Sarepta Announces First Clinical Data from siRNA Pipeline Targeting FSHD1 and DM1

Early results from Phase 1/2 ascending dose studies of SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP-1003 for myotonic dystrophy type 1 (DM1) demonstrated dose- dependent muscle exposure, early biomarker effects, and favorable tolerability, reinforcing scientific confidence in the potential for differentiated benefits of the v6 integrin-targeted delivery platform. In addition, the Company has generated proof-of-concept data which found that after a single dose, both SRP-1001 and ...