Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) is a commercial-stage biopharmaceutical company focused on discovering and developing RNA-targeted therapeutics, gene therapy, and other genetic therapeutic modalities for the treatment of rare diseases. The company's website is www.sarepta.com.

Sarepta operates as a precision genetic medicine company with a diversified portfolio spanning multiple therapeutic modalities. The company has developed and commercialized four approved products for Duchenne muscular dystrophy: EXONDYS 51, VYONDYS 53, AMONDYS 45, and ELEVIDYS. EXONDYS 51, launched in 2016, targets approximately 13 percent of Duchenne patients amenable to exon 51 skipping. VYONDYS 53, launched in 2019, addresses approximately 8 percent of patients amenable to exon 53 skipping, while AMONDYS 45, launched in 2021, targets approximately 8 percent of patients amenable to exon 45 skipping. ELEVIDYS, an AAV-based gene therapy launched in 2023, represents the company's entry into gene therapy treatment for Duchenne.

The company's core technology platforms include phosphorodiamidate morpholino oligomer chemistry for exon skipping, small interfering RNA programs developed through collaboration with Arrowhead Pharmaceuticals, and adeno-associated virus gene therapy utilizing the AAVrh.74 vector. Beyond Duchenne, Sarepta is developing therapeutic candidates for limb-girdle muscular dystrophy, facioscapulohumeral muscular dystrophy, myotonic dystrophy type 1, spinocerebellar ataxia, idiopathic pulmonary fibrosis, and Huntington's disease.

For the fiscal year ended December 31, 2025, Sarepta recorded net revenues of $1,864.3 million. The company maintains strategic partnerships with major pharmaceutical and biotechnology entities, including F. Hoffmann-La Roche Ltd for ELEVIDYS commercialization outside the United States and Arrowhead Pharmaceuticals for siRNA program development. Manufacturing and supply operations leverage partnerships with contract manufacturing organizations including Catalent and Aldevron to support both clinical development and commercial distribution.

Sarepta distributes its PMO products through specialty pharmacy providers and hospital networks in the United States, with international distribution through early access programs in select countries. The company's pipeline includes multiple programs in various stages of clinical development, reflecting its commitment to advancing precision genetic medicine across multiple rare disease indications.