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Main focus: Gene therapies and gene editing for the treatment of muscular dystrophy
Company stage: Clinical
Diseases: Duchenne Muscular Dystrophy (DMD)
Genome editing tool: CRISPR-Cas9
Funding stage: Public (NASDAQ:SRPT)
Location: Cambridge, MA, USA
Partnerships: Genevant Sciences, GenEdit
Sarepta Therapeutics is a clinical stage, publicly traded biotech company. The company focuses on many biotherapeutic areas, some of which include gene editing. Sarepta is using CRISPR-Cas9 as a tool for treating Duchenne muscular dystrophy. It has a collaboration with Genevant Sciences for the delivery of CRISPR constructs and collaborations with Duke University and Harvard University to develop gene-editing methods.