Généthon is a non-profit research organization focused on the development of gene therapies for rare diseases. It was created by the patient-led organization AFM-Téléthon, which has invested almost 800 million euros in gene therapy research. Thousands of patients worldwide are already benefiting from its research through traditional licensing agreements, such as the first gene therapy approved in 2019 for spinal muscular atrophy type 1. Généthon is leading pivotal clinical trials of gene therapy for Crigler-Najjar syndrome, a life-threatening liver disease that affects approximately 1 in 750,000 to 1 million people worldwide. This therapy would be a first for this ultra-rare disease. For ultra-rare diseases, it proposes alternative approaches to patients, such as providing access through publicly funded clinical trials instead of the traditional approval process. CEO Frédéric Revah emphasizes that despite the challenges in gaining the interest of pharmaceutical companies and investors, Généthon will not abandon patients with rare and ultra-rare diseases.[1][2][3]