REGENXBIO Inc.
For the period ended Sep 30, 2026. Filed Nov 5, 2026.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2026 · Filed Nov 5, 2026
RGNX$7.62+3.3%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2026-06-30; filed 2026-08-06.
0001193125-26-336725
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-09-30; filed 2026-11-05.
0001193125-26-448581
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 157 previous paragraphs and 161 current paragraphs.
104 unchanged; 46 reworded; 10 added; 5 removed; 2 previous and 1 current paragraphs rearranged.
Tables are not compared: 3 previous and 3 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (10)
Current report
The clinical development program evaluating subretinal sura-vec in wet AMD encompasses multiple clinical studies and sub-studies which together are designed to establish a broad and robust body of clinical evidence. The program has enrolled a diverse spectrum of patients, including those with high disease activity and bilateral disease, with the intent of supporting the potential applicability of sura-vec across a broad patient population. The program also includes ODYSSEY, a retrospective comparative analysis evaluating outcomes in sura-vec-treated patients against matched real-world controls. Additionally, AbbVie is enrolling ACHIEVE, a global, randomized, controlled Phase 3b study to assess the injection burden, efficacy, safety and long-term preservation of vision in a real-world context.
Current report
In October 2026, we held an investor event during which we reviewed the clinical development to date, including data from select Phase I/II studies, and discussed the unmet need and potential role of sura-vec in wet AMD with retina experts. A copy of that presentation is available on our website.
Current report
License and Royalty Revenue. License and royalty revenue decreased by $18.4 million, from $23.6 million for the three months ended September 30, 2025 to $5.2 million for the three months ended September 30, 2026. The decrease was primarily attributable to Zolgensma royalty revenues for the third quarter of 2026.
Current report
Research and Development Expense. Research and development expenses increased by $4.9 million, from $56.1 million for the three months ended September 30, 2025 to $61.0 million for the three months ended September 30, 2026. The increase was primarily attributable to a $7.1 million increase in costs associated with clinical trials and regulatory activities, largely driven by expenses for RGX-202 clinical trials. The increase in research and development expenses was partially offset by a decrease of $1.0 million in personnel-related costs, largely driven by a $0.9 million decrease in stock-based compensation expense.
Current report
Gain on Litigation Settlement. In September 2026, we entered into a settlement agreement with Sarepta Therapeutics, Inc. (Sarepta) to resolve all patent litigation matters between the parties. Pursuant to the settlement agreement, we received a $39.0 million settlement payment from Sarepta, which was recorded as other current assets as of September 30, 2026 and received in full in October 2026. We recorded a gain of $38.0 million in the third quarter of 2026 related to the settlement, which consisted of the $39.0 million settlement payment from Sarepta, net of proceeds payable by us to certain licensors as a result of the settlement. For further information regarding the settlement agreement with Sarepta, please refer to Note 8, “Commitments and Contingencies—Sarepta Therapeutics Settlement Agreement” to the accompanying unaudited consolidated financial statements.
Current report
Interest Expense. Interest expense decreased by $5.7 million, from $13.2 million for the three months ended September 30, 2025 to $7.5 million for the three months ended September 30, 2026. The decrease was primarily attributable to lower interest costs under our royalty monetization liabilities driven by reduced principal outstanding under the 2020 Royalty Purchase Agreement.
Current report
Comparison of the Nine Months Ended September 30, 2026 and 2025
Current report
Gain on Litigation Settlement. In September 2026, we entered into a settlement agreement with Sarepta to resolve all patent litigation matters between the parties. Pursuant to the settlement agreement, we received a $39.0 million settlement payment from Sarepta, which was recorded as other current assets as of September 30, 2026 and received in full in October 2026. We recorded a gain of $38.0 million in the third quarter of 2026 related to the settlement, which consisted of the $39.0 million settlement payment from Sarepta, net of proceeds payable by us to certain licensors as a result of the settlement. For further information regarding the settlement agreement with Sarepta, please refer to Note 8, “Commitments and Contingencies—Sarepta Therapeutics Settlement Agreement” to the accompanying unaudited consolidated financial statements.
Current report
Interest Expense. Interest expense decreased by $8.8 million, from $32.7 million for the nine months ended September 30, 2025 to $24.0 million for the nine months ended September 30, 2026. The decrease was primarily attributable to lower interest cost under our royalty monetization liabilities driven by reduced principal outstanding under the 2020 Royalty Purchase Agreement. The decrease in interest expense was partially offset by an increase in interest expense under the 2025 Royalty Bond since its issuance in May 2025.
Current report
In September 2026, we entered into a settlement agreement with Sarepta to resolve all patent litigation matters between the parties. Pursuant to the settlement agreement, we received a $39.0 million settlement payment from Sarepta in October 2026.
Removed paragraphs (5)
Previous report
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 report
The decrease in research and development expenses was partially offset by an increase of $0.9 million in costs associated with preclinical activities and other early-stage research and development.
Previous report
Comparison of the Six Months Ended June 30, 2026 and 2025
Previous report
an increase of $1.8 million in costs associated with preclinical activities and other early-stage research and development.
Previous report
General and Administrative Expense. General and administrative expenses increased by $2.7 million, from $40.2 million for the six months ended June 30, 2025 to $42.9 million for the six months ended June 30, 2026. The increase was largely driven by personnel-related costs, commercialization expenses, consulting and other corporate advisory services.
Reworded paragraphs (46)
Previous wording
ATMOSPHERE® and ASCENT®are multi-center, randomized, active-controlled trials evaluating sura-vec versus ranibizumab and aflibercept, respectively. The primary endpoint is non-inferiority based on change from baseline in best-corrected visual acuity (BCVA) at 54 weeks and one year, respectively. Secondary endpoints include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections in the treatment arms.
Current wording
ATMOSPHERE® and ASCENT®are multi-center, randomized, active-controlled trials evaluating sura-vec versus monthly ranibizumab and aflibercept, respectively. The primary endpoint in both studies is non-inferiority based on change from baseline in best-corrected visual acuity (BCVA) at 54 weeks. Secondary endpoints include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections in the treatment arms.
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, 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.
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 1,300 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
The AAVIATE® trial is a multi-center, open label, randomized, controlled, dose-escalation Phase II trial to evaluate the efficacy, safety and tolerability of suprachoroidal delivery of sura-vec for the treatment of wet AMD. Based on the favorable safety profile observed as of July 29, 2024, the Phase II AAVIATE trial enrolled a cohort to evaluate sura-vec at dose level 4 (1.5x10e12 GC/eye). Patients in this cohort received short course prophylactic steroid eye drops. Enrollment of the AAVIATE trial has been completed.
Current wording
The AAVIATE® trial is a multi-center, open label, randomized, controlled, dose-escalation Phase II trial to evaluate the efficacy, safety and tolerability of suprachoroidal delivery of sura-vec for the treatment of wet AMD. Based on the favorable safety profile observed as of July 29, 2024, the Phase II AAVIATE trial enrolled a cohort to evaluate sura-vec at dose level 4 (1.5x1012 GC/eye). Patients in this cohort received short course prophylactic steroid eye drops. Enrollment of the AAVIATE trial has been completed.
Previous 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.
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 to evaluate sura-vec for the treatment of DR. 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 Phase III trial led by AbbVie.
Previous wording
In July 2026, we presented long-term follow-up data from the Phase II ALTITUDE trial that showed, among other findings, that participants at dose level 3 (1.0×10¹² GC/eye) with short-course prophylactic topical steroids, the same dose being evaluated in the Phase IIb/III NAAVIGATE trial, maintained a durable safety and efficacy profile through 2.5 years. 55% of participants achieved >2-step improvement on the DRSS without additional treatment, and 70% of participants experienced no vision-threatening events. These data are consistent with previously presented two-year dose level 3 NPDR data from the ALTITUDE trial and support the potential of one-time in-office sura-vec to modify the underlying disease and decrease risk of vision-threatening events.
Current wording
In 2026, we presented long-term follow-up data from the Phase II ALTITUDE trial that showed, among other findings, that participants at dose level 3 (1.0×10¹² GC/eye) with short-course prophylactic topical steroids, the same dose being evaluated in the Phase IIb/III NAAVIGATE trial, maintained a durable safety and efficacy profile through 2.5 years and 3 years. At 2.5 years, 55% of dose level 3 participants achieved >2-step improvement on the DRSS without additional treatment, and 70% of participants experienced no vision-threatening events. 60% of all dose level 3 participants with three-year visits (n=6/10) achieved >2-step improvement on the DRSS without additional treatment for diabetic retinopathy. These participants did not experience any vision-threatening events. Additionally, the majority of participants (n=3/4) who achieved a 1-step DRSS improvement at one year without supplemental anti-VEGF injections went on to achieve a >2-step DRSS improvement by three years without additional treatment for diabetic retinopathy. These data are consistent with previously presented two-year dose level 3 NPDR data from the ALTITUDE trial and support the potential of one-time in-office sura-vec to modify the underlying disease and decrease risk of vision-threatening events.
Previous wording
AFFINITY DUCHENNE® is an ongoing 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 ambulatory patients with Duchenne aged 1 to 11 years old. Phase I/II results, reported between November 2024 and March 2026, demonstrated RGX-202 to be well tolerated with no serious adverse events or adverse events of special interest among all patients (n=13). Additionally, we reported positive biomarker data showing consistent, high expression and transduction of RGX-202 microdystrophin, with all patients exceeding 10%. Pivotal dose (2x1014 GC/kg) participants in the Phase I/II exceeded expected disease trajectory on the North Star Ambulatory Assessment (NSAA) and other timed function tests at one year using multiple validated methods to estimate expected disease progression without treatment (n=7). Notably, five participants for whom functional data has been reported were aged eight or older at dosing, when functional decline is expected.
Current wording
AFFINITY DUCHENNE® is an ongoing 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 ambulatory patients with Duchenne aged 1 to 11 years old. Phase I/II results, reported between November 2024 and March 2026, demonstrated RGX-202 to be well tolerated with no serious adverse events or adverse events of special interest among all patients (n=13). Additionally, we reported positive biomarker data showing consistent, high expression and transduction of RGX-202 microdystrophin, with all patients exceeding 10%. Pivotal dose (2x1014 GC/kg) participants in the Phase I/II exceeded expected disease trajectory on the North Star Ambulatory Assessment (NSAA) and other timed function tests at one year using multiple validated methods to estimate expected disease progression without treatment (n=7). Notably, five participants for whom functional data have been reported were aged eight or older at dosing, when functional decline is typically expected.
Previous 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.
Current wording
Given the positive topline pivotal data, continued favorable safety profile, and statistically significant correlation between microdystrophin and functional improvement, we expect to hold a pre-Biologics License Application (BLA) meeting with the FDA in the fourth quarter of 2026 and submit a BLA in the first quarter of 2027 under the accelerated approval pathway, supporting potential FDA approval in the second half of 2027.
Previous wording
In June 2026, we announced we had completed dosing in the confirmatory study of RGX-202 and our plan to include in our planned BLA a safety dataset from the AFFINITY DUCHENNE pivotal and confirmatory studies (n=63) as well as efficacy data from the pivotal portion (n=30), including 12-month functional data for at least half of the total participants in the pivotal study.
Current wording
In June 2026, we announced we had completed dosing in the confirmatory study of RGX-202. We expect the BLA to include a safety dataset from the AFFINITY DUCHENNE pivotal and confirmatory studies (n=63) as well as efficacy data from the pivotal portion (n=30), including 12-month functional data for at least half of the total participants in the pivotal study.
Previous wording
A BLA for RGX-121 seeking accelerated approval was submitted to the FDA in March 2025. The FDA subsequently granted priority review of the BLA and successfully completed mid-cycle meeting, Pre-license inspection (PLI) and Bioresearch monitoring information (BIMO) inspections. The PLI and BIMO inspections were completed with no observations. In August 2025, we announced that the FDA review timeline had been extended following submission of 12-month clinical data for all patients in the pivotal study of RGX-121 (n=13) in response to an FDA information request.
Current wording
A BLA for RGX-121 seeking accelerated approval was submitted to the FDA in March 2025. The FDA subsequently granted priority review of the BLA and successfully completed mid-cycle meeting, Pre-license inspection (PLI) and Bioresearch monitoring information (BIMO) inspections. The PLI and BIMO inspections were completed with no observations. In August 2025, we announced that the Prescription Drug User Fee Act (PDUFA) goal date was extended from November 9, 2025 to February 8, 2026 following submission of 12-month clinical data for all patients in the pivotal study of RGX-121 (n=13) in response to an FDA information request.
Previous wording
The initial Prescription Drug User Fee Act (PDUFA) goal date was extended from November 9, 2025 to February 8, 2026. In January 2026, we announced that the FDA placed the RGX-121 program on partial clinical hold in relation to a serious adverse event in a patient treated in the Phase I/II trial of RGX-111, discussed in further detail below. The FDA cited the similarities in products, study populations, and shared risk between the clinical studies. The partial clinical hold on RGX-121 was lifted by the FDA on April 30, 2026.
Current wording
In January 2026, we announced that the FDA placed the RGX-121 program on partial clinical hold in relation to a serious adverse event in a patient treated in the Phase I/II trial of RGX-111, discussed in further detail below. The FDA cited the similarities in products, study populations, and shared risk between the clinical studies. The partial clinical hold on RGX-121 was lifted by the FDA on April 30, 2026.
Previous wording
In February 2026, we announced that the FDA issued a Complete Response Letter (CRL) for the RGX-121 BLA. The FDA stated in the CRL that it had agreed to the study protocol in principle and outlined several reasons for not approving the gene therapy, including uncertainty regarding the study eligibility criteria to adequately define a population with neuronopathic disease (vs. attenuated disease), the comparability of the natural history external control to the study population, and the appropriateness of CSF HS D2S6 as a surrogate endpoint reasonably likely to predict clinical benefit. The CRL lists several potential paths forward, including a new study, treating additional patients and conducting longer-term follow up, and using an untreated control arm. Throughout active discussions during the BLA process, we believed we had addressed the points raised in the CRL through the submission of additional data and responses to numerous information requests. The FDA did not agree the data set provided substantial evidence of effectiveness to support approval of RGX-121 for the treatment of MPS II.
Current wording
In February 2026, we announced that the FDA issued a Complete Response Letter (CRL) for the RGX-121 BLA. The FDA stated in the CRL that it had agreed to the study protocol in principle and outlined several reasons for not approving the gene therapy, including uncertainty regarding the study eligibility criteria to adequately define a population with neuronopathic disease (vs. attenuated disease), the comparability of the natural history external control to the study population, and the appropriateness of CSF HS D2S6 as a surrogate endpoint reasonably likely to predict clinical benefit.
Previous wording
Following the CRL, we entered into discussions with FDA senior leadership in March 2026 and filed a Formal Dispute Resolution Request.
Current wording
In March 2026, we entered into discussions with FDA senior leadership and filed a Formal Dispute Resolution Request.
Previous wording
In June 2026, we announced alignment with the FDA regarding the next steps needed for a potential accelerated approval of RGX-121. During those discussions, the FDA acknowledged that the existing RGX-121 clinical data is sufficient to be considered for the accelerated approval pathway and that the Company does not need to enroll additional patients or conduct additional studies, including the FDA’s previously recommended incorporation of an untreated control arm. The FDA asked the Company to request a Type A meeting to review existing longer-term biomarker and clinical data and to resubmit the BLA following this meeting. The FDA stated that it would review our resubmission on an expedited basis, with labeling discussions to begin shortly following the resubmission.
Current wording
In June 2026, we announced alignment with the FDA regarding the next steps needed for a potential accelerated approval of RGX-121. During those discussions, the FDA acknowledged that the existing RGX-121 clinical data was sufficient to be considered for the accelerated approval pathway and that the Company did not need to enroll additional patients or conduct additional studies, including the FDA’s previously recommended incorporation of an untreated control arm.
Previous wording
In July 2026, we and the FDA held a positive Type A meeting during which the FDA reaffirmed that no additional studies of RGX-121 are required for BLA resubmission. We and the FDA aligned on resubmission requirements and we plan to resubmit the RGX-121 BLA in the third quarter of 2026. The resubmission will include longer-term efficacy and safety data, including participant imaging that has been submitted to FDA and continues to be collected and analyzed as part of ongoing RGX-121 safety monitoring. A post-approval confirmatory study will be discussed as part of BLA review.
Current wording
In July 2026, we and the FDA held a positive Type A meeting during which the FDA reaffirmed that no additional studies of RGX-121 are required for BLA resubmission and aligned on resubmission requirements.
Previous 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.
Current wording
In August 2026, we announced that the FDA placed a clinical hold on RGX-121 following the discovery of asymptomatic spine MRI findings in five participants in the CAMPSIITE study. We continue to collect and evaluate additional patient imaging and long-term follow-up data. 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
In June 2026, we dosed the first patient in the NAAVIGATE trial, resulting in a $100.0 million development milestone payment from AbbVie which was fully recognized as license and royalty revenue in the second quarter of 2026. The $100.0 million milestone payment was recorded as accounts receivable as of June 30, 2026 and was received from AbbVie in July 2026.
Current wording
In June 2026, we dosed the first patient in the NAAVIGATE trial, resulting in a $100.0 million development milestone payment from AbbVie which was fully recognized as license and royalty revenue in the second quarter of 2026 and received from AbbVie in July 2026.
Previous wording
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 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 and collaborators.
Current wording
We have licensed 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 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 and collaborators.
Previous wording
Future revenues under our license and collaboration arrangements 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 under our license or collaboration agreements that is contemplated on optional goods and services, development and sales-based 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 collaborators and the arrangements are terminable at the option of the counterparty. The termination of our license and collaborations arrangements may materially impact the amount of revenue we recognize in future periods.
Current wording
Future revenues under our license and collaboration arrangements 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 under our license or collaboration agreements that is contemplated on optional goods and services, development and sales-based 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 collaborators and the arrangements are terminable at the option of the counterparty. The termination of our license and collaboration arrangements may materially impact the amount of revenue we recognize in future periods.
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 and six months ended June 30, 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 nine months ended September 30, 2026 and 2025 (in thousands):
Previous 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.
Current wording
Direct expenses related to the development of sura-vec include $9.3 million and $34.0 million for the three and nine months ended September 30, 2026, respectively, and $17.7 million and $49.5 million for the three and nine months ended September 30, 2025, respectively, in net cost reimbursement from AbbVie under our sura-vec 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
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.
Current wording
Combined Zolgensma and Itvisma royalties decreased by $18.3 million, from $23.6 million for the third quarter of 2025 to $5.2 million for the third quarter of 2026. Novartis reported combined Zolgensma and Itvisma sales of $404 million for the third quarter of 2026, as compared to $301 million for the third quarter of 2025. Per Novartis, sales growth was driven by continued launch momentum from Itvisma, while Zolgensma maintained market leadership in the incident SMA population. Zolgensma royalties for the third quarter of 2026 were $2.4 million, a decrease of $21.2 million from the third 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 third quarter of 2026 were $2.8 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 EU, Japan and multiple Gulf countries. 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
License and Royalty Revenue. License and royalty revenue increased by $3.4 million, from $105.5 million for the six months ended June 30, 2025 to $108.9 million for the six months ended June 30, 2026. The increase was primarily attributable to $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 AbbVie for the first patient dosed in the NAAVIGATE trial. The increase in license and royalty revenue was partially offset by $70.0 million of non-recurring, 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 half of 2026.
Current wording
License and Royalty Revenue. License and royalty revenue decreased by $14.9 million, from $129.1 million for the nine months ended September 30, 2025 to $114.2 million for the nine months ended September 30, 2026. The decrease was primarily attributable to $70.0 million of non-recurring, upfront license revenue recognized under our collaboration with Nippon Shinyaku in the first quarter of 2025 and a decrease in Zolgensma royalty revenues for the first nine months of 2026. The decrease in license and royalty revenue was partially offset by $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 AbbVie for the first patient dosed in the NAAVIGATE trial.
Previous wording
Combined Zolgensma and Itvisma royalties decreased by $26.5 million, from $35.4 million for the first half of 2025 to $8.9 million for the first half of 2026. Novartis reported combined Zolgensma and Itvisma sales of $667 million for the first half of 2026, as compared to $624 million for the first half of 2025. Per Novartis, sales growth was driven by continued launch momentum from Itvisma, and Zolgensma sales remained stable. Zolgensma royalties for the first half of 2026 were $6.5 million, a decrease of $28.9 million from the first half 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 first half of 2026 were $2.4 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.
Current wording
Combined Zolgensma and Itvisma royalties decreased by $44.9 million, from $59.0 million for the nine months ended September 30, 2025 to $14.1 million for the nine months ended September 30, 2026. Novartis reported combined Zolgensma and Itvisma sales of $1.07 billion for the nine months ended September 30, 2026, as compared to $925 million for the nine months ended September 30, 2025. Per Novartis, sales growth was driven by continued launch momentum from Itvisma, while Zolgensma maintained market leadership in the incident SMA population. Zolgensma royalties for the nine months ended September 30, 2026 were $8.9 million, a decrease of $50.1 million from the nine months ended September 30, 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 nine months ended September 30, 2026 were $5.2 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 EU, Japan and multiple Gulf countries. 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
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.
Current wording
Cost of License and Royalty Revenues. Cost of license and royalty revenues decreased by $1.1 million, from $14.4 million for the nine months ended September 30, 2025 to $13.3 million for the nine months ended September 30, 2026. The decrease was largely driven by lower upstream royalties on net sales of Zolgensma, consistent with the decrease in Zolgensma royalty revenues. The decrease in cost of license and royalty revenues was partially offset 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.
Previous wording
Research and Development Expense. Research and development expenses increased by $0.8 million, from $112.6 million for the six months ended June 30, 2025 to $113.4 million for the six months ended June 30, 2026. The increase was primarily attributable to the following:
Current wording
Research and Development Expense. Research and development expenses increased by $5.7 million, from $168.7 million for the nine months ended September 30, 2025 to $174.4 million for the nine months ended September 30, 2026. The increase was primarily attributable to the following:
Previous 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.
Current wording
an increase of $6.9 million in costs associated with clinical trials and regulatory activities, largely driven by expenses for RGX-202 clinical trials; and
Previous wording
an increase of $1.8 million in personnel-related costs, net of a $0.3 million decrease in stock-based compensation expense; and
Current wording
an increase of $0.8 million in personnel-related costs, net of a $1.2 million decrease in stock-based compensation expense.
Previous wording
The increase in research and development expenses was partially offset by a decrease of $2.1 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates.
Current wording
The increase in research and development expenses was partially offset by a decrease of $1.9 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates.
Previous 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.
Current wording
General and Administrative Expense. General and administrative expenses increased by $3.0 million, from $60.5 million for the nine months ended September 30, 2025 to $63.5 million for the nine months ended September 30, 2026. The increase was largely driven by personnel-related costs and commercialization expenses.
Previous 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.
Current wording
As of September 30, 2026, we had cash, cash equivalents and marketable securities of $251.0 million, which were primarily derived from our royalty monetization with HCR in May 2025, milestone payments under our license and collaboration agreements, public offerings of common stock and pre-funded warrants, and sales of common stock under our at-the-market offering program.
Previous wording
In June 2026, we dosed the first patient in the NAAVIGATE trial, resulting in a $100.0 million development milestone payment due to us under our sura-vec collaboration with AbbVie. The $100.0 million milestone payment was recorded as accounts receivable as of June 30, 2026 and was received from AbbVie in July 2026.
Current wording
In June 2026, we dosed the first patient in the NAAVIGATE trial, resulting in a $100.0 million development milestone payment under our sura-vec collaboration with AbbVie. The $100.0 million milestone payment was received from AbbVie in July 2026.
Previous wording
In July 2026, we completed a public offering of 11,671,139 shares of our common stock (inclusive of 1,667,250 shares pursuant to the full exercise by the underwriters of their option to purchase additional shares) at a price of $9.00 per share and 1,111,111 pre-funded warrants to purchase shares of our common stock at a price of $8.9999 per pre-funded warrant, which equaled the public offering price per share of the common stock less the $0.0001 exercise price of each pre-funded warrant. The aggregate net proceeds received from the offering were approximately $107.8 million, net of underwriting discounts and commissions and estimated offering expenses.
Current wording
In July 2026, we completed a public offering of 11,671,139 shares of our common stock at a price of $9.00 per share and 1,111,111 pre-funded warrants to purchase shares of our common stock at a price of $8.9999 per pre-funded warrant, which equaled the public offering price per share of the common stock less the $0.0001 exercise price of each pre-funded warrant. The aggregate net proceeds received from the offering were $107.8 million, net of underwriting discounts and commissions and offering expenses.
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 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.
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 second quarter of 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 $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.
Current wording
We expect that our cash, cash equivalents and marketable securities of $251.0 million as of September 30, 2026, along with the $39.0 million in settlement proceeds received from Sarepta in October 2026, are sufficient to fund our operating expenses and capital expenditure requirements, and to meet our financial commitments and obligations, into the late fourth quarter of 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 and nine months ended September 30, 2026 included in 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.
Previous 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.
Current wording
Our net cash used in operating activities for the nine months ended September 30, 2026 increased by $28.1 million from the nine months ended September 30, 2025. We received a $100.0 million milestone payment from AbbVie in July 2026 and a $110.0 million up-front payment from Nippon Shinyaku in March 2025, each of which partially offset our net cash used in operating activities for the respective periods. 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 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.
Current wording
For the nine months ended September 30, 2026, our net cash used in operating activities of $99.8 million consisted of a net loss of $108.1 million and unfavorable changes in operating assets and liabilities of $42.9 million, offset by adjustments for non-cash items of $51.1 million. The changes in operating assets and liabilities include an increase in other current assets of $35.4 million, which was primarily attributable to the $39.0 million settlement payment due from Sarepta as of September 30, 2026 and received in October 2026. The unfavorable changes in operating assets and liabilities also include a decrease in accrued expenses and other current liabilities of $9.1 million, which was driven largely by decreases in accrued personnel-related expenses, royalties and sublicense fees, and external research and development services, as well as a decrease in deferred revenue of $7.2 million driven by service revenue recognized under our collaboration with Nippon Shinyaku. The unfavorable changes in operating assets and liabilities were partially offset by a decrease in accounts receivable of $19.8 million, which was driven primarily by a decrease in royalties due from Novartis. 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 $25.8 million, depreciation and amortization expense of $11.1 million and non-cash interest expense of $15.9 million.
Previous 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.
Current wording
For the nine months ended September 30, 2025, our net cash used in operating activities of $71.7 million consisted of a net loss of $126.7 million, offset by adjustments for non-cash items of $41.5 million and favorable changes in operating assets and liabilities of $13.6 million. Adjustments for non-cash items primarily consisted of stock-based compensation expense of $26.2 million and depreciation and amortization expense of $11.7 million. The changes in operating assets and liabilities include an increase in deferred revenue of $34.3 million, which was primarily attributable to the deferred portion of the $110.0 million up-front payment received under our collaboration with Nippon Shinyaku in the first quarter of 2025. The favorable changes in operating assets and liabilities were partially offset by an increase in prepaid expenses and other current assets of $9.5 million, which was driven largely by an increase in net cost reimbursement due from AbbVie under our ABBV-RGX-314 collaboration, and an increase in accounts receivable of $7.1 million, which was driven largely by reimbursable costs due from Nippon Shinyaku under our collaboration for RGX-121 and RGX-111. Other changes in operating working capital occurred in the normal course of business.
Previous 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.
Current wording
For the nine months ended September 30, 2026, our net cash provided by investing activities consisted of $191.3 million in sales and maturities of marketable debt securities, offset by $138.7 million used to purchase marketable debt securities and $2.2 million used to purchase property and equipment.
Previous 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.
Current wording
For the nine months ended September 30, 2025, our net cash used in investing activities primarily consisted of $269.8 million used to purchase marketable debt securities and $1.9 million used to purchase property and equipment, partially offset by $218.3 million in maturities of marketable debt securities.
Previous 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.
Current wording
For the nine months ended September 30, 2026, our net cash provided by financing activities primarily consisted of $107.8 million in proceeds received from the public offering of common stock and pre-funded warrants completed in July 2026, net of underwriting discounts and commissions and other offering expenses paid during the period, and $18.9 million in proceeds received from sales of common stock under the ATM Program, net of commissions and offering expenses paid during the period. Net cash provided by financing activities was partially offset by $17.2 million of royalties paid, net of interest, under our royalty monetization liabilities.
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Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
Future Liquidity and Ability to Continue as a Going Concern
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
License and Royalty Revenue. License and royalty revenue increased by $85.4 million, from $18.5 million for the three months ended June 30, 2025 to $103.8 million for the three months ended June 30, 2026. The increase was primarily attributable to $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 AbbVie for the first patient dosed in the NAAVIGATE trial. The increase in license and royalty revenue was partially offset by a decrease in royalty revenues for the second quarter of 2026.
Previous report — wording carried elsewhere
a decrease of $3.0 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates; and