Sarepta Therapeutics, Inc. (NASDAQ: SRPT)

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapeutics, siRNA knockdown therapies, gene therapy and other genetic modalities for the treatment of rare diseases. The company's primary emphasis is on Duchenne muscular dystrophy, for which it has commercialized four products, and it also advances programs in facioscapulohumeral muscular dystrophy, myotonic dystrophy type 1, spinocerebellar ataxia, idiopathic pulmonary fibrosis and Huntington's disease.

Revenue is generated principally from the sale of its approved Duchenne therapies: EXONDYS 51, VYONDYS 53, AMONDYS 45 and the gene therapy ELEVIDYS. Product sales to patients, hospitals and specialty pharmacy providers accounted for net revenues of $1,864.3 million in 2025, $1,788.0 million in 2024 and $1,144.9 million in 2023. The company also earns royalties and milestone payments from collaboration partners, although product sales constitute the core of its revenue stream.

Sarepta Therapeutics, Inc. holds a leading position in the Duchenne muscular dystrophy market as the developer of the first FDA-approved exon-skipping therapies and the first gene therapy, ELEVIDYS, for this indication. Its competitors include Nippon Shinyaku with its exon 53 skipping drug VILTEPSO, Italfarmaco with the steroid Givinostat, and several biotech firms pursuing alternative exon-skipping, gene editing and siRNA approaches. Competitive advantages stem from its proprietary phosphorodiamidate morpholino chemistry, the Arrowhead siRNA platform, the AAVrh.74 vector used in its gene therapy candidates, and a portfolio of orphan drug and regulatory exclusivities that protect its marketed products.

The company serves patients diagnosed with Duchenne and other rare neuromuscular disorders, delivering its therapies through hospitals, outpatient infusion centers and a limited network of home infusion specialty pharmacy providers in the United States. Outside the U. S., distribution occurs via third-party distributors and service providers under expanded access programs or in countries where its products have received marketing approval. Specific customer names are not disclosed in the filing.