Dyne’s Investigational DMD Drug DYNE-251 Gets Japan Orphan Drug Status After Strong Trial Data
Core Insights - Dyne Therapeutics Inc. has received Orphan Drug designation in Japan for its investigational drug DYNE-251, aimed at treating Duchenne muscular dystrophy (DMD) with specific gene mutations [1][3] - The ongoing DELIVER trial has shown significant and sustained functional improvement over 18 months, attributed to notable dystrophin expression [2][3] - DYNE-251 is currently in a Phase 1/2 clinical trial, which is randomized, placebo-controlled, and double-blind, indicating a rigorous testing process [3] Company Overview - Dyne Therapeutics is a clinical-stage company focused on developing therapeutics for neuromuscular diseases in the United States [4]