Dyne Therapeutics Reports Positive Results for Duchenne Drug DYNE-251, Plans FDA Approval Submission
Dyne Therapeutics has received FDA Breakthrough Therapy Designation for DYNE-251, an investigational exon 51 skipping therapy for Duchenne muscular dystrophy (DMD).
The designation is based on encouraging early results from the Phase 1/2 DELIVER trial, showing sustained functional improvements such as faster time to rise and improved walking speed over 18 months.
DYNE-251 is designed to enable production of near full-length dystrophin, a key protein for muscle function.
Full data from the Phase 1/2 registrational expansion cohort of the DELIVER trial are expected in late 2025.
Dyne plans to submit a Biologics License Application (BLA) for U.S. accelerated approval in early 2026, with some reports indicating a target in the second quarter of 2026.
DYNE-251 remains investigational and has not yet been approved by the FDA or other regulatory agencies.
Dyne is also advancing preclinical programs for other DMD exons (53, 45, 44) and pursuing international regulatory pathways.