Sarepta Therapeutics Inc (Sarepta) is a commercial-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapeutics, gene therapy, and other genetic therapeutic modalities for treating rare diseases. The company has developed multiple approved products for Duchenne muscular dystrophy and is working on potential therapeutic candidates for a range of diseases, including other neuromuscular disorders and central nervous system-related disorders. Sarepta is also developing potential therapeutic candidates through its partnered program with Arrowhead for the treatment of Facioscapulohumeral muscular dystrophy (FSHD), myotonic dystrophy type 1 (DM1), Spinocerebellar ataxia (SCA), Idiopathic Pulmonary Fibrosis (IPF), Huntington's disease and other neuromuscular and skeletal diseases. Its RNA technologies include phosphorodiamidate morpholino oligomers (PMOs) and peptide phosphorodiamidate morpholino oligomers (PPMOs). PMO chemistry forms the basis of its technology platform, which powers the design and development of novel treatments for rare, infectious and other diseases.

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Sarepta's R&D arm focuses on developing genetic medicine pipelines, including gene therapy and RNA-based therapies. The company is actively engaged in research, development, and commercialization alliances with various entities such as universities, hospitals, and pharmaceutical companies to enhance and exploit its core technologies. Sarepta is also pursuing opportunities to access intellectual property rights that complement its internal portfolio through license agreements or other arrangements. Sarepta's R&D activities are managed through its internal expertise in process development, analytical development, quality control and assurance, and pre-clinical manufacturing. The company has built competencies around these areas to support the development and manufacturing of multiple products in parallel. The company's pipeline products include iRNA therapies like SRP-1001 for FSHD1, SRP-1003 for DM1, SRP-1004 for SCA2, and SRP-1002 for IPF. It also offers gene therapies such as SRP-9003, SRP-1004, SRP-1005, and SRP-1007. In FY2025, Sarepta spent US$1,522.1 million on its R&D activities, which as a percentage of revenue stood at 69.2%. The company holds numerous issued patents and exclusive rights to issued patents. It has 593 employees that are engaged directly in research and development activities. Sarepta pipeline comprised of programs in various stages of development, including one gene therapy and three RNA-based therapies on the market in the United States. The company has entered into long-term partnerships for large-scale supply and collaborates with partners like Aldevron, Catalent, and PPD for manufacturing and analytic testing.