Sarepta Therapeutics, Inc. (NASDAQ: SRPT; LSE: 0L35) is a commercial-stage biopharmaceutical company that discovers, develops, and commercializes precision genetic medicines for rare neuromuscular and central nervous system disorders, with a primary focus on Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and other rare genetic diseases; its approved products include ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy for ambulatory and non-ambulatory DMD patients aged four and older, EXONDYS 51 (eteplirsen), VYONDYS 53 (golodirsen), AMONDYS 45 (casimersen), and SRPT-600, an investigational DMD therapy, alongside a broad pipeline leveraging gene therapy, RNA technology including phosphorodiamidate morpholino oligomers (PMOs) and siRNA platforms, and gene editing across skeletal muscle, cardiac, and CNS targets. Founded in 1980 and headquartered in Cambridge, Massachusetts, with manufacturing facilities in Columbus, Ohio, and operations primarily in the United States serving patients through specialized sales forces and patient advocacy engagement, the company recently entered a global licensing and collaboration agreement with Arrowhead Pharmaceuticals in November 2024 for multiple clinical and preclinical siRNA programs including SRP-1003 for DM1, involving a $500 million upfront payment and $325 million equity investment, triggering a $100 million milestone in August 2025 for program advancement while selling the equity stake to fund further development; additionally, in November 2025, the FDA approved initiation of ENDEAVOR Cohort 8 to evaluate an enhanced immunosuppression regimen with sirolimus for ELEVIDYS in non-ambulatory DMD patients to mitigate acute liver injury risks, and the company completed confirmatory trials and reported third-quarter 2025 financial results supporting ongoing commercialization and pipeline expansion.