REGENXBIO Inc. reported new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202 for Duchenne muscular dystrophy on March 19, 2025. Results were presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.
New biomarker data from two additional patients showed consistent, robust expression of RGX-202 microdystrophin. A 3-year-old patient achieved microdystrophin expression of 122.3% compared to control, while a 7-year-old patient showed 31.5% expression.
RGX-202 was well tolerated with no serious adverse events or adverse events of special interest as of February 21, 2025. The company continues to advance the pivotal trial towards completing enrollment this year and a Biologics License Application submission in mid-2026.
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