Sarepta Therapeutics, Inc. (SRPT) is an American biopharmaceutical corporation which is focused on the discovery and development of unique RNA-targeted therapeutics, gene therapy, and other genetic approaches for the treatment of rare diseases. Headquartered in Cambridge, Massachusetts, Sarepta is at the forefront of precision genetic medicine, using innovative technology to develop treatments for conditions that have no current effective therapy.
Founded in 1980, Sarepta originally began under the name AntiVirals, Inc., with a primary focus on an antiviral research program. Over time, the company shifted its focus into developing a pipeline of treatments for people suffering from rare and life-threatening genetic diseases. The company's most notable achievement is the FDA approval of its flagship product, EXONDYS 51 (eteplirsen), the first FDA-approved treatment for Duchenne muscular dystrophy (DMD), and a host of other potential treatments currently in various stages of clinical trials.
Despite the challenges that come with developing treatments for rare diseases, Sarepta has proven its determination and commitment by continuing to deliver important medical breakthroughs. The company places a great emphasis on patient-centered drug discovery, aiming to give hope to patients and their families who are often left with very few options.
Sarepta has a diverse and robust pipeline, with several products at various stages of development in its mission to treat debilitating genetic diseases. In recent years, a significant portion of its R&D has also been channeled into exploring the potential applications of gene therapy in treating rare neuromuscular diseases.
Sarepta’s operating principles underline the remarkable passion for helping patients, a relentless drive for progress, and scientific rigor in everything they undertake. The company's mission is driven by its motto of "herd the hope," reflecting its unwavering commitment towards pioneering treatments that ultimately serve to give hope to patients and their families.
In a nutshell, Sarepta Therapeutics, Inc. is a research-oriented pharmaceutical company that is trailblazing in the realm of genetic medicine. By leveraging cutting-edge RNA and gene therapy technologies, Sarepta has built an impressive portfolio of treatments for rare genetic disorders, reflecting its commitment to push the boundaries of genetic medicine and transform patients' lives.