Dyne Therapeutics said its exon‑51 targeting candidate produced unprecedented dystrophin increases in a registrational cohort, prompting plans to seek accelerated FDA approval next year. The registrational expansion showed mean muscle‑content adjusted dystrophin of 5.46% of normal and unadjusted 2.87% after six months. The company contrasted those figures with historical data for Sarepta’s Exondys 51 (about 0.3% in earlier trials) and reported improvements across functional endpoints. Dyne described safety as favorable with mostly mild‑to‑moderate events. Dyne plans an accelerated submission in Q2 next year and is building commercial, medical and CMC capabilities ahead of a potential early‑2027 launch if approved.
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