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NewsA promising gene therapy for Duchenne muscular dystrophy (DMD) just cleared a major hurdle. RGX-202 met its primary end point in the Phase 3 AFFINITY DUCHENNE trial, with 93% of evaluable participants achieving microdystrophin expression above the target threshold. Early functional data at one year also showed meaningful improvements over external controls.
A gene therapy for one of the most devastating childhood muscle diseases is showing real promise. RGX-202, developed by REGENXBIO, met the primary end point of its Phase 3 AFFINITY DUCHENNE trial, with muscle biopsies from 30 participants showing mean microdystrophin expression exceeding 10% of normal levels at week 12 — the prespecified threshold for success. An impressive 93% of evaluable participants crossed that bar, with results reaching statistical significance (P < .0001). The findings were presented at the 2026 Child Neurology Society Annual Meeting in Montreal.
Functional data from 9 participants who reached one year of follow-up added to the encouraging picture, with RGX-202-treated boys outperforming external propensity score-weighted controls on the North Star Ambulatory Assessment (NSAA) and timed movement tests. Microdystrophin expression at week 12 also correlated significantly with functional improvement at one year.
By the Numbers:
Why it matters: DMD is a progressive, life-limiting disease affecting young boys with no cure. A gene therapy that durably restores even partial dystrophin function could meaningfully slow disease progression — and these Phase 3 results bring RGX-202 closer to potential regulatory approval.
