SRPT Profile
Sarepta Therapeutics, Inc., based in Cambridge, Massachusetts, is a biopharmaceutical company focused on advancing RNA-targeted therapeutics, gene therapies, and genetic therapeutic solutions to address rare diseases. Established in 1980, the company specializes in the discovery and development of innovative treatments aimed at transforming the lives of patients with debilitating genetic disorders.
At the forefront of its portfolio are EXONDYS 51 and VYONDYS 53, both approved treatments for Duchenne muscular dystrophy (Duchenne) targeting specific mutations in the dystrophin gene amenable to exon skipping strategies. Sarepta Therapeutics is also actively developing AMONDYS 45, utilizing phosphorodiamidate morpholino oligomer chemistry to skip exon 45 of the dystrophin gene, alongside SRP-5051, a peptide conjugated PMO designed to bind exon 51 of dystrophin pre-mRNA.
Further bolstering its pipeline, the company is advancing gene therapy programs including SRP-9001 for DMD micro-dystrophin and SRP-9003 for limb-girdle muscular dystrophies. Sarepta Therapeutics collaborates extensively with renowned institutions and entities such as F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Lysogene, Duke University, Genethon, and StrideBio, leveraging collective expertise to accelerate therapeutic innovations and clinical advancements.
Driven by a commitment to scientific excellence and patient-centered innovation, Sarepta Therapeutics continues to pioneer new treatments that address significant unmet medical needs in rare disease populations. Through strategic partnerships and ongoing research initiatives, the company remains dedicated to delivering transformative therapies that improve the quality of life for individuals affected by these challenging conditions.
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