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 cure 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.
Resources for Patients & Caregivers
in Response to COVID-19
Celebrating Our Sarepta Team and Extended Family
Behind the exceptional group of individuals who make up our Sarepta team is a support system of partners, parents, siblings, spouses, children and roommates. This group’s incredible support has been is more apparent and meaningful now than ever. Several teammates let us into their homes virtually to share what work and life has been like during the pandemic.
Spotlight on: Development Programs
Exploring the Potential of Gene Therapies for Limb-girdle Muscular Dystrophies
Dr. Louise Rodino-Klapac, discusses the science of limb-girdle muscular dystrophies (LGMDs) and how our gene therapy engine will help engineer potential therapies for this group of inherited neuromuscular diseases.
RNA-Targeted Therapies: A Development Strategy for Protein Upregulation
Learn more about the biochemical structure of phosphodiamidate morpholino oligomers (PMOs) and PPMOs and their mechanism.