Several elements predict exponential growth in the need for vectors. First, gene therapy is entering phase II trials, which require more patients, therefore more batches of vectors.
Then, although proof of the concept has been demonstrated in extremely rare and severe diseases such as certain immunodeficiencies, gene therapy is currently being tested for more common diseases such as blood diseases (sickle-cell anemia, beta-thalassemia). These trials must include more patients.
Lastly, although local administration of vectors was evaluated first, research is now turning to treatments using more general pathways. But you need ten to a hundred times more vectors to treat a skin disease such as epidermolysis bullosa or a neuromuscular disease such as Duchenne muscular dystrophy, spinal muscular atrophy or limb girdle dystrophy, than to treat a retinal disease.
Support the creation of a production platform
AFM has invested heavily in the installation of vector production centres. In 1997, it was the stimulus for creating a vector production network (Gene Vector Production Network, GVPN). Over a five-year period, this network has supplied nearly 3,000 batches to research teams in France and across the world. AFM has also supported the creation of several production platforms.
• Généthon: The Etablissement de Thérapie Génique et Cellulaire (ETGC – gene and cell therapy unit) (Evry)
Created in 2003, the ETGC is made up of two production sites which have been accredited by the Agence Française de Sécurité Sanitaire des Produits de Santé (AFSSAPS - French Health Products Safety Agency) for five years. The site received the AFSSAPS label in 2006. In February 2009, the ETGC was the first structure in Europe to release a batch of HIV-derived lentiviral vectors in accordance with Good Manufacturing Practices (GMP).
• Généthon Bioprod is a new site at Evry that will soon be able to produce vectors on a large scale. It is a world unique center and it’s the first time in France that an association will be allowed to produce pharmaceutical products.
• Atlantic Bio GMP (ABG), at St Herblain near Nantes (Loire-Atlantique).
This is a second centre for producing batches of viral vectors according to GMP (good manufacturing practices) standards after Généthon ETGC.
• The Etablissement de Thérapie Génique et Cellulaire (ETGC) (gene and cell therapy unit) at Nantes University Hospital.
These structures, particularly Atlantic Bio GMP and Généthon Bioprod, have been set up in order to meet the need for batches of vectors for phase II gene therapy trials. They are among the biggest vector production centres in Europe.