What changed in this filing

/stocks — the workspaceLOADING

Item 7. Management’s Discussion and Analysis

2025-03-13 compared with 2024-02-27 · 473 added, 355 removed, 1407 unchanged (37% of the section changed)

7 unchanged sentences

Overview of Product Candidates

We have developed a broad pipeline of gene therapy programs using our proprietary adeno-associated virus (AAV) gene therapy delivery platform (NAV Technology Platform) to address genetic diseases.

Our programs and product candidates are described below:

We have developed a broad pipeline of gene therapy programs using our proprietary adeno-associated virus (AAV) gene therapy delivery platform (NAV Technology Platform) as a one-time treatment to address an array of diseases.

Our lead programs and product candidates are described below:

• ABBV-RGX-314:

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

ABBV-RGX-314 is currently being evaluated in nine ongoing clinical trials in the United States and Canada.

Such ongoing clinical trials include two pivotal trials, one Phase II bridging study, one Long-term Follow-up study, and a Fellow Eye Treatment study in patients with wet AMD, all utilizing subretinal delivery, as well as two Phase II clinical trials in patients with wet AMD and DR are also ongoing along with two corresponding Long-term Follow-up studies, all utilizing in-office suprachoroidal delivery.

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).

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.

Additionally, two Phase II clinical trials in patients with wet AMD (AAVIATE) and DR (ALTITUDE) are ongoing along with two corresponding long-term follow-up studies, all utilizing in-office suprachoroidal delivery.

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

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

1 unchanged sentence

(Clearside) to deliver gene therapy treatments to the suprachoroidal space of the eye.

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

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.

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

Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in late 2025 through the first half of 2026.

We are also evaluating the pharmacodynamics, safety and efficacy of ABBV-RGX-314 in patients with wet AMD using the subretinal delivery approach in a Phase II bridging study using Good Manufacturing Practices (cGMP) material produced by our NAVXpress bioreactor platform process.

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

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

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.

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 for the treatment of DR.

We are developing RGX-202 as an investigational one-time 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 (CT) domain as well as a muscle-specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne.

AFFINITY DUCHENNE is a multicenter, open-label dose evaluation and dose expansion clinical trial to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in patients with Duchenne.

In February 2024, we reported interim data from the trial, demonstrating that RGX-202 continued to be well tolerated with

no drug-related serious adverse events in five patients at dose levels 1 and 2.

Initial biomarker data in three patients who completed three-month assessments indicate encouraging increases in expression of RGX-202 microdystrophin and reduction from baseline in serum creatinine kinase levels, supporting evidence of clinical improvement.

We expect to make a pivotal dose determination in mid-2024.

We expect to share initial strength and functional assessment data for both dose levels and the initiation of a pivotal trial in the second half of 2024.

We plan to use RGX-202 microdystrophin expression as a surrogate endpoint to support a Biologics License Application (BLA) filing using the accelerated approval pathway.

The AFFINITY BEYOND trial, an observational screening study, is also active and recruiting patients.

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.

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.

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.

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.

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.

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

The primary objective is to evaluate the prevalence of AAV8 antibodies in patients with Duchenne up to 12 years of age.

Information collected in this study may be used to identify potential participants for the AFFINITY DUCHENNE trial and potential future trials of RGX-202.

We are developing RGX-121 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.

CAMPSIITE ® is a Phase I/II/III multi-center, open-label trial to evaluate the efficacy, safety, tolerability and pharmacodynamics of RGX-121 in patients with MPS II aged 4 months up to 5 years old.

We continue to follow patients in the trial, and in February 2024, we reported that the pivotal phase of the CAMPSIITE trial achieved its primary endpoint.

We plan to use levels of cerebrospinal fluid D2S6 as a surrogate endpoint for accelerated approval and we are completing remaining activities in order to support a BLA submission in the second half of 2024.

We believe that RGX-121 is likely to be eligible for priority review, especially if no other gene therapy product for MPS II is approved before submission of a BLA for RGX-121, and potential approval of the Company's planned BLA for RGX-121 could result in receipt of a Rare Pediatric Disease Priority Review Voucher in 2025, assuming the statutory criteria are met.

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.

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

We are developing RGX-111 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 I (MPS I), also known as Hurler syndrome, using the NAV AAV9 vector to deliver the IDUA gene.

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.

Further, we will be seeking strategic alternatives, including potential partnering, for our other clinical stage product candidates:

(i) RGX-111 for the treatment of Mucopolysaccharidosis Type I (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.

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.

1 unchanged sentence

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

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).

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.

The Company will lead the manufacturing of ABBV-RGX-314 for clinical development and U.S.

commercial supply, and AbbVie will lead the global commercialization of ABBV-RGX-314.

We received an up-front fee of $370.0 million from AbbVie upon the effective date of the AbbVie Collaboration 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.

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

(Nippon Shinyaku) for the development and commercialization of RGX-121 and RGX-111 (the Nippon Shinyaku Collaboration Agreement).

Pursuant to the Nippon Shinyaku Collaboration Agreement, we are 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.

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.

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.

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

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.

Financial Overview

3 unchanged sentences

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.

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 the NAV Technology Platform and other licensed rights.

1 unchanged sentence

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 and (v) other consideration payable upon optional goods and services purchased by licensees.

(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.

2 unchanged sentences

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

Please refer to Note 2 to our audited consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K for a description of segment and geographical information regarding our revenues.

Please refer to Note 17, “Segment and Geographical Information” to the accompanying audited consolidated financial statements for a description of segment and geographical information regarding our revenues.

Zolgensma Royalties

2 unchanged sentences

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.

Collaboration and License Agreement with AbbVie

Effective in November 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).

We recognized license and royalty revenue of $370.0 million upon the effective date of the collaboration in November 2021.

The AbbVie Collaboration Agreement may materially impact our future revenues, research and development expenses, other operating expenses and operating cash flows associated with the development and commercialization of ABBV-RGX-314.

Operating Expenses

23 unchanged sentences

Unallocated Expenses

Platform and new technologies

Personnel-related

Facilities and depreciation expense

Other unallocated

Platform and early research

Stock-based compensation

Depreciation and amortization

Total unallocated expenses

Total research and development

Direct expenses related to the development of ABBV-RGX-314 for the years ended December 31, 2023, 2022 and 2021 include $74.2 million, $19.3 million and $5.9 million, respectively, in net cost reimbursement from AbbVie under our eye care collaboration which were recorded as a reduction of research and development expenses.

* 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.

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

In general, we do not allocate personnel and other internal costs, such as facilities and other overhead costs, to specific product candidates or development programs.

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

Direct expenses related to the development of RGX-111, RGX-181 and RGX-381 are included in other product candidates in the table above.

While we have discontinued internal development and are seeking strategic alternatives for these product candidates, we expect to continue to incur development expenses associated with long-term follow up studies for these product candidates.

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.

General and Administrative Expense

1 unchanged sentence

This includes certain personnel in executive, commercial, corporate development, finance, legal, human resources, information technology, facilities and administrative support functions.

Additionally, general and administrative expenses include facility-related and overhead costs not otherwise allocated to research and development expense, professional fees for accounting, legal, commercial and other advisory services, expenses associated with obtaining and maintaining patents, insurance costs, costs of our information systems and other

general corporate activities.

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

Additionally, general and administrative expenses include costs associated with accounting, legal, commercial and other corporate advisory services, obtaining and maintaining patents, insurance, information systems and other general corporate activities, as well as facility-related costs and other corporate overhead costs not otherwise allocated to research and development expense.

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

Other Income (Expense)

Interest Income from Licensing

In accordance with our revenue recognition policy, interest income from licensing consists of imputed interest recognized from significant financing components identified in our license agreements with NAV Technology Licensees as well as interest income accrued on unpaid balances due from licensees.

Investment Income

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

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.

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

9 unchanged sentences

Actual results may differ materially from these estimates under different assumptions or conditions.

Our significant accounting policies are fully described in Note 2 to our audited consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K.

Our significant accounting policies are fully described in Note 2, “Summary of Significant Accounting Policies” to our audited consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K.

We believe the following accounting policies are critical to the process of making significant judgments and estimates in the preparation of our financial statements and understanding and evaluating our reported financial results.

2 unchanged sentences

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 of ASC 606:

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

(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.

11 unchanged sentences

Consideration payable to us 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.

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.

14 unchanged sentences

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.

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.

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 includes up-front and annual fees payable during the contract term.

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

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.

23 unchanged sentences

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.

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.

6 unchanged sentences

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

For additional information regarding our collaborative arrangements, including our ABBV-RGX-314 collaboration with AbbVie which became effective in November 2021, please refer to Note 10, “License and Collaboration Agreements” to the accompanying audited consolidated financial statements.

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.

Accrued Research and Development Expenses

17 unchanged sentences

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 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.

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

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.

Due to the lack of sufficient historical data, we estimate the expected term of our employee stock options using the “simplified” method, whereby the expected term equals the arithmetic average of the vesting term and the original contractual term of the option.

31 unchanged sentences

Credit losses (recoveries)

Impairment of long-lived assets

Other operating expenses (income)

Total operating expenses

Income (loss) from operations

Loss from operations

Other Income (Expense)

3 unchanged sentences

Total other income (expense)

Income (loss) before income taxes

Income Tax Benefit (Expense)

Net income (loss)

Loss before income taxes

Income Tax Benefit

Comparison of the Years Ended December 31, 2024 and 2023

1 unchanged sentence

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 Zolgensma royalty revenues, which decreased by $16.6 million, from $101.9 million in 2022 to $85.3 million in 2023.

As reported by Novartis, sales of Zolgensma in 2023 decreased by 11% (USD) as compared to 2022, and established markets are now treating mainly incident patients.

Cost of Revenues.

Cost of revenues decreased by $17.3 million, from $54.5 million for the year ended December 31, 2022 to $37.2 million for the year ended December 31, 2023.

The decrease was largely attributable to a non-recurring charge of $9.2 million recognized in the first quarter of 2022 related to the amendment of our license agreement with The Trustees of the University of Pennsylvania (Penn) to buy out our obligation to pay sublicense fees to Penn under the license agreement.

The remaining decrease in cost of revenues was primarily attributable to a reduction in upstream royalties payable to licensors on net sales of Zolgensma during the period.

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.

Research and Development Expense.

1 unchanged sentence

The decrease was primarily attributable to the following:

• a decrease of $11.6 million in manufacturing expenses and other costs of clinical supply for our lead product candidates, largely driven by ABBV-RGX-314 and RGX-202 clinical supply;

• a decrease of $8.4 million in costs associated with clinical trial and regulatory activities, largely driven by an increase in net development cost reimbursement from AbbVie under our ABBV-RGX-314 collaboration, and partially offset by increases in clinical trial expenses for RGX-121 and RGX-202;

• a decrease of $2.2 million in costs associated with preclinical activities and other early stage research and development.

The decrease in research and development expenses was partially offset by the following:

• an increase of $7.2 million in costs for laboratories and facilities used by research and development personnel, including a $4.4 million increase in depreciation expense allocated to research and development functions, largely driven by the activation of our cGMP facility in mid-2022;

• an increase of $4.7 million in personnel-related costs for research and development personnel, net of a $0.8 million decrease in stock-based compensation expense, largely driven by $3.0 million in restructuring charges for employee severance and benefits recognized in the fourth quarter of 2023.

The decrease in research and development expenses for ABBV-RGX-314 was largely driven by a shift in the development cost sharing arrangement under our collaboration with AbbVie beginning in 2023.

In accordance with the AbbVie Collaboration Agreement, through December 31, 2022 we were responsible for development expenses related to certain ongoing clinical trials of ABBV-RGX-314 and the remaining ABBV-RGX-314 development expenses were shared with AbbVie.

Beginning in 2023, AbbVie became responsible for the majority of all ABBV-RGX-314 development expenses.

• 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.

General and Administrative Expense.

General and administrative expenses increased by $3.2 million, from $85.3 million for the year ended December 31, 2022 to $88.5 million for the year ended December 31, 2023.

The increase was primarily attributable to personnel-related costs, professional fees for corporate advisory services and other corporate overhead expenses.

Other Operating Expenses (Income).

Other operating expenses were $0.4 million for the year ended December 31, 2023, as compared to other operating income of $6.7 million for the year ended December 31, 2022.

The change was primarily attributable to proceeds of $7.5 million received under a settlement agreement with a third party in the fourth quarter of 2022 that released certain claims regarding infringement of the Company's intellectual property.

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.

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.

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

For further information regarding the settlement agreement with Abeona and the allowance for credit losses, please refer to Note 10, “License and Collaboration Agreements – Settlement Agreement with Abeona Therapeutics” to the accompanying audited consolidated financial statements.

We did not record any credit losses or recoveries during the year ended December 31, 2023.

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 a realized gain of $2.2 million recognized in 2023 upon the achievement of milestones associated with the acquisition of our non-marketable equity securities of Corlieve Therapeutics SAS (Corlieve) by uniQure N.V.

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.

Interest Expense .

Interest expense decreased by $16.4 million, from $23.3 million for the year ended December 31, 2022 to $6.9 million for the year ended December 31, 2023.

The decrease was primarily attributable to a lower balance in our liability related to the sale of future royalties resulting from Zolgensma royalties paid to HCR, as well as changes in the effective interest rate of the liability resulting from changes in the estimated royalties forecasted to be paid to HCR over the life of the royalty purchase agreement.

Liquidity and Capital Resources

Sources of Liquidity

As of December 31, 2023, we had cash, cash equivalents and marketable securities of $314.1 million, which were primarily derived from the sale of our common stock and license fees received under the AbbVie Collaboration Agreement.

We expect that our cash, cash equivalents and marketable securities as of December 31, 2023, 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.

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:

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

• 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.

• In January 2021, we completed a public offering of 4,899,000 shares of our common stock (inclusive of 639,000 shares pursuant to the full exercise by the underwriters of their option to purchase additional shares) at a price of $47.00 per share.

The aggregate net proceeds from the offering, inclusive of the underwriters’ option exercise, were $216.1 million, net of underwriting discounts and commissions and offering expenses payable by us.

• 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.

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.

While we expect the pipeline prioritization and

corporate restructuring implemented in November 2023 to result in cost savings, we may also incur other charges or cash expenditures not currently contemplated due to events that may occur as a result of, or associated with, the restructuring.

In addition, we may not achieve the expected benefits of any cost reduction measures on our currently anticipated timeline, or at all.

Furthermore, 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 Program

On September 1, 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 ATM Program).

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

As of December 31, 2023, no shares of common stock had been sold under the ATM Program.

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.

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.

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).

As of December 31, 2024, no shares of common stock had been sold under the Leerink ATM Program.

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

Private Placement

On July 7, 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.

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.

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

Years Ended December 31,

Net cash provided by (used in) operating activities

Net cash used in operating activities

Net cash provided by (used in) investing activities

2 unchanged sentences

Cash Flows from Operating Activities

Our net cash used in operating activities for the year ended December 31, 2023 increased by $10.9 million from the year ended December 31, 2022.

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.

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, 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.

The changes in operating assets and liabilities include a decrease in total accounts receivable of $9.7 million, which was driven largely by a decrease in Zolgensma royalties receivable and the full collection of amounts due under our settlement agreement with Abeona, for which we recorded a $5.0 million credit recovery during the period.

The changes in operating assets and liabilities also include a total decrease in prepaid expenses and other current assets of $12.0 million, which was driven primarily by decreases in prepaid fees to CROs and net cost reimbursement due from AbbVie under our ABBV-RGX-314 collaboration.

The favorable changes in operating assets and liabilities were partially offset by a decrease in accrued expenses and other current liabilities of $12.1 million, which was driven primarily by decreases in accruals for external research and development services and sublicense and royalties due to licensors.

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 assets and liabilities occurred in the normal course of business as a result of changes in operating working capital.

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.

For the year ended December 31, 2022, our net cash used in operating activities of $207.5 million consisted of a net loss of $280.3 million, offset by adjustments for non-cash items of $58.9 million and favorable changes in operating assets and liabilities of $14.0 million.

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.

The changes in operating assets and liabilities include an increase in other liabilities of $8.1 million, which was driven primarily by a long-term liability recorded during the period related to the amendment of the Penn License in the first quarter of 2022.

The favorable changes in operating assets and liabilities were partially offset by a net decrease in total accounts payable and accrued expenses and other current liabilities of $6.8 million, which was driven primarily by decreases in accrued sublicense fees and royalties and income taxes payable.

Other changes in operating assets and liabilities occurred in the normal course of business as a result of changes in operating working capital.

Cash Flows from Investing Activities

For the year ended December 31, 2023, our net cash provided by investing activities consisted of $285.5 million in maturities of marketable debt securities and $2.0 million in proceeds received from uniQure upon the achievement of milestones associated with their acquisition of Corlieve, offset by $86.6 million to purchase marketable debt securities and $10.0 million to purchase property and equipment.

For the year ended December 31, 2022, our net cash used in investing activities primarily consisted of $184.9 million to purchase marketable debt securities and $30.7 million to purchase property and equipment, partially offset by $203.1 million in maturities of marketable debt securities.

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, 2023, our net cash provided by investing activities consisted of $285.5 million in maturities of marketable debt securities and $2.0 million in proceeds received from uniQure upon the achievement of milestones associated with their acquisition of Corlieve, offset by $86.6 million used to purchase marketable debt securities and $10.0 million used to purchase property and equipment.

Cash Flows from Financing Activities

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

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, 2022, our net cash used in financing activities primarily consisted of $33.1 million of Zolgensma royalties paid to HCR, net of imputed interest, under our royalty purchase agreement, and was partially offset by $4.5 million in proceeds received from the exercise of stock options and issuance of common stock under our employee stock purchase plan.

Additional Capital Requirements

10 unchanged sentences

Due to the contingent nature of the payments, the amounts and timing of payments to licensors under our in-license agreements are uncertain and may fluctuate significantly from period to period.

In March 2022, we entered into a letter agreement with Penn to buy out our obligation to pay sublicense fees under the Penn License.

In March 2022, we entered into a letter agreement (the Penn Letter Agreement) with The Trustees of the University of Pennsylvania (Penn) to buy out our obligation to pay sublicense fees under our license agreement with Penn (the Penn License).

Pursuant to the letter agreement, we are obligated to pay Penn a total of $12.0 million to satisfy any other past or future obligations to pay sublicense fees under the Penn License, which is payable in four equal annual installments of $3.0 million beginning in March 2023.

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.

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

and New York, New York, as well as a number of laboratory and other equipment leases.

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

As of December 31, 2024, we had recorded total lease liabilities of $82.0 million under our operating leases.

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, 2023, the total amount of future Zolgensma royalties to be paid to HCR under the agreement was $102.0 million if paid by November 7, 2024, or $142.0 million if paid after that date.

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.

3 unchanged sentences

We do not expect to achieve such revenues, and expect to continue to incur losses, for at least the next several years.

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.

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.

8 unchanged sentences

• the costs, timing and outcome of regulatory review of our product candidates;

• the impact of any government-imposed tariffs on cost of goods and services, particularly related to partnered product candidates;

• the costs of future product sales, medical affairs, marketing, manufacturing and distribution activities for any of our product candidates for which we receive marketing approval;

3 unchanged sentences

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

• our current licensing agreements or collaborations remaining in effect, including the AbbVie Collaboration Agreement, and our ability to timely achieve any milestones set forth in such agreements or collaborations;

• 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;

• our ability to establish and maintain additional licensing agreements or collaborations on favorable terms, if at all;

• the extent to which we acquire or in-license other product candidates and technologies.

Many of these factors are outside of our control.

Identifying potential product candidates and conducting preclinical testing and clinical trials is a time-consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain regulatory and marketing approval and achieve product sales.

In addition, our product candidates, if approved, may not achieve commercial success.

Our product revenues, if any, and any commercial milestones or royalty payments under our licensing agreements, will be derived from or based on sales of products that may not be commercially available for many years, if at all.

In addition, revenue from our NAV Technology Platform licensing is dependent in part on the clinical and commercial success of our licensing partners, including the commercialization of Zolgensma, and on maintaining our license agreements with our licensor partners, including GlaxoSmithKline LLC (GSK) and Penn.

Accordingly, we will need to continue to rely on additional financing to achieve our business objectives.

The issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance, may cause the market price of our common stock to decline.

The issuance of additional securities, whether equity or debt, by us, including through our at-the-market program, or the possibility of such issuance, may cause the market price of our common stock to decline.

Adequate additional financing may not be available to us on acceptable terms, or at all.

12 unchanged sentences

government and agency securities, certificates of deposit 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, 2023, we estimate that the increase would have resulted in a hypothetical decline of $1.5 million in the net fair value of our interest-sensitive securities.

If market interest rates were to increase immediately and uniformly by 100 basis points, or one percentage point, from levels at December 31, 2024, 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, 2024.

A similar increase in market interest rates as of December 31, 2023 would have resulted in an estimated hypothetical decline of $1.5 million in the net fair value of our interest-sensitive securities as of December 31, 2023.

35 unchanged sentences

The adoption or termination of contracts, instructions or written plans for the purchase or sale of our securities by our Section 16 officers and directors for the three months ended December 31, 2024, each of which is intended to satisfy the affirmative defense of Rule 10b5-1(c) (Rule 10b5-1 Plan), were as follows:

Scheduled Expiration

Rule 10b5-1 Trading Plan

1 unchanged sentence

Securities to be

Termination Date

Expiration Date

Purchase/Sale

Purchased/Sold (b)

Stephen Pakola, M.D.

Executive Vice President, Chief Medical Officer

Entities affiliated with Allan M.

Executive Vice President, Chief Legal Officer

(a) Patrick J.

Christmas' Rule 10b5-1 Plan was terminated on December 27, 2023, prior to its scheduled expiration date of December 31, 2024 (the 2023 Plan).

Christmas terminated the 2023 Plan in order to amend certain terms, and thereafter entered into a new Rule 10b5-1 Plan on December 28, 2023 (the 2024 Plan) to reflect those amendments in the 2024 Plan.

No sales occurred under the 2023 Plan prior to its termination.

(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.

34 unchanged sentences

Consolidated Balance Sheets

Consolidated Statements of Operations and Comprehensive Income (Loss)

Consolidated Statements of Operations and Comprehensive Loss

Consolidated Statements of Stockholders’ Equity

5 unchanged sentences

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

and its subsidiaries (the “Company”) as of December 31, 2023 and 2022, and the related consolidated statements of operations and comprehensive income (loss), of stockholders’ equity and of cash flows for each of the three years in the period ended December 31, 2023, including the related notes (collectively referred to as the “consolidated financial statements”).

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).

33 unchanged sentences

Washington, District of Columbia

February 27, 2024

March 13, 2025

We have served as the Company’s auditor since 2015.

11 unchanged sentences

Marketable securities

Accounts receivable (net of allowance of $ 4,152 as of December 31, 2022)

Accounts receivable

Property and equipment, net

31 unchanged sentences

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(in thousands, except per share data)

7 unchanged sentences

Credit losses (recoveries)

Impairment of long-lived assets

Other operating expenses (income)

Total operating expenses

Income (loss) from operations

Loss from operations

Other Income (Expense)

3 unchanged sentences

Total other income (expense)

Income (loss) before income taxes

Income Tax Benefit (Expense)

Net income (loss)

Loss before income taxes

Income Tax Benefit

Other Comprehensive Income (Loss)

1 unchanged sentence

Total other comprehensive income (loss)

Comprehensive income (loss)

Net income (loss) per share:

Weighted-average common shares outstanding:

Comprehensive loss

Net loss per share, basic and diluted

Weighted-average common shares outstanding, basic and diluted

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

5 unchanged sentences

Balances at December 31, 2021

Issuance of common stock upon public offering,

net of transaction costs of $ 14,194

Vesting of restricted stock units, net of tax

Exercise of stock options, net of tax

8 unchanged sentences

stock purchase plan

Issuance of common stock upon private placement,

net of transaction costs of $ 126

Stock-based compensation expense

Unrealized loss on available-for-sale securities, net

Unrealized gain on available-for-sale securities, net

Balances at December 31, 2023

3 unchanged sentences

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

7 unchanged sentences

Cash flows from operating activities

Net income (loss)

Adjustments to reconcile net income (loss) to net cash provided by

(used in) operating activities

Adjustments to reconcile net loss to net cash used in operating activities

Stock-based compensation expense

1 unchanged sentence

Provision for credit losses (recoveries)

Net amortization of premiums on marketable debt securities

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

Net realized loss (gain) on investments

Impairment of long-lived assets

Non-cash interest expense

10 unchanged sentences

Other liabilities

Net cash provided by (used in) operating activities

Net cash used in operating activities

Cash flows from investing activities

8 unchanged sentences

Proceeds from issuance of common stock under employee stock purchase plan

Proceeds from public offering of common stock, net of underwriting discounts

and commissions

Proceeds from public offering of common stock and pre-funded warrants,

net of issuance costs

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

Offering expenses related to at-the-market offering program

Issuance costs for public offering of common stock

Transaction costs for sale of future royalties

Offering expenses related to at-the-market offering programs

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

14 unchanged sentences

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 AAV vectors, including commonly used AAV8 and AAV9.

The NAV ® Technology Platform consists 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 to other leading biotechnology and pharmaceutical companies (NAV Technology Licensees).

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.

1 unchanged sentence

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'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.

The Company may never achieve recurring profitability, and unless and until it does, the Company will continue to need to raise additional capital.

The Company may never achieve recurring profitability and, unless and until it does, will continue to need to raise additional capital through equity offerings, licensing and collaboration arrangements, or other non-dilutive financings.

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

As of December 31, 2023, the Company had cash, cash equivalents and marketable securities of $ 314.1 million, which management believes is sufficient to fund operations for at least the next 12 months from the date these consolidated financial statements were issued.

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.

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

6 unchanged sentences

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.

Use of Estimates

2 unchanged sentences

Actual results may differ materially from these estimates.

Significant 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 the fair value of financial instruments.

Reclassifications

Certain amounts reported in prior periods have been reclassified to conform to current period financial statement presentation.

These reclassifications are not material and have no effect on previously reported financial position, results of operations and cash flows.

Segment and Geographical Information

Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker (CODM), or decision-making group, in making decisions on how to allocate resources and assess performance.

The Company’s CODM, its Chief Executive Officer, views the Company’s operations and manages the business as one operating segment.

The Company’s revenues consist of license and royalty revenue.

For the year ended December 31, 2023 , 32 % of the Company’s revenues were attributed to the U.S.

and no other country 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 U.S.

and Germany, respectively, and no other countries accounted for 10 % or more of the Company’s revenues.

For the year ended December 31, 2021 , 79 % and 7 % of the Company’s revenues were attributed to Bermuda and the U.S., 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 substantial majority of the Company’s assets reside in the U.S.

Estimates are used in the following areas, among others:

Cash and Cash Equivalents

1 unchanged sentence

Restricted Cash

Restricted cash includes money market mutual funds and other deposits used to collateralize irrevocable letters of credit required under the Company’s lease agreements and certain other agreements with third parties.

Restricted cash consists of deposits held at financial institutions that are used to collateralize irrevocable letters of credit required under the Company’s lease agreements and certain other agreements with third parties.

The following table provides a reconciliation of cash and cash equivalents and restricted cash as reported on the consolidated balance sheets to the total of these amounts as reported at the end of the period in the consolidated statements of cash flows (in thousands):

20 unchanged sentences

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.

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 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.

11 unchanged sentences

The Company has adopted an investment policy which limits potential concentrations of investments and establishes minimum acceptable credit ratings, thereby reducing credit risk exposure.

With the exception of accounts receivable from Abeona Therapeutics Inc.

(Abeona), as discussed further in Note 10, the Company believes that it is not exposed to significant credit risk related to accounts receivable due to the credit quality and history of collections from its significant customers, and the Company is unaware of any concentrations of credit risk related to accounts receivable from significant customers with deteriorated credit quality.

As of December 31, 2024, the Company believes that it is not exposed to significant credit risk related to accounts receivable due to the credit quality and history of collections from its significant customers, and the Company is unaware of any concentrations of credit risk related to accounts receivable from significant customers with deteriorated credit quality.

The Company has no financial instruments with off-balance sheet risk of loss.

3 unchanged sentences

As of December 31,

* Represented less than 10%

The Company accounts for its lease arrangements in accordance with Accounting Standards Codification (ASC) 842, Leases (ASC 842).

2 unchanged sentences

Regardless of classification, the Company records a right-of-use asset and a lease liability for all leases with a term greater than 12 months.

All of the Company’s leases as of December 31, 2023 and 2022 have been classified as operating leases.

All of the Company’s leases are classified as operating leases.

Operating lease expense is recognized on a straight-line basis over the term of the lease, with the exception of variable lease expenses which are recognized as incurred.

1 unchanged sentence

The Company does not allocate lease consideration between lease and nonlease components and records a lease liability equal to the present value of the remaining fixed consideration under the lease.

interest rates implicit in the Company’s leases are generally not readily determinable.

The interest rates implicit in the Company’s leases are generally not readily determinable.

Accordingly, the Company uses its estimated incremental borrowing rate at the commencement date of the lease to determine the present value discount of the lease liability.

4 unchanged sentences

The Company excludes options to extend or terminate leases from the calculation of the lease liability unless it is reasonably certain the option will be exercised.

The Company evaluates its right-of-use assets for impairment in accordance with its policy for long-lived assets.

To the extent an impairment of a right-of-use asset is recognized, the remaining carrying value of the asset is subsequently amortized as lease expense on a straight-line basis from the date of impairment to the earlier of the end of the right-of-use asset’s useful life or the end of the lease term.

The Company determines the classification of subleases at the inception of the sublease, as well as whether the Company has been relieved of its primary obligation under the original lease.

All of the Company's subleases are classified as operating leases and, in each case, the Company has not been relieved of its primary obligation under the original lease and continues to account for the original lease as it did prior to the commencement of the sublease.

Sublease income is recognized on a straight-line basis over the term of the sublease as a reduction of the related lease expense of the original lease.

Initial direct costs of entering into a sublease are deferred and amortized on a straight-line basis over the term of the sublease as a reduction of sublease income.

Property and Equipment

13 unchanged sentences

Once implementation activities are substantially complete and the cloud-based application is ready for its intended use, capitalization ceases and amounts capitalized are amortized on a straight-line basis over the term of the hosting arrangement.

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

As of December 31, 2023 and 2022, the Company had recorded capitalized costs, net of amounts amortized, of $ 1.0 million and $ 2.4 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, 2023, 2022 and 2021 was $ 1.4 million, $ 1.3 million, and $ 0.6 million, respectively, and was included in general and administrative expenses in the consolidated statements of operations and comprehensive income (loss).

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.

Impairment of Long-lived Assets

The Company's long-lived assets consist primarily of property and equipment and operating lease right-of-use assets.

The Company evaluates its long-lived assets for impairment when events or changes in circumstances indicate the carrying value of the assets may not be recoverable.

1 unchanged sentence

If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the book value of the assets exceed their fair value, which is measured based on the projected discounted future net cash flows arising from the assets.

No material impairment losses on long-lived assets were recorded during the years ended December 31, 2023, 2022 and 2021 .

Please refer to Note 5 and Note 6 for further information on impairment of long-lived assets.

Non-marketable Equity Securities

2 unchanged sentences

Please refer to Note 4 for further information on non-marketable equity securities.

Fair Value of Financial Instruments

Fair Value Measurements

The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values.

ASC 820, Fair Value Measurements and Disclosures (ASC 820), establishes a hierarchy of inputs used in measuring fair value that maximizes the use of observable inputs and minimizes the use of unobservable inputs by requiring that the observable inputs be used when available.

ASC 820, Fair Value Measurements and Disclosures , establishes a hierarchy of inputs used in measuring fair value that maximizes the use of observable inputs and minimizes the use of unobservable inputs by requiring that the observable inputs be used when available.

Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company.

6 unchanged sentences

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment.

Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3.

A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.

The fair values of the Company’s Level 2 instruments are based on quoted market prices or broker or dealer quotations for similar assets.

Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for assets and liabilities categorized in Level 3.

The level within the fair value hierarchy of an asset or liability measured at fair value is based on the lowest level of any input that is significant to the fair value measurement.

The fair values of the Company’s Level 2 financial instruments are based on quoted market prices or broker or dealer quotations for similar assets.

These investments are initially valued at the transaction price and subsequently valued utilizing third-party pricing providers or other market observable data.

Please refer to Note 4 for further information on the fair value measurement of the Company’s financial instruments.

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

Liability Related to Sale of Future Royalties

8 unchanged sentences

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

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.

Revenue Recognition

1 unchanged sentence

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:

11 unchanged sentences

Consideration payable to the Company under its 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.

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.

12 unchanged sentences

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.

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.

2 unchanged sentences

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 includes up-front and annual fees payable during the contract term.

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

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.

20 unchanged sentences

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.

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.

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.

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.

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.

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.

25 unchanged sentences

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.

1 unchanged sentence

Compensation expense related to awards with service-based vesting conditions is recognized on a straight-line basis based on the estimated grant date fair value over the requisite service period of the award, which is generally the vesting term.

Compensation expense related to awards

with performance-based vesting conditions is recognized based on the estimated grant date fair value over the requisite service period using the accelerated attribution method to the extent achievement of the performance condition is probable.

Compensation expense related to awards with performance-based vesting conditions is recognized based on the estimated grant date fair value over the requisite service period using the accelerated attribution method to the extent achievement of the performance condition is probable.

The Company has elected to not estimate forfeitures of stock-based awards and accounts for forfeitures as they occur.

15 unchanged sentences

The Company recognizes interest and penalties related to uncertain tax positions in income tax expense.

As of December 31, 2023 and 2022 , the Company had no accrued interest or penalties related to uncertain tax positions and no amounts have been recognized in the Company’s consolidated statements of operations and comprehensive income (loss).

As of December 31, 2024 and 2023 , the Company had no accrued interest or penalties related to uncertain tax positions and no amounts have been recognized in the Company’s consolidated statements of operations and comprehensive loss.

Restructuring Expenses

3 unchanged sentences

Please refer to Note 14 for further information regarding restructuring expenses.

Net Income (Loss) Per Share

Basic net income (loss) per share is calculated by dividing net income (loss) applicable to common stockholders by the weighted-average common shares outstanding during the period, without consideration for common stock equivalents.

Diluted net income (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.

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

For purposes of the diluted net income (loss) per share calculation, common stock equivalents are excluded from the calculation of diluted net income (loss) per share if their effect would be anti-dilutive.

Comprehensive Income (Loss)

Comprehensive income (loss) includes net income (loss) as well as unrealized gains and losses on available-for-sale debt securities, net of income tax effects and reclassification adjustments for realized gains and losses.

Net Loss Per Share

Basic net loss per share is calculated by dividing net loss applicable to common stockholders by the weighted-average common shares outstanding during the period, without consideration for common stock equivalents.

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.

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.

For purposes of the diluted net loss per share calculation, common stock equivalents are excluded from the calculation of diluted net loss per share if their effect would be anti-dilutive.

Comprehensive Loss

Comprehensive loss includes net loss as well as unrealized gains and losses on available-for-sale debt securities, net of income tax effects and reclassification adjustments for realized gains and losses.

Recent Accounting Pronouncements

Recently Adopted Accounting Pronouncements

The Company did not adopt any new accounting standards during the year ended December 31, 2023 which had a material impact on the consolidated financial statements.

Recent Accounting Pronouncements Not Yet Adopted

In November 2023, the Financial Accounting Standards Board (FASB) issued Accounting Standards Update (ASU) 2023-07, Segment Reporting (Topic 280):

1 unchanged sentence

Additionally, the standard requires entities with a single reportable segment to provide all disclosures required by ASC 280, Segment Reporting .

The standard is effective for the Company for annual periods beginning January 1, 2024 and interim periods beginning January 1, 2025.

Early adoption is permitted.

The Company does not believe the application of this standard with have a material impact on its financial statement disclosures.

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

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

1 unchanged sentence

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

The Company does not believe the application of this standard with have a material impact on its financial statement disclosures.

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

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

Disaggregation of Income Statement Expenses .

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.

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

10 unchanged sentences

Corporate bonds

As of December 31, 2023 and 2022 , no available-for-sale debt securities had remaining maturities greater than three years.

As of December 31, 2024 and 2023 , no available-for-sale debt securities had remaining maturities greater than two years.

The amortized cost of marketable debt securities is adjusted for amortization of premiums and accretion of discounts to maturity, or to the earliest call date for callable debt securities purchased at a premium.

As of December 31, 2024 and 2023 , 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 income (loss) consisted of the following (in thousands):

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,

18 unchanged sentences

The Company did not recognize any impairment or credit losses on available-for-sale debt securities during the years ended December 31, 2024, 2023 and 2022 .

Fair Value of Financial Instruments

Fair Value Measurements

Financial instruments reported at fair value on a recurring basis include cash equivalents and marketable securities.

The following tables present the fair value of cash equivalents and marketable securities in accordance with the hierarchy discussed in Note 2 (in thousands):

The following tables present the fair value of cash equivalents and marketable securities in accordance with the fair value hierarchy discussed in Note 2 (in thousands):

December 31, 2024

1 unchanged sentence

Money market mutual funds

government and agency securities

Total cash equivalents

8 unchanged sentences

Money market mutual funds

Corporate bonds

Total cash equivalents

11 unchanged sentences

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.

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, 2024, 2023 and 2022.

Non-marketable Equity Securities

4 unchanged sentences

(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.

In connection with 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.

The Company recorded a realized gain of $ 5.2 million during the year ended December 31, 2021 as a result of the acquisition of Corlieve by uniQure, which is included in investment income in the consolidated statements of operations and comprehensive income (loss).

In addition to the upfront proceeds received in connection with uniQure's acquisition of Corlieve, the Company also 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, 2023, the Company received € 1.9 million in milestone payments from uniQure and recognized investment income of $ 2.2 million related to the achievement of milestones during the period.

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.

12 unchanged sentences

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.

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

In connection with the sublease, the Company recorded impairment of property and equipment of $ 0.7 million in the first quarter of 2024 related to furniture and fixtures and leasehold improvements located at the subleased facility.

Please refer to Note 6 for further information regarding the sublease agreement and associated impairment of long-lived assets.

9804 Medical Center Drive

8 unchanged sentences

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.

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, 2023, the Company had recorded property and equipment at cost of $ 132.0 million related to the buildout at 9804 Medical Center Drive, of which $ 41.2 million was placed in service upon the initial occupation of the building in 2021, $ 77.6 million was placed in service upon the activation of the manufacturing facility in 2022, $ 2.6 million was placed in service in 2023 and the remaining $ 10.6 million has not yet been placed in service.

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, 2024, the Company had recorded right-of-use assets of $ 42.9 million and lease liabilities of $ 69.0 million related to the 9804 Medical Center Drive Lease.

7 unchanged sentences

As of December 31, 2024, the Company had recorded right-of-use assets of $ 3.2 million and lease liabilities of $ 3.6 million related to the 9712 Medical Center Drive Lease.

New York Lease and Sublease

In May 2016, the Company entered into an operating lease for office space in New York, New York (the New York Lease), which has since been amended to include additional office space and extend the term of the lease.

2 unchanged sentences

As required by the New York Lease, the Company has provided the landlord with an irrevocable letter of credit of $ 0.2 million which the landlord may draw upon in the event of any uncured default by the Company under the terms of the lease.

In March 2024, the Company entered into an agreement to sublease its office space under the New York Lease (the New York Sublease) to a third-party subtenant.

The sublease term commenced in April 2024 and expires in April 2027 concurrent with the expiration of the New York Lease.

Monthly payments under the New York Sublease commenced in July 2024 and escalate annually in accordance with the sublease agreement.

As of December 31, 2024, total undiscounted future minimum lease payments to be received by the Company over the term of the New York Sublease were $ 1.2 million.

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

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.

The Company continues to account for the New York Lease as it did prior to the commencement of the sublease.

As a result of the New York Sublease, the Company determined an impairment indicator was present as of March 31, 2024 related to the long-lived asset group subject to the sublease, which included the right-of-use asset under the New York Lease, leasehold improvements and other property and equipment allocable to the New York Sublease.

The Company concluded the carrying value of the asset group as of March 31, 2024 was not recoverable, as it exceeded the sum of the estimated undiscounted cash flows to be generated by the assets over their remaining lives.

The Company estimated the fair value of the asset group as of March 31, 2024 using a discounted cash flow method, which incorporated unobservable inputs including the net identifiable cash flows over the term of the New York Sublease and an estimated borrowing rate of a market participant subtenant.

The estimated fair value of the asset group as of March 31, 2024 represents a Level 3 nonrecurring fair value measurement.

The Company concluded the carrying value of the asset group of $ 3.4 million exceeded its estimated fair value of $ 1.3 million as of March 31, 2024.

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.

As of December 31, 2024, the Company had recorded right-of-use assets of $ 0.6 million and lease liabilities of $ 2.2 million related to the New York Lease.

13 unchanged sentences

Variable lease cost

Sublease income

Total lease cost

Cash paid (received) for amounts included in operating lease liabilities

Cash paid for amounts included in operating lease liabilities

Right-of-use assets acquired through operating lease liabilities

Cash paid (received) for amounts included in operating lease liabilities for the years ended December 31, 2023, 2022 and 2021 includes $ 0.1 million, $ 1.3 million and $ 11.4 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.

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, 2024, 2023 and 2022 was not material and is included in operating lease cost in the table above.

18 unchanged sentences

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 is 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, and (ii) $ 300.0 million applicable for the period from November 8, 2024 through the effective date of termination of the Novartis License.

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.

The First Cap Amount was not achieved prior to November 7, 2024, therefore the 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.

provided, however, that with respect to a call option exercised on or before November 7, 2024, in the event that the then applicable Cap Amount minus the total amount of royalty payments received by HCR is less than $ 1.0 million, the repurchase price shall equal such difference.

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.

4 unchanged sentences

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.

As of December 31, 2023, the estimated effective interest rate under the Royalty Purchase Agreement was 4.8 % .

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.

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.

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):

25 unchanged sentences

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.

treatment of CLN2 disease.

From the inception of the agreement through December 31, 2024, the Company had incurred $ 0.5 million for development milestones achieved, or deemed probable of achievement, under the Penn License.

11 unchanged sentences

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, 2022, the Company had recorded $ 10.3 million payable under the Penn License, net of present value discount, of which $ 2.3 million was included in accounts payable and accrued expenses and other current liabilities, and $ 8.0 million was included in other liabilities on the consolidated balance sheet.

As of December 31, 2023, the Company had recorded $ 8.2 million payable under the Penn License, net of present value discount, of which $ 2.6 million was included in accrued expenses and other current liabilities, and $ 5.6 million was included in other liabilities on the consolidated balance sheet.

GlaxoSmithKline

2 unchanged sentences

Additionally, the Company is obligated to reimburse GSK for certain costs incurred related to the maintenance of the licensed patents.

The Company was also obligated to pay $ 1.5 million to GSK upon the achievement of various milestones, all of which have been achieved and paid as of December 31, 2023.

The Company was also obligated to pay $ 1.5 million to GSK upon the achievement of various milestones, all of which have been achieved and paid.

In connection with the execution of the Penn Letter Agreement in March 2022, the Company’s royalty obligations under the GSK License were assigned by GSK to Penn.

9 unchanged sentences

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, 2022, the Company had recorded $ 14.1 million payable under the GSK License, of which $ 13.8 million was included in accounts payable and accrued expenses and other current liabilities, and $ 0.2 million was included in other liabilities on the consolidated balance sheet.

As of December 31, 2023, the Company had recorded $ 12.3 million payable under the GSK License, of which $ 12.2 million was included in accrued expenses and other current liabilities, and $ 0.1 million was included in other liabilities on the consolidated balance sheet.

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.

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 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.

Clearside Biomedical

1 unchanged sentence

(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.

The Company exercised its license option in October 2019, resulting in a payment of $ 1.6 million to Clearside which was recognized as research and development expense upon exercise.

The Company exercised its license option in October 2019, resulting in a payment of $ 1.6 million to Clearside payable under the license agreement.

Additionally, the Company is obligated to pay milestone fees of up to $ 136.0 million upon the achievement of various development and sales-based milestones, as well as royalties on net sales of licensed products using the SCS Microinjector.

3 unchanged sentences

In November 2014, the Company entered into a license agreement, which has been amended from time to time, with Regents of the University of Minnesota (Minnesota), for an exclusive license to Minnesota’s interest in certain patent rights which are co-owned by Minnesota and the Company to commercialize products covered by the licensed patent rights in any country or territory in which a licensed patent has been issued and is unexpired, or a licensed patent application is pending.

Pursuant to the license agreement, the

Company is obligated to pay Minnesota annual maintenance fees, royalties on net sales, sublicense fees and fees upon the achievement of various milestones.

Pursuant to the license agreement, the Company is obligated to pay Minnesota annual maintenance fees, royalties on net sales, sublicense fees and fees upon the achievement of various milestones.

Additionally, the Company is obligated to pay for certain costs incurred related to the maintenance of the licensed patents.

15 unchanged sentences

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 Company was paid $ 7.5 million, which was recorded as other operating income in the consolidated statements of operations and comprehensive income (loss) for the year ended December 31, 2022.

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.

Capitalization

5 unchanged sentences

Reserved for issuance under employee stock purchase plan

Public Offerings

In January 2021, the Company completed a public offering of 4,899,000 shares of its common stock (inclusive of 639,000 shares pursuant to the full exercise by the underwriters of their option to purchase additional shares) at a price of $ 47.00 per share.

The aggregate net proceeds received by the Company from the offering, inclusive of the underwriters’ option exercise, were $ 216.1 million, net of underwriting discounts and commissions and offering expenses payable by the Company.

Private Placement

On July 7, 2023, the Company sold an aggregate of 257,466 shares of its common stock to Redmile Biopharma Investments III, L.P.

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.

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.

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 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).

1 unchanged sentence

The Private Placement was conducted in reliance on the exemption from registration provided by Section 4(a)(2) of the Securities Act.

At-the-Market Offering Program

On September 1, 2023, the Company entered into an ATM Equity Offering SM Sales Agreement with BofA Securities, Inc.

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

As of December 31, 2023, no shares of common stock had been sold under the ATM Program.

At-the-Market Offering Programs

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

The Company 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.

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

As of December 31, 2024, no shares of common stock had been sold under the Leerink ATM Program.

License and Collaboration Agreements

Please refer to Note 8 for information on license agreements for technology in-licensed by the Company from third parties.

License and Royalty Revenue

2 unchanged sentences

Consideration to the Company under its 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.

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.

3 unchanged sentences

Zolgensma royalties

AbbVie collaboration and license agreement

Other license and royalty revenue

3 unchanged sentences

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 will be obligated to pay sublicense fees to licensors based on a specified percentage of the fees earned by the Company.

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.

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.

15 unchanged sentences

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, 2023 consisted of research and development services to be performed by the Company related to licensed products, which will be satisfied as the research and development services are performed.

As of December 31, 2023, the aggregate transaction price of the Company's license agreements allocated to performance obligations not yet satisfied, or partially satisfied, was $ 1.2 million, which is expected to be satisfied over a period of two to three years .

Revenue recognized from performance obligations satisfied in previous periods, as presented in the table above, was primarily attributable to Zolgensma royalties, sublicense fees earned from licensees and changes in the transaction prices of the Company’s license agreements.

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, 2023, 2022 and 2021 resulting from performance obligations satisfied in previous periods includ ed $ 2.0 million, zero and $ 0.5 million, respectively, in cumulative catch-up adjustments for changes in the probability of achievement of development milestones.

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

9 unchanged sentences

Non-current accounts receivable:

Due from Abeona, net of present value discount

Other unbilled

Allowance for credit losses

Non-current accounts receivable, net

8 unchanged sentences

Changes in present value discount of receivables

Credit recoveries

Balance at December 31, 2024

The Company’s allowance for credit losses as of December 31, 2023 and 2022 was related solely to accounts receivable from Abeona.

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.

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.

The Company did no t record a provision for credit losses for the years ended December 31, 2023 and 2022.

The Company recorded credit recoveries of $ 2.6 million during the year ended December 31, 2021.

Zolgensma License with Novartis Gene Therapies

19 unchanged sentences

The Settlement Agreement resolved all arbitration and legal proceedings and mutually released each party from any and all claims under the terminated license agreement.

Pursuant to the Settlement Agreement, Abeona will pay the Company a total of $ 30.0 million as follows:

(i) $ 20.0 million which was paid in November 2021, (ii) $ 5.0 million which was paid in November 2022, and (iii) $ 5.0 million payable on the earlier of the third anniversary of the Settlement Agreement in November 2024 or the closing of a specified type of transaction by Abeona.

As of December 31, 2023 and 2022, the Company had recorded accounts receivable of $ 4.6 million and $ 4.2 million, respectively, associated with the remaining amounts due from Abeona under the Settlement Agreement.

Pursuant to the Settlement Agreement, Abeona paid the Company a total of $ 30.0 million as follows:

(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.

While the Company anticipates taking appropriate measures to enforce the full collection of all amounts due from Abeona under the Settlement Agreement, the Company assessed the collectability of the accounts receivable from Abeona as it relates to credit risk.

In performing this assessment, the Company evaluated Abeona’s credit profile and financial condition, as well its expectations regarding Abeona’s future cash flows and ability to satisfy the contractual obligations of the Settlement Agreement.

As a result of its analysis, the Company recorded an allowance for credit losses of $ 4.6 million and $ 4.2 million as of December 31, 2023 and 2022, respectively, related to the accounts receivable due from Abeona.

The Company recorded credit recoveries of $ 2.6 million during the year ended December 31, 2021 as a result of changes in estimates regarding amounts collectable from Abeona under the terminated license agreement and subsequent Settlement Agreement.

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.

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 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.

The present value discount of the receivable is accreted as interest income from licensing through the contractual due date using the effective interest method.

The Company has 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 on the Abeona receivable.

Collaboration Agreements

AbbVie Collaboration and License Agreement

Effective in November 2021, the Company entered into a collaboration and license agreement with AbbVie Global Enterprises Ltd.

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.

Pursuant to the AbbVie Collaboration Agreement, the Company granted AbbVie a co-exclusive license to develop and commercialize ABBV-RGX-314 in the United States and an exclusive license to develop and commercialize ABBV-RGX-314 outside the United States.

44 unchanged sentences

Years Ended December 31,

License and royalty revenue

Net cost reimbursement to (from) AbbVie included in:

2 unchanged sentences

Total net cost reimbursement to (from) AbbVie

Nippon Shinyaku Collaboration and License Agreement

In January 2025, the Company entered into a collaboration and license agreement with Nippon Shinyaku for the development and commercialization of RGX-121, the Company’s product candidate for the treatment of MPS II, and RGX-111, the Company's product candidate for the treatment of MPS I (the Nippon Shinyaku Collaboration Agreement).

The Nippon Shinyaku Collaboration Agreement became effective in March 2025.

Pursuant to the Nippon Shinyaku Collaboration Agreement, the Company granted Nippon Shinyaku a license to develop and exclusively commercialize RGX-121 and RGX-111 in the United States and certain countries in Asia.

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 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.

Stock-based Compensation

10 unchanged sentences

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.

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.

11 unchanged sentences

As of December 31, 2024, the Company had $ 59.7 million of unrecognized stock-based compensation expense related to stock options, restricted stock units and the 2015 Employee Stock Purchase Plan (the 2015 ESPP), which is expected to be recognized over a weighted-average period of 2.3 years.

The Company recorded aggregate stock-based compensation expense in the consolidated statements of operations and comprehensive income (loss) as follows (in thousands):

The Company recorded aggregate stock-based compensation expense in the consolidated statements of operations and comprehensive loss as follows (in thousands):

Years Ended December 31,

23 unchanged sentences

Unvested balance at December 31, 2024

The total intrinsic value of restricted stock units vested during the years ended December 31, 2023 and 2022 was $ 3.9 million and $ 2.2 million, respectively.

No restricted stock units vested during the year ended December 31, 2021.

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.

Employee Stock Purchase Plan

10 unchanged sentences

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.

The components of income (loss) before income taxes were as follows (in thousands):

The components of loss before income taxes were as follows (in thousands):

Years Ended December 31,

United States

Total income (loss) before income taxes

Total loss before income taxes

The components of the provision for income tax expense (benefit) were as follows (in thousands):

4 unchanged sentences

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 $ 107.3 million and $ 75.0 million as of December 31, 2023 and 2022, respectively, related to capitalized research and development expenses, net of amounts amortized during the periods.

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.

1 unchanged sentence

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.

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 income (loss) (in thousands):

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.

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.

Years Ended December 31,

Federal income tax expense (benefit) at statutory rate

State income tax expense (benefit), net of federal tax effect

Federal income tax benefit at statutory rate

State income tax benefit, net of federal tax effect

Research and development credits

43 unchanged sentences

The calculation of these credits requires assumptions to be made by the Company to estimate qualified research expenses.

The Company conducts formal studies to document the qualified activities and expenses used to calculate these credits, however a portion of these credits may be subject to future studies which have not yet occurred, the results of which may result in an adjustment to the Company’s credit carryforwards.

The Company conducts formal studies to document the qualified activities and expenses used to calculate these credits, however a portion of these credits may be subject to future examinations which have not yet occurred, the results of which may result in an adjustment to the Company’s credit carryforwards.

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.

No reserve for uncertain tax positions has been placed against qualified expenses for which a study has not been conducted.

However, 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.

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 does not expect that a significant portion of its unrecognized tax benefits will increase or decrease in the next 12 months as of December 31, 2023.

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.

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 for the treatment of Mucopolysaccharidosis Type II (MPS II), while pursuing strategic alternatives for the Company's other clinical stage programs.

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.

As a result of 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 income (loss).

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.

Restructuring costs 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.

The Company expects cash payments related to the restructuring costs to be completed by the fourth quarter of 2024.

The following table presents the details of the Company's restructuring liability, which is included in accounts payable and accrued expenses and other current liabilities on the consolidated balance sheet as of December 31, 2023 (in thousands):

During the year ended December 31, 2024, the Company recorded reductions in the restructuring liability of $ 0.4 million associated with changes in total estimated restructuring costs.

As of December 31, 2024, all of the restructuring costs had been paid by the Company and no restructuring liability was recorded.

The following table presents the changes in the Company's restructuring liability (in thousands):

Restructuring Liability

3 unchanged sentences

Balance at December 31, 2023

Cash payments

Other adjustments

Balance at December 31, 2024

Related Party Transactions

1 unchanged sentence

The agreement with FOXKISER was terminated effective June 2022.

Expenses incurred under the agreement with FOXKISER for the years ended December 31, 2022 and 2021 were $ 2.4 million and $ 4.8 million, respectively, and were recorded as research and development expenses in the consolidated statements of operations and comprehensive income (loss).

No expenses under the agreement with FOXKISER were incurred during the year ended December 31, 2023.

Net Income (Loss) Per Share

The computations of basic and diluted net income (loss) per share were as follows (in thousands, except per share data):

Years Ended December 31,

Basic net income (loss) per share:

Net income (loss)

Shares used in computation:

Weighted-average common shares outstanding

Basic net income (loss) per share

Diluted net income (loss) per share:

Net income (loss)

Shares used in computation:

Weighted-average common shares outstanding

Stock options

Restricted stock units

Employee stock purchase plan

Weighted-average diluted common shares

Diluted net income (loss) per share

For periods in which the Company incurred net losses, common stock equivalents were excluded from the calculation of diluted net loss per share as their effect would be anti-dilutive.

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

Since the Company incurred net losses for the years ended December 31, 2024, 2023 and 2022, common stock equivalents were excluded from the calculation of diluted net loss per share for such periods as their effect would be anti-dilutive.

Accordingly, basic and diluted net loss per share were the same for such periods.

The weighted-average number of common shares outstanding used in the basic and diluted net loss per share calculations includes the weighted-average effect of pre-funded warrants to purchase shares of the Company's common stock, as the pre-funded warrants are exercisable at any time for nominal cash consideration.

The following potentially dilutive common stock equivalents outstanding at the end of the period were excluded from the computations of weighted-average diluted common shares for the periods indicated as their effects would be anti-dilutive (in thousands):

3 unchanged sentences

Employee stock purchase plan

Segment and Geographical Information

The Company’s CODM , its Chief Executive Officer, views the Company’s operations and manages the business as one operating segment focused on the development and commercialization of gene therapies to treat an array of diseases.

The determination of a single operating segment is consistent with the consolidated financial information regularly provided to the CODM.

The CODM reviews and evaluates consolidated net income (loss) for purposes of assessing performance, making operating decisions and allocating resources.

The CODM uses net income (loss) to assess performance versus operating budgets and in the preparation of near-term and long-range operating plans to inform decisions on resource and capital allocation.

The CODM reviews consolidated cash, cash equivalents and marketable securities as a measure of segment assets.

As of December 31, 2024 and 2023, the Company’s cash, cash equivalents and marketable securities were $ 244.9 million and $ 314.1 million, respectively.

The following table presents information about the Company's segment revenues, significant segment expenses regularly provided to the CODM, other segment items and consolidated net income (loss) (in thousands):

Years Ended December 31,

Cost of revenues

Research and development expense

Direct development and support (a)

Stock-based compensation

Depreciation and amortization

Total research and development expense

General and administrative expense

Other general and administrative (b)

Stock-based compensation

Depreciation and amortization

Total general and administrative expense

Other segment items (c)

(a) Direct development and support includes external goods and services for the development of product candidates and early-stage research activities, laboratory costs, consulting, development cost reimbursement to and from collaborators and other expenses in support of research and development activities.

(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.

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 substantial majority of the Company’s assets reside in the United States.

Supplemental Disclosures

8 unchanged sentences

Accrued personnel costs

Accrued sublicense fees and royalties

Accrued external research and development expenses

Accrued sublicense fees and royalties

Accrued external general and administrative expenses

4 unchanged sentences

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, 2021 were $ 10.1 million, a net increase of $ 0.6 million from December 31, 2020.

Proceeds due to the Company for sales of non-marketable equity securities included in other current assets as of December 31, 2021 were $ 0.6 million.

No such amounts were recorded as of December 31, 2023 and 2022.

Offering expenses for the ATM Program included in accounts payable and accrued expenses and other liabilities as of December 31, 2023 were less than $ 0.1 million.

No such amounts were recorded as of December 31, 2022 and 2021.

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

Incorporated by Reference

ATM Equity Offering SM Sales Agreement, dated as of September 1, 2023, between BofA Securities, Inc.

and REGENXBIO Inc.

Sales Agreement dated December 9, 2024 between the Registrant and Leerink Partners LLC

Restated Certificate of Incorporation

2 unchanged sentences

Description of Securities

Form of Pre-funded Warrant

Form of Indemnity Agreement for directors and officers

4 unchanged sentences

2015 Employee Stock Purchase Plan

Employment Agreement effective as of June 30, 2015 between the Registrant and Kenneth T.

Employment Agreement effective as of June 30, 2015 between the Registrant and Vittal Vasista

Employment Agreement effective as of July 1, 2024 between the Registrant and Curran Simpson

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

5 unchanged sentences

Fourth Amendment to License Agreement effective April 4, 2019 between the Registrant and The Trustees of the University of Pennsylvania

Incorporated by Reference

Fifth Amendment to License Agreement effective September 11, 2020 between the Registrant and The Trustees of the University of Pennsylvania

Letter Agreement dated March 21, 2022 between the Company and the Trustees of the University of Pennsylvania

Incorporated by Reference

License Agreement dated March 6, 2009 between the Registrant and SmithKline Beecham Corporation d/b/a GlaxoSmithKline

13 unchanged sentences

First Amendment to Lease dated April 23, 2019 between the Registrant and ARE-Maryland No.

Incorporated by Reference

Second Amendment to Lease dated November 4, 2019 between the Registrant and ARE-Maryland No.

1 unchanged sentence

Royalty Purchase Agreement dated December 22, 2020 between the Registrant and entities managed by Healthcare Royalty Management, LLC

Insider Trading Policy

Subsidiaries of the Registrant

2 unchanged sentences

Certification of the Chief Financial Officer as required by Section 302 of the Sarbanes-Oxley Act of 2002

Incorporated by Reference

Certifications of the Chief Executive Officer and Chief Financial Officer as required by 18 U.S.C.

2 unchanged sentences

(i) Consolidated Balance Sheets

(ii) Consolidated Statements of Operations and Comprehensive Income (Loss)

(ii) Consolidated Statements of Operations and Comprehensive Loss

(iii) Consolidated Statements of Stockholders’ Equity

6 unchanged sentences

under the Securities Act or the Exchange Act, whether made before or after the date of this Annual Report on Form 10-K, irrespective of any general incorporation language contained in such filing.

Pursuant to the requirements of Section 13 and 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized on February 27, 2024.

REGENXBIO INC.

/s/ Kenneth T.

/s/ Curran Simpson

Curran Simpson

President and Chief Executive Officer

Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed below by the following persons on behalf of the registrant and in the capacities and on the dates indicated.

/s/ Kenneth T.

/s/ Curran Simpson

President, Chief Executive Officer and

February 27, 2024

March 13, 2025

Curran Simpson

Director (Principal Executive Officer)

/s/ Vittal Vasista

/s/ Mitchell Chan

Chief Financial Officer

February 27, 2024

Vittal Vasista

March 13, 2025

Mitchell Chan

(Principal Financial and Accounting Officer)

/s/ Kenneth T.

Chairman of the Board of Directors

February 27, 2024

March 13, 2025

/s/ Jean Bennett

February 27, 2024

March 13, 2025

/s/ Alexandra Glucksmann

February 27, 2024

March 13, 2025

Alexandra Glucksmann

“Jerry” Karabelas

February 27, 2024

March 13, 2025

“Jerry” Karabelas

/s/ George Migausky

February 27, 2024

March 13, 2025

George Migausky

February 27, 2024

March 13, 2025

/s/ Daniel Tassé

February 27, 2024

March 13, 2025

/s/ Jennifer Zachary

February 27, 2024

March 13, 2025

Jennifer Zachary

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