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Gene Therapy
Gene Therapy (VTX-801) for Wilson's Disease
Recruiting1 awardPhase 1 & 2
Dallas, Texas
This trial is testing VTX-801, a gene therapy, in adults with Wilson's Disease. The treatment involves adding new genes to help the body control copper levels. Researchers want to see if it is safe and effective over several years. VTX-801 is a mini version of the human ATP7B gene that has shown long-term correction of copper metabolism in animal studies.
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