Post by Genethon

[SYMPOSIUM] Genethon was proud to co‑organize, alongside Institut Imagine and the Broad Institute of MIT and Harvard, an exceptional symposium dedicated to the future of genetic medicine. This unique gathering brought together leading scientific, clinical and regulatory experts from these institutions to explore how the most advanced innovations in genetic medicine can be translated into transformative treatments for patients.   The symposium featured high-level presentations from pioneers and experts and, including Pr. David R. Liu (Broad Institute of MIT and Harvard, Harvard University, Howard Hughes Medical Institute (HHMI), Pr.Tim Yu, Romain Lévy, M.D, Ph.D, Marina Cavazzana, Frederic Revah, Annarita Miccio and Winston Yan, MD PhD, reflecting the depth and excellence of the discussions across disciplines.   As highlighted by Frederic Revah, “ This Symposium brought together the best experts from our institutions which are leading pioneers in the field of Genetic Medicine, and have been at the origin of world firsts in the field of gene therapy and genetic surgery from research to use in patients. »   Throughout the day, discussions also highlighted what it truly takes to move from breakthrough science to patient impact. - From the very first steps from the bench to first‑in‑human, experts, including Dr. Giuseppe Ronzitti, addressed the key challenges of preclinical proof of concept, quality control and safety. - The central role of patient organizations was also underscored, with the contribution of the Center for Therapeutic Genetics and the unique development of a cutting-edge innovation ecosystem built by AFM-Téléthon with François Lamy. - Then, the conversation extended to regulatory pathways, with a dedicated roundtable on strategies across France, Europe and the United States, moderated by Frédéric Revah, bringing together perspectives from major authorities with the major contribution of Catherine Paugam-Burtz (Catherine P.)  CEO of ANSM Agence nationale de sécurité du médicament et des produits de santé, Dr Emmely de Vries - European Medical Association (EMA). Medical Association for Doctors and Medical Industry Personnel. (EMA) and Winston Yan, MD PhD. - Finally, the question of sustainability was at the heart of discussions, with Vincent Genet contributing to a forward‑looking dialogue on building a robust economic model capable of ensuring long‑term access to genetic therapies.   At Genethon, we remain fully committed to advancing gene therapy and genome editing through collaboration, with one clear objective: bringing these innovations to as many patients as possible.   #genethon #GeneticMedicine #GeneTherapy #RareDiseases #PatientAdvocacy  Alexander Sousa Nina Frost Bernard Celli Emi Botelho Bana Jabri, Center for Therapeutic Genetics, Université Paris Cité, AP-HP, Assistance Publique - Hôpitaux de Paris

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