Resamirigene bilparvovec

Resamirigene bilparvovec is an investigational gene therapy designed to treat X-linked myotubular myopathy (XLMTM), a severe and often fatal genetic disorder primarily affecting male infants. The disease is caused by mutations in the MTM1 gene, which encodes the protein myotubularin. Resamirigene bilparvovec consists of a functional copy of the human MTM1 gene delivered via an adeno-associated virus serotype 8 (AAV8) vector, administered as a single intravenous infusion. The therapy aims to restore myotubularin expression in skeletal muscle, potentially improving motor function and reducing ventilator dependence in affected children. Development has been led by Astellas Gene Therapies (formerly Audentes Therapeutics), with preclinical work conducted in collaboration with Genethon. As of late 2023, clinical development remains on hold following safety concerns related to liver failure observed during trials[1][4][5][6][8].

Other names

AT132AT-132AT 132