This clinical trial is focused on studying Wilson’s Disease, a rare genetic disorder that affects how the body processes copper. The study involves a new treatment called VTX-801, which is a type of gene therapy. Gene therapy is a method that uses genes to treat or prevent diseases. In this case, VTX-801 is designed to help the body manage copper levels better by using a special viral vector to deliver a shortened form of the human gene ATP7B, which is important for copper regulation.

The purpose of the study is to assess the safety and tolerability of VTX-801 when given to adult patients with Wilson’s Disease. The study will involve a single dose of the treatment, administered through an intravenous infusion, which means it is given directly into a vein. Participants will be monitored over a period of five years to observe how they respond to the treatment and to ensure it is safe. The study will also involve the use of 64Cu, a form of copper, to help track how the body processes copper during the trial.

Throughout the study, various health checks will be conducted, including clinical examinations and tests like ECG (a test that records the electrical activity of the heart) and MRI (a type of scan that uses magnetic fields to create detailed images of the body). These checks will help researchers understand the effects of the treatment on the participants’ health. The study aims to provide valuable information that could lead to better treatment options for people with Wilson’s Disease in the future.