SGT-001 (Microdystrophin Gene Transfer)
Duchenne muscular dystrophy (DMD)
Stage (next event)
Phase 1/2 (Data)
Catalyst Info & Data Links
TITLE: SGT-001 for Duchenne muscular dystrophy (DMD) - Phase 1/2 Data
ClinicalTrial.gov (NCT03368742): Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)
WHAT IS THE CATALYST EVENT?
Phase 1/2 Data
WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?
Mechanism of Action
MECHANISM OF ACTION
Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy (Duchenne). Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.
SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, in the United States and Orphan Drug Designations in both the United States and European Union.
Updated by HC
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