AAV9-micro-dystrophin

AAV9-micro-dystrophin is a gene therapy platform and class of investigational recombinant adeno-associated virus serotype 9 (AAV9) vector constructs designed to deliver a shortened, functional version of the dystrophin gene (micro-dystrophin) to muscle tissues. This approach is developed to treat Duchenne muscular dystrophy (DMD), a progressive and fatal neuromuscular disorder caused by mutations in the DMD gene that lead to a lack of functional dystrophin protein. Because the full-length dystrophin gene exceeds the packaging capacity of AAV vectors, these therapies utilize a truncated micro-dystrophin transgene that retains essential functional domains, such as the neuronal nitric oxide synthase (nNOS) binding domain. Several candidates have been developed under this platform, including Solid Biosciences' SGT-001 (discontinued), Pfizer's fordadistrogene movaparvovec (PF-06939926, discontinued), and Insmed's INS1201, which is currently being evaluated in Phase 1 clinical trials using an intrathecal route of administration to improve safety and biodistribution compared to systemic intravenous delivery.

Other names

AAV9-microdystrophinAAV-9-microdystrophinAAV 9-microdystrophinAAV9-µDysrAAV9-microdystrophinrAAV-9-microdystrophinrAAV 9-microdystrophinrAAV9-µDys