SRPT
- Sarepta Therapeutics, Inc.
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Overview
Company Summary
Sarepta Therapeutics, Inc. is a biopharmaceutical company that focuses on the discovery, development, and commercialization of innovative RNA-targeted therapeutics. The company primarily focuses on treating rare neuromuscular diseases.
Sarepta Therapeutics is known for its pioneering work in the field of genetic medicine, particularly with its expertise in RNA-based technologies. The company's core technology platform is called phosphorodiamidate morpholino oligomer (PMO), which is designed to modify the messenger RNA (mRNA) involved in various genetic diseases.
By targeting and modifying specific mRNA sequences, Sarepta develops therapies that aim to address the underlying cause of genetic diseases. Using its proprietary PMO technology, the company encourages the production of functional proteins, replaces absent or defective proteins, or adjusts protein expression levels.
Sarepta's most well-known product is called Exondys 51 (eteplirsen), the first personalized medicine approved for the treatment of Duchenne muscular dystrophy (DMD) in the United States. DMD is a rare and devastating genetic disorder that primarily affects young boys, causing progressive muscle degeneration.
In addition to Exondys 51, Sarepta Therapeutics has a robust pipeline of potential therapies targeting various genetic diseases, including other forms of muscular dystrophy, rare genetic conditions, and diseases related to specific mutations within RNA. They aim to leverage their expertise in RNA technology to develop therapeutic solutions that address unmet medical needs across a range of rare diseases.
Overall, Sarepta Therapeutics is dedicated to advancing the field of genetic medicine and developing innovative treatments for rare neuromuscular diseases by targeting and manipulating RNA to potentially restore protein function and improve patients' lives.