Find treatments for rare disease


After successful tests in mice, scientists from Cima University of Navarra, Pamplona, Spain, have developed a gene therapy for a rare and potentially life-threatening liver disease.

Due to an inherited gene (ATP7B) defect, Wilson’s disease (WD) causes an accumulation of toxic copper levels in the body, mainly in the liver and brain.

The university, working with the biotech company Vivet Therapeutics, France, and in partnership with the pharmaceutical company Pfizer, developed a miniaturised version of the ATP7B gene – called VTX-801 - that aims to reduce the levels of copper.

The gene therapy has now been approved by the U.S. Food and Drug Administration for evaluation in patients in a clinical trial starting this year.

“The use of a Wilson’s disease mouse model that displays the main characteristics of human liver disease has been instrumental for the development of this Wilson’s Disease gene therapy.”

Gloria Gonzalez-Aseguinolaza, Deputy Director of the Center of applied Medical Research (CIMA) of the University of Navarra.

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