Dyne Therapeutics announces FDA BTD to DYNE-251

Dyne Therapeutics (DYN) announced that the U.S. Food and Drug Administration, FDA, has granted Breakthrough Therapy Designation, BTD, to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy, DMD, amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial. “This Breakthrough Therapy Designation for DYNE-251 is a testament to its potential as a next-generation therapy designed to bring meaningful functional improvement to individuals with DMD for whom exon 51 skipping can lead to the production of near full-length dystrophin,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “As we’ve previously disclosed, DYNE-251 has demonstrated sustained functional improvement through eighteen months, as assessed by key measures such as time to rise and stride velocity 95th centile. The level of near-full-length dystrophin expression observed marks a significant step forward to potentially deliver meaningful benefits to patients.”