Généthon produces first batch of HIV-derived vectors in Europe for
gene therapy trial
19 March 2009
Genethon, the French laboratory created and funded by the AFM (French
Association against Myopathies) using donations from Téléthon, has
announced on the 20th of February that it has produced, controlled and
released a batch of lentiviral vectors derived from the human
immunodeficiency (HIV) virus for a gene therapy trial in humans in a
rare immune deficiency.
The Etablissement de thérapie génique et cellulaire (ETGC - gene and
cell therapy unit) at Généthon thus becomes the first organisation in
Europe to have produced this type of vector in accordance with GMP (good
manufacturing practice) standards. Part of the safety controls were
carried out by GenoSafe(1), a company created by the AFM and Généthon.
Since it was founded in 2005 and officially approved by the health
authorities in 2006, the ETGC at Généthon has been working on a number
of different vectors. In particular, it carried out sterile filling
operations, quality control and batch release of AAV vectors used in the
phase I gene therapy trial conducted for a neuromuscular disease: gamma-sarcoglycanopathy.
It has also produced and released oncoretroviral vectors for future
trials in graft-versus-host disease, inclusion body myositis and
The release of this first batch of HIV-derived vectors, produced in
accordance with GMP standards, is a crucial step in the preparation of
the clinical trial authorisation application to be submitted to Afssaps
(the French drug safety authority) during the course of March. Following
approval by the health authorities, this batch will be used in a gene
therapy trial for Wiskott-Aldrich syndrome, a rare immune deficiency.
Genethon will be the sponsor of this trial, which is expected to be
launched during the last quarter of 2009.
"This first is good news for our Généthon laboratory, confirming its
capacity to serve as a major European tool to demonstrate the
effectiveness of gene therapy in the treatment of rare diseases," states
Laurence Tiennot-Herment, President of the AFM, and also President of
the laboratory since 1 January 2009.
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