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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 Therapeutics (SRPT)Sarepta TherapeuticsCompany update

Correction of MBNL- dependent mis-splicing provides functional evidence that SRP-1003 is degrading disease-causing RNA within the nucleus. In a preclinical model, SRP-1003 reduced pathogenic DMPK mRNA in both the cytoplasm and nucleus in a dose-dependent manner, achieving up to approximately 80%

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Sarepta Therapeutics

Precision Genetic Medicine Sarepta Therapeutics develops and commercializes genetic medicines for rare neuromuscular diseases, with a major focus on Duchenne muscular dystrophy. ELEVIDYS ELEVIDYS is an AAV-based gene-transfer therapy that delivers a micro-dystrophin transgene to skeletal muscle. It is indicated for ambulatory patients...

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Stock data

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18.56 USD
Market cap
1.72B USD
Exchange
NASDAQ
Sector
Gene Therapy
Location
United States