KONFERANS: ALAIN FISCHER – ANKARA

Bu etkinlik sadece İngilizcedir.

GENE THERAPY, WHERE DO WE STAND, WHAT CAN BE EXPECTED?

26 Mart 2026, 16.00
Ankara Üniversitesi
Ord. Prof. Dr. Abdülkadir Noyan Konferans Salonu

Based on the advances in molecular biology over the last 50 years, it is now feasible to transfer genes into cells either ex vivo or in vivo through viral vehicles. Such strategies have now been succesfully used over the last 25 year to treat twenty or so inherited diseases from primary immunodeficiencies, to hemoglobinopathies, storage diseases, neuromuscular diseases, blindness or hemophilias. In addition, development of chimeric antigen receptor (CAR)-modified T lymphocytes has been implemented to treat refractory malignancies of B lymphocytes (leukemias, lymphomas, myeloma) and possiby severe autoimmune diseases. Gene edition,i.e gene modification (“surgery”) based on the CRISPR-CAS9 technology that enables precise intervention into the genome opens new avenues to treat more inherited diseases, but perhaps also more common diseases. Today, 17 gene therapy drugs have been marketed which is a testimony of a medical succes. Nevertheless, accessibility remains problematic because of the excessively high prices at which these these drugs are sold. This should require some form of regulation.

Alain Fischer obtained his medical degree (pediatrics) in 1979. He became a professor of immunology at Paris Descartes University, and then, in 1991, became director of an INSERM unit on “Normal and Pathological Development of the Immune System.” He was head of the “Pediatric Immunology and Hematology” unit at Necker Enfants-malades Hospital (AP-HP) from 1996 to 2012. In November 2002, he was elected a member of the French Academy of Sciences and in 2011 a member of the French Academy of Medicine. He was a member of the French National Consultative Ethics Committee from 2003 to 2009. He was the founding director of the Imagine Institute for Genetic Diseases (2007-2016). Alain Fischer was elected a member of the US National Academy of Medicine in 2017 and the US National Academy of Sciences in 2019. Alain Fischer held the Chair of Experimental Medicine at the Collège de France from 2014 to 2020. His work has been recognized with numerous awards, including the Jeantet Prize (2001), the INSERM Grand Prize (2008), and the Japan Prize (2015). Alain Fischer’s work has been devoted to the study of the human immune system through the characterization of numerous genetic defects and the understanding of their pathophysiology. He successfully developed the first gene therapy trials. In 2016, he chaired the steering committee for the citizen consultation on infant vaccination and, from December 2020 to July 2022, the steering committee for the anti-Covid vaccination strategy. From January 2023 to 2025, he chaired the French Academy of Sciences.