Sarepta Therapeutics Inc (NASDAQ: SRPT) is a biopharmaceutical company focused on the discovery, development, and commercialization of RNA-targeted therapies and gene therapies for rare neuromuscular diseases. Revenue is generated through product sales of approved therapies, primarily targeting Duchenne muscular dystrophy (DMD) and related rare genetic conditions. The company holds a portfolio of approved and investigational treatments, including exon-skipping therapies and adeno-associated virus (AAV)-based gene therapies. Sarepta operates in a specialized rare disease segment where FDA approval pathways, patent protection, and reimbursement from government and commercial payors are central to commercial success. As of the 10-K filed March 2, 2026, CEO Douglas S. Ingram leads the company, with Ryan H. Wong serving as Executive Vice President and Chief Financial Officer. The company is subject to ongoing intellectual property litigation and holds patents in the United States and Europe with expirations ranging from 2028 through 2038, covering compositions of matter and methods of use across its product programs.

Revenue model

Product sales of approved RNA-targeted and gene therapies for rare neuromuscular diseases, with pricing subject to third-party payor reimbursement including government programs and commercial insurers.

Products and services

Approved and investigational exon-skipping therapies for Duchenne muscular dystrophy; investigational AAV-based gene therapies for limb-girdle muscular dystrophies (LGMD) including SRP-9003 (bidridistrogene xeboparvovec, LGMD2E/R4), SRP-9004 (patidistrogene bexoparvovec, LGMD2D), SRP-6004 (LGMD2B/R2), and SRP-9005 (LGMD2C/R5); and SRP-1001, an RNAi-based investigational therapy for facioscapulohumeral muscular dystrophy (FSHD). As of July 2025, FDA placed a clinical hold on LGMD gene therapy trials following a patient death in the SRP-9004 Phase 1/2 trial; the hold remained in place as of December 2025.

Customers and end markets

Patients with rare neuromuscular diseases, primarily Duchenne muscular dystrophy and limb-girdle muscular dystrophies. Products are sold through channels reimbursed by government healthcare programs and commercial insurers. Healthcare professionals and specialty pharmacies serve as the distribution interface.

Value-chain role

Drug developer and commercial-stage manufacturer of RNA-targeted therapies and gene therapies; holds owned and licensed patents in the U.S. and Europe covering compositions of matter and methods of use. Relies on third-party manufacturing relationships and is subject to FDA and international regulatory oversight.

Geographic exposure

Primary commercial operations in the United States; international operations subject to foreign regulatory approval processes, pricing and reimbursement requirements, and U.S. law including the Foreign Corrupt Practices Act.

Source: SEC 10-K, filed 2026-03-02

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