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Sarepta Therapeutics
Sarepta Therapeutics operates as a commercial-stage biopharmaceutical business based in Cambridge, Massachusetts.
Sarepta Therapeutics
Sarepta Therapeutics operates as a commercial-stage biopharmaceutical business based in Cambridge, Massachusetts. The company's core focus is developing precision genetic medicines — including antisense oligonucleotides and gene therapies — to address rare diseases with high unmet medical need. Sarepta's investment posture centers on in-house R&D and strategic partnerships. It holds US accelerated approval for three exon-typing agents for Duchenne muscular dystrophy: Exondys 51 (eteplirsen), Vyondys 53 (golodirsen), and Amondys 45 (casimersen). The pipeline extends to limb-girdle muscular dystrophy and other neuromuscular disorders via gene therapy candidates from its internal platform and a collaboration with Roche that closed in 2019. The firm's geographic footprint focuses on the United States but includes partnerships covering ex-US commercialization rights. As a publicly traded company (NASDAQ: SRPT), Sarepta does not disclose AUM or operator metrics typical of an asset manager. It employed over 1,400 people as of its most recent annual report. The firm maintains no disclosed philanthropic or adjacent investment vehicles separate from its corporate R&D operations. Sarepta's structural differentiator is its dual-platform strategy combining RNA-based exon skipping with gene therapy capabilities, a rare combination among rare-disease biotechs. Succession and governance follow standard public-company board structures; founder Chris Garabedian left the CEO role in 2015, but the scientific leadership remains intact.
General information
Firm type
other
Year founded
—
AUM
Undisclosed
Location
Region
North America
Country
United States
City
Cambridge
Corporate office
Cambridge, MA, United States
Sector focus
Frequently asked questions
What type of entity is Sarepta Therapeutics?
Sarepta Therapeutics is a publicly traded biopharmaceutical company (NASDAQ: SRPT), not a family office or asset manager. It develops genetic medicines for rare diseases.
Does Sarepta Therapeutics manage outside capital?
No. Sarepta is a corporate biotech — it funds operations through revenue from approved therapies, equity offerings, and partnership payments (e.g., a $1.1B upfront-plus-milestones deal with Roche in 2019 for its DMD gene therapy program). There is no disclosed AUM or external LP capital.
What are Sarepta's approved therapies?
Three exon-skipping drugs for Duchenne muscular dystrophy: Exondys 51 (approved 2016), Vyondys 53 (2019), and Amondys 45 (2021). All target specific mutations in the DMD gene. All three have US accelerated approval, and confirmatory trials are ongoing.
Who leads Sarepta Therapeutics?
The CEO is Douglas Ingram, appointed in 2017. The chief scientific officer is Mendell R. R. Mendell, a neurologist who joined from Nationwide Children's Hospital. The board includes scientific and industry figures; no controlling family or individual owner is disclosed beyond public filings.
What is Sarepta's relationship with Roche?
In 2019, Sarepta licensed ex-US rights to its SRP-9001 DMD gene therapy to Roche in a deal valued at up to $1.7B in milestones plus royalties. Roche co-funds development and holds commercialization rights outside the US. The therapy received accelerated approval in the US in June 2023.
Does Sarepta invest in other biotechs or venture funds?
Sarepta does not operate a corporate venture arm or make disclosed direct investments in external companies. Its capital is deployed internally on R&D, manufacturing capacity, and partnership prepayment obligations.
Where does Sarepta's cash come from?
Revenue from its three approved DMD therapies, plus funding from strategic partnerships (notably Roche), equity offerings, and debt facilities. As of its most recent 10-K, the company held approximately $1.8B in cash, cash equivalents, and investments, primarily in government and corporate bonds.
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