GNT0008

GNT0008 is an adeno-associated virus (AAV) vector-based gene therapy being developed by Genethon for the treatment of Calpainopathy, also known as Limb-Girdle Muscular Dystrophy Recessive 1 (LGMDR1) or LGMD2A. This condition is caused by mutations in the CAPN3 gene, which leads to a deficiency in the calpain-3 protein, resulting in progressive muscle weakness. GNT0008 is designed to deliver a functional copy of the CAPN3 gene to skeletal muscle cells to restore calpain-3 protein levels and address the underlying cause of the disease. The therapy is currently in preclinical development, with the goal of providing a systemic treatment for patients affected by this common LGMD subtype.

Other names

Genethon CAPN3 gene therapyAAV-CAPN3 gene therapyAAV-CAPN-3 gene therapyAAV-CAPN 3 gene therapy