Genethon and Children’s Hospital Boston get FDA approval for a Wiskott Aldrich gene therapy trial

The US Food & Drug Administration (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.