G\u00e9n\u00e9thon starts a European phase I\/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease.<\/p>\n
This European trial will include 17 patients over 10 years old, (the age when the liver reaches maturity); it aims to assess the tolerance of the product, determine the optimal dose, and assess the therapeutic efficacy of the drug candidate. The trial takes place in four investigation centres in Europe: France (Prof Labrune – H\u00f4pital B\u00e9cl\u00e8re in Clamart), Italy (Prof Brunetti-Pierri \u2013 H\u00f4pital Federico II, Prof d\u2019Antiga – Azienda Ospedaliera Papa Giovanni XXIII in Bergamo) and the Netherlands (Prof Beuers – Academic Medical Center in Amsterdam).
\nThe technology used in the trial was developed by the \u2018Immunology and Gene Therapy for Liver Disease\u2019 team at G\u00e9n\u00e9thon, headed by Dr Federico Mingozzi. In partnership with research teams in Italy and the Netherlands, G\u00e9n\u00e9thon has developed an Adeno-Associated Viral (AAV) vector that is able to transfer a copy of the UGT1A1 gene (coding for the production of bilirubin-GT) to the liver cells. Tested first of all in the Gunn rat, an animal model of the disease, a single injection of this drug candidate has enabled the long-term correction of the disease in murine models (Molecular Therapy Methods and Clinical Development \u2013 July 2016).<\/p>\n
\u201cWe are delighted to be starting a clinical trial of the gene therapy product we have designed to treat Crigler Najjar Syndrome, a very serious liver disease. The trial was made possible by G\u00e9n\u00e9thon’s pioneering research into gene therapy for rare diseases, over a period of almost 20 years. Today, seven clinical trials are taking place across the world, involving products that G\u00e9n\u00e9thon\u2019s researchers either developed themselves or participated closely in developing \u201d explains Fr\u00e9d\u00e9ric Revah, CEO of G\u00e9n\u00e9thon.<\/p>\n","protected":false},"excerpt":{"rendered":"
G\u00e9n\u00e9thon starts a European phase I\/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease. This European trial will include 17 patients over 10 years old, (the age when the liver reaches maturity); it aims to assess the tolerance of the product, determine the optimal dose, and assess the therapeutic efficacy … [Read more]<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3],"tags":[],"class_list":["post-5995","post","type-post","status-publish","format-standard","hentry","category-news"],"_links":{"self":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5995","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/comments?post=5995"}],"version-history":[{"count":1,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5995\/revisions"}],"predecessor-version":[{"id":5996,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5995\/revisions\/5996"}],"wp:attachment":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/media?parent=5995"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/categories?post=5995"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/tags?post=5995"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}