FDA Places Clinical Hold on Sarepta DMD Micro-Dystrophin Gene Therapy Trial

Sarepta Therapeutics is promising a rapid response to the FDA’s placing a clinical hold on the company’s Phase I/IIa trial of its Duchenne Muscular Dystrophy (DMD) gene therapy candidate AAVrh74.MHCK7.micro-Dystrophin—a month after the company trumpeted the study’s first positive results.Sarepta acknowledged that the trial ( NCT03375164 ) was placed on clinical hold after a “trace” amount of DNA fragment surfaced in research-grade raw material plasmid supplied