SGT-001

SGT-001 is an investigational gene therapy developed for the treatment of Duchenne muscular dystrophy (DMD). It is a systemically administered adeno-associated virus serotype 9 (AAV9) vector-based gene therapy designed to deliver and express a synthetic microdystrophin gene in skeletal and cardiac muscle. The microdystrophin transgene encodes a shortened but functional version of the dystrophin protein, including the neuronal nitric oxide synthase (nNOS) binding domain, which is critical for muscle function. By restoring microdystrophin expression, SGT-001 aims to stabilize essential associated proteins and improve muscle strength and function in patients with DMD. The drug has been evaluated in Phase 1/2 clinical trials (IGNITE DMD), showing durable production of microdystrophin, improvements in motor and pulmonary function, as well as patient-reported outcomes. Adverse events have included complement activation-related reactions that resolved with medical management[1][5][8][9].

Other names

AAV9-microdystrophin-5AAV-9-microdystrophin-5AAV 9-microdystrophin-5