For the first time, a non-profit organization, created by a patient association, has received the French Galien Prize. Frédéric Revah, Chief Executive Officer of Généthon, said “This prestigious prize confirms the incontestable role of Généthon as world leader in the field of gene therapy, and recognizes its major contribution to medicine in general and to the treatment of rare diseases specifically.”
Created in 1990 by a patient association, the AFM-Téléthon, and 90% financed by donations from the French Téléthon, Généthon is dedicated to the development of gene therapy treatments for rare diseases - from their discovery through production and through their pre-clinical and clinical development.
Laurence Tiennot-Herment, Chairman of the AFM-Téléthon and of Généthon, said ““I would like to dedicate this prize to the donors and partners of the Téléthon who have supported Généthon since its inception and who have made possible its development. Without them this incredible adventure never could have happened. Thanks to their continued support we have tackled the most audacious challenges, from producing the first maps of the human genome to the production today of medicines using gene therapy.”
Généthon is recognized for creating the first maps of the human genome which immediately were made available to the world scientific community. Généthon also made history in gene therapy with major breakthroughs which included exon-skipping by AAV, the first gene therapy treatment of a neuromuscular disease (gamma sarcoglycanopathy) and the first trans-Atlantic (France/England/USA) multicenter, gene therapy clinical trial in immunodeficiency.
To respond to the needs of production, Généthon has evolved and now has the largest bio-production center in the world for gene therapy products suitable for use in humans, Généthon-Bioprod. This facility is unique and essential to respond to the needs of researchers conducting human trials for rare diseases. The creation of Généthon-Bioprod has transformed Généthon which now has moved from the age of research into the age of pre-industrialization.