REGENXBIO Inc. has announced positive interim data from its Phase I/II AFFINITY DUCHENNE trial involving RGX-202, a potential gene therapy for Duchenne muscular dystrophy. The trial results indicate consistent functional, safety, and biomarker benefits, with participants in dose level 2 surpassing external natural history controls on key functional measures. Biomarker data showed robust microdystrophin expression, with one participant achieving 118.6% expression compared to controls. The trial, which aims to enroll around 30 patients in the U.S. and Canada by 2025, supports a potential Biologics License Application submission using the accelerated approval pathway in mid-2026. REGENXBIO plans to release top-line data in the first half of 2026. A webcast discussing these developments is scheduled for 8:00 a.m. EST, accessible via the company's website.
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