AT-GTX-701

AT-GTX-701 is an investigational gene therapy based on an adeno-associated virus (AAV) vector designed for the treatment of Fabry disease. It utilizes an engineered human alpha-galactosidase A (hGLA) transgene optimized for enhanced stability and substrate reduction relative to wildtype hGLA. Preclinical studies demonstrated robust reduction of pathologic substrate (lyso-Gb3/GL-3) in relevant Fabry disease tissues, including kidney, heart, and dorsal root ganglia. The therapy is a collaborative development between Amicus Therapeutics and the University of Pennsylvania, leveraging Amicus’ protein engineering expertise and Penn’s AAV vector technology. AT-GTX-701 has shown greater efficacy than wildtype in preclinical in vivo models, leading to near-complete substrate clearance at higher doses. The intent is to provide stable, long-lasting production of the functional enzyme in target tissues, thus addressing the underlying enzymatic deficiency responsible for Fabry disease[1][3][4][7][9].