Thermo Fischer Scientific 2009 prize

Anne Galy, winner of the Biotherapy prize for her project “gene therapy in Wiskott-Aldrich syndrome”

Généthon, the laboratory created and funded by the AFM (French Association against Myopathies) using donations from Téléthon, has announced 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 GenoSafe1, 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 autoimmune diseases. 

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. Généthon 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.