NASDAQ

SAREPTA THERAPEUTICS INC (SRPT)

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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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