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.
On October 1, 2020, Solid Biosciences Inc. shared that the FDA has lifted the Clinical Hold on the IGNITE DMD Clinical Trial.
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