SGT-003

SGT-003 is an investigational gene therapy developed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes a next-generation adeno-associated virus (AAV) vector, specifically the proprietary AAV-SLB101 capsid, to deliver a transgene encoding a differentiated microdystrophin protein. This microdystrophin includes R16 and R17 domains that enable binding to neuronal nitric oxide synthase (nNOS), which is important for normal muscle function. The therapy is designed to provide skeletal muscle tropism, enhanced durability, and improved clinical outcomes by efficiently delivering the genetic payload specifically to muscle cells. Clinical data from early-phase trials have shown significant expression of microdystrophin in treated patients and reductions in biomarkers associated with muscle injury. The therapy has received orphan drug designation, fast track designation, and rare pediatric disease designation from the FDA[1][3][5][6][7].

Brand names

SGT-003SGT003SGT 003

Other names

SGT-003SGT003SGT 003