Manufacturing Agreement for Wilson Disease Gene Therapy

According to an article from BioSpace, Vivet Therapeutics and Pfizer have entered into a manufacturing agreement to develop Vivet’s gene therapy, which treats Wilson disease. The terms of the agreement state that Pfizer is to provide clinical supply for a clinical trial of the therapy.

 About Wilson Disease

Wilson disease is characterized by the body’s inability to metabolize and excrete copper, leading to a build up in the brain, liver, and other organs. Symptoms typically appear when one is a teenager, and they include jaundice, trouble walking, abdominal swelling, fluid accumulation in the extremities, easy bruising, trouble swallowing, and a low white blood cell count. Neurological problems arise when there is copper build up in the brain, and hepatitis occurs when it accumulates in the liver. A brown ring may also appear around the iris. It is integral to seek treatment for this condition, as it can cause organ damage and be fatal. Treatment consists of chelation therapy drugs, although these often bring severe side effects. Doctors also recommend a diet that is low in copper and includes zinc supplements.

About the Agreement

Pfizer is collaborating with Vivet Therapeutics on their gene therapy for Wilson disease, VTX-801. The two companies have agreed that Pfizer will provide clinical supply for the upcoming Phase 1/2 trial, which is expected to begin in 2021. Before the trial, they will file for an Investigational New Drug (IND) application.

Both companies have released statements on their excitement to work together and the need for a Wilson disease gene therapy. They hope that their agreement will allow for a treatment that will better the lives of affected individuals.

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