Sarepta says SRP-1003 DM1 Phase 1/2 study advances after preclinical data show up to 80% DMPK RNA knockdown

PUBT · 1d ago
Sarepta says SRP-1003 DM1 Phase 1/2 study advances after preclinical data show up to 80% DMPK RNA knockdown
  • Sarepta disclosed new preclinical data on SRP-1003 for myotonic dystrophy type 1, presented at the 2026 Oligonucleotide Therapeutics Society meeting.
  • The studies showed SRP-1003 reduced the disease-driving DMPK RNA inside the cell nucleus, supporting its intended mechanism.
  • Functional readouts indicated improvement in downstream RNA processing disruptions linked to the disorder.
  • The update supports continued development in an ongoing Phase 1/2 trial, with clinical results expected later this year.


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