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Wednesday, March 25, 2026

Sarepta 1st Phase 1/2 data supporting αvβ6 integrin-targeted siRNA muscle delivery platform

 

Sarepta unveils first Phase 1/2 data for SRP-1001 and SRP-1003 supporting αvβ6 integrin-targeted siRNA muscle delivery platform

  • SRP-1001 is being developed for FSHD1 and SRP-1003 for DM1 in Phase 1/2 studies.
  • Initial clinical data show dose-dependent muscle exposure and target knockdown for both siRNA candidates.
  • Both candidates demonstrated favorable tolerability, supporting Sarepta's αvβ6 integrin-targeted delivery platform for muscle.

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