Sarepta Therapeutics, Inc. (SRPT)

Sarepta Therapeutics, Inc. (SRPT) is a commercial-stage biopharmaceutical company focused on precision genetic medicine for rare diseases, headquartered in Cambridge, Massachusetts. Led by CEO Doug Ingram, Sarepta’s strategic mission is to "unleash the power of genetic medicine to rescue lives from devastating rare diseases." The company holds a pioneering industry position as the leader in Duchenne muscular dystrophy (DMD) treatments. Sarepta’s vision is to become the "Standard of Care in Genetic Medicine." By late 2025, SRPT stock has stabilized following the successful "Cohort 8" expansion of its ELEVIDYS gene therapy, targeting non-ambulant individuals to significantly broaden its patient reach.

The business operations of Sarepta revolve around its proprietary RNA-targeted technology and gene therapy platforms. In fiscal 2025, the company reported annual revenues exceeding $2.4 billion, driven by the rapid adoption of ELEVIDYS. The 2026 strategic roadmap focuses on "Pipeline Maturity," including progress on SRP-1003 for Myotonic Dystrophy and its next-generation peptide-conjugated PMO platform. Sarepta holds a "Scientific Moat" protected by an extensive patent estate and its deep partnership with Roche for international commercialization. By late 2025, the company refinanced its debt to improve its capital structure, ensuring it has the liquidity to fund its 40+ programs in various stages of development. As gene therapy becomes more widely accepted, Sarepta remains the definitive leader in the commercialization of life-changing genetic interventions.