Partnership for a gene therapy Clinical trial signed between Genethon and Children’s Hospital Boston

A unique multicenter international study of gene therapy for a rare genetic disease (Wiskott-Aldrich syndrome) led in London, Paris and Boston.

Genethon, the not-for-profit biotherapy laboratory funded by the French Muscular Dystrophy Association (AFM) thanks to the donations collected during the French Telethon, and Children’s Hospital Boston announce that they have initiated a partnership to conduct a gene therapy clinical trial for Wiskott-Aldrich Syndrome (WAS). This rare genetic condition is a life-threatening immunodeficiency disease. Genethon is sponsoring parallel trials at Great Ormond Street Hospital in London and Hopital Necker-Enfants Malades in Paris (Press release – Feb 24, 2010) and will be supplying the vector for the trial at the US site. The vector is a latest-generation lentiviral vector that incorporates several safety features designed to avoid the
complications caused by early-generation vectors. The vector is manufactured by Genethon in Evry. Altogether, the WAS gene therapy trials in London, Paris and Boston will constitute a unique multicenter collaboration to accelerate the testing of new advanced therapies for rare conditions.