Sarepta Therapeutics
↗Cambridge, USA
Sarepta Therapeutics is a commercial-stage biopharmaceutical company and a global leader in precision genetic medicine for rare diseases. The company is primarily focused on developing therapies for Duchenne Muscular Dystrophy (DMD) and other rare neuromuscular and neurological disorders. Sarepta utilizes a multi-platform approach, including RNA-targeted technologies (PMO, PPMO) and gene therapy (AAV), to address the underlying genetic causes of disease.
Headquartered in Cambridge, Massachusetts, Sarepta has successfully brought four therapies to market in the U.S., including the first-ever gene therapy for DMD, Elevidys. The company maintains a robust pipeline of over 40 programs and has established significant strategic partnerships with global pharmaceutical leaders like Roche to expand the reach of its transformative medicines.
CLASSIFICATION
SIZE & FINANCIALS
Employees:1001-5000
Revenue:$1.5B-$2.5B
Founded:1980
Ownership:public
Status:operating
FUNDING
Stage:Public
Total Raised:$2.5B+
Investors:Vanguard Group, BlackRock, State Street Global Advisors, Perceptive Advisors
STOCK
Exchange:NASDAQ
Ticker:SRPT
Market Cap:$12.5B
PIPELINE
Stage:Commercial
Lead Drug Stage:Commercial (Elevidys, Exondys 51, Vyondys 53, Amondys 45)
Modalities:RNA (PMO/PPMO), Gene therapy (AAV), siRNA, Gene editing
Active Trials:25
Trial Phases:Phase 1: 8 | Phase 2: 10 | Phase 3: 5 | Phase 4: 2
FDA Approvals:4
EMA Approvals:0
CORPORATE STRUCTURE
Subsidiaries:Sarepta International, Myonexus Therapeutics
Key Partnerships:Roche (Ex-US rights for Elevidys), Arrowhead Pharmaceuticals (siRNA collaboration), Catalent (Gene therapy manufacturing), Chugai Pharmaceutical (Japan distribution)
COMPETITION
Position:Leader
Competitors:NS Pharma (Nippon Shinyaku), Solid Biosciences, Dyne Therapeutics, PTC Therapeutics, Italfarmaco, Regenxbio
LEADERSHIP
Key Executives:
Douglas Ingram - CEO
Ian Estepan - CFO
Louise Rodino-Klapac - CSO
Scientific Founders:R. Iversen
Board Members:Douglas Ingram, M. Kathleen McGlynn, Richard Barry, Hans Wigzell
LINKS
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