- Cambridge, MA-based Solid Biosciences (Pending:SLDB) has filed a preliminary prospectus for a $100M IPO.
- The life sciences firm says its mission is to cure Duchenne muscular dystrophy (DMD). Lead candidate is SGT-001, a gene transfer that restores functional dystrophin protein expression in muscles by delivering a synthetic transgene that produces dystrophin-like protein. The delivery vector is an adeno-associated virus (AAV) that contains a muscle-specific promoter.
- SGT-001 has Rare Pediatric Disease and Orphan Drug designations in the U.S. A Phase 1/2 study, IGNITE DMD, was initiated last quarter. According to ClinicalTrials.gov, the estimated primary completion date is March 2020.
- 2017 Financials (9 mo.)($M): Operating Expenses: 39.7 (+134.9%); Net Loss: (39.7) (-134.9%); Cash Burn: (29.2) (-101.4%).
- DMD-related tickers: SRPT BIIB CAPR WVE PTCT CATB SMMT BMRN
- Now read: Another Threat For Biogen's MS Franchise
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