A major research centre
Today Genethon devotes it time to developing gene therapy for rare diseases.
Through its budget and its workforce, it is one of the leading centres in the world for research and development of treatments for rare diseases, as well as a major player in the field of biotherapies.
AFM-Téléthon is still the major financier, with €25.1 million given in 2015.
A policy of partnerships in Europe and the United States enables the Genethon to maintain the highest standards of preclinical and clinical development for each disease in question.
Examples: setting up an international network to treat Wiskott-Aldrich syndrome and a "gene therapy for the eye" network with the aim of stepping up the development of treatments for rare eye diseases.