Simone Spuler, MD
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Company type: Therapeutics
Main focus: Engineered muscle satellite cells
Company stage: Clinical
Diseases: Muscular dystrophies
Genome-editing tool: CRISPR mRNA
Funding stage: Seeding
Location: Berlin, Germany
MyoPax is a clinical-stage company focused on the isolation and engineering of primary human satellite cells (PHSats). CRISPR-Cas-engineered PHSats are deployed to treat muscle impairment that can arise through congenital malformation, trauma, hereditary muscular dystrophies, and acute and/or critical conditions including cancer and aging. The company's pipeline contains two clinical-stage programmes for the treatment of Exstrophy-Epispadias-Complex (EEC) and Limb Girdle Muscular Dystrophies (LGMD).