MWAV201

MWAV201 is an investigational adeno-associated virus (AAV) gene therapy being developed for the treatment of Wilson disease, a rare autosomal recessive disorder characterized by the toxic accumulation of copper in the liver, brain, and other organs. The therapy is designed to deliver a functional copy of the human *ATP7B* gene to hepatocytes using an AAV vector. Wilson disease is caused by mutations in the *ATP7B* gene, which encodes the copper-transporting ATPase 2 protein responsible for biliary copper excretion and the incorporation of copper into ceruloplasmin. By restoring the expression of a functional ATP7B protein, MWAV201 aims to normalize copper homeostasis and prevent the progressive tissue damage associated with the disease. It is typically administered as a single, peripheral intravenous infusion.