REGENXBIO Inc.

Previous wording

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-vec is currently being evaluated in multiple clinical trials, including two pivotal trials (ATMOSPHERE and ASCENT) where enrollment has been completed, 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 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.

Current wording

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). In wet AMD, sura-vec is currently being evaluated in multiple clinical 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-study. In DR, we are actively enrolling a pivotal, two-part Phase IIb/III study (NAAVIGATE) using suprachoroidal delivery. 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). 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.

Previous wording

Enrollment in the ATMOSPHERE® and ASCENT® pivotal trials for the treatment of patients with wet AMD using subretinal delivery was completed in October 2025. These trials are expected to support global regulatory submissions with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Topline data from these trials are expected to be shared in the fourth quarter of 2026 in partnership with AbbVie, with global regulatory submissions expected in 2027.

Current wording

Enrollment in the ATMOSPHERE and ASCENT pivotal trials for the treatment of patients with wet AMD using subretinal delivery was completed in October 2025. These trials, 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 announced in the fourth quarter of 2026 in partnership with AbbVie, with global regulatory submissions expected in 2027.

Previous wording

Concurrent with the two-year data announcement in 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 sura-vec in subjects with NPDR without center-involved diabetic macular edema (CI-DME). The primary endpoint is > 2-step improvement on the DRSS at one year. Following an interim analysis of part one (Phase IIb) portion of the NAAVIGATE trial, we and AbbVie will 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.

Current wording

In 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) of the NAAVIGATE trial, we and AbbVie expect 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.

Previous wording

The ALTITUDE® trial is a multi-center, open label, randomized, controlled, dose-escalation Phase II trial to evaluate the efficacy, safety and tolerability of sura-vec using suprachoroidal delivery for the treatment of DR. In November 2023, we announced data showing sura-vec 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 positive 2-year data showing sura-vec was well tolerated in subjects with non-proliferative diabetic retinopathy (NPDR) at dose levels 1, 2, and 3. There were no drug-related serious adverse events and no intraocular inflammation was observed through two years at dose level 3 (1.0x1012 GC/eye) (n = 15) with short-course topical prophylactic steroids. 50% of dose level 3 patients achieved at least a two-step improvement without need for any supplemental treatment.

Current wording

Concurrent with the August 2025 announcement, we announced positive two-year data from the Phase II ALTITUDE® trial. The ALTITUDE trial is a multi-center, open label, randomized, controlled, dose-escalation Phase II trial to evaluate the efficacy, safety and tolerability of sura-vec using suprachoroidal delivery for the treatment of DR. The positive two-year data showed sura-vec was well tolerated in subjects with NPDR at dose levels 1, 2, and 3. There were no drug-related serious adverse events and no intraocular inflammation was observed through two years at dose level 3 (1.0x1012 GC/eye) (n = 15) with short-course topical prophylactic steroids. Additionally, 50% of dose level 3 patients achieved at least a two-step improvement without need for any supplemental treatment.

Previous wording

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

Current wording

In October 2025, we announced that enrollment had completed in the pivotal portion of AFFINITY DUCHENNE (n=approximately 30).

Previous wording

In recent discussions with the FDA, the agency 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.

Current wording

As of May 2026, in recent discussions, the FDA 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.

Previous wording

Given the positive topline pivotal data, continued favorable safety profile, and statistically significant correlation between microdystrophin and functional improvement, we plan to pursue accelerated approval for RGX-202 and are preparing for a potential commercial launch in 2027.

Current wording

Given the positive topline pivotal data, continued favorable safety profile, and statistically significant correlation between microdystrophin and functional improvement, we plan to initiate a Biologics License Application (BLA) submission in the third quarter of 2026 under the accelerated approval pathway to support potential FDA approval in the second half of 2027.

Previous wording

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.

Current wording

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.

Previous wording

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

Current wording

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.), 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 (the Novartis License) for the development and commercialization of treatments for SMA using the NAV Technology Platform.

Previous wording

In mid-January 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. Patents covering the use of Itvisma have issued in the United States and certain other countries and are licensed to Novartis Gene Therapies under the Novartis License. We are entitled to ongoing royalties on certain net sales of Itvisma in these territories.

Current wording

In January 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. Licensed patents covering the use of Itvisma have issued in the United States and certain other countries, and we are entitled to ongoing royalties on certain net sales of Itvisma in these territories.

Previous wording

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, 2026 and 2025 (in thousands):

Current wording

We expect to continue to incur significant research and development expenses for the foreseeable future as we continue the development of our product candidates and engage in early research and development for prospective product candidates and new technologies. The following table summarizes our research and development expenses incurred during the three and six months ended June 30, 2026 and 2025 (in thousands):

Previous wording

Direct expenses related to the development of ABBV-RGX-314 include $12.6 million and $14.7 million for the three months ended March 31, 2026 and 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, 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.

Current wording

Direct expenses related to the development of sura-vec include $12.1 million and $24.7 million for the three and six months ended June 30, 2026, 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 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 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.

Previous wording

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.

Current wording

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 sura-vec product candidates.

Previous wording

License and Royalty Revenue. License and royalty revenue decreased by $82.0 million, from $87.0 million for the three months ended March 31, 2025 to $5.1 million for the three months ended March 31, 2026. The decrease was primarily attributable to $70.0 million of upfront license revenue recognized under our collaboration with Nippon Shinyaku in the first quarter of 2025, as well as a decrease in royalty revenues for the first quarter of 2026. Combined Zolgensma and Itvisma royalties decreased by $11.9 million, from $17.0 million for the first quarter of 2025 to $5.1 million for the first quarter of 2026. Novartis reported combined Zolgensma and Itvisma sales of $302 million for the first quarter of 2026, as compared to $327 million for the first quarter of 2025. Zolgensma royalties for the first quarter of 2026 were $4.7 million, a decrease of $12.2 million from the first quarter of 2025. The decrease was primarily attributable to the expiration of licensed patents in the United States in mid-January 2026. We are entitled to continued royalties on net sales of Zolgensma in approximately 20 countries where licensed patents remain active. Itvisma royalties for the first quarter of 2026 were $0.3 million. Itvisma was approved in the United States in the fourth quarter of 2025, with U.S. sales commencing in the first quarter of 2026. Licensed patents covering the use of Itvisma have issued in the United States and certain other countries, and we are entitled to ongoing royalties on certain net sales of Itvisma in these territories.

Current wording

Combined Zolgensma and Itvisma royalties decreased by $14.6 million, from $18.4 million for the second quarter of 2025 to $3.8 million for the second quarter of 2026. Novartis reported combined Zolgensma and Itvisma sales of $365 million for the second quarter of 2026, as compared to $297 million for the second quarter of 2025. Per Novartis, sales growth was driven by continued launch momentum from Itvisma, and Zolgensma sales remained stable. Zolgensma royalties for the second quarter of 2026 were $1.8 million, a decrease of $16.7 million from the second quarter of 2025, which was primarily attributable to the expiration of licensed patents in the United States in January 2026. We are entitled to continued royalties on net sales of Zolgensma in approximately 20 countries where licensed patents remain active. Itvisma royalties for the second quarter of 2026 were $2.1 million. Itvisma was approved in the United States in the fourth quarter of 2025, with U.S. sales commencing in the first quarter of 2026. Itvisma is now also approved in the UAE, Japan, Qatar and the EU. Licensed patents covering the use of Itvisma have issued in the United States and certain other countries, and we are entitled to ongoing royalties on certain net sales of Itvisma in these territories.

Previous wording

Research and Development Expense. Research and development expenses increased by $4.3 million, from $53.1 million for the three months ended March 31, 2025 to $57.3 million for the three months ended March 31, 2026. The increase was primarily attributable to the following:

Current wording

Research and Development Expense. Research and development expenses decreased by $3.4 million, from $59.5 million for the three months ended June 30, 2025 to $56.1 million for the three months ended June 30, 2026. The decrease was primarily attributable to the following:

Previous wording

an increase of $0.9 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates; and

Current wording

a decrease of $3.0 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates; and

Previous wording

an increase of $1.3 million in costs associated with clinical trials and regulatory activities, largely driven by RGX-202 pivotal trials;

Current wording

a decrease of $1.5 million in costs associated with clinical trials and regulatory activities, largely driven by a decrease in expenses for sura-vec and RGX-121 pivotal trials, and partially offset by an increase in pivotal trial expenses for RGX-202.

Previous wording

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

Current wording

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

Previous wording

Cost of License and Royalty Revenues. Cost of license and royalty revenues increased by $7.6 million, from $3.4 million for the three months ended March 31, 2025 to $11.1 million for the three months ended March 31, 2026. The increase was largely driven by a non-recurring charge of $10.0 million in the first quarter of 2026 related to a settlement with GlaxoSmithKline LLC (GSK) to resolve a dispute over sublicense fee obligations under our license agreement with GSK. For further information regarding the settlement agreement with GSK, please refer to Note 8, “Commitments and Contingencies—GlaxoSmithKline—GSK Settlement Agreement” to the accompanying unaudited consolidated financial statements.

Current wording

Cost of License and Royalty Revenues. Cost of license and royalty revenues increased by $3.5 million, from $8.6 million for the six months ended June 30, 2025 to $12.1 million for the six months ended June 30, 2026. The increase was largely driven by a non-recurring charge of $10.0 million in the first quarter of 2026 related to a settlement with GlaxoSmithKline LLC (GSK) to resolve a dispute over sublicense fee obligations under our license agreement with GSK. For further information regarding the settlement agreement with GSK, please refer to Note 8, “Commitments and Contingencies—GlaxoSmithKline—GSK Settlement Agreement” to the accompanying unaudited consolidated financial statements. The increase in cost of license and royalty revenues was partially offset by a decrease in upstream royalties on net sales of Zolgensma, consistent with the decrease in Zolgensma royalty revenues.

Previous wording

an increase of $1.1 million in personnel-related costs due to increased headcount of development personnel, net of a $0.2 million decrease in stock-based compensation expense;

Current wording

an increase of $1.8 million in personnel-related costs, net of a $0.3 million decrease in stock-based compensation expense; and

Previous wording

an increase of $0.9 million in costs associated with preclinical activities and other early-stage research and development.

Current wording

an increase of $1.8 million in costs associated with preclinical activities and other early-stage research and development.

Previous wording

As of March 31, 2026, we had cash, cash equivalents and marketable securities of $150.5 million, which were primarily derived from our royalty monetization with HCR in May 2025 and the up-front payment received under the Nippon Shinyaku Collaboration Agreement in March 2025.

Current wording

As of June 30, 2026, we had cash, cash equivalents and marketable securities of $105.5 million, which were primarily derived from our royalty monetization with HCR in May 2025 and sales of common stock under our at-the-market offering program.

Previous wording

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, 2026, no shares of common stock had been sold under the

Current wording

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 ATM Program). During the three and six months ended June 30, 2026, we sold 2,318,735 shares of common stock under the 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 ATM Program prior to the second quarter of 2026.

Previous wording

We expect that our cash, cash equivalents and marketable securities of $150.5 million as of March 31, 2026 will enable us to fund our operating expenses and capital expenditure requirements, and are sufficient to meet our financial commitments and obligations into early 2027. This estimate is based on our current operating plan, and excludes the potential effects of any future financings or material milestone payments that may be received under our licensing and collaboration arrangements. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our capital resources sooner than expected. These conditions raise substantial doubt about our ability to continue as a going concern within 12 months from the issuance date of our consolidated financial statements for the 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.

Current wording

We expect that our cash, cash equivalents and marketable securities of $105.5 million as of June 30, 2026, along with the $100.0 million milestone payment received from AbbVie in July 2026 and the $107.8 million in net proceeds received from the public offering of common stock and pre-funded warrants in July 2026, are sufficient to fund operations into the fourth quarter of 2027. As such, we believe we have the ability to meet our obligations as they become due for at least the next 12 months from the date of this report. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our capital resources sooner than expected. 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.

Previous wording

Our net cash used in operating activities for the three months ended March 31, 2026 increased by $109.8 million from the three months ended March 31, 2025, largely driven by the $110.0 million up-front fee received from Nippon Shinyaku in March 2025 and an increase in operating expenses in the first quarter of 2026. 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.

Current wording

Our net cash used in operating activities for the six months ended June 30, 2026 increased by $122.7 million from the six months ended June 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 half of 2026. We expect to continue to incur regular net cash outflows from operations for the foreseeable future, 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.

Previous wording

For the three months ended March 31, 2026, our net cash used in operating activities of $76.2 million consisted of a net loss of $90.1 million and unfavorable changes in operating assets and liabilities of $1.5 million, offset by adjustments for non-cash items of $15.4 million. The changes in operating assets and liabilities include a decrease in accrued expenses and other current liabilities of $16.7 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 million, depreciation and amortization expense of $3.9 million and non-cash interest expense of $3.8 million.

Current wording

For the six months ended June 30, 2026, our net cash used in operating activities of $138.4 million consisted of a net loss of $67.3 million and unfavorable changes in operating assets and liabilities of $104.8 million, offset by adjustments for non-cash items of $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 $15.6 million, which was driven largely by decreases in accrued personnel-related expenses, royalties and sublicense fees, and external research and development services. 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 $17.7 million, depreciation and amortization expense of $7.6 million and non-cash interest expense of $9.7 million.

Previous wording

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

Current wording

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 sura-vec 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.

Previous wording

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

Current wording

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

Previous wording

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.

Current wording

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.

Previous wording

For the three months ended March 31, 2026, our net cash used in financing activities primarily consisted of $13.4 million of royalties paid, net of interest, under our royalty monetization liabilities.

Current wording

For the six months ended June 30, 2026, our net cash provided by financing activities primarily consisted of $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.

Previous wording

We have incurred cumulative losses since our inception and had an accumulated deficit of $1.22 billion as of March 31, 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.

Current wording

We have incurred cumulative losses since our inception and had an accumulated deficit of $1.19 billion as of June 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.

Previous wording

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

Current wording

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