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