Sarepta Therapeutics
Sarepta Therapeutics is a precision genetic medicine company that develops and commercializes AAV gene therapies and antisense oligonucleotide treatments for rare neuromuscular diseases, primarily Duchenne muscular dystrophy, serving patients through specialty pharmacy channels in the US and via Roche/Chugai internationally.
- Company typePublic
- Founded1987
- HeadquartersCambridge, United States
- Headcount501–1,000
- GTM typeB2B
- OfferingHardware or Manufacturing
What Sarepta Therapeutics does
Sarepta Therapeutics is a commercial-stage precision genetic medicine company developing therapeutics for rare neuromuscular diseases. Its portfolio centers on Duchenne muscular dystrophy (DMD), with four FDA-approved therapies: ELEVIDYS, an AAV-mediated gene therapy delivering micro-dystrophin; and three antisense oligonucleotide (PMO) exon-skipping products (EXONDYS 51, AMONDYS 45, VYONDYS 53) addressing distinct DMD mutations. The company is headquartered in Cambridge, Massachusetts, and operates gene therapy manufacturing in Columbus, Ohio (Genetic Therapies Center of Excellence within the $1.1B Nationwide Children's Innovation District) and Andover, Massachusetts. Its pipeline is expanding beyond DMD through an αvβ6 integrin-targeted siRNA delivery platform acquired from Arrowhead Pharmaceuticals in 2024 for $500M, supporting investigational programs in FSHD1 (SRP-1001), DM1 (SRP-1003), and Huntington's disease (SRP-1005).
Sarepta generates revenue primarily through one-time/perpetual pricing on ELEVIDYS and recurring pricing on its exon-skipping products, distributed via a US specialty pharmacy and treating-center channel and ex-US through the Roche/Chugai partnership (Japan commercial launch completed February 2026). FY2025 net product revenue reached $1.86B (up 16% YoY), with ELEVIDYS contributing $898.7M and total revenue including collaboration reaching approximately $2.2B. Customer support is delivered through SareptAssist and SareptAlly programs. International commercialization rights for ELEVIDYS are held by Roche, with a $40M milestone triggered by first Japan sale and a $325M declined-option payment recorded in FY2025 collaboration revenue.
The company faces a significant operational reset in 2025-2026 following patient deaths linked to ELEVIDYS: an FDA boxed warning for acute liver injury, shipment suspension for non-ambulatory patients, a CHMP negative opinion in the EU, a >35% workforce reduction, ENVISION study pause, revocation of the FDA platform technology designation, and the announced 2026 retirement of CEO Doug Ingram. Cash position of $953.8M at year-end 2025 and a $700M refinancing of convertibles to 2030 provide runway to navigate the transition while advancing the siRNA pipeline and resuming ex-US expansion.
Sarepta Therapeutics firmographics
Firmographics- Name
- Sarepta Therapeutics
- Legal name
- Sarepta Therapeutics, Inc.
- Website
- https://sarepta.com
- Company type
- Public
- Founded year
- 1987
- Operating status
- Operating
- Headcount range
- 501–1,000 employees
- Short description
- Sarepta Therapeutics is a precision genetic medicine company that develops and commercializes AAV gene therapies and antisense oligonucleotide treatments for rare neuromuscular diseases, primarily Duchenne muscular dystrophy, serving patients through specialty pharmacy channels in the US and via Roche/Chugai internationally.
- Ownership category
- akta.pro rank
Sarepta Therapeutics industry classification
Industry- Product category
- Rare Disease Genetic Medicine
- NAICS
- Biological Product (except Diagnostic) Manufacturing (325414), Scientific Research and Development Services (5417)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)
- akta.pro secondary industries
- Neurology/CNS Gene Therapies (HLAAACAH), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD), Neuromuscular Medicine (ALS, Myasthenia, Neuropathy) (HLAKAIAD)
Keywords
Where Sarepta Therapeutics is headquartered
LocationHeadquarters
- HQ city
- Cambridge
- HQ country
- United States
- HQ region
- North America
Offices5 records
Markets served
Sarepta Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales, Others
Revenue model
- Product Sales: Net product revenue from four FDA-approved therapies: ELEVIDYS (gene therapy), EXONDYS 51, AMONDYS 45, VYONDYS 53 (exon-skipping). FY2025 net product revenues of $1.86 billion. Q1 2026 net product revenues of $330.5 million.
- Collaboration Revenue: Includes Roche declined option payment ($325 million), milestone payments from partners ($40M from Roche for Japan commercial launch). Royalty revenue from international sales through Chugai.
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| One time/ perpetual license | One time/ perpetual license | ELEVIDYS gene therapy pricing |
Go-to-market motion1 record
Distribution channels2 records
Marketing channels5 records
Sarepta Therapeutics product offering
Product offeringCore offering
Sarepta Therapeutics is a precision genetic medicine company that discovers, develops, and commercializes therapies for rare neuromuscular diseases. The company has four FDA-approved products for Duchenne muscular dystrophy (DMD): ELEVIDYS gene therapy, and three antisense oligonucleotide exon-skipping therapies (EXONDYS 51, AMONDYS 45, VYONDYS 53). It is also advancing investigational siRNA programs for facioscapulohumeral muscular dystrophy type 1 (FSHD1), myotonic dystrophy type 1 (DM1), and Huntington's disease.
Product overview
Sarepta Therapeutics is a precision genetic medicine company with multiple approved therapies and an advancing pipeline. The company's commercial portfolio consists of four FDA-approved products for Duchenne muscular dystrophy: ELEVIDYS (gene therapy), EXONDYS 51, AMONDYS 45, and VYONDYS 53 (all exon-skipping ASO therapies targeting different DMD mutations). The pipeline includes investigational siRNA programs (SRP-1001 for FSHD1, SRP-1003 for DM1, SRP-1005 for Huntington's disease) developed on the company's RNA platform with proprietary αvβ6 integrin-targeted delivery technology. The company also maintains a gene therapy platform for AAV-mediated gene delivery.
Differentiator
Problem solved
Functional benefit
Brands
- ELEVIDYS: Gene therapy for Duchenne muscular dystrophy (delandistrogene moxeparvovec-rokl)
- EXONDYS 51
- VYONDYS 53
- AMONDYS 45
- SareptAssist
- SareptAlly
Products and services
- ELEVIDYS (delandistrogene moxeparvovec-rokl) AAV-mediated gene therapy for Duchenne muscular dystrophy that delivers a gene encoding a shortened functional dystrophin protein (micro-dystrophin) to muscle cells. Approved for ambulatory patients aged 4 and older with confirmed DMD gene mutation; second-best selling gene therapy globally with 2025 sales of approximately $898.7 million.
- AMONDYS 45 (casimersen) Antisense oligonucleotide (ASO) exon-skipping therapy for Duchenne muscular dystrophy, designed to skip exon 45 of the DMD gene to produce a truncated but functional dystrophin protein. Approved under FDA accelerated approval pathway; sNDA for traditional approval conversion planned by April 2026.
- VYONDYS 53 (golodirsen) Antisense oligonucleotide (ASO) exon-skipping therapy for Duchenne muscular dystrophy, designed to skip exon 53 of the DMD gene to produce a truncated but functional dystrophin protein. Approved under FDA accelerated approval pathway; sNDA for traditional approval conversion planned by April 2026.
- EXONDYS 51 (eteplirsen) Antisense oligonucleotide (ASO) exon-skipping therapy for Duchenne muscular dystrophy, designed to skip exon 51 of the DMD gene to produce a truncated but functional dystrophin protein. Sarepta's first approved therapy, approved under FDA accelerated approval pathway.
- SRP-1001 Investigational siRNA therapy using the αvβ6 integrin-targeted delivery platform for facioscapulohumeral muscular dystrophy type 1 (FSHD1), designed to reduce production of the DUX4 protein believed to play a central role in the disease. Currently in Phase 1/2 clinical trials.
- SRP-1003 Investigational siRNA therapy for myotonic dystrophy type 1 (DM1), targeting and suppressing DMPK (dystrophia myotonica protein kinase) in skeletal muscle. Licensed from Arrowhead Pharmaceuticals; currently in Phase 1/2 clinical trials.
- SRP-1005 Investigational siRNA therapeutic for Huntington's disease, with FDA approval of clinical trial application announced. Targets the huntingtin disease gene using the proprietary αvβ6 integrin-targeted delivery platform.
Quantifiable outcome
- 3-year EMBARK: Statistically significant and clinically meaningful efficacy across NSAA, TTR, and 10MWR vs external control group
- +3 more outcomes
Companies that use Sarepta Therapeutics
Customer profileNamed customers1 record
Segments4 records
Ideal customer profiles2 records
Sarepta Therapeutics technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Sarepta Therapeutics partnerships and signals
Strategic signalPartnerships
Four partnerships are on record, tiered core and minor.
- Roche / Chugai PharmaceuticalcoreRoche holds commercial rights for ELEVIDYS outside the United States. Chugai Pharmaceutical (Roche Group company) handles Japan commercialization with reimbursement listing and commercial launch in February 2026. First commercial sale triggered $40M milestone payment to Sarepta. Roche is also conducting additional global Phase 3 trial for European approval resubmission.
- Arrowhead PharmaceuticalscoreSarepta acquired exclusive rights to seven siRNA programs from Arrowhead Pharmaceuticals for $500 million in 2024. Programs include SRP-1001 (FSHD1) and SRP-1003 (DM1) which are now in Phase 1/2 clinical trials. Arrowhead's αvβ6 integrin-targeted delivery technology enables high muscle concentrations for siRNA therapeutics.
- Nationwide Children's Hospital (Columbus, Ohio)corePartnership for gene therapy research and manufacturing. Sarepta established Genetic Therapies Center of Excellence (GTCOE) in Columbus as part of $1.1 billion Innovation District. 25% of world's first eight gene therapy treatments originated at Nationwide Children's Hospital. Louise Rodino-Klapac was previously head of Laboratory for Gene Therapy Research at Nationwide Children's before joining Sarepta.
- Cooperative International Neuromuscular Research Group (CINRG)minorPPMD partnered with CINRG and global sponsors to advance expanded Duchenne Natural History Study (eDNHS). Study aims to gather long-term data on DMD to facilitate therapy development. Active enrollment across North America, Australia, and India.
Scale indicators7 records
Recent moves6 records
Expansion highlights6 records
Sarepta Therapeutics competitors and assessment
Company assessmentDirect peers
- Regenxbio: AAV gene therapy platform company that has been both a Sarepta licensor and patent litigant, developing DMD and other rare disease gene therapies using NAV vectors.
- PTC Therapeutics: Direct competitor in Duchenne muscular dystrophy with Translarna (ataluren) and Emflaza (deflazacort), serving the same DMD patient population with disease-modifying and symptomatic therapies.
- Solid Biosciences: Clinical-stage gene therapy developer focused on Duchenne muscular dystrophy with SGT-501, directly competing in AAV-microdystrophin approaches to DMD.
- Capricor Therapeutics: DMD-focused biotech developing CAP-1002 cell therapy for Duchenne, addressing overlapping patient segments and pursuing complementary disease-modifying mechanisms.
- Ionis Pharmaceuticals: Antisense oligonucleotide pioneer and direct competitor in exon-skipping chemistry for DMD, with overlapping ASO platform technology.
- Arrowhead Pharmaceuticals: siRNA platform company that licensed its αvβ6 integrin-targeted programs to Sarepta for $500M, providing both the underlying delivery technology and an ongoing co-development pipeline relationship.
Broad incumbents
- BioMarin Pharmaceutical: Established rare disease biopharma with significant gene therapy experience (Roctavian for hemophilia A), competing in the broader rare neuromuscular and AAV gene therapy space.
- Alnylam Pharmaceuticals: siRNA platform leader and category-defining company; Sarepta's αvβ6 integrin delivery tech is licensed from Arrowhead, making Alnylam the closest RNA-interference analog for platform comparison.
- Vertex Pharmaceuticals: Large-cap rare disease biopharma with a precision medicine and genetic therapy focus (including Casgevy in sickle cell/beta-thalassemia), representing a benchmark for scaled rare-disease commercial execution.
Emerging players
- uniQure: Gene therapy company developing AMT-130 for Huntington's disease, directly competing with Sarepta's SRP-1005 program in the same indication.
Market position
Strengths4 records
Weaknesses5 records
Competitive moat7 records
Key risks7 records
Key highlights7 records
Customer concentration
Sarepta Therapeutics social profiles
Digital presenceSarepta Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Sarepta Therapeutics leadership team
Management profileNumber of profiles
Profiles20 records
Sarepta Therapeutics subsidiaries and ownership
Company hierarchySubsidiaries1 record
Sarepta Therapeutics funding detail
Funding detailFunding overview
Funding rounds11 records
Investors12 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Sarepta Therapeutics M&A and investment
M&A and investmentM&A3 records
Investments6 records
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Sarepta Therapeutics
What does Sarepta Therapeutics do?
Sarepta Therapeutics is a precision genetic medicine company that discovers, develops, and commercializes therapies for rare neuromuscular diseases. The company has four FDA-approved products for Duchenne muscular dystrophy (DMD): ELEVIDYS gene therapy, and three antisense oligonucleotide exon-skipping therapies (EXONDYS 51, AMONDYS 45, VYONDYS 53). It is also advancing investigational siRNA programs for facioscapulohumeral muscular dystrophy type 1 (FSHD1), myotonic dystrophy type 1 (DM1), and Huntington's disease.
Is Sarepta Therapeutics a public or private company?
Sarepta Therapeutics is a public company. It is classified as public and is currently operating.
When was Sarepta Therapeutics founded?
Sarepta Therapeutics was founded in 1987. It employs 501 to 1,000 people.
Where is Sarepta Therapeutics based?
Sarepta Therapeutics is headquartered in Cambridge, United States, in the North America region.
How does Sarepta Therapeutics make money?
Two revenue lines are on record. Product Sales are the primary driver. The others are collaboration Revenue.
Who are Sarepta Therapeutics's main competitors?
Direct peers on record are Regenxbio, PTC Therapeutics, Solid Biosciences, Capricor Therapeutics, Ionis Pharmaceuticals and Arrowhead Pharmaceuticals. Broad incumbents are BioMarin Pharmaceutical, Alnylam Pharmaceuticals and Vertex Pharmaceuticals. uniQure is listed as an emerging player.
Does Sarepta Therapeutics have an API?
No public API is recorded for Sarepta Therapeutics.
What industry is Sarepta Therapeutics in?
Sarepta Therapeutics's product category is Rare Disease Genetic Medicine. Its primary akta.pro industry code is HLAIAIAA, Genetic & Genomic Rare Disease Therapeutics, with a secondary code of HLAAACAH, Neurology/CNS Gene Therapies. Its NAICS code is 325414 and its SIC code is 2836.