AAV-miniATP7B

AAV-miniATP7B (also known as VTX-801) is an investigational, liver-directed recombinant adeno-associated virus (rAAV) gene therapy developed by Vivet Therapeutics, in collaboration with Pfizer, for the treatment of Wilson's disease. Wilson's disease is a rare, autosomal recessive genetic disorder caused by mutations in the ATP7B gene, which encodes a copper-transporting ATPase, leading to toxic copper accumulation in the liver and central nervous system. AAV-miniATP7B utilizes a hepatotropic AAV capsid to deliver a codon-optimized, shortened version of the human ATP7B gene (miniATP7B) that lacks four of the six metal-binding domains, allowing it to fit within the packaging capacity of the AAV vector. Once inside hepatocytes, the transgene is expressed to restore functional copper transport and biliary excretion. Clinical development of VTX-801 in the Phase 1/2 GATEWAY trial was terminated by Vivet Therapeutics in October 2024 due to insufficient efficacy at the tested doses and the need for additional funding to escalate to higher doses.

Other names

AAV-miniATP7BAAV-miniATP-7BAAV-miniATP 7BAAV8-miniATP7BAAV-8-miniATP7BAAV 8-miniATP7BAAVAnc80-miniATP7BAAVAnc-80-miniATP7BAAVAnc 80-miniATP7B