• In early clinical results, Sarepta's αvβ6 integrin-targeted siRNA approach achieves high muscle concentrations without dose limiting toxicity for FSHD1 and DM1
  • Company to host investor call on March 25, 2026, at 8:30 a.m. Eastern time

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared the first clinical results from two of its siRNA programs for neuromuscular diseases.

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 αvβ6 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 SRP-1003 support reduction, or knockdown, of the target protein or mRNA. In both studies, the majority of adverse events were mild to moderate and were not dose dependent.

For rare, genetic diseases, such as FSHD and DM1, which are caused by overexpression of mutant proteins or toxic mRNA, RNA-targeted therapies hold significant promise but have been limited by rapid degradation of drug before it reaches the intended cells. SRP-1001 and SRP-1003 are each designed with an optimized siRNA chemistry and a proprietary, αvβ6 integrin-targeted ligand, intended to enable the siRNA to enter the cell and penetrate muscle tissue. Through this targeted delivery approach, these investigational treatments aim to overcome some of the delivery and safety challenges of other approaches.