MD, Director of the INSERM lab NeuroGenCell (Gene and cell Therapy for neurodegenerative diseases of adults and children) at Institute for Brain and Spine (ICM) in Hospital Pitié Salpêtrière in Paris.
Engaged in the development of gene therapy applications for neurodegenerative diseases (genetic leukodystrophies, Huntington’s disease, Alzheimer disease, Amyotrophic Lateral Sclerosis) Including all translation steps from preclinical proof of concept in animal models to clinical trials in human patients; development of clinical protocol and regulatory aspects.
Investigator in the first gene therapy trial based on autologous transplantation of corrected cells with an HIV vector to correct cerebral forms of Adrenoleukodytrophy and Investigator in the gene therapy trial using AAV gene transfer of arylsulfatase A in brai parenchyma to cure metachromatic leukodystrophy.
Teatching activity :
Professor at the Faculty of Pharmaceutical Sciences (Université Paris Descartes 2010-2013, University Paris Sud since) : Genetics, medical semiology, Biotherapies
Director of the Biotherapy course in the Magistère of Genetics Paris 5-Paris 7
Visiting Professor, University of Massachusetts Medical School : Gene therapy
Expert activity
President of the Société Française de Thérapie Cellulaire et Génique (SFTCG , 2010-2014)
Coordinator of the Domaine d’Intérêt Majeur (DIM) of the Ile de France region for biothérapies (since 2011)
Expert in the French committee for the follow up of clinical trials in Biotherapy if INSERM (COSSEC) since 2008
Expert at INSERM national specialized committee for biotherapies (CSS8, 2008-2013)
Member of the Scientific committee of INSERM (2013-2018)
Former President of the scientific committee of the Fondation Maladies Rares
Member of the scientific committee of AFM (Association Française contre les Myopathies)
Corresponding member of the French Académie Nationale de Médecine (section biology)
Member of the Gene Therapy Working Party (GTWP) European Medicine Agency (EMA)
Member of the scientific committee of the Janssen research fund (2017)
Member of the scientific committee of the Hanover Medical School (2015-current)
Former President of the European Society for Cell and Gene Therapy ( ESGCT, European Society for cell and Gene Therapy) : 2014-2016
Honors
Jean Valade award (Fondation de France) (2004)
Thermo Biotherapie award (2007)
Sisley-Lejeune award (2010)
Drieu – Cholet (Académie de Médecine) award (2010)
Chevalier de la Légion d’Honneur (French Government award) (2010)
Mémain-Pelletier de l’Académie des Sciences award (2011)
Corresponding member of the French Academy of Medicine (2012)
Grand Prix de la Fondation pour la Recherche médicale (2019)
Grand prix Lazorthes de l’Académie des Sciences 2019