SAREPTA THERAPEUTICS INC (SRPT)
Currency USD
Company Overview
Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company that focuses on the discovery and development of RNA-based therapeutics targeting rare and infectious diseases. It concentrates on the development of exon-skipping drug candidates targeting Duchenne muscular dystrophy (DMD), a rare genetic disorder affecting children. The company received accelerated approval for Exondys 51, Amondys 45 (casimersen) and Vyondys 53 (golodirsen). Exondys 51 is Sarepta's first PMO-based technology to be granted accelerated approval by the FDA. The company is conducting a confirmatory study on the clinical benefits of these drugs for gaining continued approval. The rest of its exon-skipping DMD pipeline includes a PPMO-based, exon 51 skipping candidate, SRP-5051. Sarepta is also developing gene therapies for treating DMD, Limb-girdle muscular dystrophy and central nervous system disorders including Mucopolysaccharidosis type IIIA and Pompe Disease.
Contact Details
- Address
- 215 FIRST STREET SUITE 415
- City
- CAMBRIDGE
- State
- MA
- Country
- USA
- Zip Code
- 02142
- Phone Number
- 617-274-4000
- Website
- -
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Latest Analysis
View MoreSRPT Stock: Sarepta's Elevidys Gets Green Light for Duchenne Patients
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