Vivet Therapeutics’ Wilson Disease Gene Therapy Clinical Trial Update

On behalf of the Vivet Therapeutics Wilson disease study team, we are happy to share updates about the ongoing and enrolling GATEWAY clinical trial.

GATEWAY is an international gene therapy clinical trial for people living with Wilson disease assessing the safety, efficacy and durability (how long-lasting) of study medication VTX-801.

What is VTX-801? In people living with Wilson disease, the ATP7B gene – normally active in the liver – is malfunctioning. VTX-801 is an investigational gene therapy containing a corrective version of the ATP7B gene; this gene is packaged within a vector, which is the empty (non-infectious) shell of a virus engineered to deliver the corrective gene into liver cells via the blood. The treatment is administered as a single intravenous infusion.

Where is this study taking place? There are now ten participating sites open across the United States and Europe: 6 sites in the U.S. (California, Connecticut, Florida, Michigan, Texas and North Carolina) and 4 sites in Europe (Denmark, Germany and the UK). For eligible patients, assistance with travel and study-related expenses will be available.

Who is sponsoring the study? Vivet Therapeutics is sponsoring the GATEWAY clinical trial. Vivet is dedicated to the development of innovative therapies for people affected by rare liver disorders such as Wilson disease.

Visit www.gatewaytrialwilsondisease.com/ for more information about eligibility and clinical trial sites.