the Cap Amount) equal to (i) $ 260.0 million applicable for the period from the effective date of the Royalty Purchase Agreement through November 7, 2024 (the First Cap Amount), and (ii) $ 300.0 million applicable for the period from November 8, 2024 through the effective date of termination of the Novartis License (the Second Cap Amount).

If, on or prior to the defined dates for each Cap Amount, the total amount of royalty payments received by HCR equals or exceeds the Cap Amount applicable to such date, the Royalty Purchase Agreement will automatically terminate and all rights to the Zolgensma royalty payments will revert back to the Company.

Under the 2020 Royalty Purchase Agreement, HCR purchased the Company’s rights to a capped amount of Zolgensma and Itvisma royalty payments under the Company’s license agreement with Novartis Gene Therapies (the Novartis License), including $ 4.0 million of royalty payments received by the Company in the fourth quarter of 2020.

In consideration for these rights, HCR paid the Company $ 200.0 million (the Purchase Price), less $ 4.0 million representing the payment of the royalties received in the fourth quarter of 2020 to HCR.

Beginning upon the effective date of the 2020 Royalty Purchase Agreement, Zolgensma and Itvisma royalty payments, up to a specified threshold, shall be paid to HCR, net of upstream royalties payable by the Company to certain licensors in accordance with existing license agreements.

Pursuant to the 2020 Royalty Purchase Agreement, the total amount of Zolgensma and Itvisma royalty payments to be paid to HCR was subject to an increasing cap (the Cap Amount) equal to (i) $ 260.0 million applicable for the period from the effective date of the 2020 Royalty Purchase Agreement through November 7, 2024 (the First Cap Amount), and (ii) $ 300.0 million applicable for the period from November 8, 2024 through the effective date of termination of the Novartis License (the Second Cap Amount).

If, on or prior to the defined dates for each Cap Amount, the total amount of royalties paid to HCR equals or exceeds the Cap Amount applicable to such date, the 2020 Royalty Purchase Agreement will automatically terminate.

The First Cap Amount was not achieved prior to November 7, 2024, therefore the 2020 Royalty Purchase Agreement will remain in effect until the achievement of the Second Cap Amount or the termination of the Novartis License, if earlier.

The Company has no obligation to repay any amounts to HCR if total future Zolgensma royalty payments are not sufficient to achieve the applicable Cap Amount prior to the termination of the Novartis License.

The Company has a call option to repurchase its rights to the purchased royalties from HCR for a repurchase price equal to, as of the option exercise date, $ 300.0 million minus the total amount of royalty payments received by HCR.

The proceeds received from HCR of $ 196.0 million were recorded as a liability, net of transaction costs of $ 3.5 million, which is amortized over the estimated life of the arrangement using the effective interest method.

In order to determine the amortization of the liability, the Company is required to estimate the total amount of future royalty payments to be received by HCR, subject to the Cap Amount, over the life of the arrangement.

The total amount of royalty payments received by HCR under the Royalty Purchase Agreement, less the net proceeds received by the Company of $ 192.5 million, is recorded as interest expense over the life of the arrangement using the effective interest method.

Due to its continuing involvement in the Novartis License, the Company continues to recognize royalty revenue on net sales of Zolgensma and records the royalty payments to HCR as a reduction of the liability when paid.

As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the Royalty Purchase Agreement.

The Company estimates the effective interest rate used to record interest expense under the Royalty Purchase Agreement based on its estimate of future royalty payments to be received by HCR.

At each reporting date, the Company reassesses its estimate of total future royalty payments to be received by HCR at the applicable Cap Amount, and prospectively adjusts the effective interest rate and amortization of the liability as necessary.

Over the life of the arrangement, the actual effective interest rate will be affected by the amount and timing of the royalty payments received by HCR and changes in the Company’s forecasted royalties.

The estimated interest rate in effect as of December 31, 2024 and 2023 was 65.5 % and 4.8 %, respectively, which was based on the amortized balance of the liability and the estimated remaining royalties to be paid to HCR under the arrangement.

This interest rate is subject to adjustments in the future based on actual royalties paid to HCR and changes in the royalty forecast.

The interest rate increased during the year ended December 31, 2024 as a result of changes in the Company's forecasted royalties for territories outside the United States.

As of December 31, 2024, the estimated effective interest rate over the life of the Royalty Purchase Agreement, taking into account actual royalties paid to date and the estimated remaining royalties to be paid under the arrangement, was 16.0 %.

The following table presents the changes in the liability related to the sale of future royalties under the Royalty Purchase Agreement with HCR (in thousands):

Liability Related to

Sale of Future Royalties

The Company has no obligation to repay any amounts to HCR under the 2020 Royalty Purchase Agreement if future Zolgensma and Itvisma royalty payments are not sufficient to achieve the applicable Cap Amount prior to the termination of the Novartis License.

The Company has a call option to repurchase its rights to the royalties under the 2020 Royalty Purchase Agreement for a repurchase price equal to, as of the option exercise date, $ 300.0 million minus the total amount of royalty payments paid to HCR.

The proceeds received from HCR under the 2020 Royalty Purchase Agreement of $ 196.0 million were recorded as a liability, net of transaction costs of $ 3.5 million, which is amortized over the estimated life of the arrangement using the effective interest method.

In order to determine the amortization of the liability, the Company is required to estimate the total amount of future royalty payments to be paid to HCR, subject to the Cap Amount, over the life of the arrangement.

The total amount of royalty payments paid to HCR, less the net proceeds received by the Company of $ 192.5 million, is recorded as interest expense over the life of the arrangement using the effective interest method.

Due to its continuing involvement in the Novartis License, the Company continues to recognize royalty revenue on net sales of Zolgensma and Itvisma and records the royalty payments to HCR as a reduction of the liability when paid.

As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the arrangement.

The Company estimates the effective interest rate used to record interest expense under the 2020 Royalty Purchase Agreement based on its estimate of total Zolgensma and Itvisma royalties to be paid HCR under the arrangement.

At each reporting date, the Company reassesses its estimate of total future royalty payments to be paid to HCR at the applicable Cap Amount, and prospectively adjusts the effective interest rate and amortization of the liability as necessary.

Over the life of the arrangement, the actual effective interest rate will be affected by the amount and timing of royalty payments actually paid to HCR, which may differ from the Company’s forecasts.

The estimated effective interest rate in effect as of December 31, 2025 and 2024 was 85.2 % and 65.5 %, respectively, which was based on the amortized balance of the liability and the estimated remaining royalties to be paid to HCR under the arrangement.

The estimated interest rate is subject to adjustments in the future based on actual royalties paid to HCR and changes in the royalty forecast.

As of December 31, 2025, the estimated effective interest rate over the life of the 2020 Royalty Purchase

Agreement, taking into account actual royalties paid to date and the estimated remaining royalties to be paid under the arrangement, was 16.3 %.

The following table presents the changes in the royalty monetization liability under the 2020 Royalty Purchase Agreement with HCR (in thousands):

Purchase Agreement

Balance at December 31, 2023

Zolgensma royalties paid to HCR

Novartis royalties paid to HCR

Interest expense recognized

Balance at December 31, 2024

Zolgensma royalties paid to HCR

Novartis royalties paid to HCR

Interest expense recognized

Balance at December 31, 2025

Current portion of liability related to sale of future royalties

Liability related to sale of future royalties, non-current

Current portion

Non-current portion

2025 Royalty Bond

In May 2025, the Company entered into a loan agreement with HCR pursuant to which HCR will provide the Company with an aggregate limited recourse loan of up to $ 250.0 million (the 2025 Royalty Bond).

The 2025 Royalty Bond is disbursable to the Company in three tranches, with $ 150.0 million funded on the closing date in May 2025, $ 50.0 million available to be funded if sales of a specified product exceed a specified threshold prior to December 31, 2026, and $ 50.0 million available to be funded if both parties exercise an option in 2027.

Loan proceeds under the 2025 Royalty Bond are funded to the Company net of an original issue discount of 2.25 % and reimbursement of certain expenses to HCR.

Proceeds received by the Company from the initial funding tranche of the 2025 Royalty Bond in May 2025, net of discounts and transaction costs, were $ 144.5 million.

Prior to the maturity date, interest and principal under the 2025 Royalty Bond shall be paid quarterly to HCR solely using proceeds received from certain specified royalties, milestone payments, license fees and other consideration payable to the Company under specified license agreements (collectively, the Royalty Interest), including (i) the Novartis License for Zolgensma and Itvisma, (ii) the collaboration and license agreement with Nippon Shinyaku for RGX-121 and RGX-111, and (iii) NAV Technology Platform license agreements with Rocket Pharmaceuticals, Inc.

and Ultragenyx Pharmaceutical Inc.

Zolgensma and Itvisma royalties earned under the Novartis License shall only be included in the Royalty Interest after full repayment of the applicable Cap Amount under the 2020 Royalty Purchase Agreement with HCR.

The Royalty Interest excludes, and the Company retains the rights to, certain other consideration payable under the license agreements including certain milestone payments, license fees and reimbursement of costs as applicable.

The Royalty Interest is payable to HCR net of upstream royalty and sublicense fee obligations payable by the Company to applicable licensors.

The 2025 Royalty Bond is collateralized by a security interest and lien on the Royalty Interest.

The 2025 Royalty Bond bears interest at a rate of 9.75 % plus the 3-month secured overnight financing rate as administered by the Federal Reserve Bank of New York ( SOFR ), with a minimum interest rate of 14.0 %.

Interest payments are due quarterly using proceeds received under the Royalty Interest.

At each payment date, any proceeds received under the Royalty Interest in excess of the interest payment due will be applied to outstanding principal.

If the proceeds received under the Royalty Interest are insufficient to pay the interest due, unpaid interest will accrue to the principal balance.

The 2025 Royalty Bond matures in May 2035 , subject to potential extension, unless repaid in full at an earlier date.

The maturity date may be extended by two years to May 2037 subject to a potential patent term extension of a specific patent.

Upon maturity, the outstanding principal and interest shall be due and payable to HCR.

Additionally, upon repayment in full prior to the maturity date, or at the maturity date, the Company shall pay to HCR an additional amount equal to 5.0 % of the total outstanding principal as of the applicable determination date.

Other than through the payment of proceeds received under the Royalty Interest, the 2025 Royalty Bond may not be prepaid prior to maturity.

In connection with the loan agreement for the 2025 Royalty Bond, the Company also issued HCR warrants to purchase 268,096 shares of its common stock at an exercise price per share of $ 14.92 (the May 2025 Warrants).

The May 2025 Warrants are exercisable upon issuance and expire 10 years from the closing date of the 2025 Royalty Bond.

The Company evaluated the May 2025 Warrants and concluded the warrants are indexed to the Company's common stock and meet the criteria to be classified as equity.

The net proceeds received by the Company under the loan agreement of $ 144.5 million were allocated between the 2025 Royalty Bond and

the May 2025 Warrants based on their relative fair values.

The fair value of the 2025 Royalty Bond was determined based on the carrying amount of the loan on the closing date.

The fair value of the May 2025 Warrants was determined using a Black-Scholes option-pricing model on the closing date, resulting in an estimated fair value of the warrants of $ 1.7 million.

Based on the relative fair values of these instruments, $ 1.6 million of the net proceeds were allocated to the warrants and recorded as additional paid-in capital.

The net proceeds allocated to the 2025 Royalty Bond were $ 142.9 million, resulting in a total debt discount of $ 7.1 million which is recorded as a reduction of the carrying value of the debt and will be amortized as interest expense over the life of the 2025 Royalty Bond.

The effective interest rate of the 2025 Royalty Bond is partially estimated based on the Company's estimate of future payments under the Royalty Interest to be paid HCR.

At each reporting date, the Company reassesses its estimate of total future payments to HCR under the arrangement and prospectively adjusts the effective interest rate and amortization of the debt and associated discount as necessary.

Over the life of the arrangement, the actual effective interest rate will be affected by the amount and timing of the actual Royalty Interest payments to HCR and fluctuations in the variable interest rate, which may differ from the Company’s forecasts.

The estimated effective interest rate in effect as of December 31, 2025 was 15.1 %, which was based on the amortized balance of the liability, the current coupon rate and the estimated remaining payments to HCR under the arrangement.

The following table presents the changes in the royalty monetization liability under the 2025 Royalty Bond with HCR (in thousands):

2025 Royalty Bond

Balance at December 31, 2024

Proceeds from royalty bond, net of discount and issuance costs

Royalty interest paid to HCR

Portion of payments representing interest

Unpaid interest accrued to principal

Amortization of debt discount and issuance costs

Balance at December 31, 2025

Current portion

Non-current portion

Commitments and Contingencies

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The Trustees of the University of Pennsylvania

In February 2009, the Company entered into a license agreement, which has been amended from time to time (as amended, the Penn License), with The Trustees of the University of Pennsylvania (together with the University of Pennsylvania, Penn) for exclusive, worldwide rights to certain patents owned by Penn underlying the Company’s NAV Technology Platform, as well as exclusive rights to certain data, results and other information.

In February 2009, the Company entered into a license agreement, which has been amended from time to time (as amended, the Penn License), with The Trustees of the University of Pennsylvania (Penn) for exclusive, worldwide rights to certain patents owned by Penn underlying the Company’s NAV Technology Platform, as well as exclusive rights to certain data, results and other information.

Pursuant to the originally agreed upon Penn License, the Company was obligated to pay Penn royalties on net sales of licensed products and sublicense fees.

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In April 2019, the Penn License was amended to include exclusive license rights to certain patent rights and know-how, including research data and other information, relating to the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.

In consideration for the additional licensed rights, and in addition to any consideration owed under the license prior to the amendment, the Company paid Penn an up-front fee and is obligated to pay milestone fees of up to $ 20.5 million upon the achievement of various development and sales-based milestones and additional royalties on net sales of licensed products for the

treatment of CLN2 disease.

From the inception of the agreement through December 31, 2025, the Company had incurred $ 0.5 million for development milestones achieved, or deemed probable of achievement, under the Penn License.

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Years Ended December 31,

Cost of revenues

Research and development

General and administrative

Interest expense

As of December 31, 2024, the Company had recorded $ 5.8 million payable under the Penn License, net of present value discount, of which $ 2.9 million was included in accrued expenses and other current liabilities, and $ 2.9 million was included in other liabilities on the consolidated balance sheet.

As of December 31, 2025, the Company had recorded $ 3.0 million payable under the Penn License, net of present value discount, which was included in accounts payable and accrued expenses and other current liabilities on the consolidated balance sheet.

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Years Ended December 31,

Cost of revenues:

Royalties on net sales of Zolgensma

Other cost of revenues

Total cost of revenues

Cost of license and royalty revenues:

Royalties on net sales of Zolgensma and Itvisma

Total cost of license and royalty revenues

General and administrative

As of December 31, 2024, the Company had recorded $ 6.3 million payable under the GSK License, of which $ 6.2 million was included in accrued expenses and other current liabilities, and $ 0.1 million was included in other liabilities on the consolidated balance sheet.

As of December 31, 2025, the Company had recorded $ 5.7 million payable under the GSK License, of which $ 5.7 million was included in accrued expenses and other current liabilities, and less than $ 0.1 million was included in other liabilities on the consolidated balance sheet.

The Company has been notified of a potential dispute with GSK over the amount of sublicense fees paid by the Company to GSK under the GSK License.

The Company has been notified of a dispute with GSK over the amount of sublicense fees paid by the Company to GSK under the GSK License.

GSK claims there has been a significant underpayment by the Company as they are entitled to a sublicense payment on all amounts received by the Company from sublicensees, including royalties, and not just amounts received for GSK's sublicensed patents.

The Company disagrees with GSK's interpretation of the GSK License.

The Company disagrees with GSK's interpretation of the GSK License and engaged in non-binding mediation with GSK but the dispute has not yet been resolved.

The Company does not believe that a loss is probable, and no reasonable range of loss is estimable, related to this matter.

No liabilities related to this matter have been recorded as of December 31, 2024.

No liabilities related to this matter were recorded as of December 31, 2025 and 2024.

Emory University

In August 2018, the Company entered into a license agreement (the Emory License) with Emory University (Emory) for an exclusive license to Emory’s interest in certain patent rights, which are co-owned by Emory and the Company, to commercialize products covered by the licensed patent rights in any country or territory.

Patent rights licensed under the Emory License include certain rights which have been sublicensed by the Company to Novartis and are applicable to Itvisma for the treatment of certain patients with SMA.

Pursuant to the Emory License, the Company is obligated to reimburse Emory for patent prosecution and maintenance expenses and pay Emory annual maintenance fees under certain circumstances, royalties on net sales, sublicense fees and fees upon the achievement of various milestones for the first licensed product.

During the years ended December 31, 2025 and 2024, the Company incurred $ 0.1 million and $ 0.1 million, respectively, under the Emory License for milestone payments and patent maintenance costs.

Clearside Biomedical

In August 2019, the Company entered into an option and license agreement with Clearside Biomedical, Inc.

(Clearside) pursuant to which the Company was granted an option to exclusively license the worldwide rights to certain patents related to Clearside’s proprietary, in-office SCS Microinjector for the delivery of ABBV-RGX-314 to the suprachoroidal space to treat wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other diseases.

(Clearside) pursuant to which the Company was granted an option to exclusively license the worldwide rights to certain patents related to Clearside’s proprietary, in-office SCS Microinjector ® for the delivery of ABBV-RGX-314 to the suprachoroidal space to treat wet AMD, DR and other diseases.

The Company exercised its license option in October 2019, resulting in a payment of $ 1.6 million to Clearside payable under the license agreement.

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From the inception of the agreement through December 31, 2025, the Company had incurred $ 3.0 million for development milestones achieved, or deemed probable of achievement, under the agreement.

In November 2025, Clearside announced that it filed a voluntary petition under Chapter 11 of the U.S.

Bankruptcy Code in the District of Delaware and that it was seeking authorization to sell all or substantially all of its assets in a court-supervised auction and sale process under Section 363 of the U.S.

Bankruptcy Code.

Clearside subsequently announced that it entered into an asset purchase agreement with a stalking horse bidder, Health Ocean Pharma (Eye) Limited, for the sale of substantially all of its assets, including its remaining rights with respect to the SCS Microinjector.

The sale process is still ongoing and is subject to pending objections by parties in interest, and, therefore, the ultimate outcome of that process is unknown.

Other Licenses

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Additionally, the Company is obligated to pay for certain costs incurred related to the maintenance of the licensed patents.

In August 2018, the Company entered into a license agreement with Emory University (Emory) for an exclusive license to Emory’s interest in certain patent rights which are co-owned by Emory and the Company to commercialize products covered by the licensed patent rights in any country or territory.

Pursuant to the license agreement, the Company is obligated to reimburse Emory for patent prosecution and maintenance expenses and pay Emory annual maintenance fees under certain circumstances, royalties on net sales, sublicense fees and fees upon the achievement of various milestones for the first licensed product.

In June 2022, the Company entered into a license agreement with Johns Hopkins University (JHU) for an exclusive license to JHU's interest in certain patent rights which are co-owned by JHU and the Company to commercialize products covered by the licensed patent rights in any country or territory.

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As of December 31, 2025 and 2024 , the Company did no t have any material indemnification claims that were probable or reasonably possible and consequently had not recorded any related liabilities.

Settlement Agreement

In the fourth quarter of 2022, the Company entered into a settlement agreement with a third party pursuant to which the Company released certain claims regarding infringement of the Company's intellectual property.

In consideration for the release of claims made by the Company, the third party paid $ 7.5 million to the Company which was recorded as other operating income in the consolidated statements of operations and comprehensive loss for the year ended December 31, 2022.

On or about February 13, 2026, a putative securities class action complaint was filed by Andre Kuik against the Company and certain of its current officers and directors in the United States District Court for the District of Maryland.

The complaint asserts claims under Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder, on behalf of a putative class of persons who purchased or otherwise acquired the Company's securities during the period from February 9, 2022 through January 27, 2026.

The complaint alleges that the Company misled investors concerning the viability and safety of RGX-111 study, and that the Company's stock price declined following the announcement of the clinical holds imposed by the FDA on the Company’s RGX-111 and RGX-121 programs on January 28, 2026.

The plaintiff seeks unspecified compensatory damages, attorneys' fees, expert fees and other costs, and other relief as the court may deem just and proper.

The Company believes that it has meritorious defenses to the claims asserted and intends to vigorously defend against them.

The Company does not believe that a loss is probable, and no reasonable range of loss is estimable, related to this matter.

No liabilities related to this matter were recorded as of December 31, 2025.

Capitalization

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Reserved for issuance under employee stock purchase plan

Reserved for exercise of pre-funded warrants outstanding

March 2024 Public Offering

In March 2024, the Company completed a public offering (March 2024 Offering) of 4,565,260 shares of its common stock at a price of $ 23.00 per share and 1,521,740 pre-funded warrants to purchase shares of its common stock at a price of $ 22.9999 per pre-funded warrant, which equaled the public offering price per share of the common stock less the $ 0.0001 exercise price of each pre-funded warrant.

Reserved for exercise of warrants outstanding

May 2025 Warrants

In May 2025, in connection with issuance of the 2025 Royalty Bond, the Company issued to HCR the May 2025 Warrants to purchase 268,096 shares of its common stock at an exercise price per share of $ 14.92 .

The May 2025 Warrants are exercisable upon issuance and have a contractual term of 10 years.

The Company evaluated the May 2025 Warrants and concluded the warrants are indexed to the Company's common stock, meet the criteria to be classified as equity and are not subject to remeasurement.

The Company allocated $ 1.6 million of the net proceeds from the 2025 Royalty Bond to the issuance of the May 2025 Warrants, which were recorded as additional paid-in capital.

Please refer to Note 7 for further information on the May 2025 Warrants issued in connection with the 2025 Royalty Bond.

As of December 31, 2025, no ne of the May 2025 Warrants had been exercised and 268,096 of the May 2025 Warrants remained outstanding.

March 2024 Public Offering and Pre-funded Warrants

In March 2024, the Company completed a public offering of 4,565,260 shares of its common stock at a price of $ 23.00 per share and 1,521,740 pre-funded warrants (the March 2024 Pre-funded Warrants) to purchase shares of its common stock at a price of $ 22.9999 per pre-funded warrant, which equaled the public offering price per share of the common stock less the $ 0.0001 exercise price of each pre-funded warrant.

The aggregate net proceeds received by the Company from the offering were $ 131.1 million, net of underwriting discounts and commissions and offering expenses.

The rights and privileges of the pre-funded warrants issued under the March 2024 Offering are set forth in the warrant agreement between the Company and each of the respective warrant holders.

The pre-funded warrants are exercisable at the option of the warrant holder at any time and do not expire.

The rights and privileges of the March 2024 Pre-funded Warrants are set forth in the warrant agreement between the Company and each of the respective warrant holders.

The March 2024 Pre-funded Warrants are exercisable at the option of the warrant holder at any time and do not expire.

However, as set forth in the warrant agreements with each holder, the number of pre-funded warrants that may be exercised at any given time may be limited if, upon exercise, the warrant holder and any of its affiliates would beneficially own more than 9.99 % of the Company’s common stock, or have voting power of more than 9.99 % of the Company's common stock.

The limitation threshold may be increased or decreased by the warrant holder, with advance notice to the Company, to any other percentage not less than 4.99 % nor in excess of 19.99 %.

Pre-funded warrants do not provide any of the rights or privileges provided by the Company's common stock, including any voting rights, until the pre-funded warrants are exercised and settled in underlying shares of common stock.

The Company evaluated the pre-funded warrants issued under the March 2024 Offering and concluded the warrants are indexed to the Company's common stock, meet the criteria to be classified as equity and are not subject to remeasurement.

The March 2024 Pre-funded Warrants do not provide any of the rights or privileges provided by the Company's common stock, including any voting rights, until the pre-funded warrants are exercised and settled in underlying shares of common stock.

The Company evaluated the March 2024 Pre-funded Warrants and concluded the warrants are indexed to the Company's common stock, meet the criteria to be classified as equity and are not subject to remeasurement.

The proceeds received from the issuance of the pre-funded warrants were recorded as additional paid-in capital.

The Company issued 197,000 shares of common stock upon the exercise of pre-funded warrants during the year ended December 31, 2024.

As of December 31, 2024, 1,324,740 pre-funded warrants remained outstanding.

July 2023 Private Placement

In July 2023, the Company sold an aggregate of 257,466 shares of its common stock to Redmile Biopharma Investments III, L.P.

(Redmile) at a purchase price of $ 19.42 per share, which was the closing price of the common stock on July 6, 2023 (the Private Placement).

The Company received aggregate net proceeds from the Private Placement of $ 4.9 million, net of offering expenses.

The Private Placement was conducted in reliance on the exemption from registration provided by Section 4(a)(2) of the Securities Act.

During the years ended December 31, 2025 and 2024, the Company issued 455,137 shares and 197,000 shares, respectively, of common stock upon the exercise of March 2024 Pre-funded Warrants.

As of December 31, 2025, 869,603 of the March 2024 Pre-funded Warrants remained outstanding.

At-the-Market Offering Programs

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License and Collaboration Agreements

License and Royalty Revenue

As of December 31, 2024 , the Company’s NAV Technology Platform was being applied by NAV Technology Licensees in one commercial product, Zolgensma, and in the development of a number of other licensed products.

Additionally, the Company has licensed intellectual property rights to collaborators for the joint development of certain product candidates.

Consideration to the Company under its license agreements may include:

License and Collaboration Revenues

As of December 31, 2025 , the Company’s NAV Technology Platform was being applied by NAV Technology Licensees in two commercial products, Zolgensma and Itvisma, and in the development of various other licensed products.

Additionally, the Company has licensed intellectual property rights to collaborators for the joint development and commercialization of certain product candidates.

Consideration payable to the Company under its license and collaboration agreements may include:

(i) up-front and annual fees, (ii) milestone payments based on the achievement of certain development and sales-based milestones, (iii) sublicense fees, (iv) royalties on sales of licensed products, (v) fees for services related to the development and manufacturing of licensed products and (vi) other consideration payable upon optional goods and services purchased by licensees and collaborators.

Sublicense fees vary by license and range from a mid-single digit percentage to a low-double digit percentage of license fees received by licensees as a result of sublicenses.

Royalties on net sales of commercialized products vary by license and range from a mid-single digit percentage to a low double-digit percentage of net sales by licensees.

License and royalty revenue consisted of the following (in thousands):

Revenues earned under license and collaboration agreements consisted of the following (in thousands):

Years Ended December 31,

Zolgensma royalties

Other license and royalty revenue

License and royalty revenue:

Zolgensma and Itvisma royalties

Nippon Shinyaku licenses

Total license and royalty revenue

Outstanding development milestone payments are evaluated each reporting period and are only included in the transaction price of each license and recognized as license revenue to the extent the milestones are considered probable of achievement.

Service revenue:

Nippon Shinyaku services

Total service revenue

Total revenues

Outstanding development milestone payments are evaluated each reporting period and are only included in the transaction price of each license to the extent the milestones are considered probable of achievement.

Sales-based milestones are excluded from the transaction price of each license agreement and recognized as royalty revenue in the period of achievement.

As of December 31, 2024, the Company’s license agreements, excluding additional licenses that could be granted upon the exercise of options by licensees, contained unachieved milestones which could result in aggregate milestone payments to the Company of up to $ 1.51 billion, including (i) $ 524.9 million upon the commencement of various stages of clinical trials, (ii) $ 113.8 million upon the submission of regulatory approval filings or upon regulatory approval of licensed products, and (iii) $ 870.0 million upon the achievement of specified sales targets for licensed products, including milestones payable upon the first commercial sale of licensed products.

To the extent the milestone payments are realized by the Company, the Company may be obligated to pay sublicense fees to licensors based on a specified percentage of the fees earned by the Company.

As of December 31, 2025, the Company’s license and collaboration agreements contained unachieved milestones which could result in aggregate milestone payments to the Company of up to $ 2.18 billion, including (i) $ 546.7 million upon the commencement of various stages of clinical trials, (ii) $ 106.3 million upon the submission of regulatory approval filings or upon regulatory approval of licensed products, and (iii) $ 1.53 billion upon the achievement of specified sales targets for licensed products, including milestones payable upon the first commercial sale of licensed products.

To the extent the Company realizes the milestone payments, the Company may be obligated to pay sublicense fees to licensors based on a specified percentage of the fees earned by the Company.

The achievement of these milestones is highly dependent on the successful development and commercialization of licensed products and it is at least reasonably possible that some or all of the milestone fees will not be realized by the Company.

Changes in Accounts Receivable, Contract Assets and Deferred Revenue

The following table presents the balances of the Company’s net accounts receivable, contract assets and deferred revenue, as well as other information regarding revenue recognized, during the periods presented (in thousands):

Accounts Receivable, Contract Assets and Deferred Revenue

The following table presents the balances of the Company’s accounts receivable, contract assets and deferred revenue, as well as other information regarding revenue recognized, during the periods presented (in thousands):

Years Ended December 31,

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End of period

Deferred revenue:

Deferred revenue, current and non-current:

Beginning of period

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Performance obligations satisfied in previous periods

As of December 31, 2024 , the Company had recorded deferred revenue of $ 0.1 million which represents consideration received or unconditionally due from licensees for performance obligations that have not yet been satisfied by the Company.

Unsatisfied performance obligations as of December 31, 2024 consisted of development services to be performed by the Company related to licensed products, which will be satisfied as the services are performed.

As of December 31, 2024, the aggregate transaction price of the Company's license agreements allocated to performance obligations not yet satisfied, or partially satisfied, was $ 1.1 million, which is expected to be satisfied over a period of approximately two years .

Revenue recognized from performance obligations satisfied in previous periods, as presented in the table above, was primarily attributable to Zolgensma royalties and changes in the transaction prices of the Company’s license agreements.

Changes in transaction prices were primarily attributable to development milestones achieved or deemed probable of achievement during the periods which were previously not considered probable of achievement, resulting in a cumulative catch-up adjustment to revenue.

Revenue recognized during the years ended December 31, 2024, 2023 and 2022 includ ed zero , $ 2.0 million and zero , respectively, in cumulative catch-up adjustments for changes in the probability of achievement of development milestones.

Accounts Receivable, Contract Assets and the Allowance for Credit Losses

Accounts receivable, net consisted of the following (in thousands):

Revenue recognized from performance obligations satisfied in previous periods, as presented in the table above, was primarily attributable to Zolgensma and Itvisma royalties and changes in the transaction prices of the Company’s license agreements.

Changes in transaction prices were primarily attributable to development milestones achieved or deemed probable of achievement during the periods, resulting in a cumulative catch-up adjustment to revenue.

Revenue recognized during the year ended December 31, 2025, included $ 1.5 million in cumulative catch-up adjustments for changes in the probability of achievement of development milestones.

No cumulative catch-up adjustments for development milestones were recorded in revenue during the year ended December 31, 2024.

As of December 31, 2025 , the Company had recorded deferred revenue of $ 29.4 million which represents consideration received or unconditionally due from licensees and collaboration partners for performance obligations that have not yet been satisfied by the Company.

Unsatisfied performance obligations as of December 31, 2025 consisted of (i) development services to be performed related to licensed products, which will be satisfied as the services are performed, and (ii) material rights granted to purchase commercial supply of licensed products, which will be satisfied upon delivery of the commercial supply.

As of December 31, 2025, the aggregate transaction price of the Company’s license and collaboration agreements allocated to performance obligations not yet satisfied or partially satisfied was $ 30.4 million, primarily associated with development services under the Company's collaboration and license agreement with Nippon Shinyaku, the substantial majority of which is expected to be satisfied over a period of approximately five years .

Accounts receivable consisted of the following (in thousands):

As of December 31,

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Billed to customers

Unbilled Zolgensma royalties

Due from Abeona, net of present value discount

Unbilled Novartis royalties

Unbilled Nippon Shinyaku services

Other unbilled

Allowance for credit losses

Current accounts receivable, net

Current accounts receivable

Non-current accounts receivable:

Unbilled Nippon Shinyaku services

Other unbilled

Non-current accounts receivable, net

Total accounts receivable, net

Non-current accounts receivable

Total accounts receivable

The following table presents the changes in the allowance for credit losses related to accounts receivable and contract assets for the years ended December 31, 2025 and 2024 (in thousands):

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Balance at December 31, 2023

Provision for credit losses

Changes in present value discount of receivables

Credit recoveries

Balance at December 31, 2024

Provision for credit losses

Changes in present value discount of receivables

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Balance at December 31, 2025

The Company’s allowance for credit losses during the years ended December 31, 2024, 2023 and 2022 was related solely to accounts receivable from Abeona Therapeutics Inc.

The Company’s allowance for credit losses during the year ended December 31, 2024 was related solely to accounts receivable from Abeona Therapeutics Inc.

Please refer to the section below, "Settlement Agreement with Abeona Therapeutics," for further information regarding amounts due from Abeona and the associated allowance for credit losses.

Zolgensma License with Novartis Gene Therapies

Zolgensma and Itvisma License with Novartis Gene Therapies

In March 2014, the Company entered into an exclusive license agreement (as amended, the Novartis License) with Novartis Gene Therapies.

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In consideration for the rights granted under the license, Novartis Gene Therapies paid the Company (i) an up-front fee of $ 2.0 million upon the execution of the agreement in 2014, (ii) license fees totaling $ 180.0 million upon the amendment of the agreement in January 2018 and the subsequent acquisition of AveXis, Inc.

(now Novartis Gene Therapies) by Novartis in May 2018, (iii) total cumulative payments of $ 12.3 million upon the achievement of various development milestones, and (iv) a sales-based milestone payment of $ 80.0 million upon the achievement of $ 1.0 billion in cumulative net sales of Zolgensma in the third quarter of 2020.

In addition to the consideration above, Novartis Gene Therapies is obligated to pay to the Company fixed annual fees, royalties on net sales of licensed products and a percentage of any sublicense fees received by Novartis Gene Therapies from sublicensees for the licensed intellectual property rights.

Royalties are payable by Novartis Gene Therapies at a mid-single to low double-digit percentage of net sales of licensed products using the NAV AAV9 vector, and a low double-digit percentage of net sales of licensed products using a licensed vector other than NAV AAV9, and are subject to reduction in specified circumstances.

In 2019, Novartis Gene Therapies launched commercial sales of Zolgensma, a licensed product under the Novartis License.

In accordance with the Novartis License, the Company receives royalties on net sales of Zolgensma.

All development and sales-based milestones under the Novartis License have been achieved and there are no further milestone payments payable to the Company under the license agreement.

In 2019, Novartis Gene Therapies launched commercial sales of Zolgensma for the treatment of SMA in patients under the age of two years old.

In the fourth quarter of 2025, Novartis Gene Therapies launched commercial sales of Itvisma for the treatment of SMA in patients two years and older.

Zolgensma and Itvisma are licensed products under the Novartis License, pursuant to which the Company receives royalties on net sales of the licensed products.

The Company recognized the following amounts under the Novartis License (in thousands):

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Zolgensma royalties

Itvisma royalties

Other license revenue

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Interest income from licensing

As of December 31, 2024 and 2023 , the Company had recorded total accounts receivable of $ 20.4 million and $ 24.3 million, respectively, from Novartis Gene Therapies under the Novartis License, which consisted primarily of Zolgensma royalties receivable.

The Zolgensma royalties receivable recorded as of December 31, 2024 included $ 14.4 million expected to be paid to HCR in accordance with the Royalty Purchase Agreement discussed in Note 7.

As of December 31, 2025 and 2024 , the Company had recorded total accounts receivable of $ 24.1 million and $ 20.4 million, respectively, from Novartis Gene Therapies under the Novartis License, which consisted primarily of Zolgensma and Itvisma royalties receivable.

Royalties receivable from Novartis recorded as of December 31, 2025 included $ 18.3 million expected to be paid to HCR in accordance with the 2020 Royalty Purchase Agreement discussed in Note 7.

Settlement Agreement with Abeona Therapeutics

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(i) $ 20.0 million paid in November 2021, (ii) $ 5.0 million paid in November 2022, and (iii) $ 5.0 million paid in November 2024.

As of December 31, 2024, all amounts due from Abeona under the Settlement Agreement had been paid in full and no further amounts were due to the Company from Abeona.

As of December 31, 2023, the Company had recorded accounts receivable of $ 4.6 million associated with the remaining amounts due from Abeona under the Settlement Agreement.

The receivable of $ 4.6 million as of December 31, 2023 consisted of the $ 5.0 million payment due by November 2024, net of discount to present value.

The Company assessed the credit risk of the receivable from Abeona as of December 31, 2023.

Based on its evaluation of Abeona's credit profile and financial condition, and its expectations regarding Abeona's future cash flows and ability to satisfy the contractual obligations of the Settlement Agreement, the Company recorded an allowance for credit losses of $ 4.6 million as of December 31, 2023 related to the accounts receivable due from Abeona.

As of December 31, 2025 and 2024, all amounts due from Abeona under the Settlement Agreement had been paid in full and no further amounts were due to the Company from Abeona.

As of December 31, 2023, the Company had recorded accounts receivable of $ 4.6 million associated with the remaining amounts due from Abeona under the Settlement Agreement, which consisted of the $ 5.0 million payment due by November 2024, net of discount to present value.

Based on the Company's evaluation of Abeona's credit profile and financial condition, and its expectations regarding Abeona's future cash flows and ability to satisfy the contractual obligations of the Settlement Agreement, the Company recorded an allowance for credit losses of $ 4.6 million as of December 31, 2023 related to the accounts receivable due from Abeona.

Prior to collection, the present value discount of the Abeona receivable was accreted as interest income from licensing through the contractual due date using the effective interest method.

The Company elected to record increases in the allowance for credit losses associated with the accretion of the present value discount as a reduction of the associated interest income, resulting in no interest income recognized during the periods related to the accretion of the present value discount.

The Company elected to record increases in the allowance for credit losses associated with the accretion of the present value discount as a reduction of the associated interest income, resulting in no interest income recognized related to the accretion of the present value discount.

The Company collected the final payment of $ 5.0 million due from Abeona under the Settlement Agreement upon its contractual due date in November 2024.

As a result of the full collection of the receivable, the Company recorded a credit recovery of $ 5.0 million during the year ended December 31, 2024 against the associated allowance for credit losses.

No credit losses or recoveries were recorded on the Abeona receivable during the years ended December 31, 2023 and 2022.

Collaboration Agreements

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In September 2021, the Company entered into a collaboration and license agreement with AbbVie Global Enterprises Ltd.

(AbbVie), a subsidiary of AbbVie Inc., to jointly develop and commercialize ABBV-RGX-314, the Company’s product candidate for the treatment of wet AMD, DR and other chronic retinal diseases (the AbbVie Collaboration Agreement).

The AbbVie Collaboration Agreement became effective in November 2021.

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The Company and AbbVie will collaborate to develop ABBV-RGX-314 in the United States, and AbbVie will be responsible for the development of ABBV-RGX-314 in specified markets outside the United States.

Through December 31, 2022, the Company was responsible for the development expenses related to certain ongoing clinical trials of ABBV-RGX-314 and the parties shared the additional development expenses related to ABBV-RGX-314.

Beginning on January 1, 2023, AbbVie became responsible for the majority of all ABBV-RGX-314 development expenses.

Global development expenses for ABBV-RGX-314 are shared by the parties in accordance with the AbbVie Collaboration Agreement, with AbbVie being responsible for the majority of total development expenses.

The Company will lead the manufacturing of ABBV-RGX-314 for clinical development and U.S.

commercial supply, and AbbVie will lead the manufacturing of ABBV-RGX-314 for commercial supply outside the United States.

Manufacturing expenses will be allocated between the parties in accordance with the terms of the AbbVie Collaboration Agreement and supply agreements determined in accordance with the agreement.

If requested by AbbVie, the Company will manufacture up to a specified portion of ABBV-RGX-314 for commercial supply outside the United States at a price specified in the agreement.

Manufacturing expenses will be allocated between the parties in accordance with the terms of the AbbVie Collaboration Agreement and mutually agreed supply agreements.

If requested by AbbVie, the Company will manufacture up to a specified portion of ABBV-RGX-314 commercial supply for sales outside the United States at a price specified in the agreement.

AbbVie will lead the commercialization of ABBV-RGX-314 globally, and the Company will participate in U.S.

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AbbVie is also required to pay to the Company tiered royalties on net sales of ABBV-RGX-314 outside the United States at percentages in the mid-teens to low twenties, subject to specified offsets and reductions.

In August 2025, the Company and AbbVie entered into an amendment to the AbbVie Collaboration Agreement which modified the development plan and milestone payment structure for the ABBV-RGX-314 DR program.

Under the amendment, the Company will conduct the first registration enabling trial for DR suprachoroidal (SCS) treatment as a combined Phase IIb/III trial (NAAVIGATE) which will be performed in two parts (Part 1 and Part 2), and AbbVie will conduct the second registration enabling trial as a separate, standalone Phase III trial.

In lieu of a $ 200.0 million development milestone payable to the Company under the original AbbVie Collaboration Agreement upon first patient dosed in the first registration enabling trial for DR SCS treatment, AbbVie will pay the Company $ 100.0 million upon first patient dosed in the NAAVIGATE trial and an additional $ 100.0 million upon first patient dosed in the subsequent Phase III trial.

The Company will be responsible for its development expenses to conduct Part 1 of the NAAVIGATE trial and the parties will share the development expenses related to Part 2 of the NAAVIGATE trial and the subsequent Phase III trial for DR in accordance with the existing terms of the AbbVie Collaboration Agreement.

AbbVie will be responsible for all development expenses related to the ACHIEVE study.

The AbbVie Collaboration Agreement contains provisions for termination, including termination for convenience by AbbVie.

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The intellectual property licensed to AbbVie includes the rights to certain patents, data, know-how and other rights developed and owned by the Company, as well as other intellectual property rights exclusively licensed by the Company from various third parties.

The Company evaluated options granted to AbbVie under the agreement and determined that the options do not represent material rights, and therefore are not considered separate performance obligations under the current contract.

Specifically, the Company concluded that the option granted to AbbVie to purchase commercial supply of ABBV-RGX-314 from the Company for a portion of sales outside the United States does not convey a material right, as the option is not priced at an incremental discount to the standalone selling price of the underlying goods and services.

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In accordance with the sale- or usage-based royalty exception under ASC 606, royalties on net sales and sales-based milestones will be recognized as revenue in the period the underlying sales occur or milestones are achieved.

There were no changes in the transaction price of the AbbVie Collaboration Agreement during the years ended December 31, 2024, 2023 and 2022.

There were no changes in the transaction price of the AbbVie Collaboration Agreement, and no revenue was recognized, during the years ended December 31, 2025 and 2024.

The Company applied the requirements of ASC 808 to the AbbVie Collaboration Agreement for the units of account which were deemed to be a collaborative arrangement.

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The Company is responsible for the development of RGX-121 and RGX-111 in the United States, and Nippon Shinyaku is responsible for development in licensed territories outside the United States.

The Company is responsible for the manufacturing of RGX-121 and RGX-111 for clinical development and commercial supply, and manufacturing expenses will be allocated between the parties in accordance with the terms of the Nippon Shinyaku Collaboration Agreement and supply agreements determined in accordance with the agreement.

Nippon Shinyaku will be responsible, at its sole cost, for the commercialization of RGX-121 and RGX-111 in the licensed territories.

The Company reserves the right to develop and commercialize RGX-121 and RGX-111 in countries outside the licensed territories.

In consideration for the rights granted and services to be performed under the Nippon Shinyaku Collaboration Agreement, Nippon Shinyaku is required to pay an up-front fee of $ 110.0 million to the Company following the effective date of the agreement, and the Company is eligible to receive up to $ 700.0 million from Nippon Shinyaku upon the achievement of specified development and sales-based milestones, of which $ 40.0 million are based on development milestones and $ 660.0 million are sales-based milestones.

The Company is also eligible to receive double-digit royalties on net sales of RGX-121 and RGX-111 by Nippon Shinyaku, subject to specified offsets and reductions.

The Nippon Shinyaku Collaboration Agreement contains provisions for termination, including termination for convenience by Nippon Shinyaku.

In consideration for the rights granted and services to be performed under the Nippon Shinyaku Collaboration Agreement, Nippon Shinyaku paid the Company an up-front fee of $ 110.0 million upon the effective date of the agreement in March 2025 and is required to pay to the Company up to $ 700.0 million upon the achievement of specified development and sales-based milestones, of which $ 40.0 million are based on development milestones and $ 660.0 million are sales-based milestones.

Nippon Shinyaku is also

required to pay to the Company double-digit royalties on net sales of RGX-121 and RGX-111 in the licensed territories, subject to specified offsets and reductions.

The Company retains all rights to, and any proceeds related to the sale of, any priority review vouchers that may be issued upon the potential approvals of RGX-121 and RGX-111.

The Company evaluated its various commitments under the Nippon Shinyaku Collaboration Agreement and identified the distinct units of account under the arrangement.

For each of the distinct units of account identified, the Company determined whether the transactions should be accounted for as a contract with a customer within the scope of ASC 606 or as a collaborative arrangement within the scope of ASC 808.

The Company concluded that each of the distinct units of account identified should be accounted for as revenue under ASC 606, as Nippon Shinyaku is deemed to be a customer for each of the various transactions.

The Company identified the following material performance obligations under the agreement:

The intellectual property licensed to Nippon Shinyaku includes the rights to certain patents, data, know-how and other rights developed and owned by the Company, as well as other intellectual property rights exclusively licensed by the Company from various third parties.

In determining the distinct performance obligations under the agreements, the Company concluded that the licenses granted to Nippon Shinyaku to develop and commercialize RGX-121 and RGX-111 are distinct from the other goods and services promised under the agreement, as Nippon Shinyaku can benefit from the licenses on a standalone basis and, based on the stage of development of the product candidates, the underlying licensed products and know-how are not expected to be significantly modified as a result of other goods and services promised under the agreement.

The Company evaluated all options granted to Nippon Shinyaku under the agreement to determine whether the options represent material rights.

Management concluded the options to purchase commercial supply convey material rights granted to Nippon Shinyaku, and therefore are accounted for as separate performance obligations under the current arrangement.

The Company identified various promises under the Nippon Shinyaku Collaboration Agreement which were determined to be immaterial in the context of the contract and will not be accounted for as separate performance obligations.

As of December 31, 2025, the transaction price of the Nippon Shinyaku Collaboration Agreement included fixed consideration of $ 110.0 million for the up-front payment and variable consideration of $ 4.1 million for estimated reimbursable costs of manufacturing and other services which are deemed not to be constrained.

Variable consideration which has been excluded from the transaction price includes $ 40.0 million in payments for development milestones that have not yet been achieved and were not considered probable of achievement, and reimbursable costs of manufacturing and other services which are contingent on events occurring that are outside the Company’s control and are deemed to be constrained.

The transaction price also excludes sales-based milestone payments of $ 660.0 million and royalties on net sales of RGX-121 and RGX-111 in the United States and Asia territories.

Development milestones will be added to the transaction price upon achievement, or if deemed probable of achievement, and other variable consideration for manufacturing and other services may be added to the transaction price in the future as uncertainties regarding payment of the consideration are resolved.

There were no changes in the fixed transaction price of the Nippon Shinyaku Collaboration Agreement between the effective date of the agreement and December 31, 2025.

The fixed transaction price of the Nippon Shinyaku Collaboration Agreement of $ 110.0 million was allocated to the various performance obligations based on their relative standalone selling prices, which requires significant judgment.

The selling prices of the intellectual property licenses were determined based on discounted cash flow models for each of the licensed products in the respective licensed territories and were adjusted for the probability of developmental, regulatory and commercial success.

The selling prices of development services and commercial supply were determined based on the expected cost plus a reasonable margin.

The selling prices of the material rights to purchase commercial supply were determined based on the incremental discount to the standalone selling prices of the commercial supply and were adjusted to consider the likelihood of exercise of the material rights.

Significant assumptions and judgments were required to estimate the future costs of development and manufacturing, an appropriate margin for such services, and the likelihood of exercise of the material rights to purchase commercial supply.

The $ 4.1 million of variable consideration included in the transaction price is allocated directly to performance obligations for the manufacturing of commercial supply prior to regulatory approval and other service obligations since the consideration is directly associated with the performance of such services and reimbursement of applicable costs.

Consideration contingent upon the future exercise of options to purchase commercial supply is excluded from the transaction price until exercised.

The portion of the $ 110.0 million fixed transaction price allocated to the delivery of the intellectual property licenses was recognized as license and royalty revenue upon the delivery of the licenses to Nippon Shinyaku in March 2025.

The portion of the fixed transaction price allocated to development services will be recognized as service revenue as the services are performed using an input method based on costs incurred versus total estimated costs to perform the services, which is re-assessed at each reporting date.

The portion of the fixed transaction price allocated to material rights to purchase commercial supply will be recognized as revenue proportionally with the total expected commercial supply revenue expected to be recognized under the arrangement, which is re-assessed at each reporting date.

Commercial supply revenue will be recognized as revenue upon delivery to Nippon Shinyaku, or otherwise upon transfer of control of commercial supply to Nippon Shinyaku as defined in the associated supply agreements.

The Company recognized the following amounts under the Nippon Shinyaku Collaboration Agreement (in thousands):

Years Ended December 31,

License and royalty revenue

Service revenue

Total revenues

As of December 31, 2025, the Company had recorded total accounts receivable of $ 4.1 million for reimbursable costs of manufacturing and other services under the Nippon Shinyaku Collaboration Agreement, of which $ 2.3 million was included in current assets and $ 1.9 million was included in non-current assets.

As of December 31, 2025, the Company had recorded total deferred revenue of $ 29.4 million for development services and material rights which have not yet been satisfied under the Nippon Shinyaku Collaboration Agreement, of which $ 10.5 million was included in current liabilities and $ 18.9 million was included in non-current liabilities.

Stock-based Compensation

In September 2014, the Board of Directors adopted the 2014 Stock Plan (the 2014 Plan).

In June 2015, the Board of Directors adopted the 2015 Equity Incentive Plan (the 2015 Plan), which became effective upon the Company’s initial public offering in September 2015.

The 2015 Plan replaced the 2014 Plan, and as of the effective date of the 2015 Plan, no further awards may be issued under the 2014 Plan.

Any options or awards outstanding under the 2014 Plan as of the effective date of the 2015 Plan remained outstanding and effective.

The number of authorized shares under the 2015 Plan automatically increases annually on the first business day of each fiscal year, by the lesser of (i) 4 % of the total number of shares of common stock outstanding on December 31 of the prior year, or (ii) a number of common shares determined by the Board of Directors.

As of December 31, 2024, the total number of shares of common stock authorized for issuance under the 2015 Plan and the 2014 Plan was 19,118,989 , of which 1,512,367 remained available for future grants under the 2015 Plan.

An additional 1,981,975 shares were authorized for issuance under the 2015 Plan effective in January 2025.

The 2014 Plan and 2015 Plan provide for the issuance of stock options, stock appreciation rights, restricted and unrestricted stock and unit awards, and performance cash awards to employees, members of the Board of Directors and consultants of the Company.

Since the inception of the plans, the Company has issued only stock options and restricted stock units under the plans.

Stock options under the 2014 Plan and 2015 Plan generally expire 10 years following the date of grant.

Options typically vest over a four-year period, but vesting provisions can vary by award based on the discretion of the Board of Directors.

Certain stock option

awards granted by the Company may include performance conditions that must be achieved in order for vesting to occur.

Stock options under the 2014 Plan and 2015 Plan have an exercise price at least equal to the estimated fair value of the Company’s common stock on the date of grant.

In September 2014, the Company adopted the 2014 Stock Plan (the 2014 Plan).

In June 2015, the Company adopted the 2015 Equity Incentive Plan (the 2015 Plan), which replaced the 2014 Plan effective upon the Company’s initial public offering in September 2015.

Upon the effective date of the 2015 Plan, no further awards may be granted under the 2014 Plan.

As of December 31, 2025, there were no awards outstanding under the 2014 Plan.

Effective in January 2025, an additional 1,981,975 shares were authorized for issuance under the 2015 Plan.

The 2015 Plan expired in June 2025 , upon which no further awards may be granted under the plan.

Any awards outstanding under the 2015 Plan as of the plan expiration date shall remain outstanding and effective pursuant to the contractual terms of the awards.

In May 2025, the Company adopted the 2025 Equity Incentive Plan (the 2025 Plan), which replaced the 2015 Plan upon its expiration in June 2025.

The total number of shares of common stock authorized for issuance under the 2025 Plan upon its adoption was 5,500,000 .

The number of shares authorized for issuance under the 2025 Plan shall automatically increase for any shares of common stock underlying awards outstanding under the 2015 Plan, as of the adoption date of the 2025 Plan, which are not issued due to forfeiture, expiration, termination or cancellation of the award.

Shares of common stock that are withheld, tendered, or otherwise not issued in connection with the settlement of awards outstanding under the 2015 Plan do not increase the number of shares authorized for issuance under the 2025 Plan.

As of December 31, 2025, the total number of shares of common stock reserved for issuance under the 2025 Plan and 2015 Plan was 20,133,416 , of which 5,717,654 remained available for future grants under the 2025 Plan.

The 2014 Plan, 2015 Plan and 2025 Plan provide for the issuance of stock options, stock appreciation rights, restricted and unrestricted stock and unit awards, and performance cash awards to employees, members of the Board of Directors and consultants of the Company.

As of December 31, 2025, the Company has issued only stock options and restricted stock units under the plans.

Stock options generally expire 10 years following the date of grant.

Stock options typically vest over a four-year period, but vesting provisions can vary by award based on the discretion of the Board of Directors.

Stock options have an exercise price at least equal to the estimated fair value of the Company’s common stock on the date of grant.

Restricted stock units typically vest over a four-year period, but vesting provisions can vary by award based on the discretion of the Board of Directors.

Upon vesting, restricted stock units are settled in common stock of the Company.

Shares of common stock underlying awards previously issued under the 2014 Plan and 2015 Plan which are reacquired by the Company, withheld by the Company in payment of the purchase price, exercise price or withholding taxes, expired, cancelled due to forfeiture or otherwise terminated other than by exercise or settlement, are added to the number of shares of common stock available for issuance under the 2015 Plan.

Shares available for issuance under the 2015 Plan may be either authorized but unissued shares of the Company’s common stock or common stock reacquired by the Company and held in treasury.

The 2015 Plan expires in June 2025 , 10 years from the date it was adopted by the Board of Directors, unless earlier terminated.

Awards granted under the 2025 Plan generally have a minimum vesting requirement of one year from the grant date.

Shares of common stock underlying awards granted under the 2025 Plan which are not issued due to forfeiture, expiration, termination or cancellation of the award are added to the number of shares of common stock available for issuance under the 2025 Plan, except for shares that are withheld, tendered or otherwise not issued in connection with the settlement of the award.

Shares available for issuance under the 2025 Plan and 2015 Plan may be either authorized but unissued shares of the Company’s common stock or common stock reacquired by the Company and held in treasury.

The 2025 Plan expires in May 2035 , 10 years from its adoption date, unless earlier terminated.

Stock-based Compensation Expense

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Stock Options

The following table summarizes stock option activity under the 2014 Plan and 2015 Plan (in thousands, except per share data):

The following table summarizes stock option activity under the Company's equity incentive plans (in thousands, except per share data):

Outstanding at December 31, 2024

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The weighted-average grant date fair value per share of options granted during the years ended December 31, 2025 and 2024 was $ 4.96 and $ 9.98 , respectively.

During the years ended December 31, 2024, 2023 and 2022, the total number of stock options exercised was 290,753 , 222,935 and 331,912 , respectively, resulting in total proceeds of $ 1.5 million, $ 1.5 million and $ 2.8 million, respectively.

The total intrinsic value of options exercised during the years ended December 31, 2024, 2023 and 2022 was $ 3.1 million, $ 2.9 million and $ 6.9 million, respectively.

During the years ended December 31, 2025 and 2024, the total number of stock options exercised was 67,735 and 290,753 , respectively, resulting in total proceeds of $ 0.3 million and $ 1.5 million, respectively.

The total intrinsic value of options exercised during the years ended December 31, 2025 and 2024 was $ 0.3 million and $ 3.1 million, respectively.

The fair values of options granted were estimated at each grant date using the Black-Scholes valuation model with the following weighted-average assumptions:

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Restricted Stock Units

The following table summarizes restricted stock unit activity under the 2015 Plan (in thousands, except per share data):

The following table summarizes restricted stock unit activity under the Company's equity incentive plans (in thousands, except per share data):

Weighted-average

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Unvested balance at December 31, 2025

The total intrinsic value of restricted stock units vested during the years ended December 31, 2024, 2023 and 2022 was $ 6.7 million, $ 3.9 million and $ 2.2 million, respectively.

The total intrinsic value of restricted stock units vested during the years ended December 31, 2025 and 2024 was $ 6.0 million and $ 6.7 million, respectively.

Employee Stock Purchase Plan

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The Company matches employee deferrals up to a specified percentage of eligible compensation.

For the years ended December 31, 2024, 2023 and 2022, the Company incurred expenses of $ 3.1 million, $ 3.3 million and $ 3.0 million, respectively, for matching contributions to the 401(k) Plan.

For the years ended December 31, 2025 and 2024, the Company incurred expenses of $ 3.4 million and $ 3.1 million, respectively, for matching contributions to the 401(k) Plan.

The components of loss before income taxes were as follows (in thousands):

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Total loss before income taxes

The components of the provision for income tax expense (benefit) were as follows (in thousands):

Due to taxable losses and a full valuation allowance against its deferred tax assets, the Company did no t record a provision for income taxes in the U.S.

(federal or state) or any foreign jurisdictions for the years ended December 31, 2025 and 2024.

Net income taxes paid (net refunds received) were as follows (in thousands):

Years Ended December 31,

Total current

Total deferred

Total income tax expense (benefit)

Beginning in 2022, the Tax Cuts and Jobs Act of 2017 (the TCJA) eliminated the option to deduct research and development expenses currently and requires taxpayers to amortize such costs over a period of five years for expenses incurred in the United States and a period of 15 years for expenses incurred outside the United States.

This provision of the TCJA resulted in deferred tax assets of $ 122.1 million and $ 107.3 million as of December 31, 2024 and 2023, respectively, related to capitalized research and development expenses, net of amounts amortized to date.

There was no material impact to the Company's current or deferred tax provision or operating cash flows during the years ended December 31, 2024, 2023 and 2022 as a result of this provision of the TCJA given the Company incurred net operating losses (NOLs) during the periods and has recorded a full valuation allowance against its deferred tax assets.

The Inflation Reduction Act (the IRA) was enacted in August 2022 and contains revenue-raising provisions to include a book-income alternative minimum tax and an excise tax on stock buybacks, among other provisions.

Based on the thresholds established by the IRA and a review of the Company’s transactions, the enactments of the IRA did not have an impact on the Company’s income tax provision for the years ended December 31, 2024, 2023 and 2022.

Total net income taxes paid (net refunds received):

Beginning in 2022, the Tax Cuts and Jobs Act of 2017 (the TCJA) eliminated the option to deduct research and development expenses currently and required taxpayers to amortize such costs over a period of five years for expenses incurred in the United States and a period of 15 years for expenses incurred outside the United States.

This provision of the TCJA resulted in deferred tax assets of $ 122.1 million as of December 31, 2024 related to capitalized research and development expenses, net of amounts amortized to date.

The One Big Beautiful Bill Act (OBBBA) was enacted in July 2025.

The OBBBA amended U.S.

federal tax laws by restoring the option for immediate expense recognition of research and development expenses incurred in the United States and making permanent the ability to claim 100% bonus depreciation on qualified property, among other changes.

The Company currently expects to elect these options and deduct U.S.

research and development expenses incurred in the current period, along with 100% bonus depreciation on eligible property placed in service, beginning in 2025.

Research and development expenses incurred outside the United States will continue to be capitalized and amortized over a period of 15 years for tax purposes.

Unamortized U.S.

research and development expenses incurred prior to 2025 may be amortized over their remaining life, or over a period of one or two years beginning in 2025.

The Company currently expects to amortize these amounts over two years beginning in 2025.

The enactment of the OBBBA resulted in a decrease in deferred tax assets for capitalized research and development expenses and an increase in deferred tax assets for net operating loss (NOL) carryforwards during the year ended December 31, 2025.

However, due to taxable losses and a full valuation allowance against its deferred tax assets, the enactment of the OBBBA had no material impact to the Company's income tax provision for the year ended December 31, 2025.

The Company continues to evaluate the impact the OBBBA will have on its consolidated financial statements in future periods.

The Organization for Economic Co-operation and Development (OECD) has introduced BEPS Pillar 2 rules that impose a global minimum tax rate of 15 %.

Numerous countries have enacted corresponding legislation that is effective beginning January 1, 2024.

Numerous countries enacted corresponding legislation that is effective at various dates beginning as early as January 1, 2024.

These rules generally apply to multinational companies with consolidated revenue of at least € 750.0 million in at least two of the four preceding fiscal years.

Based on the revenue thresholds established in the BEPS Pillar 2 rules, these changes do not have an impact on the Company’s income tax provision for the year ended December 31, 2024.

The following table presents a reconciliation of income tax expense (benefit) computed at the statutory federal income tax rate of 21 % to income tax expense (benefit) reported in the consolidated statements of operations and comprehensive loss (in thousands):

Years Ended December 31,

Federal income tax benefit at statutory rate

State income tax benefit, net of federal tax effect

Research and development credits

Based on the revenue thresholds established in the BEPS Pillar 2 rules, these changes do not have an impact on the Company’s income tax provision for the years ended December 31, 2025 and 2024.

Further, under administrative guidance issued in January 2026, even if the Company were to exceed the applicable revenue threshold in future periods, it would be eligible to elect the Side-by-Side Safe Harbor, which is expected to mitigate the impact of the Pillar 2 rules on the Company’s income tax provision.

The following table presents a reconciliation of the U.S.

federal statutory tax rate to the Company's effective tax rate for the years ended December 31, 2025 and 2024, presented in accordance with ASU 2023-09 (dollars in thousands):

Year Ended December 31, 2025

Year Ended December 31, 2024

federal statutory tax rate

State and local income tax benefit, net of federal tax effect

Foreign tax effects:

Effect of changes in tax laws or rates enacted in the current period

Effects of cross-border tax laws

Research and development and orphan drug tax credits

Other tax credits

Change in valuation allowance

Nontaxable or nondeductible items:

Stock-based compensation

Executive compensation

Other non-deductible expenses and reconciling items

Change in corporate tax rates

Change in valuation allowance

Total income tax expense (benefit)

Changes in unrecognized tax benefits

Other adjustments

Effective tax rate

The significant components of the Company’s net deferred tax assets were as follows (in thousands):

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Based on the Company’s history of operating losses, and other relevant facts and circumstances, the Company concluded that it was more likely than not that the benefit of its deferred tax assets will not be realized.

Accordingly, the Company provided a full valuation allowance for its net deferred tax assets as of December 31, 2024 and 2023.

Accordingly, the Company provided a full valuation allowance for its deferred tax assets as of December 31, 2025 and 2024.

The valuation allowance increased by $ 36.1 million and $ 57.9 million during the years ended December 31, 2025 and 2024, respectively.

The increases in the valuation allowance during the years ended December 31, 2024 and 2023 were due primarily to research and development expenses incurred during the periods which were capitalized for tax purposes, and federal and state NOLs and research and development tax credits generated during the periods.

The increase in the valuation allowance during the year ended December 31, 2025 was due primarily to federal and state NOLs and research and development tax credits generated during the period, partially offset by a decrease in capitalized research and development expenses as a result of the enactment of the OBBBA.

The increase in the valuation allowance during the year ended December 31, 2024 was

due primarily to an increase in capitalized research and development expenses, and federal and state NOLs and research and development tax credits generated during the period.

The following table presents the Company's U.S.

federal and state NOL and tax credit carryforwards, net of unrecognized tax benefits, which may be available to offset future income tax liabilities (in thousands):

federal and state NOL and tax credit carryforwards which may be available to offset future income tax liabilities (in thousands):

As of December 31, 2025

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As of December 31, 2025 , the Company had U.S.

federal and state research and development tax credit carryforwards of approximately $ 87.2 million, net of unrecognized tax benefits of $ 0.1 million, which may be available to reduce future income tax liabilities.

federal and state research and development tax credit carryforwards of approximately $ 95.1 million which may be available to reduce future income tax liabilities.

The calculation of these credits requires assumptions to be made by the Company to estimate qualified research expenses.

1 unchanged sentence

The Company accounts for uncertain tax positions in accordance with the requirements of ASC 740, and recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized.

As of December 31, 2024 and 2023 , the Company had total unrecognized tax benefits of $ 0.1 million and $ 0.1 million, respectively, which were reserved against its research and development tax credit carryforwards as uncertain tax positions.

Further, a full valuation allowance has been provided against the net credit carryforwards and, if an adjustment is required upon the completion of the study, this adjustment would be offset by an adjustment to the deferred tax asset established for the research and development credit carryforwards and the valuation allowance.

If these unrecognized tax benefits were to be recognized, the impact would be offset by an adjustment to the valuation allowance, resulting in no impact on the Company’s effective tax rate.

The Company expects that all of its unrecognized tax benefits as of December 31, 2024 will reverse in the next 12 months due to the expiration of the associated statute of limitations.

As of December 31, 2024 , the Company had total unrecognized tax benefits of $ 0.1 million which were reserved against its research and development tax credit carryforwards as uncertain tax positions.

The Company released all of its reserves for unrecognized tax benefits during the year ended December 31, 2025 due to the expiration of the associated statute of limitations, and had no unrecognized tax benefits as of December 31, 2025.

Under the provisions of the Internal Revenue Code, the Company’s NOL and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities.

8 unchanged sentences

Restructuring

In November 2023, the Company implemented a strategic pipeline prioritization and corporate restructuring designed to reduce operating expenses and prioritize the development of ABBV-RGX-314, RGX-202 for the treatment of Duchenne muscular dystrophy, and RGX-121, while pursuing strategic alternatives for the Company's other clinical stage programs.

The restructuring included a reduction in workforce and other planned operating expenses, primarily in rare neurodegenerative disease development, early research and other general and administrative areas.

In connection with the restructuring, the Company implemented a reduction in workforce of approximately 15 %, which was substantially completed in the fourth quarter of 2023.

The Company recorded restructuring costs of $ 3.7 million during the year ended December 31, 2023, of which $ 3.0 million is included in research and development expense and $ 0.7 million is included in general administrative expense in the consolidated statements of operations and comprehensive loss.

Restructuring costs primarily consisted of employee severance, continuing healthcare benefits and other employee-related costs.

The Company recorded restructuring costs of $ 3.7 million during the year ended December 31, 2023, which primarily consisted of employee severance, continuing healthcare benefits and other employee-related costs.

Restructuring costs associated with one-time termination benefits were recorded pursuant to ASC 420, while restructuring costs associated with ongoing benefit arrangements were recorded pursuant to ASC 712.

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As of December 31, 2024, all of the restructuring costs had been paid by the Company and no restructuring liability was recorded.

No restructuring costs were recorded during the years ended December 31, 2025 and 2024.

The following table presents the changes in the Company's restructuring liability (in thousands):

3 unchanged sentences

Cash payments

Other adjustments

Balance at December 31, 2024

Restructuring charges

Cash payments

Other adjustments

Balance at December 31, 2025

Related Party Transactions

From 2016 until June 2022, the Company was a party to professional services agreements with FOXKISER LLP (FOXKISER), an affiliate of certain stockholders of the Company and an affiliate of a member of the Company’s Board of Directors, pursuant to which the Company paid a fixed monthly fee in consideration for certain strategic services provided by FOXKISER.

The agreement with FOXKISER was terminated effective June 2022.

Expenses incurred under the agreement with FOXKISER for the year ended December 31, 2022 were $ 2.4 million and were recorded as research and development expenses in the consolidated statements of operations and comprehensive loss.

No expenses under the agreement with FOXKISER were incurred during the years ended December 31, 2024 and 2023.

Net Loss Per Share

7 unchanged sentences

Employee stock purchase plan

May 2025 Warrants outstanding

Segment and Geographical Information

8 unchanged sentences

Years Ended December 31,

Cost of revenues

Cost of license and royalty revenues

Research and development expense

11 unchanged sentences

(b) Other general and administrative expenses include professional and administrative services, consulting, commercial cost reimbursement to and from collaborators and other corporate overhead expenses.

(c) Other segment items include credit losses (recoveries), impairment of long-lived assets, other operating expenses (income), interest income from licensing, investment income, interest expense and income tax benefit.

The Company’s interest income during the years ended December 31, 2024, 2023 and 2022 included interest income from licensing as presented in the consolidated statements of operations and comprehensive loss, as well as interest income from investments of $ 12.1 million, $ 9.1 million and $ 5.5 million, respectively, which is included within investment income in the consolidated statements of operations and comprehensive loss.

The Company’s revenues consist of license and royalty revenue.

(c) Other segment items include credit losses (recoveries), impairment of long-lived assets, other operating expenses, interest income from licensing, investment income and interest expense.

The Company’s interest income during the years ended December 31, 2025 and 2024 included interest income from licensing as presented in the consolidated statements of operations and comprehensive loss, as well as interest income from investments of $ 12.2 million and $ 12.1 million, respectively, which is included within investment income in the consolidated statements of operations and comprehensive loss.

For the year ended December 31, 2025 , 44 % and 25 % of the Company’s revenues were attributed to Japan and the United States, respectively, and no other countries accounted for 10 % or more of the Company’s revenues.

For the year ended December 31, 2024 , 36 %, 10 % and 10 % of the Company’s revenues were attributed to the United States, Germany and United Arab Emirates, respectively, and no other countries accounted for 10 % or more of the Company’s revenues.

For the year ended December 31, 2023 , 32 % of the Company’s revenues were attributed to the United States and no other countries accounted for 10 % or more of the Company’s revenues.

For the year ended December 31, 2022 , 35 % and 10 % of the Company’s revenues were attributed to the United States and Germany, respectively, and no other countries accounted for 10 % or more of the Company’s revenues.

The country of origin for license revenue is determined based on the country of domicile of the licensee.

The country of origin for royalty revenue is determined based on the location of the underlying net sales of licensed products.

The country of origin for service revenue is determined based on the location where the Company principally performs the services.

The substantial majority of the Company’s assets reside in the United States.

3 unchanged sentences

As of December 31,

Net cost reimbursement due from collaborators

Net cost reimbursement due from AbbVie

Accrued interest on investments

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Accrued purchases of property and equipment

Other accrued expenses and current liabilities

Supplemental Disclosures of Non-cash Investing and Financing Activities

Purchases of property and equipment included in accounts payable and accrued expenses and other current liabilities as of December 31, 2024 were $ 0.3 million, a net decrease of $ 0.1 million from December 31, 2023.

Purchases of property and equipment included in accounts payable and accrued expenses and other current liabilities as of December 31, 2023 were $ 0.4 million, a net decrease of $ 2.1 million from December 31, 2022.

Purchases of property and equipment included in accounts payable and accrued expenses and other current liabilities as of December 31, 2022 were $ 2.5 million, a net decrease of $ 7.5 million from December 31, 2021.

Offering expenses for at-the-market offering programs included in accounts payable and accrued expenses and other liabilities as of December 31, 2024 and 2023 were $ 0.2 million and less than $ 0.1 million, respectively.

No such amounts were recorded as of December 31, 2022.

During the year ended December 31, 2024, the Company derecognized $ 0.6 million in deferred offering costs upon the termination of the BofA ATM Program in November 2024.

EXHIBIT INDEX

6 unchanged sentences

Form of Pre-funded Warrant

REGENXBIO Inc.

Warrant to Purchase Common Stock

Form of Indemnity Agreement for directors and officers

2014 Stock Plan, as amended

2015 Equity Incentive Plan

1 unchanged sentence

Form of Stock Option Award Agreement for the 2015 Equity Incentive Plan

REGENXBIO Inc.

2025 Equity Incentive Plan

Form of Restricted Stock Unit Award Agreement for the 2025 Equity Incentive Plan

Form of Stock Option Award Agreement for the 2025 Equity Incentive Plan

2015 Employee Stock Purchase Plan

1 unchanged sentence

Form of Employment Agreement for Executive Vice Presidents

Consulting and Employment Separation Agreement effective as of July 1, 2024 between the Registration and Kenneth T.

Employment Separation Agreement effective as of September 21, 2024 between the Registrant and Vittal Vasista

Consulting Agreement effective as of September 21, 2024 between the Registrant and Vittal Vasista

Compensation Program for Non-Employee Directors

13 unchanged sentences

Collaboration and License Agreement dated September 10, 2021 between the Registrant and AbbVie Global Enterprises Ltd.

First Amendment to Collaboration and License Agreement dated August 5, 2025 between the Registrant and AbbVie Global Enterprises Ltd.

Collaboration and License Agreement dated January 14, 2025 between the Registrant and Nippon Shinyaku Co., Ltd.

Lease dated March 6, 2015 between the Registrant and BMR-Medical Center Drive LLC

12 unchanged sentences

Royalty Purchase Agreement dated December 22, 2020 between the Registrant and entities managed by Healthcare Royalty Management, LLC

Loan Agreement dated May 16, 2025 between REGENXBIO RS LLC and affiliate of Healthcare Royalty Management, LLC

Insider Trading Policy

54 unchanged sentences

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 7. Management’s Discussion and Analysis