As a pioneer in the discovery and development of gene therapies for rare diseases, Généthon is a non-profit laboratory that was established by AFM-Téléthon. A first gene therapy for spinal muscular atrophy to which Généthon contributed has obtained a product license. With more than 200 scientists and professional staff, Généthon is pursuing its aim to develop therapies which change the lives of patients suffering from rare genetic diseases. Thirteen products developed by Généthon are in clinical trial for diseases of the liver, blood, immune system, muscles and eyes. Seven other products could enter clinical trial over the next five years.