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.

Généthon, 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.

The Wiskott Aldrich Syndrome (WAS) is a rare primary immune deficiency disease causing significant bleeding due to low platelets and increased numbers 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/Généthon, Université 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.

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 of Translational Research for Children’s Hospital Boston, will serve as sponsor-investigator.

The London WAS trial ongoing at Great Ormond Street Hospital/University College of London Institute of Child Health is conducted by Adrian J Thrasher MD, Professor of Paediatric Immunology and H. Bobby Gaspar MD Professor of Paediatrics and Immunology.

The trial ongoing at Necker-Enfants Malades in Paris is conducted by Marina Cavazzana-Calvo MD, Head of Clinical Investigation Center Biotherapy, Necker, Alain Fischer MD, Professor of Pediatric Immunology, Scientific Director of the Imagine Foundation and Salima Hacein-Bey Abina PhD, Professor of Immunology and Head of the Gene Therapy Laboratory.

“This gene therapy trial (being run simultaneously in London, Paris and Boston) wouldn’t have been possible without the Telethon. The AFM is taking on a huge challenge – biotherapeutics. This means new hope for millions of patients and new medical tools for the good of all.” comments Laurence Tiennot-Herment, Chairperson, AFM and Généthon

“We are delighted with this new collaboration with 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, we are committed to utilizing state-of-the-art cell and molecular therapies to treat devastating pediatric diseases. The trial in WAS is particularly noteworthy as it represents a continuing transatlantic scientific and clinical research collaborative effort in gene therapy and huge multi-disciplinary team effort at Children’s” comments Dr. David A. Williams, Sponsor-Investigator of study.