Sarepta Therapeutics
SRPTSRPT · Stock Price
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Overview
Sarepta Therapeutics is a commercial-stage biotech leader with an urgent mission to engineer and deliver precision genetic medicines for rare diseases. The company has achieved significant commercial success with multiple FDA-approved RNA-based exon-skipping therapies for Duchenne muscular dystrophy (DMD) and the landmark approval of ELEVIDYS, a gene therapy for DMD. Its strategy leverages a deep, multi-modal pipeline, a hybrid manufacturing model for scale, and a global commercial footprint to solidify its position as a dominant force in rare disease therapeutics.
Technology Platform
Sarepta's technology is built on two complementary platforms: a proprietary RNA-targeted Phosphorodiamidate Morpholino Oligomer (PMO) chemistry for exon-skipping therapies and an adeno-associated virus (AAV)-based gene therapy engine designed to deliver functional transgenes to muscle tissue.
Pipeline
34| Drug | Indication | Stage | Watch |
|---|---|---|---|
| Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53] | Duchenne Muscular Dystrophy | Approved | |
| Eteplirsen | Muscular Dystrophy, Duchenne | Phase 3 | |
| SRP-9005 + Corticosteroid | Muscular Dystrophies, Limb-Girdle | Phase 3 | |
| Casimersen + Golodirsen | Duchenne Muscular Dystrophy | Phase 3 | |
| SRP-9003 + Glucocorticoid | Limb-girdle Muscular Dystrophy | Phase 3 |
Funding History
3FDA Approved Drugs
3
Company Timeline
Founded in Cambridge, United States
IPO — $45.0M
FDA Approval: EXONDYS 51
PIPE: $400.0M
FDA Approval: VYONDYS 53
Debt: $550.0M