#rAAVrh74

U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4 CAMBRIDGE, Mass., June 04, 2025--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the rAAVrh74 viral vector used in the invest...

#FDA Sarepta's #rAAVrh74 viral vector in the limb girdle muscular dystrophy program receives #platform designation.

This is another sign that 'new' FDA is serious in accelerating rare disease drug development + bodes well for agency's view of recent DMD death: finance.yahoo.com/news/u-fda-g...