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.
|Microdystrophin Gene Transfer Study in Adolescents and Children With DMD|
Webinar: Solid Biosciences Presents Safety & Efficacy Update
On 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.
Solid BioSciences Presents at the PPMD 2019 Annual Conference
Solid BioSciences Presents at the PPMD 2018 Annual Conference