SGT-003 is an adeno-associated virus (AAV) mediated gene transfer that contains an engineered version of the dystrophin gene (microdystrophin). SGT-003 uses a novel AAV capsid designed by Solid Biosciences. The microdystrophin gene in SGT-003 has been designed to produce a functional form of dystrophin protein containing the nNos domain in skeletal and cardiac muscles.
A Phase 1/2 trial is currently recruiting.
This program is sponsored by Solid Biosciences.
RECRUITING | A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) |
