Some see slow and steady scientific progress.
We see a revolution.
Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases.
We’re ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the world’s largest gene therapy manufacturing capacity, and rethinking access and reimbursement models for revolutionary new treatments. We are in a daily race to transform genetic understanding into genetic medicine. Because every day is an opportunity to change the lives of people living with rare disease.
Some think a treatment for a rare disease is a miracle.
Our scientists would disagree.
Sarepta is engineering solutions for rare diseases with science that is on the forefront of precision genetic medicine. Our platforms include: gene therapy, RNA technologies, and gene editing. We are in a daily race to save lives stolen or impacted by rare disease.
Patients can’t wait for the next breakthrough in medical research.
So neither will we.
While there may be as many as 7,000 rare diseases, only a small percentage have treatments. That is why we are relentless in our dedication: Leverage the best science to help as many patients as possible. Today, we are doing just that in Duchenne muscular dystrophy, in six forms of limb-girdle muscular dystrophy, in Charcot-Marie-Tooth disease, and in Mucopolysaccharidosis type IIIA (MPS IIIA), among others. Sarepta will always follow the science and continuously evaluate other diseases and modalities to pursue.
Who We Are
Sarepta is a global biotechnology company on an urgent mission: to engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We’re ushering in a new era of drug development with the goal of driving efficiencies, including shortening the time from lab to patient and building the world’s largest gene therapy manufacturing capacity. We’re collaborating with health networks and payers, rethinking pricing models for revolutionary new treatments in development. We are in a daily race to transform genetic understanding into genetic medicine. Because every day is an opportunity to save lives stolen by rare disease.
Armed with the most advanced science in genetic medicine, we are in a daily race to rescue lives otherwise stolen by rare disease. At Sarepta, every day is another twenty-four hours to stand up for patients, advance technology, challenge convention, and drag tomorrow into today.
Sarepta is pursuing the development of precision genetic medicine at the forefront of biotechnology for rare diseases: gene therapy, RNA-targeted exon skipping, and gene editing. And we’re constantly looking for new ways to tackle rare genetic diseases, which include developing drugs faster with more predictability, differentiated manufacturing processes, and novel reimbursement models.
Sarepta Clinical Trials
Sarepta is committed to creating breakthrough treatments for people whose lives are being cut short and burdened by rare diseases. Fundamental to that mission are carefully designed and conducted clinical trials.
Sarepta is at the forefront of precision genetic medicine, with over 40 therapies in various stages of development. The company’s programs and research focus span several therapeutic modalities, including gene therapy, RNA technologies, and gene editing.
The groups below represent a few of the organizations that help connect patients and families to education, research, support services, and the broader muscular dystrophy community.
Duchenne Muscular Dystrophy Resources
Sarepta International UK Ltd.
Hill House, 1 Little New Street
London, EC4A 3TR United Kingdom
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- Registered number: Company No. 10987747
- Place of registration: England and Wales
- Registered office address:
9 Greyfriars Road
RG1 1NU United Kingdom
- Tax ID Number: 37612 11842
- VAT number of business: 292 5942 68
Please note we are no longer using the Hill House address. All mail should be sent to the 9 Greyfriar Road address.