# Sarepta Therapeutics

> Sarepta Therapeutics develops and commercializes genetic medicines for rare diseases. Its four marketed Duchenne muscular dystrophy therapies span mutation-specific RNA exon skipping and one-time gene transfer, while an investigational siRNA portfolio extends its research into other muscle, neurological and pulmonary diseases.

- **Founded:** 1980
- **Headquarters:** Cambridge, United States
- **Founders:** James E. Summerton (Founder; former Chairman, CEO and Chief Scientific Officer)
- **Products:** EXONDYS 51 (eteplirsen), VYONDYS 53 (golodirsen), AMONDYS 45 (casimersen), ELEVIDYS (delandistrogene moxeparvovec-rokl), SareptAssist
- **Notable:** Brought three mutation-specific exon-skipping medicines to the U.S. market between 2016 and 2021., ELEVIDYS became the first FDA-approved Duchenne gene therapy in 2023., Reached $2.198 billion in reported total annual revenue in 2025.

## Products & services

- **EXONDYS 51 (eteplirsen)** — Weekly intravenous RNA exon-skipping medicine for Duchenne patients with a confirmed mutation amenable to exon 51 skipping; received accelerated FDA approval.
- **VYONDYS 53 (golodirsen)** — Weekly intravenous RNA exon-skipping medicine for Duchenne mutations amenable to exon 53 skipping; accelerated approval, with a traditional-approval application under review as of October 2026.
- **AMONDYS 45 (casimersen)** — Weekly intravenous RNA exon-skipping medicine for Duchenne mutations amenable to exon 45 skipping; accelerated approval, with a traditional-approval application under review as of October 2026.
- **ELEVIDYS (delandistrogene moxeparvovec-rokl)** — Single-infusion AAV gene-transfer therapy, initially approved in 2023. Current U.S. use is limited to ambulatory patients aged four and older with a confirmed DMD mutation, subject to contraindications and a boxed liver-injury warning.
- **SareptAssist** — Patient support program for treatment access, insurance navigation and support during the treatment journey.
- **Investigational siRNA portfolio** — Research programs include SRP-1001 for FSHD and SRP-1003 for DM1; these are investigational medicines, not approved products.

## Achievements

- Brought three mutation-specific exon-skipping medicines to the U.S. market between 2016 and 2021.
- ELEVIDYS became the first FDA-approved Duchenne gene therapy in 2023.
- Reached $2.198 billion in reported total annual revenue in 2025.

## Latest updates

- **2026-10** — October 14: company reported preclinical SRP-1003 results showing reduction of pathogenic DMPK RNA in nuclear and cytoplasmic compartments; human efficacy is not established by these findings.
- **2026-09** — September 30: company presented older-ambulatory ELEVIDYS analyses using an external comparison group at World Muscle Society; these are company-reported findings.
- **2026-08** — Second-quarter net product revenue was $328.7 million: $230.6 million from PMOs and $98.1 million from ELEVIDYS.
- **2026-07** — Michael Severino became CEO effective July 28, succeeding Doug Ingram.
- **2026-06** — FDA accepted applications seeking traditional approval for AMONDYS 45 and VYONDYS 53; target action date February 28, 2027. Acceptance is not approval.

## Links

- Website: https://www.sarepta.com/
- LinkedIn: https://www.linkedin.com/company/sarepta-therapeutics
- Twitter/X: https://twitter.com/sarepta
- Instagram: https://www.instagram.com/sareptatherapeutics/
- Facebook: https://www.facebook.com/Sarepta-Therapeutics-208950446535427/

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Profile page: https://yespress.io/sarepta-therapeutics
Published by YesPress — https://yespress.io
Last updated: 2026-10-23