FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne MD

Dyne Therapeutics, Inc.

• FDA granted Breakthrough Therapy Designation to DYNE-251 for DMD amenable to exon 51 skipping.
• Designation based on DELIVER trial data showing sustained functional improvement over 18 months.
• DYNE-251 now joins DYNE-101 (DM1) as Dyne's second compound to receive this designation.
• Registrational expansion cohort data expected late 2025; BLA submission for accelerated approval anticipated early 2026.
• Benefits include rolling review, Priority Review eligibility, and enhanced FDA support.