Sarepta Therapeutics Announces Initiation of EMBARK, Study of SRP-9001, a Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
October 04, 2021 at 04:30 pm IST
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Sarepta Therapeutics, Inc. announced the initiation, in partnership with Roche, in the U.S. and countries around the world, of study SRP-9001-301, also known as EMBARK, a pivotal study of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) for the treatment of Duchenne muscular dystrophy. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.
Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company. The Company is focused on helping patients through the discovery and development of ribonucleic acid (RNA)-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. It has developed multiple approved products for the treatment of Duchenne muscular dystrophy (Duchenne) and is developing potential therapeutic candidates for a range of diseases and disorders, including Duchenne, Limb-girdle muscular dystrophies (LGMDs) and other neuromuscular and central nervous system (CNS) related disorders. It has developed and commercialized four approved products for the treatment of Duchenne: EXONDYS 51 (eteplirsen), Injection (EXONDYS 51), VYONDYS 53 (golodirsen) Injection (VYONDYS 53), AMONDYS 45 (casimersen) Injection (AMONDYS 45), and ELEVIDYS. Its pipeline includes approximately 40 programs at various stages of discovery, pre-clinical and clinical development.