RGNX 10-K & 10-Q changes, risk factors and insider trading

We may encounter substantial delays in ourOur planned clinical trials,trials may be substantially delayed, or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.

Delays in obtaining regulatory approval of our manufacturing process and facility or disruptions in our manufacturing process and product testing may delay or disrupt our commercialization efforts.

Third parties we rely upon to conductmanufacture and supply materials for our programs including ingredients and key components for our product manufacturingcandidates and to perform quality testing may not perform satisfactorily.

Issued patents covering our NAV Technology Platform or our product candidates could be found invalidinvalid, unenforceable or unenforceable.found to lack patent eligibility by the courts or patent offices.

Changes in U.S. patent law including appellate interpretation of patent eligibility in biotechnology could diminish the value of patents in general,general and biotechnology patents specifically, thereby impairing our ability to protect our products.

Our certificate of incorporation includes an exclusive forum clausesclause for certain litigation.

Our business could be negatively affected as a result of the actions of activist stockholders.stockholders or stockholder litigation.

Our gene therapy product candidates are based on a novel technology that makes it difficult to predict the time and cost of development and of subsequently obtaining regulatory approval. Only a fewsmall number of gene therapy products have been approved in the United States, the European Union or elsewhere.

We have concentrated our research and development efforts on our proprietary AAV gene delivery platform (our NAV Technology Platform), and we have granted licenses to certain intellectual property related to our NAV Technology Platform to our NAV Technology Licensees.Licensees and collaborators. Our future success depends on our and our NAV Technology Licensees’ and collaborators’ successful development and commercialization of viable gene therapy product candidates. There can be no assurance that we or our NAV Technology Licensees and collaborators will not experience problems or delays in developing current or future product candidates or that such problems or delays will not cause unanticipated costs, or that any such development problems can be solved. We also may experience unanticipated problems or delays in expanding our manufacturing capacity, and this may prevent us from completing our clinical trials, meeting the obligations of our collaborations or commercializing our products on a timely or profitable basis, if at all. For example, we, a partner or another group may uncover one or more previously unknown risks associated with AAV or our NAV Technology Platform, and this may prolong the period of observation required for obtaining regulatory approval, necessitate additional clinical testing or invalidate our NAV Technology.

In addition, the clinical trial requirements of the FDA, the EMA and other regulatory authorities and the criteria these regulators use to determine the quality, safety and efficacy of a product candidate vary substantially according to the type, complexity, novelty and intended use and market of such product candidates. The regulatory approval process for novel product candidates such as ours can be significantly more expensive and take longer than for other,product candidates from categories that are better known or morethat extensivelyhave been studied productmore candidates.extensively. Only a fewsmall number of gene therapy products have been approved in the United States, the European Union or elsewhere.elsewhere, including less than ten AAV-based gene therapies approved in the United States as of the end of 2025. It is difficult to determine how long it will take or how much it will cost to obtain regulatory approvals for our product candidates in the United States, the European Union or elsewhere, or how long it will take to commercialize our product candidates, if approved. Furthermore, approvals by one regulatory authority may not be indicative of what other regulatory authorities may require for approval, and approvals of ex vivo gene therapy products (such as autologous CAR T-cell therapies) may not be indicative of what may be required for approval of in vivo gene therapy products.products (such as directly administered AAV-based gene therapies).

Our business depends substantially on the success of our lead product candidates. If we are unable to obtain regulatory approval for, or successfully commercialize, our lead product candidates, our business will be materially harmed.

Our product candidates will require substantial clinical development and testing, manufacturing bridging studies and process validation and regulatory approval prior to commercialization. Successful continued development and ultimate regulatory approval of our lead product candidates is critical for our future business success and our ability to generate product revenue. We have invested, and will continue to invest, a significant portion of our financial resources in the development of our lead product candidates. We will need to raise sufficient funds for, and successfully complete, our clinical trials of our lead product candidates in appropriate subjects. The future regulatory and commercial success of these product candidates is subject to a number of risks, including the following:

we may not successfully establish commercial manufacturing capabilities for each of our products;

Of the large number of biologics and drugs in development in the biopharmaceutical industry, only a small percentage result in the submission of a BLA to the FDA or marketing authorization application (MAA) to the EMA and even fewer are expected to be approved for commercialization. Furthermore, even if we do receive regulatory approval to market our lead product candidates, any such approval may be subject to limitations on the indicated use or uses for which we may market the product. Accordingly, even if we are able to obtain the requisite financing to continue to fund our development programs, we cannot assureprovide youany assurance that our lead product candidates will be successfully developed or commercialized. If we or any of our future development partners are unable to develop, obtain regulatory approval for, or, if approved, successfully commercialize, our lead product candidates, we may not be able to generate sufficient revenue to continue our business.

WeClinical programs in rare diseases have limited clinical results for some of our product candidatesdatasets and success in preclinical studies or early clinical trials may not be indicative of results obtained in later trials.

Gene therapy development has inherent risks. Some of ourOur lead product candidates in the rare disease space have limited clinical and preclinical results and we may experience unexpected results in the future. We or any of our future development partners will be required to demonstrate through adequate and well-controlled clinical trials that our product candidates containing our proprietary vectors are safe and effective, with a favorable benefit-risk profile, for use in their target indications before we can seek regulatory approvals for their commercial sale. Drug development is a long, expensive and uncertain process, and delay or failure can occur at any stage of development, including after commencement of any of our clinical trials.

The results of preclinical studies and early clinical trials are not always predictive of future results. Preclinical studies have experimental and animal model limitations, may not detect or predict human adverse effects, and are not powered to detect rare events. Any product candidate we or any of our future development partners advance into clinical trials, including our lead product candidates, may not have favorable results in later clinical trials, if any, or receive regulatory approval. There is a high failure rate for drugs and biologic products proceeding through clinical trials. Data obtained from preclinical and clinical activities are subject to varying interpretations that may delay, limit or prevent regulatory approval. In addition, we may experience regulatory delays or rejections as a result of many factors, including due to changes in regulatory policy during the period of our product candidate development. Any such delays could materially harm our business, financial condition, results of operations and prospects.

During the FDAclinical reviewdevelopment process, we will need to identify success criteria and endpoints for our clinical trials such that the FDA will be able to subsequently evaluate the clinical efficacy and safety profile of our product candidates. As we are developing novel treatments for diseases in which there is little clinical experience with new endpoints and methodologies, there is heightened risk that the FDA, the EMA or other regulatory bodies may not consider the clinical trial endpoints that we select to providedirectly clinicallyreflect meaningful results (reflectinghow a tangibletreatment benefitimpacts toa patients).disease. In addition, we may not successfully achieve the statistical criteria to reflect what is clinically meaningful, or the resulting clinical data and results may be difficult to analyze. EvenFor if the FDA does find our success criteria to be sufficiently validated and clinically meaningful, we may not achieve the pre-specified endpoints to a degree of statistical significance. Further,example, even if we do achieve the pre-specified criteria, we may produce results that are unpredictable or inconsistent with the results of the non-primary endpoints or other relevant data. Further, we may be required to produce more data, including enhanced functional data, increased number of patients and/or data from untreated control arm patients to achieve FDA support. The FDA also weighs the benefits of a product against its risks, and the FDA may view the efficacy results in the context of safety as not being supportive of regulatory approval. The EMA and other regulatory authorities in the European Union and other countries may make similar comments with respect to these endpoints and data, which may jeopardize or preclude our ability to obtain regulatory approvals in the European Union and other jurisdictions.

While we believe our product candidates should be applicable for the treatment of patients with certain conditions, the results from our preclinical and planned clinical trials may not support as broad of a marketing approval as we seek. EvenThis could result in significant cost increases and substantial delays in obtaining, or never obtaining, marketing approval for our product candidates to treat patients. In addition, even if we obtain regulatory approval for our product candidates, we may be required by the FDA, the EMA or other regulatory bodies to conduct additional clinical trials to support approval of our product candidates for patients diagnosed with different mutations of the respective diseases to which our product candidates relate. ThisIn couldaddition, resultwe inmay significantbe costapproved increasesonly andfor substantialsevere delaysforms inof obtaining,a disease or never obtaining, marketing approval for our product candidates to treat patients.condition. The inability to market our product candidates to treat patients for the intended indications would materially harm our business, financial condition, results of operations and prospects.

We may encounter substantial delays in ourOur planned clinical trials,trials may be substantially delayed, or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.

delays in reaching a consensus with or changing requirements by regulatory authorities on trial design;

imposition of a clinical hold by regulatory authorities in trials in addition to RGX-121 and RGX-111;

Any inability to successfully complete research studies and preclinical and clinical development could result in additional costs to us or impair our ability to generate revenues from product sales, regulatory and commercialization milestones and royalties. In addition, if we make manufacturing or formulation changes to our product candidates, we may need to conduct additional studies to bridge our modified product candidates to earlier versions. Clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize our product candidates, if approved, or allow our competitors to bring products to market before we do, which could impair our ability to successfully commercialize our product candidates, if approved, and may harm our business, financial condition, results of operations and prospects. Furthermore, unpredictability in the regulatory environment and the regulatory requirements to obtain approval of our product candidates may adversely impact our ability to raise the capital needed to finance our operations.

receive a clinical hold for a particular product candidate, in addition to RGX-111 and RGX-121, which, if not lifted, could require that we discontinue development of a product candidate;

There have been several significant adverse side effects in gene therapy treatments in the past, including reported cases of leukemia in trials using lentivirus vectors and death seen in trials sponsored by other companies using adenovirus vectors and AAV vectors, including NAV vectors. Gene therapy is still a relatively new approach to disease treatment and additional adverse side effects could be identified. There also is the potential risk of delayed adverse events following exposure to gene therapy products due to persistent biologic activity of the genetic material or other components of products used to carry the genetic material. Possible adverse side effects that could occur with treatment with gene therapy products include an immunologic reaction early after administration which could substantially limit the effectiveness of the treatment. In previous clinical trials involving AAV vectors for gene therapy, some subjects experienced the development of a T-cell response, whereby after the vector is within the target cell, the cellularsubject’s immune response system triggers the removal of transduced cells by activated T-cells. Approved labels for certain gene therapies state that AAV vector DNA can integrate into the host genome at low frequency and that such integration may carry a theoretical risk of malignancy, including hepatocellular carcinoma. Furthermore, in clinical trials sponsored by other companies involving AAV vectors administered intravitreally for the treatment of retinal conditions, serious adverse reactions, such as panuveitis and loss of vision, have occurred. In addition to side effects caused by product candidates, the administration process or related procedures also can cause adverse side effects. If any such adverse events occur in our or third-party trials, our clinical trials could be suspended or terminated. Additionally, if clinically meaningful AAV integration events or an increased incidence of cancer were observed in our studies or in the broader gene therapy field, the FDA or other regulatory authorities could impose additional requirements, place our clinical trials on hold, restrict dosing or patient populations, require enhanced warnings or other labeling limitations, or delay or deny approval of our product candidates. In the RGX-121 CRL, FDA noted that we should include a safety post marketing requirement in the resubmission.

Additionally, if any of our product candidates receives marketing approval, the FDA could require us to adopt a Risk Evaluation and Mitigation Strategy (REMS) and other regulatory authorities could impose other specific obligations as a condition of approval to ensure that the benefits of our product candidates outweigh their risks, which could delay approval of our product candidates. A REMS may include, among other things, a medication guide outlining the risks of the product for distribution to patients; a communication plan to health care practitioners or patients; and elements to assure safe use, which can severely restrict the distribution of a product by, for example, requiring that health care providers receive particular training and obtain special certification prior to prescribing and dispensing the product, limiting the healthcare settings in which the product may be dispensed and subjecting patients to monitoring and enrollment in a registry. If the FDA requires us to adopt a REMS program for our products and we are unable to comply with its requirements, the FDA may deem our products to be misbranded and we may be subject to civil money penalties. The European Commission, the EMA and other regulatory authorities may, following grant of marketing authorization in their territory, impose similar obligations.

We cannot commercialize a product candidate until the appropriate regulatory authorities have reviewed and approved the product candidate. Even if our product candidates meet their safety and efficacy endpoints in clinical trials, the regulatory authorities may not complete their review processes in a timely manner or we may not be able to obtain regulatory approval. The ability and willingness of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory and policy changes. Average review times at the FDA have fluctuated in recent years as a result. Disruptions at the FDA and other agencies may also slow the time necessary for new product candidates to be reviewed and approved by necessary government agencies, which could adversely affect our business. In addition, government funding of other government agencies on which our operations may rely, including those that fund research and development activities, is subject to the political process, which is inherently fluid and unpredictable. Additional delays may result if an FDA Advisory Committee or other regulatory authority recommends non-approval or restrictions on approval. The FDA has recently delayed approvals or issued CRLs for a number of rare disease therapies, including those seeking accelerated approval. Heightened regulatory scrutiny may delay or prevent our ability to resolve the deficiencies identified in the RGX-121 CRL and obtain approval of our product candidates. In addition, we may experience delays or rejections based on additional government regulation from future legislation or administrative action or based on changes in regulatory authority policy during the period of product development, clinical trials and the review process.

Further, the regulatory authorities may require concurrent approval or the CE mark (a mandatory conformity assessment marking for certain products sold within the European Economic Area (the EEA)) of a companion diagnostic device, since it may be necessary to use FDA-cleared or FDA-approved, or CE-marked, diagnostic tests or diagnostic tests approved by other comparable foreign regulatory authorities to diagnose patients or to assure the safe and effective use of our product candidates in trial subjects. FDA refers to such tests as in vitro companion diagnostic devices. The FDA has articulated a policy position that, when safe and effective use of a therapeutic product depends on a diagnostic device, the FDA generally will require approval or clearance of the companion diagnostic device at the same time that the FDA approves the therapeutic product. The FDA’s guidance allows for twoan exceptionsexception to the general rule of concurrent drug/device approval, namely,approval when the therapeutic product is intended to treat serious and life-threatening conditions for which no satisfactory alternative exists,treatment exists and whenthe abenefits seriousfrom safetythe issueuse arisesof for an approvedthe therapeutic agent,product andare so pronounced as to outweigh the fact that no FDA-cleared or FDA-approved companion diagnostic test is yet available. It is unclear how the FDA will apply this policy to our current or future gene therapy product candidates. Should the FDA deem genetic tests used for diagnosing patients for our therapies to be in vitro companion diagnostics requiring FDA clearance or approval, we may face significant delays or obstacles in obtaining approval of a BLA for our product candidates. In this regard, in a 2024 case a U.S. District Court vacated FDA’s final rule to regulate laboratory developed test services, and FDA declined to appeal, creating uncertainty for FDA’s continued regulation of certain tests as companion diagnostics. Uncertainty about the legal status of certain companion diagnostics, including the possibility of additional law or further regulations or guidance from FDA, may create uncertainty that could lead to delay in the development and approval of our product candidates.

We face significant competition in an environment of rapid technological change and there is a possibility that our competitors may achieve regulatory approval before us or develop products that are safer, less expensive or more convenient or effective than ours, any of which may harm our financial condition and our ability to successfully market or commercialize our product candidates.

We have incurred cumulative net losses and have had few profitable quarters since inception.inception Weand expect to regularly incur losses until we have successfully commercialized one or more product candidates and may never achieve or maintain profitability in the future.

Since inception, we have incurred cumulative net losses. We have historically financed our operations primarily through private and public offerings of our equity securities, collaborations and licensing rights to our NAV Technology Platform, including milestone payments and royalties from our NAV Technology Licensees.Licensees and collaborators. We have devoted substantially all of our efforts to research and development, including preclinical and clinical development of our product candidates, and licensing our NAV Technology Platform, as well as to building out our team. We expect that it could be years before we commercialize most of our product candidates, and we can provide no assurance that we will ever be able to do so. We license certain intellectual property related to our NAV Technology Platform to our NAV Technology Licensees and collaborators. Our NAV Technology Licensees and collaborators have multiple preclinical studies and clinical trials in progress. However, only onetwo gene therapy productproducts based on our licensing program, Novartis AG’s Zolgensma,Zolgensma hasand Itvisma, have been approved or commercialized. Other than revenue in connection with sales of Zolgensma,Zolgensma and Itvisma, we may generate only limited recurring revenue in the near term from our current NAV Technology Licensees and collaborators. We expect to continue to incur significant expenses and regularly incur operating losses for the foreseeable future. The net losses we incur may fluctuate significantly from quarter to quarter. We anticipate that our expenses will increase substantially if, and as, we:

We will need to raise additional funding, which may not be available on acceptable terms, or at all.all, Failureand failure to obtaindo this necessary capital when neededso may force us to delay, limit or terminate certain of our licensing activities, product development and commercialization efforts or other operations.

We expect to require substantial future capital in order to complete research studies, preclinical and clinical development for our current product candidates and any future product candidates, and potentiallyto commercialize these product candidates, if approved. We expect our spending levels to increase in connection with our preclinical and clinical trials of our product candidates. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant expenses related to product sales, medical affairs, marketing, manufacturing and distribution. Accordingly, we will need to obtain substantial additional funding in connection with our continuing operations. Increased unpredictability in the regulatory environment and the regulatory requirements to obtain approval of our product candidates may adversely impact our ability to raise the capital needed to finance our operations. If we are unable to raise capital when needed or on favorable terms, we could be forced to delay, reduce or eliminate certain of our licensing activities, our research and development programs or other operations.

revenue received from commercial sales of Zolgensma and Itvisma, and the timing and amount of Zolgensma and Itvisma royalties paid to Healthcare Royalty Management, LLC (collectively and with other affiliated entities, HCR) under our royalty purchasemonetization agreementagreements;

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 collaborationscollaborations, and the timing and amount of any such revenues payable to HCR under our royalty monetization agreements;

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

Although we have generated significant revenues from licensing our NAV Technology Platform and our other intellectual property, such as our licensing pursuant to the AbbVie Collaboration and License Agreement and may do so under the Nippon Shinyaku Collaboration and License Agreement, we have never generated revenue from sales of our product candidates and may never do so in the future.

We have generated significant revenues from licensing our NAV Technology Platform, including sublicense fees, milestone payments and royalties on net sales of a licensed product,products, Zolgensma,Zolgensma and Itvisma, and licensing our intellectual property to AbbViecollaborators pursuantunder toour thelicense and collaboration agreements with AbbVie Collaboration and License Agreement and may do so in the future pursuant to the Nippon Shinyaku Collaboration and License Agreement.Shinyaku. However, our ability to generate revenue from sales of our internal product candidates will depend on our ability, alone or with partners, to successfully complete the development of, and obtain the regulatory approvals necessary to commercialize, our product candidates.

Many of these factors as they relate to our licensees’ and collaborators’ products, including Zolgensma,Zolgensma and Itvisma, will be outside our control, and future revenues in connection with sales of such products may be precluded or limited by any of these factors.

For a full discussion and analysis of financial condition and results of operations for the year ended December 31, 2023,2024, including a year-over-year comparison to the year ended December 31, 2022,2023, please read the “Management's Discussion and Analysis of Financial Condition and Results of Operations” section of our Annual Report on Form 10-K for the year ended December 31, 2023,2024, which we filed with the SEC on FebruaryMarch 27,13, 2024.2025.

ABBV-RGX-314: We are developing ABBV-RGX-314 (surabgene lomparvoveclomparvovec, 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. 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). Additionally, we are planning a Phase IIb/III program in DR and expect to dose the first patient in a two-part Phase IIb/III study (NAAVIGATE) in the second quarter of 2026. ABBV-RGX-314 uses the NAV® AAV8 vector to deliver a gene encoding a therapeutic antibody fragment to inhibit vascular endothelial growth factor (VEGF). We have licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. (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 for the treatment of patients with wet AMD using subretinal delivery.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.the fourth quarter of 2026 in partnership with AbbVie.

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. BasedIn onNovember 2023, we announced data showing ABBV-RGX-314 was well tolerated at dose levels 1 and 2 and positive interim results from this trial, we, with partner AbbVie, conducted an End-of-Phase II meeting with the FDA in the fourth quartersignals of 2024.efficacy, Inincluding January20.8% 2025,of wepatients andexhibiting AbbVie> announced2-step weDiabetic willRetinopathy planSeverity aScale Phase(DRSS) IIIimprovement programwithout foradditional ABBV-RGX-314DR intreatment DR.at Theone program is expected to support global regulatory submissions.year.

The ALTITUDE trial is now enrollingincludes a new cohort of patients with center-involved DME.DME evaluating ABBV-RGX-314 at dose level 4. Enrollment completed in this cohort in June 2025. DME is a vision-threatening complication of DR; an estimated 34 million people globally have DME. Patients will receivereceived 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.

RGX-202: We are developing RGX-202 as an investigational AAV therapeutic for the treatment of Duchenne muscular dystrophy (Duchenne), using the NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal domain as well as a muscle-specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne. Other differentiating elements of RGX-202 include the proactive immune suppression regimen and in-house, state-of-the-art manufacturing that has demonstrated leading purity levels in Duchenne (>80% full capsids).

AFFINITY DUCHENNE® is a multicenter, open-label Phase I/II/III trial to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous dose of RGX-202 in patients with Duchenne aged one and older. The initiation of the pivotal study as well as positive safety and efficacy data from the Phase I/II portion of the study, including the first functional data, were announced in November 2024. In March 2025, we announced that the pivotal study was nearly 50% enrolled, and we expectdesigned to completeenroll enrollmentapproximately 30 patients in the study in 2025, share top line data in the first half of 2026U.S. and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026.Canada.

As of March 2026, we plan to work with the FDA to address the clinical hold and CRL, and discuss potential paths forward for the program, Potential approval of the BLA for RGX-121 could result in receipt of a Rare Pediatric Disease Priority Review Voucher in 2025,(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.

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 areas set to be reinitiated following our announcement in January 2025part of athe strategic partnership with Nippon Shinyaku.Shinyaku are ongoing.

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 (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. 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.

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 beis 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 receivereceived 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 Licensing Platform

In addition to our internal product development efforts, we also selectively license the NAV Technology Platform and other intellectual property rights to other leading biotechnology and pharmaceutical companies, which we refer to as NAV Technology Licensees. As of December 31, 2024,2025, our NAV Technology Platform was being applied in onetwo commercial product,products, Zolgensma® and Itvisma®, and the preclinical and clinical development of a number ofvarious 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.

Our revenues to date consisthave been primarily of license and royalty revenue resultinggenerated from the licensing of our NAV Technology Platform and other intellectual property rights.rights to NAV Technology Licensees and collaborators. We have not generated any revenues from commercial sales of our own products. If we fail to complete the development of our product candidates in a timely manner or obtain regulatory approval and adequate labeling, our ability to generate future revenues will be materially compromised.

We license our NAV Technology Platform and other intellectual property rights to other biotechnology and pharmaceutical companies, including collaborators for the joint development and commercialization of our product candidates. The terms of the licenses vary, and licenses may be exclusive or non-exclusive and may be sublicensable by the licensee. Licenses may grant intellectual property rights for purposes of internal and preclinical research and development only, or may include the rights, or options to obtain future rights, to commercialize drug therapies for specific diseases using the NAV Technology Platform and other licensed rights. License agreements generally have a term at least equal to the life of the underlying patents, but are terminable at the option of the licensee. Consideration frompayable licenseesto us under our license and collaboration agreements may include: (i) up-front and annual fees, (ii) milestone payments based on the achievement of certain development and sales-based milestones, (iii) sublicense fees, (iv) royalties on sales of licensed products, (v) fees for services related to the development and manufacturing of licensed products and (vi) other consideration payable upon optional goods and services purchased by licensees.licensees and collaborators.

Future revenues under our license and royaltycollaboration revenuesarrangements are dependent on the successful development and commercialization of licensed products, which is uncertain, and revenues may fluctuate significantly from period to period. Additionally, we may never receive consideration inunder our license or collaboration agreements that is contemplated on optionoptional fees,goods and services, development and sales-based milestone payments,milestones, royalties on sales of licensed products or sublicense fees, given the contingent nature of these payments. Our revenues are concentrated among a low number of licensees and licensescollaborators and the arrangements are terminable at the option of the licensee.counterparty. The termination of our licenseslicense byand licenseescollaborations arrangements may materially impact the amount of revenue we recognize in future periods. Please refer to Note 17,16, “Segment and Geographical Information” to the accompanying audited consolidated financial statements for a description of segment and geographical information regarding our revenues.

Zolgensma and Itvisma Royalties

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma,Zolgensma and Itvisma, which isare marketed by Novartis Gene Therapies, Inc. (formerly AveXis, Inc.) (Novartis Gene Therapies), a wholly owned subsidiary of Novartis AG (Novartis), for the treatment of spinal muscular atrophy (SMA). Zolgensma isand aItvisma are licensed productproducts under our license agreement with Novartis Gene Therapies for the development and commercialization of treatments for SMA using the NAV Technology Platform.

Our operating expenses consist primarily of cost of license and royalty revenues, research and development expenses and general and administrative expenses. Personnel costs including salaries, wages, benefits, bonuses and stock-based compensation expense, comprise a significant component of research and development and general and administrative expenses. We allocate indirect expenses associated with our facilities, information technology costs, depreciation and other overhead costs between research and development and general and administrative categories based on employee headcount and the nature of work performed by each employee or using other reasonable allocation methodologies.

Cost of License and Royalty Revenues

Our cost of license and royalty revenues consists primarily of upstream fees due to our licensors as a result of revenue generated from the licensing of our NAV Technology Platform and other intellectual property rights, including sublicense fees and royalties on net sales of licensed products. Sublicense fees are based on a percentage of license fees received by us from licensees and are recognized in the period that the underlying license revenue is recognized. Royalties are based on a percentage of net sales of licensed products by licensees and are recognized in the period that the underlying sales occur. Future costs of revenues are uncertain due to the nature of our license agreements and significant fluctuations in cost of license and royalty revenues may occur from period to period.

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 years ended December 31, 2024, 20232025 and 20222024 (in thousands):

Direct expenses related to the development of ABBV-RGX-314 for the years ended December 31, 2024, 20232025 and 20222024 include net cost reimbursement from AbbVie under our eye care collaboration of $78.3 million, $74.2$60.2 million and $19.3$78.3 million, respectively, which were recorded as a reduction of research and development expenses. NetIn addition to reimbursement of direct development expenses, net cost reimbursement from AbbVie includes reimbursement of personnel and overhead costs attributable to the development of ABBV-RGX-314, the underlying costs of which are reported as unallocated expenses in the table above. We typically utilize our employee and infrastructure resources across our development programs. As a result, we generally do not allocate personnel and other internal costs, such as facilities and other overhead costs, to specific product candidates or development programs.

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

Our general and administrative expenses consist primarily of salaries, wages and personnel-related costs, including benefits, travel and stock-based compensation, for employees performing functions other than research and development. 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 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.

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. Marketable securities are comprised of available-for-sale debt securities.

We license our NAV Technology Platform and other intellectual property rights to other biotechnology and pharmaceutical companies.companies, including collaborators for the joint development and commercialization of our product candidates. The terms of the licenses vary, and licenses may be exclusive or non-exclusive and may be sublicensable by the licensee. Licenses may grant intellectual property rights for purposes of internal and preclinical research and development only, or may include the rights, or options to obtain future rights, to commercialize drug therapies for specific diseases using ourthe NAV Technology Platform and other licensed rights. License agreements generally have a term at least equal to the life of the underlying patents, but are terminable at the option of the licensee. Consideration payable to us under our license and collaboration agreements may include: (i) up-front and annual fees, (ii) milestone payments based on the achievement of certain development and sales-based milestones, (iii) sublicense fees, (iv) royalties on sales of licensed products, (v) fees for services related to the development and manufacturing of licensed products and (vi) other consideration payable upon optional goods and services purchased by licensees.licensees and collaborators.

Our license and collaboration agreements are accounted for as contracts with customers within the scope of ASC 606, with the exception of transactions for which the counterparty is determined not to be a customer. At the inception of each license agreement, we determine the contract term for purposes of applying the requirements of ASC 606. Licenses are generally terminable at the option of the licensee with advance notice to us. For each license granted, including licenses granted upon the exercise of license options, we evaluate these termination rights to determine whether a substantive termination penalty would be incurred by the licensee upon termination. If the licensee incurs a substantive termination penalty upon termination, the contract term for revenue recognition purposes is generally equal to the stated term of the license, which is the life of the underlying licensed patents. Alternatively, if the licensee does not incur a substantive termination penalty upon termination, the contract term for revenue recognition purposes may be shorter than the stated term of the license, in which case the termination rights may be accounted for as contract renewal options. The determination of whether a substantive termination penalty is associated with the termination rights requires significant judgment. In making this determination, we consider, among other things, the nature of the intellectual property rights that would be returned to us upon termination, including the exclusivity of the licensed rights and the stage of development of the licensed products, the payment terms, including the amount and timing of non-refundable or guaranteed payments, and the business purpose of the termination rights granted to the licensee. Generally, the most significant judgment in determining whether a substantive termination penalty exists relates to the amount of any up-front or guaranteed non-refundable payments relative to the amount of annual payments that may be avoided by the licensee upon termination of the license. We consider all of the facts and circumstances relevant to each license when making this determination.

Performance obligations under our license and collaboration agreements may include (i) the delivery of intellectual property licenses, (ii) options granted to licensees to acquire additional licenses, to the extent the options represent material rights to the licensee,development and (iii) research and developmentmanufacturing services to be performed by us related to licensed products.products Licenseand agreements may provide licensees with contract renewal(iii) options or optionsgranted to acquirepurchase additional licenses, goods orand other services. Options are evaluated at the inception of the license agreement to determine whether they provide material rightsservices, to the licensee. In making this determination, we consider whetherextent 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 aconvey material right to the licensee and is accounted for as a separate performance obligation under the current license agreement.rights. At the inception of each license agreement which contains performance obligations for researchdevelopment, andmanufacturing developmentor other services, we evaluate whether the license is distinct from the research and development services, which requires judgment. In making this determination, we consider, among other things, the stage of development of the licensed products and whether the research and development services will significantly impact further development of the licensed products. 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. Agreements may provide licensees and collaborators with options to purchase additional goods or other services, including options to purchase commercial supply of licensed products. Options are evaluated at the inception of the agreement to determine whether they provide material rights to the customer. In making this determination, we consider whether the options are priced at an incremental discount to the standalone selling price of the underlying goods or services, in which case the option is considered to be a material right. Material rights are accounted for as separate performance obligations under the current arrangement.

We evaluate the transaction price of our license and collaboration agreements at thecontract inception of each agreement and at each reporting date. The transaction price includes the fixed consideration payable to us duringover the contract term, as well as any variable consideration to the extent that it is probable that a significant reversal of revenue will not occur in the future. Fixed consideration under the license agreements may include up-front and annual fees payable duringto us over the contract term and fixed fees for developmentdevelopment, servicesmanufacturing relatedand toother licensed products.services. Variable consideration under the license agreements may include development and sales-based milestone payments, payments for development, manufacturing and other services, sublicense fees and royalties on sales of licensed products. Consideration contingent upon the exercise of options by athe licenseecustomer is excluded from the transaction price and not accounted for as part of the license agreementarrangement until the option is exercised.

The transaction price forof eachour license agreementand collaboration arrangements is allocated to the underlying performance obligations based on their relative standalone selling prices and recognized as revenue when (or as) the performance obligations are satisfied. Variable consideration payable based on services performed is allocated directly to the performance obligation for such services. Consideration allocated to performance obligations for the delivery of an intellectual property licenselicenses is recognized as license and royalty revenue in full upon the delivery of the license to the licensee.license. Consideration allocated to performance obligations for licensedevelopment, optionsmanufacturing and other services is recognized as service revenue inas fullwe uponperform the earlier of the option exercise or expiration. The exercise of a license option by a licensee is accounted for as a new license for revenue recognition purposes.services. Consideration allocated to performance obligations for researchmaterial rights to purchase additional goods and development services is recognized as revenue asupon the satisfaction of the performance obligations underlying the optional goods and services purchased by the customer. Service revenue is recognized using a measure of progress that best reflects the pattern of satisfaction of the performance obligations. At each reporting date, we re-evaluate the measure of progress and adjust service revenue on a cumulative catch-up basis to reflect our best estimate of the services areperformed to date versus the total services to be performed byunder us.the arrangement.

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:below.

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). Sura-vecIn wet AMD, sura-vec is currently being evaluated in multiple clinical trials,trials utilizing subretinal delivery, including two pivotal trials (ATMOSPHERE and ASCENT) where enrollment has been completed, one long-term follow-up study and a fellow eye sub-studysub-study. inIn patientsDR, withwe wetare AMD,actively allenrolling utilizinga subretinalpivotal, two-part Phase IIb/III study (NAAVIGATE) using suprachoroidal delivery. Additionally,In addition to these late-stage pivotal programs, sura-vec is being evaluated in two Phase II clinical trials in patients with wet AMD (AAVIATE) and DR (ALTITUDE), which 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 sura-vec in diabetic macular edema (DME). Additionally, we have activated U.S. clinical sites and initiated enrollment of a pivotal trial in DR and expect to dose the first patient in the two-part Phase IIb/III study (NAAVIGATE) in the second quarter of 2026. Sura-vec uses the NAV® AAV8 vector to deliver a gene encoding a therapeutic antibody fragment to inhibit vascular endothelial growth factor (VEGF). We have licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. (Clearside) to deliver gene therapy treatments to the suprachoroidal space of the eye.

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 trialstrials, which together enrolled over 1,200 participants across more than 200 sites, are expected to support global regulatory submissions including with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Topline data from these trials are expected to be sharedannounced in the fourth quarter of 2026 in partnership with AbbVie, with global regulatory submissions expected in 2027.

Concurrent with the two-year data announcement inIn August 2025, we and AbbVie announced an amendment to our collaboration agreement and plans to initiate a pivotal program consisting of a Phase IIb/III trial (NAAVIGATE) as well as a second Phase III trial. NAAVIGATE is a two-part, multicenter, randomized, masked, sham-controlled Phase IIb/III study to evaluate the safety and efficacy of a one-time, in-office administration of sura-vec in subjects with non-proliferative diabetic retinopathy (NPDR) without center-involved diabetic macular edema (CI-DME). The primary endpoint is > 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) at one year. We are actively enrolling the Phase IIb/III NAAVIGATE trial and, in June 2026, dosed the first patient in the Phase IIb portion of the trial, upon which we earned a $100.0 million milestone payment that was received from AbbVie in July 2026. Following an interim analysis of part one (Phase IIb) portion of the NAAVIGATE trial, we and AbbVie willexpect to initiate a Phase III expansion, including part two (Phase III) of the U.S. NAAVIGATE trial and a parallel global trial led by AbbVie. We have activated U.S. clinical sites and initiated enrollment of the Phase IIb/III NAAVIGATE trial and expect to dose the first patient in the second quarter of 2026, upon which we are entitled to a $100.0 million milestone payment from AbbVie.

In October 2025, we announced that enrollment had completed in the pivotal portion of AFFINITY DUCHENNE,DUCHENNE which was designed to enroll (n=approximately 30 patients in the U.S. and Canada, and that we continue to enroll participants in the ongoing confirmatory trial.).

InAs of May 2026, in recent discussions withdiscussions, the FDA, the agencyFDA shared that the use of RGX-202 microdystrophin expression as a surrogate endpoint will be based on the correlation analysis with clinical outcomes, which has been clearly demonstrated in our interim data. While the FDA has recommended a randomized controlled trial, it has guided that externally controlled trials may be adequate for demonstrating substantial evidence of effectiveness, especially when the treatment effect is sufficiently large enough to overcome limitations of externally controlled trials. The FDA offered to review the RGX-202 data and alternative proposals. We plan to discuss this data with the FDA at a future meeting. We are also finalizing the trial design for a study of RGX-202 outside the United States to support global regulatory submissions.

Given the positive topline pivotal data, continued favorable safety profile, and statistically significant correlation between microdystrophin and functional improvement, we plan to pursueinitiate a Biologics License Application (BLA) submission in the third quarter of 2026 under the accelerated approval forpathway RGX-202to and are preparing for asupport potential commercialFDA launchapproval in the second half of 2027.

Following the CRL, we entered into discussions with FDA senior leadership in March 2026 and filed a Formal Dispute Resolution Request which is pending. We plan to continue to work with the FDA to address the CRL and discuss potential paths forward for the RGX-121 program. Potential approval of the BLA for RGX-121 could result in receipt of a Rare Pediatric Disease Priority Review Voucher (PRV), assuming the statutory criteria are met. If approved, RGX-121 would be the first approved gene therapy and one-time treatment for MPS II.

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 MarchJune 31,30, 2026, our NAV Technology Platform was being applied in two commercial products, Zolgensma® and Itvisma®, and the preclinical and clinical development of various other licensed products. 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.

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma and Itvisma, which are marketed by Novartis Innovative Technologies Inc. (formerly, Novartis Gene Therapies, Inc. (Novartis Gene Therapies), a wholly owned subsidiary of Novartis AG (Novartis), for the treatment of spinal muscular atrophy (SMA). Zolgensma and Itvisma are licensed products under our license agreement with Novartis Gene Therapies (the Novartis License) for the development and commercialization of treatments for SMA using the NAV Technology Platform.

In mid-JanuaryJanuary 2026, licensed patents for Zolgensma under the Novartis License expired in the United States. We are entitled to continued royalties on net sales of Zolgensma in approximately 20 countries where licensed patents remain active. Licensed product made prior to patent expiration but sold after expiration may also be subject to royalties. PatentsLicensed patents covering the use of Itvisma have issued in the United States and certain other countriescountries, and are licensed to Novartis Gene Therapies under the Novartis License. Wewe are entitled to ongoing royalties on certain net sales of Itvisma in these territories.

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 MarchJune 31,30, 2026 and 2025 (in thousands):

Direct expenses related to the development of ABBV-RGX-314sura-vec include $12.6$12.1 million and $14.7$24.7 million for the three and six months ended MarchJune 31,30, 20262026, respectively, and $17.1 million and $31.7 million for the three and six months ended June 30, 2025, 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,sura-vec, the underlying costs of which are reported as unallocated expenses in the table above. We typically utilize our employee and infrastructure resources across our development programs. As a result, we generally do not allocate personnel and other internal costs, such as facilities and other overhead costs, to specific product candidates or development programs.

Our general and administrative expenses consist primarily of salaries, wages and personnel-related costs, including benefits, travel and stock-based compensation, for employees performing functions other than research and development. 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 our commercial partners prepare for and carry out product launch efforts, in particular for the potential commercialization of our RGX-202 and ABBV-RGX-314sura-vec product candidates.

Comparison of the Three Months Ended MarchJune 31,30, 2026 and 2025

ServiceLicense and Royalty Revenue. ServiceLicense and royalty revenue decreasedincreased by $0.7$85.4 million, from $2.0$18.5 million for the three months ended MarchJune 31,30, 2025 to $1.3$103.8 million for the three months ended MarchJune 31,30, 2026. The decreaseincrease was primarily attributable to service$100.0 million of license revenue recognized in the second quarter of 2026 upon the achievement of a development milestone under our sura-vec collaboration with Nippon Shinyaku, which decreased from $1.8 millionAbbVie for the first quarterpatient ofdosed 2025in tothe $1.0NAAVIGATE milliontrial. The increase in license and royalty revenue was partially offset by a decrease in royalty revenues for the firstsecond quarter of 2026, largely driven by the performance of RGX-121 development and manufacturing services.2026.

Research and Development Expense. Research and development expenses increaseddecreased by $4.3$3.4 million, from $53.1$59.5 million for the three months ended MarchJune 31,30, 2025 to $57.3$56.1 million for the three months ended MarchJune 31,30, 2026. The increasedecrease was primarily attributable to the following:

ana increasedecrease of $0.9$3.0 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates; and ana increasedecrease of $0.9$1.5 million in costs associated with preclinicalclinical activitiestrials and otherregulatory early-stageactivities, researchlargely driven by a decrease in expenses for sura-vec and development.RGX-121 pivotal trials, and partially offset by an increase in pivotal trial expenses for RGX-202.

General and Administrative Expense. General and administrative expenses increased by $1.0$1.7 million, from $20.3$19.9 million for the three months ended MarchJune 31,30, 2025 to $21.3$21.6 million for the three months ended MarchJune 31,30, 2026. The increase was largely driven by personnel-related costs, commercialization expenses, consulting and other corporate advisory services.

As of MarchJune 31,30, 2026, we had cash, cash equivalents and marketable securities of $150.5$105.5 million, which were primarily derived from our royalty monetization with HCR in May 2025 and thesales up-frontof paymentcommon receivedstock under theour Nipponat-the-market Shinyakuoffering Collaboration Agreement in March 2025.program.

In December 2024, we entered into a Sales Agreement with Leerink Partners LLC (Leerink) pursuant to which we may offer and sell shares of our common stock having an aggregate offering price of up to $150.0 million from time to time through Leerink, acting as our sales agent (the Leerink ATM Program). AsDuring ofthe Marchthree 31,and six months ended June 30, 2026, nowe sold 2,318,735 shares of common stock hadunder beenthe ATM Program for aggregate net proceeds of $18.9 million, after deducting commissions and offering expenses. No shares of common stock were sold under the Leerink ATM Program.Program We intendprior to use proceeds obtained from the salesecond quarter of shares under the Leerink ATM Program, if any, for general corporate purposes.2026.

We expect that our cash, cash equivalents and marketable securities of $150.5$105.5 million as of MarchJune 31,30, 2026, along with the $100.0 million milestone payment received from AbbVie in July 2026 willand enablethe us$107.8 tomillion fundin ournet operatingproceeds expensesreceived from the public offering of common stock and capitalpre-funded expenditurewarrants requirements,in andJuly 2026, are sufficient to fund operations into the fourth quarter of 2027. As such, we believe we have the ability to meet our financial commitments and obligations intoas earlythey 2027.become Thisdue estimatefor isat based on our current operating plan, and excludesleast the potentialnext effects12 months from the date of anythis future financings or material milestone payments that may be received under our licensing and collaboration arrangements.report. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our capital resources sooner than expected. These conditions raise substantial doubt about our ability to continue as a going concern within 12 months from the issuance date of our consolidated financial statements for the three months ended March 31, 2026, which accompany this Quarterly Report on Form 10-Q. Our ability to continue as a going concern will depend heavily on the successful development, approval and commercialization of our product candidates and our ability to raise additional capital to fund operations. If we are unable to raise capital sufficient to meet our working capital needs in the future, we may be forced to delay expenditures, reduce the scope of our development activities or make other changes to our operating plans.

Our net cash used in operating activities for the threesix months ended MarchJune 31,30, 2026 increased by $109.8$122.7 million from the threesix months ended MarchJune 31,30, 2025, largely driven by the $110.0 million up-front fee we received from Nippon Shinyaku in March 2025 and an increase in operating expenses in the first quarterhalf of 2026. We expect to continue to incur regular net cash outflows from operations for the foreseeable futurefuture, which from time to time may be offset by non-recurring payments received under our license and collaboration arrangements, as we continue the development and advancement of our product candidates and other research programs.

For the threesix months ended MarchJune 31,30, 2026, our net cash used in operating activities of $76.2$138.4 million consisted of a net loss of $90.1$67.3 million and unfavorable changes in operating assets and liabilities of $1.5$104.8 million, offset by adjustments for non-cash items of $15.4$33.7 million. The changes in operating assets and liabilities include an increase in accounts receivable of $78.5 million, which was driven primarily by the $100.0 million development milestone due from AbbVie as of June 30, 2026 and received in July 2026, and was partially offset by a $19.7 million decrease in royalties due from Novartis. The unfavorable changes in operating assets and liabilities also include a decrease in accrued expenses and other current liabilities of $16.7$15.6 million, which was driven largely by decreases in accrued personnel-related expenses, royalties and sublicense fees, and external research and development services. The unfavorable changes in operating assets and liabilities were partially offset by a decrease in accounts receivable of $18.2 million, which was driven largely by royalties receivable on net sales of Zolgensma. 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 $8.6$17.7 million, depreciation and amortization expense of $3.9$7.6 million and non-cash interest expense of $3.8$9.7 million.

For the threesix months ended MarchJune 31,30, 2025, our net cash providedused byin operating activities of $33.6$15.7 million consisted of a net incomeloss of $6.1$64.8 million, adjustmentsoffset for non-cash items of $11.1 million andby favorable changes in operating assets and liabilities of $16.4$24.0 million.million Adjustmentsand adjustments for non-cash items primarily consisted of stock-based$25.1 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.million. The changes in operating assets and liabilities include an increase of $39.8 million in deferred revenue,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 a decrease in total accounts payable and accrued expenses and other current liabilities of $18.1 million, which was driven largely by decreases in accrued personnel-related expenses, royalties and external research and development services, as well as a totalan increase in prepaid expenses and other current assets of $4.3$7.3 million, which was driven primarily by increasesan increase in prepaid software licenses and net cost reimbursement due from AbbVie under our ABBV-RGX-314sura-vec collaboration.collaboration and increases in prepaid clinical trial services and software licenses. 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 $17.2 million and depreciation and amortization expense of $7.9 million.

For the threesix months ended MarchJune 31,30, 2026, our net cash provided by investing activities consisted of $81.7$148.3 million in sales and maturities of marketable debt securities, offset by $9.8 million used to purchase marketable debt securities and $1.2$2.0 million used to purchase property and equipment.

For the threesix months ended MarchJune 31,30, 2025, our net cash providedused byin investing activities consisted of $64.9 million in maturities of marketable debt securities, offset by $19.2$230.3 million used to purchase marketable debt securities and $1.0$1.4 million used to purchase property and equipment.equipment, offset by $136.0 million in maturities of marketable debt securities.

For the threesix months ended MarchJune 31,30, 2026, our net cash usedprovided inby financing activities primarily consisted of $13.4$19.1 million in proceeds received from sales of common stock under the ATM Program, net of commissions and offering expenses paid during the period, and was partially offset by $15.2 million of royalties paid, net of interest, under our royalty monetization liabilities.

For the threesix months ended MarchJune 31,30, 2025, our net cash usedprovided inby financing activities primarily consisted of $5.6$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.

We have incurred cumulative losses since our inception and had an accumulated deficit of $1.22$1.19 billion as of MarchJune 31,30, 2026. 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. 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. Subject to obtaining regulatory approval for our product candidates, we expect to incur significant commercialization expenses for product sales, marketing, manufacturing and distribution. Additionally, we expect to continue to incur capital expenditures associated with building out additional laboratory and manufacturing capacity to further support the development of our product candidates and potential commercialization efforts. As a result, we will need significant additional capital to fund our operations, which we may obtain through one or more equity offerings, debt financings or other third-party funding, including potential strategic alliances and licensing or collaboration arrangements.