Wilson's disease research

Wilson's disease is an inherited disorder with an incidence of 1 case per 30,000 inhabitants. It is caused by mutation of the ATP7B gene, responsible for copper metabolism. Its mutation causes this metal to accumulate in the liver and then, through the bloodstream, it can reach other tissues, damaging the brain, kidneys and eyes.

Cima initiated a line of research on this disease in 2014 that resulted in the design and innovation by gene therapy of a viral vector, VTX-801, capable of correcting the specific cause of Wilson's disease (copper accumulation) and reversing the symptoms and pathological alterations associated with this pathology.

In 2016 Cima licensed the vector to the biotech company Vivet Therapeutics, which continues with the development of gene therapy with adeno-associated vector technologies (AAVs) for this and other diseases.

Cima is committed to continuing research into the molecular mechanisms associated with Wilson's disease.

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Our goal: to stop neuronal death

REFERENCES IN GENE THERAPY

Hepatic Diseases Gene Therapy Research Group

Reference team in the research of liver diseases, in particular those hereditary diseases that can be cured by liver transplantation. We work to understand the pathophysiology of some of these rare diseases and to develop new therapies through gene therapy and gene editing that can reach patients.

It is part of the Gene Therapy and Expression of Gene Regulation Program.