Dyne Therapeutics receives FDA Orphan Drug Designation for DYNE-251

Dyne Therapeutics announced that DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy mutations amenable to exon 51 skipping, was granted U.S. Food and Drug Administration, FDA, orphan drug and rare pediatric disease designations. DYNE-251 is being evaluated in the Phase 1/2 DELIVER clinical trial. "These regulatory designations highlight the urgent and critical need for new and better therapeutic options for people living with this fatal disease," said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. "We are excited about DYNE-251 which we believe has the potential to transform the lives of people with DMD. We continue to advance our DELIVER clinical trial and look forward to sharing initial clinical data later this year."