VTX-801

VTX-801 is a novel, investigational gene therapy designed for the treatment of Wilson disease, a rare genetic disorder caused by mutations in the ATP7B gene that impair copper metabolism and lead to severe hepatic and neurological complications. The therapy uses a recombinant adeno-associated virus (rAAV) vector to deliver a functional, miniaturized ATP7B transgene into liver cells. This approach aims to restore copper homeostasis, reverse liver pathology, and reduce copper accumulation in affected tissues. VTX-801 is administered as a single intravenous infusion and has shown promising preclinical results in restoring physiological copper metabolism in animal models. It is being developed by Vivet Therapeutics in collaboration with Pfizer and has received Orphan Drug Designation from both the FDA and European Commission as well as Fast Track designation from the FDA[1][3][4][5][6].