2023-12-07 08:56:41 ET
- Solid Biosciences ( NASDAQ: SLDB ) rose about 6% in premarket trading as the company announced the receipt of Fast Track designation for the U.S. FDA for SGT-003, its Duchenne muscular dystrophy gene therapy candidate.
- The Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.
- Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age.
- SLDB's SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding a shortened form of the dystrophin protein (microdystrophin), containing the R16-R17 nNOS binding domain.
- Source: Press Release
For further details see:
Solid Biosciences receives FDA fast track designation for Duchenne gene therapy candidate