Sarepta (SRPT) Therapeutics 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 alphavbeta6 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.
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