Dyne Therapeutics Announces FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne

Dyne Therapeutics (NASDAQ:DYN) has received FDA Breakthrough Therapy Designation for DYNE-251, targeting Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER trial, where DYNE-251 demonstrated sustained functional improvement through 18 months in key measures like time to rise and stride velocity.

The company has completed enrollment of 32 patients in the DELIVER registrational expansion cohort, with data expected in late 2025. Dyne plans to submit a Biologics License Application (BLA) for U.S. accelerated approval in early 2026. DYNE-251 has also received Fast Track, Orphan Drug, and Rare Pediatric disease designations from the FDA.

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