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
Key Highlights
- ➤SRP-1003 achieved up to approximately 80% DMPK mRNA knockdown in nuclear and cytoplasmic compartments
- ➤SRP-1003 corrected MBNL-dependent mis-splicing, providing functional evidence of nuclear activity
- ➤Nuclear target engagement was confirmed using ddPCR, RNA FISH, and RNAscope methodologies
- ➤SRP-1003 remained detectable across multiple muscle groups for up to 16 weeks after one intravenous dose
- ➤Sarepta expects to share Phase 1/2 clinical study results later this year
Expert Statements
Louise Rodino-Klapac, President, Research & Development and Technical Operations, Sarepta Therapeutics
“For an siRNA approach to address the underlying biology of DM1, it must reduce the toxic DMPK in the nucleus.”
Louise Rodino-Klapac, President, Research & Development and Technical Operations, Sarepta Therapeutics
“These results provide two complementary lines of evidence that SRP-1003 effectively and efficiently gets into the nucleus.”
Louise Rodino-Klapac, President, Research & Development and Technical Operations, Sarepta Therapeutics
“We observed correction of MBNL-dependent mis-splicing, a functional readout that requires degradation of the nuclear transcript and measured comparable levels of DMPK knockdown in the nucleus and cytoplasm.”
Louise Rodino-Klapac, President, Research & Development and Technical Operations, Sarepta Therapeutics
“Together with the reductions in toxic nuclear RNA foci across multiple models and methods, these data strengthen the biological rationale for SRP-1003 and support the continued advancement of the program in our ongoing Phase 1/2 clinical study, from which we expect to share results later this year.”
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
* Correction of MBNL- dependent mis-splicing provides functional evidence that SRP-1003 is degrading disease-causing RNA within the nucleus.
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