NasdaqGS:SRPTBiotechs
Sarepta Therapeutics (SRPT) Is Up 16.3% After Early siRNA Muscle Data Validates Avß6 Platform
In late March 2026, Sarepta Therapeutics reported first-in-human Phase 1/2 data for its SRP-1001 and SRP-1003 siRNA programs in facioscapulohumeral muscular dystrophy type 1 and myotonic dystrophy type 1, showing dose-dependent muscle exposure, early biomarker effects and generally favorable tolerability using its avß6 integrin-targeted delivery platform.
The early proof-of-concept knockdown of target protein or mRNA not only supports these rare disease programs, but also strengthens the...