Dyne's DYNE-251 Secures Orphan Drug Designation For DMD Treatment, Plans To Submit BLA In Early 2026

(RTTNews) - Dyne Therapeutics, Inc. (DYN), Thursday announced that the European Commission has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy or DMD.

The company added that DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial in individuals with DMD, who are amenable to exon 51 skipping.

Notably, long-term clinical data from the trial showed unprecedented and sustained functional improvement in the participants at the selected registrational dose.

Following this designation, the company plans to share data from the cohort in late 2025 and expects to move forward with Biologics License Application submission in early 2026.

In the pre-market hours, Dyne's stock is trading at $11.25, up 4.36 percent on the Nasdaq.