Généthon and Children’s Hospital Boston partner in gene therapy
trial to treat WAS
24 Jan 2011
Généthon, the not-for-profit biotherapy laboratory funded by
the French Muscular Dystrophy Association (AFM), and Children's Hospital
Boston have announced 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 already sponsoring parallel trials at Great Ormond
Street Hospital in London and Hopital Necker-Enfants Malades in
Paris 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 Généthon 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
“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
"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
“At Children’s Hospital Boston, we are committed to utilizing
state-of-the-art cell and molecular therapies to treat devastating
paediatric 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,” said Dr. David A.
Williams, Sponsor-Investigator of the study.