What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-08-07 compared with 2025-05-12 · 122 added, 71 removed, 266 unchanged (42% of the section changed)

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• ABBV-RGX-314:

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

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

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

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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).

Based on this favorable safety profile, the Phase II AAVIATE trial continues to enroll 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 enrolling a new cohort of patients with center-involved DME.

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

In August 2025, we announced new data from the ALTITUDE trial and plans to initiate a pivotal program.

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.

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.

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

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

Enrollment completed in this cohort in June 2025.

DME is a vision-threatening complication of DR;

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

The initiation of the pivotal study, which is expected 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.

In March 2025, we presented additional positive Phase I/II biomarker data, including the first biomarker data from the cohort of patients aged 1-3, at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.

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

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

As of May 2025, we had reported positive microdystrophin data on 12 patients and positive initial functional data from five patients.

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.

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 also expect to share additional efficacy and safety data from the Phase I/II study, including additional functional data, in the first half of 2025.

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.

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

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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 FDA approval of RGX-121 in the second half of 2025.

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.

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

Collaboration and License Agreement with AbbVie

AbbVie Collaboration for ABBV-RGX-314

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

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

Collaboration and License Agreement with Nippon Shinyaku

In August 2025, we and AbbVie entered into an amendment to the AbbVie Collaboration Agreement which modifies 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.

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.

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

Nippon Shinyaku Collaboration for RGX-121 and RGX-111

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

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We 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 $71.8 million of revenue under the Nippon Shinyaku Collaboration Agreement in the first quarter of 2025.

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

Overview of Our NAV Technology Platform

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

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

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

NAV Technology Licensing Platform

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

As of March 31, 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 potential additional revenue opportunities.

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.

Financial Overview

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

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

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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 months ended March 31, 2025 and 2024 (in thousands):

Three Months Ended March 31,

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,

Direct Expenses

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* 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 three months ended March 31, 2025 and 2024 include $14.7 million and $25.0 million, respectively, in net cost reimbursement from AbbVie under our eye care collaboration, which were recorded as a reduction of research and development expenses.

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

Direct expenses related to the development of ABBV-RGX-314 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.

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

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

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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 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.

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

Other Income (Expense)

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Interest Expense

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

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

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

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.

Critical Accounting Policies and Estimates

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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:

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

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

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

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

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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.

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

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

License and royalty revenue

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Total operating expenses

Income (loss) from operations

Loss from operations

Other Income (Expense)

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Total other income (expense)

Net income (loss)

Comparison of the Three Months Ended March 31, 2025 and 2024

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

License and Royalty Revenue.

License and royalty revenue increased by $71.7 million, from $15.3 million for the three months ended March 31, 2024 to $87.0 million for the three months ended March 31, 2025.

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

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.

Service Revenue.

Service revenue increased by $1.7 million, from $0.3 million for the three months ended March 31, 2024 to $2.0 million for the three months ended March 31, 2025.

The increase was primarily attributable to $1.8 million of development service revenue recognized under our collaboration with Nippon Shinyaku in the first quarter of 2025.

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.

Research and Development Expense.

Research and development expenses decreased by $1.8 million, from $54.8 million for the three months ended March 31, 2024 to $53.1 million for the three months ended March 31, 2025.

The decrease was primarily attributable to a $4.1 million decrease in costs associated with clinical trial activities, largely driven by clinical trial expenses for ABBV-RGX-314 and RGX-121 pivotal trials, and a $1.4 million decrease in preclinical activities and other early-stage research and development.

The decrease in research and development expenses was partially offset by an increase of $3.2 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates.

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.

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.

General and Administrative Expense.

General and administrative expenses increased by $2.1 million, from $18.3 million for the three months ended March 31, 2024 to $20.3 million for the three months ended March 31, 2025.

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

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.

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.

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

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.

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.

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.

The increase was primarily attributable to the following:

• an increase of $8.9 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.

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.

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.

Liquidity and Capital Resources

Sources of Liquidity

As of March 31, 2025, we had cash, cash equivalents and marketable securities of $272.7 million, which were primarily derived from the sale of our common stock and pre-funded warrants in March 2024 and the up-front payment received under the Nippon Shinyaku Collaboration Agreement in March 2025, as described below.

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

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

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

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

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

Prior to the maturity date, interest and principal under the 2025 Royalty Bond 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.

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.

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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 March 31, 2025, no shares of common stock had been sold under the Leerink ATM Program.

As of June 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):

Three Months Ended March 31,

Net cash provided by (used in) operating activities

Net cash provided by investing activities

Net cash provided by (used in) financing activities

Six Months Ended June 30,

Net cash used in operating activities

Net cash provided by (used in) investing activities

Net cash provided by financing activities

Net increase in cash and cash equivalents and restricted cash

Cash Flows from Operating Activities

Our net cash provided by operating activities for the three months ended March 31, 2025 increased by $89.1 million from the three months ended March 31, 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 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.

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 three months ended March 31, 2025, our net cash provided by operating activities of $33.6 million consisted of net income of $6.1 million, adjustments for non-cash items of $11.1 million and favorable changes in operating assets and liabilities of $16.4 million.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $8.5 million and depreciation and amortization expense of $4.0 million, partially offset by the accretion of discounts on marketable debt securities during the period.

The changes in operating assets and liabilities include an increase of $39.8 million in deferred revenue, 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 a decrease in total accounts payable and accrued expenses and other current liabilities of $18.1 million, which was largely driven by decreases in accrued personnel-related expenses, royalties and external research and development services, as well as a total increase in prepaid expenses and other current assets of $4.3 million, which was driven primarily by increases in prepaid software licenses and net cost reimbursement due from AbbVie under our ABBV-RGX-314 collaboration.

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 three months ended March 31, 2024, our net cash used in operating activities of $55.5 million consisted of a net loss of $63.3 million and unfavorable changes in operating assets and liabilities of $8.2 million, offset by adjustments for non-cash items of $16.0 million.

The changes in operating assets and liabilities include a net decrease in total accounts payable and accrued expenses and other current liabilities of $8.6 million, which were driven primarily by decreases in accrued sublicense fees, royalties and personnel-related expenses, and an increase in other current assets of $6.8 million, which was driven primarily by an increase in net

cost reimbursement due from AbbVie under our ABBV-RGX-314 collaboration.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $17.2 million and depreciation and amortization expense of $7.9 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.

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

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Cash Flows from Investing Activities

For the three months ended March 31, 2025, our net cash provided by investing activities consisted of $64.9 million in maturities of marketable debt securities, offset by $19.2 million used to purchase marketable debt securities and $1.0 million used to purchase property and equipment.

For the three months ended March 31, 2024, our net cash provided by investing activities consisted of $68.9 million in maturities of marketable debt securities, offset by $55.2 million used to purchase marketable debt securities and $0.6 million used to purchase property and equipment.

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.

Cash Flows from Financing Activities

For the three months ended March 31, 2025, our net cash used in financing activities primarily consisted of $5.6 million of Zolgensma royalties paid, net of imputed interest, under our royalty purchase agreement with HCR.

For the three months ended March 31, 2024, our net cash provided by financing activities primarily consisted of $131.6 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 $1.3 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 $11.2 million of Zolgensma royalties paid, net of imputed interest, under our royalty purchase agreement with HCR.

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.

Our net cash provided by financing activities was partially offset by $20.5 million of royalties paid, net of interest, under our royalty monetization liabilities.

Additional Capital Requirements

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

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

Our material commitments and obligations are further described in Item 7, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” of our Annual Report on Form 10-K for the year ended December 31, 2024, and in the notes to the audited consolidated financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2024.

Other than the changes described in the notes to the unaudited consolidated financial statements accompanying this Quarterly Report on Form 10-Q, including Note 8, “Commitments and Contingencies,” there have been no material changes to our commitments and obligations since December 31, 2024.

Other than the changes described in the notes to the unaudited consolidated financial statements accompanying this Quarterly Report on Form 10-Q, including Note 7, "Royalty Monetization Liabilities," and Note 8, “Commitments and Contingencies,” there have been no material changes to our commitments and obligations since December 31, 2024.

Future Funding Requirements

We have incurred cumulative losses since our inception and had an accumulated deficit of $926.1 million as of March 31, 2025.

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

14 unchanged sentences

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

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

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

• revenue received from commercial sales of Zolgensma;

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

• the timing and amount of Zolgensma royalties and other specified royalties, milestones and consideration under our license agreements and collaborations paid to HCR under our royalty monetization agreements;

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

9 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 three months ended March 31, 2025.

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