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

f Translational Research for Children's Hospital Boston, will serve as sponsor-investigator. The gene insertion of patient cells will be accomplished in the Connell O'Reilly Family Good Manufacturing facility at the Dana-Farber Cancer Institute in collaboration with the Center for Human Cell Therapy at Harvard Medical School.

Wiskott Aldrich Syndrome (WAS) is a rare primary immune deficiency disease causing significant bleeding due to low platelet count and increased incidence of serious infections.  Most patients have mild to severe eczema and are also at a higher risk of developing autoimmune disorders and malignancies such as lymphoma.  The forthcoming clinical trial results from a research program initiated in 2002 by the group of Anne Galy at Genethon (Inserm UMR951/Genethon, Universite d'Evry Val d'Essonne, EPHE), which has developed an ex vivo approach that uses an HIV-derived lentiviral vector to transfer genes into autologous CD34+ hematopoietic stem cells from WAS patients.

"This authorization constitutes a new key stage for the project WAS initiated in 2002 by Anne Galy and her team at the Genethon. It is also an international recognition of the quality of the work of Genethon. Without the donations collected during the French Telethon, this international gene therapy trial could not exist," states Laurence Tiennot-Herment, Chairperson, AFM and Genethon. 

"We are very happy with the approval given by the FDA for the extension of the WAS clinical trial by the group headed by Prof. Williams. It demonstrates again the ability of Genethon to bring its research projects to the clinic in collaboration with the best clinical teams worldwide, for the benefit of patients. It reflects our cutting edge expertise in the field of translational research, bioproduction and preparation and implementation of clinical trials," emphasizes Frederic Revah, CEO Genethon.

"At Children's Hospital Boston,
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SOURCE Children's Hospital Boston
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