Quick Read
- Sarepta Therapeutics is laying off 36% of its workforce, affecting 500 employees, to save $400 million annually.
- The layoffs follow safety concerns with Elevidys, a gene therapy for Duchenne muscular dystrophy, linked to two patient deaths.
- The company is pivoting its research focus from gene therapies to RNA-based treatments using its siRNA platform.
- Sarepta reported $513 million in total revenue for Q2 2025 but suspended its financial guidance amid ongoing challenges.
- Leadership changes were announced, including Ian Estepan as COO and Louise Rodino-Klapac as President of R&D.
Sarepta Therapeutics, a biotechnology company specializing in treatments for rare diseases, has announced a significant restructuring plan, affecting 36% of its workforce and pivoting its research priorities. The company aims to address financial pressures and operational challenges, particularly those linked to its flagship gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD).
Massive Layoffs and Strategic Restructuring
On July 16, 2025, Sarepta Therapeutics revealed that it would lay off approximately 500 employees, or 36% of its workforce. This move is part of a broader effort to save $400 million annually and ensure the company’s long-term viability. According to Stat News, these layoffs come amidst a challenging year, with the company’s stock price plummeting nearly 88% and trading at its 52-week low of $16.88. The workforce reduction is expected to save $120 million annually, while an additional $300 million will come from reprioritizing research efforts.
CEO Doug Ingram stated, “Faced with environmental changes, we have decided to act decisively, implementing a focused strategy to ensure Sarepta remains a vibrant, financially enduring, patient-centric organization.” The company’s revised strategy aims to meet its 2027 financial obligations while preserving its ability to develop innovative therapies for rare diseases.
Elevidys Safety Concerns and FDA Scrutiny
The restructuring follows a series of setbacks involving Elevidys, the first gene therapy approved in the U.S. for DMD. The therapy’s rollout faced significant challenges after two teenage patients died from acute liver failure within two months of treatment. These incidents prompted the U.S. Food and Drug Administration (FDA) to request a black box warning for Elevidys, highlighting the risk of acute liver injury.
In response, Sarepta halted Elevidys administration to non-ambulatory DMD patients, who are deemed at higher risk. The company also paused a phase 3 trial, ENVISION, and is working on a new protocol with enhanced immunosuppressive measures to manage liver toxicity. According to Fierce Biotech, Sarepta plans to submit this updated protocol to the FDA in the coming months.
Despite these challenges, Elevidys continues to generate significant revenue. Sarepta reported $282 million in Elevidys sales during the second quarter of 2025, although this represents a decline from $375 million in the previous quarter. The company projects annualized sales of $500 million through 2027 for ambulatory DMD patients, who remain eligible for the therapy.
Research and Development Shift
As part of its restructuring, Sarepta is pivoting its research focus from gene therapies for limb-girdle muscular dystrophies (LGMD) to RNA-based treatments, particularly those utilizing its siRNA platform. The company believes this approach offers “tremendous near-term potential” and is concentrating on conditions like myotonic dystrophy type 1, Huntington’s disease, and facioscapulohumeral muscular dystrophy.
Sarepta’s collaboration with Arrowhead Pharmaceuticals to develop skeletal muscle disease therapies will continue. The company also plans to seek strategic partnerships for deprioritized programs, including some LGMD gene therapies. According to Investing.com, Sarepta aims to submit a Biologics License Application for SRP-9003, a gene therapy for LGMD type 2E/R4, later this year.
Financial and Leadership Adjustments
Sarepta’s financial outlook remains a key concern. The company has suspended its 2025 revenue guidance of $2.3–$2.6 billion and faces $1 billion in senior notes due in 2027. However, Sarepta remains profitable, reporting $513 million in total net product revenue for the second quarter of 2025, with $231 million coming from its RNA-based therapies. The company held $850 million in cash and investments as of June 30, 2025.
Alongside these financial measures, Sarepta announced several leadership changes. Ian Estepan has been promoted to Chief Operating Officer, while Louise Rodino-Klapac has been named President of Research & Development and Technical Operations. These appointments are intended to strengthen Sarepta’s leadership team during this transitional period.
Industry Implications and Future Outlook
Sarepta’s restructuring reflects broader trends in the biotech industry, including declining interest in gene therapies and rising workforce reductions. The company’s challenges with Elevidys underscore the complexities of developing and commercializing advanced therapies, particularly for rare diseases.
Despite these hurdles, Sarepta remains committed to its mission of delivering transformative treatments. The company’s pivot to RNA-based therapies and its continued investment in high-impact programs signal a focused approach to navigating its current challenges and securing long-term growth.
Sarepta’s journey highlights both the promise and the perils of innovation in genetic medicine. As the company adapts to its evolving landscape, it remains to be seen how its strategic decisions will shape its future and impact the lives of patients worldwide.