What changed in this filing

/stocks — the workspaceLOADING

Item 2. Management’s Discussion and Analysis

2025-11-06 compared with 2025-08-07 · 87 added, 78 removed, 310 unchanged (35% of the section changed)

17 unchanged sentences

Subretinal Delivery

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

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

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

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

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

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

Suprachoroidal Delivery

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

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

In January 2024, we announced data from the AAVIATE trial demonstrating ABBV-RGX-314 suprachoroidal delivery was well tolerated across 106 patients with no drug-related serious adverse events (SAEs) were reported.

Patients treated with ABBV-RGX-314 continued to demonstrate stable best corrected visual acuity (BCVA) and central retinal thickness (CRT) at six months.

In addition, a meaningful reduction in anti-VEGF treatment burden was observed following administration of ABBV-RGX-314.

The highest reduction was seen in dose level 3, demonstrating an 80% reduction in annualized injection rate with 50% of patients remaining injection-free.

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 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.

6 unchanged sentences

New ALTITUDE trial data demonstrate a durable safety and efficacy profile observed in patients with non-proliferative DR through two years with a single, in-office injection.

As of June 9, 2025, ABBV-RGX-314 was well tolerated at dose levels 1, 2 and 3, with no drug-related serious adverse events.

As of June 9, 2025, ABBV-RGX-314 was well tolerated at dose levels 1, 2 and 3, with no drug-related SAEs.

No intraocular inflammation was observed through two years at dose level 3 (1.0x10e12 GC/eye) (n=15) with short-course topical prophylactic steroids.

In August 2025, we and AbbVie executed an amendment to our collaboration agreement and announced plans to initiate a pivotal two-part placebo-controlled Phase IIb/III trial, with the primary endpoint being ≥2-step DRSS improvement at one year.

In August 2025, we and AbbVie executed an amendment to our collaboration agreement and announced plans to initiate a pivotal two-part sham injection-controlled Phase IIb/III trial, with the primary endpoint being ≥2-step DRSS improvement at one year.

Site selection for the Phase IIb/III trial is in progress.

5 unchanged sentences

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

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

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

The initiation of the pivotal study, which is expected to enroll approximately 30 patients in the U.S.

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

and Canada, as well as positive interim safety and efficacy data from the Phase I/II portion of the study were announced in November 2024.

These data included positive biomarker data from the first nine patients, which demonstrated consistent, robust microdystrophin and transduction, as well as positive initial functional data.

Subsequent findings were presented in March 2025 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference and in June 2025 via a Company webcast.

Subsequent findings were presented in March 2025 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference, in June 2025 via a Company webcast and in October 2025 at the International Congress of the World Muscle Society.

In sum, these data were positive and demonstrate potential for RGX-202 to serve as a differentiated gene therapy for Duchenne.

1 unchanged sentence

We also reported a favorable safety profile with no serious adverse events or adverse events of special interest observed (n=13).

As of May 2025, the pivotal study was beyond 50% enrolled.

In August 2025, we announced expectations to complete enrollment by October 2025, earlier than previous guidance of year-end 2025.

Upon completion of enrollment in the pivotal trial, we expect to continue enrollment to support a planned confirmatory trial.

We expect to share topline data in the first half of 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026.

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

We also announced that the first batches of RGX-202 intended for commercial supply have been manufactured at our Manufacturing Innovation Center and that we expect to imminently complete the Process Performance Qualification (PPQ) campaign.

We expect to share topline data in early second quarter 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026.

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

2 unchanged sentences

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 February 2024, we announced that, 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 February 2024, we announced that, 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 (HS) 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.

We plan to use levels of cerebrospinal fluid HS 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.

In May 2025, the FDA granted priority review of the BLA and a Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025 was assigned.

meeting and pre-license and Bioresearch Monitoring inspections were successfully completed in July and August 2025, respectively.

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.

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

The PLI and BIMO inspections were completed with no observations.

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

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

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

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

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

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

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

5 unchanged sentences

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

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

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

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

4 unchanged sentences

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 unaudited consolidated financial statements.

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

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

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

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

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

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

Also pursuant to the amendment, AbbVie will lead a new Phase III randomized controlled study (the ACHIEVE Study) to assess the injection burden, adverse events,

change in disease activity, and long-term preservation of visual acuity of ABBV-RGX-314 in adult participants with neovascular AMD.

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

9 unchanged sentences

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

We recognized $74.5 million of revenue under the Nippon Shinyaku Collaboration Agreement in the first half of 2025.

We recognized $80.4 million of revenue under the Nippon Shinyaku Collaboration Agreement during the nine months ended September 30, 2025.

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

4 unchanged sentences

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 June 30, 2025, 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.

As of September 30, 2025, 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 additional revenue opportunities.

Financial Overview

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

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

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

33 unchanged sentences

We expect to continue to incur significant research and development expenses for the foreseeable future as we continue the development of our product candidates and engage in early research and development for prospective product candidates and new technologies.

The following table summarizes our research and development expenses incurred during the three and six months ended June 30, 2025 and 2024 (in thousands):

Three Months Ended June 30,

Six Months Ended June 30,

The following table summarizes our research and development expenses incurred during the three and nine months ended September 30, 2025 and 2024 (in thousands):

Three Months Ended September 30,

Nine Months Ended September 30,

Direct Expenses

8 unchanged sentences

* 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 include $17.1 million and $31.7 million for the three and six months ended June 30, 2025, respectively, and $21.3 million and $46.2 million for the three and six months ended June 30, 2024, respectively, in net cost reimbursement from AbbVie under our eye care collaboration, which were recorded as a reduction of research and development expenses.

Direct expenses related to the development of ABBV-RGX-314 include $17.7 million and $49.5 million for the three and nine months ended September 30, 2025, respectively, and $20.1 million and $66.3 million for the three and nine months ended September 30, 2024, respectively, in net cost reimbursement from AbbVie under our eye care collaboration, which were recorded as a reduction of research and development expenses.

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

16 unchanged sentences

Interest Expense

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

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

20 unchanged sentences

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

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

option of the licensee.

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

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

21 unchanged sentences

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

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

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

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

8 unchanged sentences

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

If it is probable that

a significant revenue reversal will not occur in the future, milestone payments are included in the transaction price.

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

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

14 unchanged sentences

Our consolidated results of operations were as follows (in thousands):

Three Months Ended June 30,

Six Months Ended June 30,

Three Months Ended September 30,

Nine Months Ended September 30,

License and royalty revenue

6 unchanged sentences

Impairment of long-lived assets

Other operating expenses (income)

Other operating expenses

Total operating expenses

5 unchanged sentences

Total other income (expense)

Comparison of the Three Months Ended June 30, 2025 and 2024

Comparison of the Three Months Ended September 30, 2025 and 2024

License and Royalty Revenue.

License and royalty revenue decreased by $3.4 million, from $21.8 million for the three months ended June 30, 2024 to $18.5 million for the three months ended June 30, 2025.

The decrease was primarily attributable to Zolgensma royalty revenues, which decreased by $3.3 million, from $21.8 million for the second quarter of 2024 to $18.4 million for the second quarter of 2025.

Novartis reported Zolgensma sales of $297 million for the second quarter of 2025, a decrease of 15% from the second quarter of 2024, driven by lower incidence of SMA during the period.

License and royalty revenue decreased by $0.4 million, from $24.0 million for the three months ended September 30, 2024 to $23.6 million for the three months ended September 30, 2025.

The decrease was primarily attributable to Zolgensma royalty revenues, which decreased by $0.3 million, from $23.9 million for the third quarter of 2024 to $23.6 million for the third quarter of 2025.

Novartis reported Zolgensma sales of $301 million for the third quarter of 2025, a decrease of 2% from the third quarter of 2024, driven by lower incidence of SMA during the period.

Service Revenue.

Service revenue increased by $2.4 million, from $0.4 million for the three months ended June 30, 2024 to $2.9 million for the three months ended June 30, 2025.

The increase was primarily attributable to $2.7 million of development service revenue recognized under our collaboration with Nippon Shinyaku in the second quarter of 2025.

Service revenue increased by $5.9 million, from $0.2 million for the three months ended September 30, 2024 to $6.1 million for the three months ended September 30, 2025.

The increase was primarily attributable to $5.9 million of development service revenue recognized under our collaboration with Nippon Shinyaku in the third quarter of 2025.

Research and Development Expense.

Research and development expenses increased by $10.6 million, from $48.9 million for the three months ended June 30, 2024 to $59.5 million for the three months ended June 30, 2025.

Research and development expenses increased by $1.7 million, from $54.4 million for the three months ended September 30, 2024 to $56.1 million for the three months ended September 30, 2025.

The increase was primarily attributable to the following:

• an increase of $5.7 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates, largely driven by ABBV-RGX-314 clinical supply and purchases of raw materials;

• an increase of $3.2 million in costs associated with clinical trials and regulatory activities, largely driven by clinical trial expenses for ABBV-RGX-314 and RGX-202 pivotal trials;

• an increase of $1.4 million in personnel-related costs as a result of increased headcount of development personnel, net of a $0.7 million decrease in stock-based compensation expense.

• an increase of $2.3 million in personnel-related costs due to increased headcount of development personnel, net of a $0.1 million decrease in stock-based compensation expense;

• an increase of $1.9 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates, largely driven by manufacturing of RGX-121;

• an increase of $0.5 million in preclinical activities and other early-stage research and development.

The increase in research and development expenses was partially offset by a decrease of $3.7 million in costs associated with clinical trials and regulatory activities, largely driven by a decrease in clinical trial expenses for ABBV-RGX-314 pivotal trials.

General and Administrative Expense.

General and administrative expenses increased by $1.0 million, from $18.9 million for the three months ended June 30, 2024 to $19.9 million for the three months ended June 30, 2025.

The increase was primarily attributable to personnel-related costs for general and administrative personnel and professional services and consulting fees, including legal and other corporate advisory services.

General and administrative expenses increased by $0.8 million, from $19.4 million for the three months ended September 30, 2024 to $20.3 million for the three months ended September 30, 2025.

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

Interest Expense.

Interest expense increased by $10.5 million, from $0.4 million for the three months ended June 30, 2024 to $11.0 million for the three months ended June 30, 2025.

Interest expense increased by $12.3 million, from $0.8 million for the three months ended September 30, 2024 to $13.2 million for the three months ended September 30, 2025.

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

Comparison of the Six Months Ended June 30, 2025 and 2024

Comparison of the Nine Months Ended September 30, 2025 and 2024

License and Royalty Revenue.

License and royalty revenue increased by $68.3 million, from $37.2 million for the six months ended June 30, 2024 to $105.5 million for the six months ended June 30, 2025.

The increase was primarily attributable to $70.0 million of upfront license revenue recognized under our collaboration with Nippon Shinyaku in the first quarter of 2025.

License and royalty revenue increased by $67.9 million, from $61.2 million for the nine months ended September 30, 2024 to $129.1 million for the nine months ended September 30, 2025.

The increase was primarily attributable to $70.0 million of up-front license revenue recognized under our collaboration with Nippon Shinyaku in the first quarter of 2025.

Service Revenue.

Service revenue increased by $4.1 million, from $0.7 million for the six months ended June 30, 2024 to $4.9 million for the six months ended June 30, 2025.

The increase was primarily attributable to $4.5 million of development service revenue recognized under our collaboration with Nippon Shinyaku in the first half of 2025.

Service revenue increased by $10.0 million, from $0.9 million for the nine months ended September 30, 2024 to $11.0 million for the nine months ended September 30, 2025.

The increase was primarily attributable to $10.4 million of development service revenue recognized under our collaboration with Nippon Shinyaku in the first nine months of 2025.

Research and Development Expense.

Research and development expenses increased by $8.9 million, from $103.7 million for the six months ended June 30, 2024 to $112.6 million for the six months ended June 30, 2025.

Research and development expenses increased by $10.5 million, from $158.1 million for the nine months ended September 30, 2024 to $168.7 million for the nine months ended September 30, 2025.

The increase was primarily attributable to the following:

• an increase of $10.9 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates, largely driven by ABBV-RGX-314 clinical supply and purchases of raw materials;

• an increase of $2.0 million in personnel-related costs as a result of increased headcount of development personnel, net of a $1.7 million decrease in stock-based compensation expense.

The increase in research and development expenses was partially offset by a decrease of $2.7 million in overall costs for clinical trials, preclinical activities and other early-stage development.

• an increase of $4.2 million in personnel-related costs due to increased headcount of development personnel, net of a $1.7 million decrease in stock-based compensation expense.

The increase in research and development expenses was partially offset by a decrease of $4.6 million in costs associated with clinical trials and regulatory activities, largely driven by a decrease in clinical trial expenses for ABBV-RGX-314 and RGX-121 pivotal trials.

General and Administrative Expense.

General and administrative expenses increased by $3.1 million, from $37.1 million for the six months ended June 30, 2024 to $40.2 million for the six months ended June 30, 2025.

General and administrative expenses increased by $3.9 million, from $56.6 million for the nine months ended September 30, 2024 to $60.5 million for the nine months ended September 30, 2025.

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

Interest Expense.

Interest expense increased by $17.1 million, from $2.4 million for the six months ended June 30, 2024 to $19.6 million for the six months ended June 30, 2025.

Interest expense increased by $29.5 million, from $3.2 million for the nine months ended September 30, 2024 to $32.7 million for the nine months ended September 30, 2025.

1 unchanged sentence

Sources of Liquidity

As of June 30, 2025, we had cash, cash equivalents and marketable securities of $363.6 million, which were primarily derived from the royalty monetization in May 2025 and the up-front payment received under the Nippon Shinyaku Collaboration Agreement in March 2025, each as described below, and the sale of our common stock and pre-funded warrants in March 2024.

We expect that our cash, cash equivalents and marketable securities as of June 30, 2025 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 September 30, 2025, we had cash, cash equivalents and marketable securities of $302.0 million, which were primarily derived from the royalty monetization in May 2025 and the up-front payment received under the Nippon Shinyaku Collaboration Agreement in March 2025, each as described below, and the sale of our common stock and pre-funded warrants in March 2024.

We expect that our cash, cash equivalents and marketable securities as of September 30, 2025 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.

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

2 unchanged sentences

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

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

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

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

6 unchanged sentences

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 June 30, 2025, no shares of common stock had been sold under the Leerink ATM Program.

As of September 30, 2025, 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.

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

Six Months Ended June 30,

Nine Months Ended September 30,

Net cash used in operating activities

3 unchanged sentences

Cash Flows from Operating Activities

Our net cash used in operating activities for the six months ended June 30, 2025 decreased by $85.2 million from the six months ended June 30, 2024, largely as a result of the $110.0 million up-front fee received from Nippon Shinyaku in March 2025.

Our net cash used in operating activities for the nine months ended September 30, 2025 decreased by $69.8 million from the nine months ended September 30, 2024, largely as a result of the $110.0 million up-front fee received from Nippon Shinyaku in March 2025.

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

For the six months ended June 30, 2025, our net cash used in operating activities of $15.7 million consisted of a net loss of $64.8 million, offset by favorable changes in operating assets and liabilities of $24.0 million and adjustments for non-cash items of $25.1 million.

The changes in operating assets and liabilities include an increase in deferred revenue of $37.7 million, which was driven primarily by the deferred portion of the $110.0 million up-front payment received under our collaboration with Nippon Shinyaku in the first quarter of 2025.

The favorable changes in operating assets and liabilities were partially offset by an increase in prepaid expenses and other current assets of $7.3 million, which was driven primarily by an increase in net cost reimbursement due from AbbVie under our ABBV-RGX-314 collaboration and increases in prepaid clinical trial services and software licenses.

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

For the nine months ended September 30, 2025, our net cash used in operating activities of $71.7 million consisted of a net loss of $126.7 million, offset by adjustments for non-cash items of $41.5 million and favorable changes in operating assets and liabilities of $13.6 million.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $26.2 million and depreciation and amortization expense of $11.7 million.

For the six months ended June 30, 2024, our net cash used in operating activities of $101.0 million consisted of a net loss of $116.3 million and unfavorable changes in operating assets and liabilities of $12.2 million, offset by adjustments for non-cash items of $27.6 million.

The changes in operating assets and liabilities include a decrease in total accounts payable and accrued expenses and other current liabilities of $14.0 million, which was driven largely by decreases in accrued sublicense fees, royalties and personnel-related expenses.

The changes in operating assets and liabilities include an increase in deferred revenue of $34.3 million, which was primarily attributable to the deferred portion of the $110.0 million up-front payment received under our collaboration with Nippon Shinyaku in the first quarter of 2025.

The favorable changes in operating assets and liabilities were partially offset by an increase in prepaid expenses and other current assets of $9.5 million, which was largely driven by an increase in net cost reimbursement due from AbbVie under our ABBV-RGX-314 collaboration, and an increase in accounts receivable of $7.1 million, which was driven largely by reimbursable costs due from Nippon Shinyaku under our collaboration for RGX-121 and RGX-111.

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

For the nine months ended September 30, 2024, our net cash used in operating activities of $141.5 million consisted of a net loss of $175.9 million and unfavorable changes in operating assets and liabilities of $6.1 million, offset by adjustments for non-cash items of $40.5 million.

The changes in operating assets and liabilities include a decrease in total accounts payable, accrued expenses and other current liabilities, and other liabilities of $7.5 million, which was driven largely by decreases in accrued personnel costs, sublicense fees payable to licensors and amounts payable to suppliers as of the end of the period.

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 $28.9 million and depreciation and amortization expense of $12.2 million.

Cash Flows from Investing Activities

For the six months ended June 30, 2025, our net cash used in investing activities consisted of $230.3 million used to purchase marketable debt securities and $1.4 million used to purchase property and equipment, offset by $136.0 million in maturities of marketable debt securities.

For the six months ended June 30, 2024, our net cash provided by investing activities consisted of $151.7 million in maturities of marketable debt securities, offset by $137.7 million used to purchase marketable debt securities and $1.0 million used to purchase property and equipment.

For the nine months ended September 30, 2025, our net cash used in investing activities primarily consisted of $269.8 million used to purchase marketable debt securities and $1.9 million used to purchase property and equipment, partially offset by $218.3 million in maturities of marketable debt securities.

For the nine months ended September 30, 2024, our net cash provided by investing activities consisted of $238.4 million in maturities of marketable debt securities, offset by $173.9 million used to purchase marketable debt securities and $1.4 million used to purchase property and equipment.

Cash Flows from Financing Activities

For the six months ended June 30, 2025, our net cash provided by financing activities primarily consisted of $144.5 million in proceeds received from the issuance of the 2025 Royalty Bond and warrants to HCR in May 2025, net of discounts and transaction costs paid during the period, and was partially offset by $10.9 million of royalties paid, net of interest, under our royalty monetization liabilities.

For the six months ended June 30, 2024, our net cash provided by financing activities primarily consisted of $131.4 million in proceeds received from the public offering of common stock and pre-funded warrants completed in March 2024, net of underwriting discounts and commissions and other offering expenses paid during the period, and $1.5 million in proceeds received from the exercise of stock options and issuance of common stock under our employee stock purchase plan.

For the nine months ended September 30, 2025, our net cash provided by financing activities primarily consisted of $144.5 million in proceeds received from the issuance of the 2025 Royalty Bond and warrants to HCR in May 2025, net of discounts and transaction costs paid during the period, and was partially offset by $18.4 million of royalties paid, net of interest, under our royalty monetization liabilities.

For the nine months ended September 30, 2024, our net cash provided by financing activities primarily consisted of $131.1 million in proceeds received from the public offering of common stock and pre-funded warrants completed in March 2024, net of underwriting discounts and commissions and other offering expenses paid during the period, and $2.7 million in proceeds received from the exercise of stock options and issuance of common stock under our employee stock purchase plan.

Our net cash provided by financing activities was partially offset by $32.2 million of royalties paid, net of interest, under our royalty monetization liabilities.

4 unchanged sentences

Future Funding Requirements

We have incurred cumulative losses since our inception and had an accumulated deficit of $996.9 million as of June 30, 2025.

We have incurred cumulative losses since our inception and had an accumulated deficit of $1.06 billion as of September 30, 2025.

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

28 unchanged sentences

For information regarding market risk, refer to Item 7A, “Quantitative and Qualitative Disclosures About Market Risk,” included in our Annual Report on Form 10-K for the year ended December 31, 2024.

There have been no material changes to our exposure to market risk during the six months ended June 30, 2025.

There have been no material changes to our exposure to market risk during the nine months ended September 30, 2025.

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-Q

Item 2. Management’s Discussion and Analysis