At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of development—many already in late-stage clinical trials. In many cases, development is being accelerated by our gene therapy engine, which potentially provides a more efficient model for drug design.
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.