Vivet's Gene Therapy for Wilson Disease

Gene therapy is designed to bring the corrective gene into sick cells. An engineered viral vector transfers the corrective gene into the cells through the bloodstream but is not infectious. The goal with gene therapy for Wilson disease is to restore - possibly permanently - excess copper elimination so the patient could withdraw from chelation and/or zinc therapy. The FDA approved human trials to begin in 2021 that are designed to test for safety and to determine if it works to correct the genetic defect. If the gene therapy works it could prevent disease complications, improve existing symptoms and quality of life.
Dr. Bénichou currently holds the position of Chief Medical Officer at Vivet therapeutics SAS. He has been working on rare inherited human disorders since 1989. He spent the last 25 years in the Clinical research department of different biotech and pharma companies, including 14 years at Sanofi Genzyme (formerly Genzyme Corporation), where he was instrumental in the development of treatments for several lysosomal storage disorders and other rare genetic diseases. Prior to joining the industry, he spent close to 10 years in the academic environment and has been involved in gene mapping, identification and study for various inherited human disorders. Dr Bénichou received his M.D. and his Ph.D in Microbiology from the Paris VI and VII Universities, France