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 new preclinical data at the Oligonucleotide Therapeutics Society meeting showing its DM1 siRNA candidate SRP-1003 reduces pathogenic DMPK RNA in the nucleus, achieving up to approximately 80% knockdown in both nuclear and cytoplasmic compartments in a dose-dependent manner. Nuclear target engagement was confirmed by three independent methods (RNA FISH, compartment-fractionated ddPCR, and RNAscope), and MBNL-dependent mis-splicing correction provided functional evidence of nuclear activity. SRP-1003 showed sustained exposure across multiple muscle groups for up to 16 weeks after a single IV dose in preclinical models.
Key figures
- Drug Name
- SRP-1003
- Phase Of Trial
- Phase 1/2
- Dmpk knockdown
- up to approximately 80% in nucleus and cytoplasm
- Muscle exposure duration
- up to 16 weeks after single IV dose
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