REGENXBIO Reports Durable 18-Month Efficacy Data for RGX-202 AAV Gene Therapy and Outlines Multiple 2026 Regulatory Milestones

ROCKVILLE, Md. —January 11, 2026 — REGENXBIO Inc. (Nasdaq: RGNX) announced new long-term clinical data and outlined key 2026 milestones across its pipeline of AAV (adeno-associated virus) gene therapies, highlighting durable efficacy for RGX-202, an AAV gene therapy for Duchenne muscular dystrophy (DMD), alongside advancing regulatory and commercial preparations for multiple late-stage AAV programs.

New 18-month functional data from the Phase I/II portion of the AFFINITY DUCHENNE® trial showed that patients treated with the pivotal dose of RGX-202 AAV gene therapy exceeded expected disease progression on the North Star Ambulatory Assessment, demonstrating sustained and durable benefit. REGENXBIO plans to report additional safety, biomarker, and functional data for this AAV-based gene therapy at the MDA Clinical and Scientific Conference in March 2026.

The company expects to share pivotal top-line data for RGX-202 AAV gene therapy in early Q2 2026 and to submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026. Enrollment in the confirmatory trial is ongoing, with most patients expected to be dosed by the time of BLA submission, supporting potential commercial launch readiness.

Beyond Duchenne, REGENXBIO highlighted near-term regulatory milestones for clemidsogene lanparvovec (RGX-121), an AAV gene therapy for MPS II (Hunter syndrome), with an FDA PDUFA target date of February 8, 2026. If approved, this AAV gene therapy would generate a Priority Review Voucher, while partner Nippon Shinyaku prepares for commercialization and REGENXBIO leads in-house AAV manufacturing.

In retinal disease, surabgene lomparvovec (ABBV-RGX-314), an AAV gene therapy for wet age-related macular degeneration and diabetic retinopathy, continues to advance in collaboration with AbbVie. Top-line pivotal data are expected in Q4 2026, and a Phase IIb/III trial using suprachoroidal delivery for diabetic retinopathy is planned, potentially making it the first AAV gene therapy for a non-rare disease.

REGENXBIO emphasized its fully integrated, end-to-end AAV gene therapy capabilities, spanning capsid discovery, AAV vector engineering, and commercial-ready manufacturing. The company also reported progress toward IND readiness for an additional AAV gene therapy for geographic atrophy, leveraging a next-generation capsid designed to enhance transgene expression via suprachoroidal delivery.

Collectively, the updates position REGENXBIO for multiple FDA decisions, pivotal data readouts, and potential AAV gene therapy launches between 2026 and 2028.