isaralgagene civaparvovec

Isaralgagene civaparvovec is an investigational gene therapy for Fabry disease. It uses a recombinant adeno-associated virus (rAAV) 2/6 vector to deliver a functional copy of the human *GLA* gene to liver cells. This enables continuous production of alpha-galactosidase A (α-Gal A), the enzyme deficient in Fabry disease, thereby reducing toxic accumulation of globotriaosylceramide (Gb3) and related symptoms. The therapy is administered as a single intravenous infusion and aims to provide durable benefit with one treatment, potentially eliminating the need for ongoing enzyme replacement therapy. Developed by Sangamo Therapeutics, it has received Fast Track designation from the FDA and other regulatory designations in Europe and the UK[1][2][4][5][7].

Other names

isaralgagene civaparvovec