REGENXBIO Targets Q1 2027 for Duchenne Muscular Dystrophy Treatment Filing
Gene therapy developer REGENXBIO has updated its regulatory timeline for its lead muscular dystrophy candidate while awaiting key data for AMD trials.
Maryland-based biotechnology firm REGENXBIO announced it remains on schedule to submit a Biologics License Application (BLA) for its RGX-202 gene therapy candidate in the first quarter of 2027. The company is pursuing an accelerated approval pathway for the Duchenne muscular dystrophy treatment, with a potential regulatory decision projected for the second half of 2027.
In its ophthalmology division, the company expects topline results from the pivotal ATMOSPHERE and ASCENT studies by the end of 2026. These trials evaluate subretinal treatment for wet age-related macular degeneration (wet AMD). Concurrently, the firm continues its phase IIb/III NAAVIGATE clinical trial targeting diabetic retinopathy.
These clinical milestones represent significant operational hurdles for the company as it seeks to validate its gene delivery platform. The regulatory progress remains the primary focus for stakeholders monitoring the company’s transition toward potential commercial-stage asset development.