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Vivet : FDA Authorizes To Proceed With Phase 1/2 Study On Gene Therapy For Wilson Disease

Vivet Therapeutics, a privately held gene therapy biotech company, and Pfizer Inc. (PFE) said Wednesday that the U.S. Food and Drug Administration has cleared Vivet's Investigational New Drug or IND application for the GATEWAY study for the potential treatment of Wilson disease, a rare and potentially life-threatening liver disorder.

The study is a phase 1/2 clinical trial that will evaluate Vivet's proprietary, investigational gene therapy vector, VTX-801. Vivet expects to enroll the first patient in early 2021.

VTX-801 is a novel, investigational rAAV-based gene therapy vector designed to deliver a miniaturized ATP7B transgene encoding, a functional protein that has been shown to restore copper homeostasis, reverse liver pathology and reduce copper accumulation in the brain of a mouse model of Wilson disease.

The GATEWAY trial will measure relevant biomarkers to evaluate physiological restoration of copper elimination and transport in patients.

In March 2019, Pfizer acquired a minority equity interest in Vivet and secured an exclusive option to acquire all outstanding shares.

In September 2020, Vivet and Pfizer signed an agreement for the manufacture by Pfizer of the VTX-801 vector for the GATEWAY study.

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