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Genethon and Children's Hospital Boston Get FDA approval for a Wiskott Aldrich Gene Therapy Trial
Date:7/21/2011

BOSTON, July 21, 2011 /PRNewswire-USNewswire/ -- The U.S. 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.  

Earlier this year, 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 announced that they have 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 fifteen patients, five for each 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 multicenter collaboration to accelerate the testing of new advanced therapies for rare conditions.

The Boston trial will be funded by the National Heart, Lung and Blood Institute (NHLBI) through its Gene Therapy Resource Program (GTRP). Principal investigators at Children's are Sung-Yun Pai, MD, Division of Hematology/Oncology, and Luigi Notarangelo, MD, director, Research and Molecular Diagnosis Program on Primary Immunodeficiencies, Division of Immunology. David A. Williams, MD, chief of the Division of Hematology/Oncology and director o
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SOURCE Children's Hospital Boston
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