REGENXBIO Inc.

REGENXBIO Presents Positive Three-Year Durability Data for Surabgene Lomparvovec in Diabetic Retinopathy at Retina Society Annual Scientific Meeting

REGENXBIO presented three-year follow-up data from the Phase II ALTITUDE trial showing durable efficacy of surabgene lomparvovec (sura-vec) in non-proliferative diabetic retinopathy. Sixty percent of Dose Level 3 participants with three-year visits (6 of 10) achieved greater than 2-step DRSS improvement after a single in-office treatment, with no vision-threatening events and no additional DR treatment. No new safety signals and no intraocular inflammation were observed through three years with short-course prophylactic topical steroids, and Dose Level 3 is now being evaluated in the Phase IIb/III NAAVIGATE trial.

•1mo ago

REGENXBIO Strengthens Board of Directors with Appointment of Greg Ciongoli

REGENXBIO appointed Greg Ciongoli to its Board of Directors effective August 25, 2026, adding expertise in biotechnology investing and capital allocation to support its commercial transition. Concurrent with the appointment, independent directors Jerry Karabelas and Jean Bennett are retiring from the board after serving since 2015 and 2021, respectively. Mr. Ciongoli is the Founder of Adiumentum Capital Management and a former Partner at The Baupost Group, currently serving as Board Chairman of Atara Biotherapeutics and as a board member at Zymeworks.

•2mo ago

REGENXBIO Reports Second Quarter 2026 Financial Results and Operational Highlights

REGENXBIO reported Q2 revenue of $108.0 million, up from $21.4 million a year ago, driven by a $100 million milestone payment from AbbVie and swinging the company to a net income of $22.7 million. Operationally, the Phase III AFFINITY DUCHENNE trial met its primary endpoint with high statistical significance, supporting a BLA submission in Q3 2026, while the FDA reaffirmed the path for RGX-121 with BLA resubmission also planned for Q3. The company strengthened its balance sheet with over $200 million in new capital during July, extending its cash runway into Q4 2027 with pro forma cash exceeding $310 million.

•2mo ago

REGENXBIO Presents Positive Long-Term Data for Surabgene Lomparvovec in Wet AMD and Diabetic Retinopathy at American Society of Retina Specialists Annual Meeting

REGENXBIO presented positive long-term follow-up data for surabgene lomparvovec in wet AMD and diabetic retinopathy at the ASRS annual meeting, demonstrating durable efficacy and safety across multiple delivery routes. In wet AMD, patients maintained stable to improved vision through five years with no drug-related intraocular inflammation observed at doses matching the pivotal trials. In diabetic retinopathy, 55% of participants achieved a greater than 2-step improvement on the DRSS scale and 70% experienced no vision-threatening events at 2.5 years. The company remains on track to announce topline data from the pivotal ATMOSPHERE and ASCENT trials in wet AMD during the fourth quarter of 2026.

•3mo ago

REGENXBIO Announces First Patient Dosed in Phase IIb/III NAAVIGATE Trial of Surabgene Lomparvovec in Diabetic Retinopathy; Company to Receive $100 Million Milestone

REGENXBIO announced the first patient dosed in the Phase IIb/III NAAVIGATE trial of surabgene lomparvovec for diabetic retinopathy, triggering a $100 million milestone payment from partner AbbVie. The pivotal study is evaluating the one-time gene therapy in patients with non-proliferative diabetic retinopathy without center-involved diabetic macular edema, targeting a primary endpoint of improvement on the diabetic retinopathy severity scale at one year. Management also plans to present long-term follow-up data from the ALTITUDE trial at the ASRS Annual Meeting in July 2026 and expects topline data from the ATMOSPHERE and ASCENT pivotal trials in wet AMD in Q4 2026.

•4mo ago

REGENXBIO Completes Dosing in Confirmatory Study of RGX-202, Marking Completion of Registrational Development Program and Supporting Planned BLA Submission in Q3 2026

REGENXBIO completed dosing in the confirmatory study of RGX-202 for Duchenne muscular dystrophy, marking the end of the registrational development program ahead of schedule. The company plans to submit a Biologics License Application (BLA) in Q3 2026 under the accelerated approval pathway, targeting potential FDA approval in the second half of 2027. Pivotal data showed RGX-202 met its primary endpoint with over 93% of patients achieving 10% microdystrophin expression at Week 12.

•4mo ago

REGENXBIO Announces Alignment with FDA on Path Forward for NAVSUNLI™ BLA Resubmission for Accelerated Approval; First Potential Gene Therapy for MPS II

REGENXBIO secured a critical regulatory win, aligning with the FDA on an accelerated approval path for NAVSUNLI without requiring additional clinical studies or a control arm. The company plans to request a Type A meeting in July and resubmit the BLA in Q3 2026, with the FDA committing to an expedited review of the existing data. This resolves a key hurdle from a February Complete Response Letter and clears the way for what could be the first gene therapy for MPS II (Hunter syndrome).

•4mo ago

REGENXBIO Announces Positive Topline Results from Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202

REGENXBIO announced positive topline results from the pivotal Phase III AFFINITY DUCHENNE study of RGX-202, a gene therapy for Duchenne Muscular Dystrophy. The trial met its primary endpoint with 93% of participants achieving microdystrophin expression above 10% at Week 12 (p<0.0001). Additionally, 80% of participants achieved microdystrophin expression above 40%. RGX-202 demonstrated a statistically significant correlation between microdystrophin expression and functional improvement, as measured by the North Star Ambulatory Assessment (NSAA) and timed function tests. The company plans to pursue accelerated approval for RGX-202 and is preparing for a potential commercial launch in 2027, based on the positive topline data and favorable safety profile.

•5mo ago