SGT-001 is an adeno-associated virus (AAV) mediated gene transfer that contains an engineered version of the dystrophin gene (microdystrophin). The microdystrophin gene in SGT-001 has been designed to produce a functional form of dystrophin protein in skeletal and cardiac muscles.
A Phase 1/2 trial is actively recruiting.
This program is sponsored by Solid Biosciences.
ACTIVELY RECRUITING | Microdystrophin Gene Transfer Study in Adolescents and Children With DMD |
MAR 2021 | Webinar: Solid Biosciences Presents Safety & Efficacy UpdateOn March 16, 2021, Solid Biosciences joined PPMD for a webinar to provide a safety and efficacy update on the ongoing IGNITE DMD phase I/II clinical study of SGT-001 microdystrophin gene therapy in patients with Duchenne muscular dystrophy. |
JUNE 2019 | Solid BioSciences Presents at the PPMD 2019 Annual Conference |
JUNE 2018 | Solid BioSciences Presents at the PPMD 2018 Annual Conference |
