SRPT 📈 Sarepta Therapeutics - Overview

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company that has been at the forefront of developing innovative RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities. The company's primary focus is on treating rare diseases, and its product portfolio reflects this commitment. For instance, EXONDYS 51 injection is designed to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the dystrophin gene that can be addressed through exon 51 skipping. This approach has shown promise in helping patients with this debilitating disease. Additionally, the company offers VYONDYS 53 for DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 53 skipping, as well as AMONDYS 45 for those with a confirmed mutation of the dystrophin gene.

The company's research and development pipeline is robust, with several promising candidates in various stages of development. One notable example is ELEVIDYS, an adeno-associated virus-based gene therapy designed to treat ambulatory pediatric patients aged 4 through 5 years with DMD and a confirmed mutation in the dystrophin gene. This gene therapy has the potential to revolutionize the treatment of DMD by addressing the root cause of the disease. Furthermore, Sarepta Therapeutics is developing SRP-5051, a peptide-conjugated phosphorodiamidate morpholino oligomer (PMO) that binds to exon 51 of dystrophin pre-mRNA, as well as SRP-9003, a limb-girdle muscular dystrophies gene therapy program. These initiatives demonstrate the company's dedication to advancing the field of genetic therapies and improving the lives of patients with rare diseases.

Sarepta Therapeutics has established a strong network of collaborations and license agreements with prominent institutions and companies, including F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Genevant Sciences, and Duke University, among others. These partnerships enable the company to leverage the expertise and resources of its collaborators, accelerating the development of its therapeutic candidates. With a rich history dating back to 1980, Sarepta Therapeutics is headquartered in Cambridge, Massachusetts, and is listed on the NASDAQ stock exchange under the ticker symbol SRPT. The company's common stock is classified under the GICS Sub Industry: Biotechnology, reflecting its focus on developing innovative biopharmaceutical solutions.