inlezifigene civaparvovec

Inlezifigene civaparvovec (formerly known as isaralgagene civaparvovec or ST-920) is an investigational liver-tropic gene therapy designed for the treatment of Fabry disease. It utilizes a recombinant adeno-associated virus serotype 2/6 (AAV2/6) vector to deliver a functional copy of the human alpha-galactosidase A (GLA) gene. Following a single intravenous infusion, the vector targets hepatocytes, which then serve as a "biofactory" to continuously produce and secrete the alpha-galactosidase A enzyme into the bloodstream. This approach aims to provide stable, long-term enzyme levels, potentially eliminating the need for bi-weekly enzyme replacement therapy (ERT) and reducing the accumulation of disease substrates such as globotriaosylceramide (Gb3) and lyso-Gb3 in tissues. The therapy is currently being evaluated in the registrational Phase 1/2 STAAR study.

Other names

isaralgagene civaparvovec