The FDA approved the launching in the U.S. of a clinical trial for gene therapy for a rare immunodeficiency, Wiskott-Aldrich syndrome (WAS). After its implementation in Paris and London, this trial based on preclinical research performed at Genethon (Evry, France) which also manufactures the GMP gene therapy product, is now going to be launched in Boston. It's one of the first international clinical trials using a gene therapy treatment for a rare disease.
Genethon, a not-for-profit biotherapy laboratory funded by the French Muscular Dystrophy Association (AFM) and Children's Hospital Boston, has initiated a partnership to conduct a gene therapy clinical trial for Wiskott-Aldrich Syndrome (WAS), a severe immunodeficiency disease leading to death before adulthood. Genethon is sponsoring parallel trials at Great Ormond Street Hospital in London and Hopital Necker-Enfants Malades in Paris and is supplying the vector for all clinical sites. In total, this trial will involve 15 patients, five per site, who will be treated by 2013-2014. The sites in London and Paris have already started treating patients under this protocol.
Altogether, the WAS gene therapy trials in London, Paris and Boston will constitute a unique multi-center collaboration to accelerate the testing of new advanced therapies for rare conditions.