Sarepta Therapeutics Presents New Preclinical Data Demonstrating Robust Knockdown of Pathogenic DMPK RNA in the Nucleus with SRP-1003 in DM1 at 2026 Oligonucleotide Therapeutics Society Annual Meeting
Sarepta presented preclinical data at the Oligonucleotide Therapeutics Society annual meeting showing that investigational siRNA program SRP-1003 can reduce pathogenic DMPK transcripts in the nucleus in DM1.
Why it matters — The findings concern SRP-1003’s preclinical activity against a molecular driver of DM1, an area relevant to Sarepta’s development program.