Patient dosing continues in clinical trial of novel gene therapy for Wilson’s disease

Key takeaways:

• VTX-801 is an investigational gene therapy to treat Wilson’s disease.
• Cohort 2 dosing allowed after Independent Data Monitoring Committee approval.

A European clinical stage biotech company announced dosing of the first patient in cohort 2 of its ongoing phase 1/2 GATEWAY clinical trial of lead candidate VTX-801 to treat Wilson’s disease.

According to a release from Vivet Therapeutics, GATEWAY is a nonrandomized, open-label study conducted across several clinical sites in the United States, United Kingdom, Germany and Denmark. The trial seeks to assess the safety, tolerability and pharmacological activity of a single IV infusion of VTX-801, a novel, investigational gene therapy, at increasing dose levels in adult patients with Wilson’s disease.

Initiation of cohort 2 followed the successful completion of cohort 1 and subsequent approval from an independent data monitoring committee to proceed, the company said.

“We are encouraged by the initial safety, histological and pharmacodynamic data that VTX-801 has demonstrated to date, and we are pleased that the first patient has been successfully dosed in Cohort 2 in the GATEWAY clinical trial,” Jean-Philippe Combal, CEO at Vivet Therapeutics, stated in the release. “2024 is shaping up to be a transformative year for Vivet and its gene therapy platform and we look forward to providing further data on our GATEWAY clinical trial at upcoming scientific conferences.”