The Waltham, MA-based biotech develops therapies for genetically driven disorders using its FORCE platform to overcome the limitations of muscle tissue delivery and advance oligonucleotide therapeutics for muscle diseases. Top pipeline programs are myotonic dystrophy, Duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy.
2020 Financials (6 mo.): Operating Expenses: $16.5M (+251%); Net Loss: ($16.9M) (-369%); Cash Burn: ($14.6M) (-204%).
Potential competitors: Sarepta Therapeutics (NASDAQ:SRPT), NS Pharma (OTC:NPNKF), Pfizer (NYSE:PFE), Capricor Therapeutics (NASDAQ:CAPR), Catabasis Pharmaceuticals (NASDAQ:CATB), Solid Biosciences (NASDAQ:SLDB), PTC Therapeutics (NASDAQ:PTCT), Avidity Biosciences (NASDAQ:RNA), Vertex Pharmaceuticals (NASDAQ:VRTX), Fulcrum Therapeutics (NASDAQ:FULC)
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