Sarepta says SRP-1003 DM1 Phase 1/2 study advances after preclinical data show up to 80% DMPK RNA knockdown
SRPT•Sarepta said preclinical studies of SRP-1003 showed up to 80% knockdown of DMPK RNA, supporting continued development in an ongoing Phase 1/2 trial. Clinical results are expected later this year.
1. Preclinical findings
Sarepta disclosed preclinical data on SRP-1003 for myotonic dystrophy type 1 at the 2026 Oligonucleotide Therapeutics Society meeting. The studies showed reduced disease-driving DMPK RNA inside the cell nucleus and 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.
