The Foundation For Rare Diseases, the Plateforme Lilloise d'Expertise Maladies Rares PLEMaRa and the University Hospital of Lille are pleased to invite you to the Hauts-de-France Scientific Symposium. This is a unique opportunity to meet all the regional players in the field of rare diseases, to discuss research, therapies, innovation and human and social sciences!
More information on the speakers
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INTRODUCTION
Pr Eric Hachulla is Professor of Internal Medicine at the University Hospital of Lille. He is the coordinator of the Reference Center for Rare Systemic Autoimmune Diseases of the North and North-West (CeRAINO). He has published numerous works, mainly on connectivities and coordinates several Inter-University Diplomas and European courses available online. Since February 2014, he has been coordinating the Fai2r health network for rare autoimmune and autoinflammatory diseases.
Frédéric BOIRON, Director of the Strasbourg University Hospital, is a graduate of the Ecole Nationale de la Santé Publique (ENSP) in Rennes (1993), Sciences Po Bordeaux (1990) and the Bordeaux Institute of Journalism (1987). He also has a university diploma in Hospital Plus (Ecole des Hautes Etudes en Santé Publique / 2009-2010) and a post-graduate diploma in "Medical Sciences for Managers" (UFR Médecine Lariboisière / 2000- 2003). Frédéric Boiron was successively deputy director of the Laennec Hospital (1991-1995), secretary general of the Georges Pompidou European Hospital (1995-2001), deputy director and then director of the General Agency for Health Equipment and Products of the Assistance Publique-Hôpitaux de Paris (AP-HP / 2001-2003). He then left Paris to take over the management of the Provins Hospital (Seine et Marne 2003-2007) while acting as interim director of the Coulommiers Hospital in 2005 (Seine et Marne). He became director of the Beauvais Hospital (2007-2011), and at the same time was interim director of the Saint-Quentin Hospital (Aisne) in 2010 and of the Creil and Senlis Hospitals (Oise) in 2010-2011, with a view to creating a joint management structure. He directed the University Hospital of Saint-Etienne from August 2011 to May 2017. In addition, Frédéric BOIRON was President of the Association of Hospital Directors (ADH) from March 16, 2011 to February 2019. Frédéric BOIRON has been Chief Executive Officer of Lille University Hospital since May 15, 2017.
Pr Daniel Scherman is Director of Research of Exceptional Rank at the CNRS and heads the Foundation For Rare Diseases. He is a Doctor of State in Science and an engineering alumnus of the Ecole Polytechnique. Daniel Scherman received in November 2017 the Grand Prix of the Academy of Sciences "Emilia Valori" for the Application of Chemical and Biological Sciences. He is a member of the European Academy of Sciences EURASC Head of the Division "Medicine and Life sciences" . Daniel Scherman has also been distinguished with the following awards: Experimental Uveitis Research Award - Deutsche Uveitis Arbeitsgemeinschaft; Palmes Académiques; Laureate of the Grand Prix of the Académie Nationale de Médecine; Silver Medal of the CNRS; Research Prize of the Rhône-Poulenc Group; Neurobiology Prize of the Fondation pour la Recherche Médicale; Prize of the Institut de Biologie Physico-Chimique.
ACTORS AT THE SERVICE OF RARE DISEASES
Pr Eric Hachulla is Professor of Internal Medicine at the University Hospital of Lille. He is the coordinator of the Reference Center for Rare Systemic Autoimmune Diseases of the North and North-West (CeRAINO). He has published numerous works, mainly on connectivities and coordinates several Inter-University Diplomas and European courses available online. Since February 2014, he has been coordinating the Fai2r health network for rare autoimmune and autoinflammatory diseases.
Pr Alexandre BELOT is a pediatrician and researcher in immunology at the Hospices Civils de Lyon. He is the director of the RAISE-Lyon national reference center and is interested in rare pediatric onset inflammatory diseases. He co-leads the FAI2R rare diseases health network and pilots the steering committee for pediatric multisystemic inflammatory syndromes, which were identified in children in 2020 and correspond to a post-infectious inflammatory disease. His research work at the International Center for Infectious Diseases Research is focused on genetic and immunological explorations of early-onset inflammatory diseases, in particular juvenile lupus.
Dr Isabelle TALON was born in 1977 and is originally from Besançon. She is a pediatric surgeon at the Strasbourg University Hospital since 2007, joined the biomaterials and bioengineering research team in 2017 and created the DIAPID project in 2019. It is therefore a very young but solid and promising project due to its collaborative aspect. No less than 5 laboratories of excellence work hand in hand around the DIAPID innovation, 4 in Strasbourg (Inserm UMR1121, CNRS CAMB, ICube, ICPEES) and Metrics in Lille.
Anne DUBURCQ is in charge of the Public Health/Epidemiology division of CEMKA. Her activity is mainly focused on the evaluation of actions, programs and health policies, as well as on the evaluation of experiments (innovation in health with Article 51 in particular), innovative devices and care paths. Part of his activity also concerns the design and implementation of pharmaco-epidemiological observational studies and epidemiological studies or evaluations in the field of occupational health. Nearly 25 years spent at CEMKA have allowed her to acquire a solid experience, to implement diversified and complementary methodologies, both quantitative and qualitative or organizational, and to work for all types of actors. Today, she has conducted more than 200 studies in these different fields. Before joining CEMKA (EVAL) in 1994, Anne DUBURCQ worked as a statistician and study manager at INSERM (U170), devoted to epidemiological and statistical research on the environment and health. She is a graduate of ENSAE (Ecole Nationale de la Statistique et de l'Administration Economique) and holds a DEA in Public Health (University of Paris XI, Kremlin-Bicêtre) and the ECORISQUE diploma in the field of environmental risks (training coordinated by the Institut National de Veille Sanitaire, the Ecole Nationale de Santé Publique and EDF) She is also a member of the Association des Epidémiologistes de Langue Française (ADELF), the Société Française de Santé Publique (SFSP) and the Société Française d'Evaluation (SFE).
MEDICAL GENETICS, FROM RESEARCH TO DIAGNOSIS
Dr Simon BOUSSION is Head of Clinic of the Universities and Hospital Assistant at the University Hospital of Lille, in the Medical Genetics Clinic "Guy Fontaine", which is a Reference Center for Rare Diseases labelled "developmental anomalies and malformative syndromes" (CLAD). He is also a member of the research team ULR 7364 - RADEME (Maladies RAres du DEveloppement et du MEtabolisme), within the University of Lille. The main research themes of the RADEME team are intellectual disability and malformative syndromes with congenital anomalies of the limbs.
Dr. Adbellah TEBANI is a senior lecturer at the University and a hospital practitioner with international experience in care, research and teaching. He is passionate about all innovations at the intersection of medicine, artificial intelligence and digital health, a convergence that is essential for the emergence of precision medicine. Dr. Tebani has developed and implemented omic strategies for the diagnosis of Inherited Metabolic Diseases using advanced analytical technologies and machine learning techniques. His current activity includes clinical practice, laboratory management, R&D supervision and training. He co-leads a multidisciplinary team tasked with implementing precision medicine by leveraging multimodal biomedical data via computational modeling, machine learning and innovative intuitive data visualization strategies. He is co-founder of the international symposium "Pathways to Precision Medicine" on Precision Medicine.
Pr Alexandre BELOT is a pediatrician and researcher in immunology at the Hospices Civils de Lyon. He is the director of the RAISE-Lyon national reference center and is interested in rare pediatric onset inflammatory diseases. He co-leads the FAI2R rare diseases health network and pilots the steering committee for pediatric multisystemic inflammatory syndromes, which were identified in children in 2020 and correspond to a post-infectious inflammatory disease. His research work at the International Center for Infectious Diseases Research is focused on genetic and immunological explorations of early-onset inflammatory diseases, in particular juvenile lupus.
Dr Isabelle TALON was born in 1977 and is originally from Besançon. She is a pediatric surgeon at the Strasbourg University Hospital since 2007, joined the biomaterials and bioengineering research team in 2017 and created the DIAPID project in 2019. It is therefore a very young but solid and promising project due to its collaborative aspect. No less than 5 laboratories of excellence work hand in hand around the DIAPID innovation, 4 in Strasbourg (Inserm UMR1121, CNRS CAMB, ICube, ICPEES) and Metrics in Lille.
Anne DUBURCQ is in charge of the Public Health/Epidemiology division of CEMKA. Her activity is mainly focused on the evaluation of actions, programs and health policies, as well as on the evaluation of experiments (innovation in health with Article 51 in particular), innovative devices and care paths. Part of his activity also concerns the design and implementation of pharmaco-epidemiological observational studies and epidemiological studies or evaluations in the field of occupational health. Nearly 25 years spent at CEMKA have allowed her to acquire a solid experience, to implement diversified and complementary methodologies, both quantitative and qualitative or organizational, and to work for all types of actors. Today, she has conducted more than 200 studies in these different fields. Before joining CEMKA (EVAL) in 1994, Anne DUBURCQ worked as a statistician and study manager at INSERM (U170), devoted to epidemiological and statistical research on the environment and health. She is a graduate of ENSAE (Ecole Nationale de la Statistique et de l'Administration Economique) and holds a DEA in Public Health (University of Paris XI, Kremlin-Bicêtre) and the ECORISQUE diploma in the field of environmental risks (training coordinated by the Institut National de Veille Sanitaire, the Ecole Nationale de Santé Publique and EDF) She is also a member of the Association des Epidémiologistes de Langue Française (ADELF), the Société Française de Santé Publique (SFSP) and the Société Française d'Evaluation (SFE).
SPONSORED PRESENTATION: ALNYLAM PHARMACEUTICALS
Alnylam, a U.S. biotechnology company founded in Boston in 2002, has become a leader in the development of breakthrough therapeutic solutions based on RNA interference (RNAi) technology, a new class of drugs. The discovery of RNAi was awarded a Nobel Prize in Medicine in 2006.
Alnylam is one of the few companies that have been able to transform this scientific approach into a therapeutic reality for patients. Alnylam's ambition remains to find drugs that meet unmet therapeutic needs and have a high public health impact. The understanding and mastery of RNAi technology has enabled the company to create and improve its own technological platform, which is the driving force behind its research.
With a broad portfolio of approved drugs in development, Alnylam is participating in the paradigm shift in the management of rare and prevalent diseases with subcutaneous injections every 3 to 6 months.
Dr. Adbellah TEBANI is a senior lecturer at the University and a hospital practitioner with international experience in care, research and teaching. He is passionate about all innovations at the intersection of medicine, artificial intelligence and digital health, a convergence that is essential for the emergence of precision medicine. Dr. Tebani has developed and implemented omic strategies for the diagnosis of Inherited Metabolic Diseases using advanced analytical technologies and machine learning techniques. His current activity includes clinical practice, laboratory management, R&D supervision and training. He co-leads a multidisciplinary team tasked with implementing precision medicine by leveraging multimodal biomedical data via computational modeling, machine learning and innovative intuitive data visualization strategies. He is co-founder of the international symposium "Pathways to Precision Medicine" on Precision Medicine.
Pr Alexandre BELOT is a pediatrician and researcher in immunology at the Hospices Civils de Lyon. He is the director of the RAISE-Lyon national reference center and is interested in rare pediatric onset inflammatory diseases. He co-leads the FAI2R rare diseases health network and pilots the steering committee for pediatric multisystemic inflammatory syndromes, which were identified in children in 2020 and correspond to a post-infectious inflammatory disease. His research work at the International Center for Infectious Diseases Research is focused on genetic and immunological explorations of early-onset inflammatory diseases, in particular juvenile lupus.
Dr Isabelle TALON was born in 1977 and is originally from Besançon. She is a pediatric surgeon at the Strasbourg University Hospital since 2007, joined the biomaterials and bioengineering research team in 2017 and created the DIAPID project in 2019. It is therefore a very young but solid and promising project due to its collaborative aspect. No less than 5 laboratories of excellence work hand in hand around the DIAPID innovation, 4 in Strasbourg (Inserm UMR1121, CNRS CAMB, ICube, ICPEES) and Metrics in Lille.
Anne DUBURCQ is in charge of the Public Health/Epidemiology division of CEMKA. Her activity is mainly focused on the evaluation of actions, programs and health policies, as well as on the evaluation of experiments (innovation in health with Article 51 in particular), innovative devices and care paths. Part of his activity also concerns the design and implementation of pharmaco-epidemiological observational studies and epidemiological studies or evaluations in the field of occupational health. Nearly 25 years spent at CEMKA have allowed her to acquire a solid experience, to implement diversified and complementary methodologies, both quantitative and qualitative or organizational, and to work for all types of actors. Today, she has conducted more than 200 studies in these different fields. Before joining CEMKA (EVAL) in 1994, Anne DUBURCQ worked as a statistician and study manager at INSERM (U170), devoted to epidemiological and statistical research on the environment and health. She is a graduate of ENSAE (Ecole Nationale de la Statistique et de l'Administration Economique) and holds a DEA in Public Health (University of Paris XI, Kremlin-Bicêtre) and the ECORISQUE diploma in the field of environmental risks (training coordinated by the Institut National de Veille Sanitaire, the Ecole Nationale de Santé Publique and EDF) She is also a member of the Association des Epidémiologistes de Langue Française (ADELF), the Société Française de Santé Publique (SFSP) and the Société Française d'Evaluation (SFE).
THERAPEUTIC APPROACHES, FROM RESEARCH TO TREATMENT
Pr David LAUNAY is Professor of Medicine at the Department of Internal Medicine and Clinical Immunology of the University Hospital of Lille, at the Reference Center for Systemic Autoimmune Diseases and member of the European Reference Network ReCONNECT. He is also Deputy Director of the Institute for Translational Research in Inflammation(INFINITE) affiliated to INSERM and Director of the University Hospital Federative Project Precision Health in Complex Inflammatory Diseases ("PRECISE"). He is very interested in autoimmune inflammatory diseases and more particularly in the links between the immune system and fibrosis in this type of disease. His model is based on systemic sclerosis to study pulmonary fibrosis for both medical and clinical research. David Launay is the author of 291 publications, including 226 articles, 32 journals, 22 letters and 11 editorials. He is the primary author of 54 papers and the secondary author of 16 papers. Thirty-three writings are in the top 10% of InCites and three in the top 1%. His work has been cited 6727 times with an H-index of 42.
Pr Rebecca DEPREZ-POULAIN holds a BS in Chemistry (Juniata College, PA, USA 1995), an engineering degree and an MSc in Organic Chemistry (HEI and Université de Lille, 1996), and a PhD in Pharmacy (Université de Lille, 2009). She completed her PhD under the supervision of Pr Tartar (1996-1999) in combinatorial chemistry applied to drug discovery within the company CEREP.SA. After a postdoctoral fellowship at the Pasteur Institute of Lille, she was appointed Senior Lecturer in General Chemistry in 2001 and is, since 2010, Professor of Medicinal Chemistry and Drug Discovery at the Faculty of Pharmacy of Lille. Her research expertise focuses on metalloprotease inhibition for therapeutic uses. She is currently coordinating the European project CAPSTONE-ETN (www.capstone-etn.eu) dedicated to ERAP modulation in immuno-oncology and autoimmune diseases. She has published more than 70 articles in international peer-reviewed journals, including J. Med. Chem, Nat. Comm, Nat. Med. and is the inventor of several patents. Prof. Deprez-Poulain was a member of the Institut Universitaire de France (2015-2020) and has been the recipient of several awards including the SCT/Servier Research Encouragement Award (2009) and the Michel Delalande Award in Pharmacochemistry from the Académie Nationale de Pharmacie (2020). She is currently the president of the French Society of Therapeutic Chemistry.
Pr Laurent STORME is a neonatal resuscitator, Head of the Neonatology Department at the Jeanne de Flandre Hospital at the University Hospital of Lille, member of the ULR 2694 METRICS multidisciplinary research team in Public Health at the University of Lille and Coordinator of the Reference Center for Rare Disease of Diaphragmatic Cup Hernia, with a recognized expertise in neonatal resuscitation and long-term medical follow-up of children with this rare disease
Alnylam is one of the few companies that have been able to transform this scientific approach into a therapeutic reality for patients. Alnylam's ambition remains to find drugs that meet unmet therapeutic needs and have a high public health impact. The understanding and mastery of RNAi technology has enabled the company to create and improve its own technological platform, which is the driving force behind its research.
With a broad portfolio of approved drugs in development, Alnylam is participating in the paradigm shift in the management of rare and prevalent diseases with subcutaneous injections every 3 to 6 months.
Dr. Adbellah TEBANI is a senior lecturer at the University and a hospital practitioner with international experience in care, research and teaching. He is passionate about all innovations at the intersection of medicine, artificial intelligence and digital health, a convergence that is essential for the emergence of precision medicine. Dr. Tebani has developed and implemented omic strategies for the diagnosis of Inherited Metabolic Diseases using advanced analytical technologies and machine learning techniques. His current activity includes clinical practice, laboratory management, R&D supervision and training. He co-leads a multidisciplinary team tasked with implementing precision medicine by leveraging multimodal biomedical data via computational modeling, machine learning and innovative intuitive data visualization strategies. He is co-founder of the international symposium "Pathways to Precision Medicine" on Precision Medicine.
Pr Alexandre BELOT is a pediatrician and researcher in immunology at the Hospices Civils de Lyon. He is the director of the RAISE-Lyon national reference center and is interested in rare pediatric onset inflammatory diseases. He co-leads the FAI2R rare diseases health network and pilots the steering committee for pediatric multisystemic inflammatory syndromes, which were identified in children in 2020 and correspond to a post-infectious inflammatory disease. His research work at the International Center for Infectious Diseases Research is focused on genetic and immunological explorations of early-onset inflammatory diseases, in particular juvenile lupus.
Dr Isabelle TALON was born in 1977 and is originally from Besançon. She is a pediatric surgeon at the Strasbourg University Hospital since 2007, joined the biomaterials and bioengineering research team in 2017 and created the DIAPID project in 2019. It is therefore a very young but solid and promising project due to its collaborative aspect. No less than 5 laboratories of excellence work hand in hand around the DIAPID innovation, 4 in Strasbourg (Inserm UMR1121, CNRS CAMB, ICube, ICPEES) and Metrics in Lille.
Anne DUBURCQ is in charge of the Public Health/Epidemiology division of CEMKA. Her activity is mainly focused on the evaluation of actions, programs and health policies, as well as on the evaluation of experiments (innovation in health with Article 51 in particular), innovative devices and care paths. Part of his activity also concerns the design and implementation of pharmaco-epidemiological observational studies and epidemiological studies or evaluations in the field of occupational health. Nearly 25 years spent at CEMKA have allowed her to acquire a solid experience, to implement diversified and complementary methodologies, both quantitative and qualitative or organizational, and to work for all types of actors. Today, she has conducted more than 200 studies in these different fields. Before joining CEMKA (EVAL) in 1994, Anne DUBURCQ worked as a statistician and study manager at INSERM (U170), devoted to epidemiological and statistical research on the environment and health. She is a graduate of ENSAE (Ecole Nationale de la Statistique et de l'Administration Economique) and holds a DEA in Public Health (University of Paris XI, Kremlin-Bicêtre) and the ECORISQUE diploma in the field of environmental risks (training coordinated by the Institut National de Veille Sanitaire, the Ecole Nationale de Santé Publique and EDF) She is also a member of the Association des Epidémiologistes de Langue Française (ADELF), the Société Française de Santé Publique (SFSP) and the Société Française d'Evaluation (SFE).
ROUND TABLE "FROM INVENTION TO INNOVATION: OBJECTIVE OF VALORIZATION".
Pr David DEVOS is Professor in Medical Pharmacology, Doctor in Neurology, Doctor in Neuroscience at the University of Lille, Inserm, CHU of Lille. His main research focus is on the development of innovative therapeutic strategies for neurodegenerative diseases, from the concept of in vitro and in vivo research to large-scale clinical trials. He has authored more than 345 publications including 205 journals. He has conducted 30 clinical studies including 15 multi-center trials, 14 as a coordinator, 4 projects funded by the European Commission (H2020) including 2 in neuroscience (iron, ferroptotic cell death), 1 large European clinical trial FAIRPARK-2 and 1 diagnostic Covid-19. (Competitive research funding > 13M€) He is a specialist physician and researcher at the expert center for Parkinson's disease and amyotrophic lateral sclerosis (ALS) in Lille. He co-coordinates the French network for Parkinson's disease (NS-Park-F-CRIN), ALS (ACT4ALS-MND) and is involved in research at the National Expert Reference Centers for ALS and Parkinson's disease in Lille with a large cohort of patients. He coordinates the Lille Neurogenetic Center (Spinocerebellar ataxia, spastic paraplegia, Neurodegeneration with brain iron accumulation). He has received several awards: the European Pharmacology Prize, the French Prize for Pharmacological Medicine, etc.
Mr Jean Denys WAELES has been leading innovation activities in the Hauts-de-France region for Bpifrance since 2021. With an annual budget of more than €70M, Jean-Denys Waeles manages and leads a team of 6 engineers in charge of innovation financing within the Lille Regional Office. Finally, he is in charge of representing and promoting Bpifrance to institutions and partners (Banks, State, Region, Investment and Seed Funds, Investment Committee).
Ms. Brigitte COURTOIS is the head of the Research and Innovation Department of the Lille University Hospital, one of the largest structures of its kind in France with nearly 350 employees, some twenty research units (of which the University Hospital is co-supervisor), and a multi-sector CIC. It participates in the definition of the research and innovation strategy of the University Hospital, pilots the main structuring projects and is involved in the coordination of relations with the University of Lille, the EPST and EURASANTE. At the same time, she is the Director of the G4 Health Cooperation Group which associates the University Hospital Centres of Amiens, Caen, Lille and Rouen on the major themes of the HU missions. In particular, she contributed to the establishment of the first G4 University Hospital Federations and laid the foundations for a future inter-regional hub for massive health data. As a hospital director for 24 years, Brigitte COURTOIS has acquired a solid experience of the health world, both in general hospitals and in the specific HU environment.
Mr. François-Xavier Denimal is a Doctor of Pharmacy, majoring in industry, with a Master's degree in Business Administration and a Certificate of Specialized Studies in Bio-statistics. He has 25 years of experience in sales and marketing management positions in healthcare companies specialized in medical devices and/or in vitro diagnostic medical devices. He has been 3 times pharmacist in charge of primary and secondary pharmaceutical establishments. He started at SATT Nord in January 2013 as the sole business developer (all technology areas), then managed the medical devices and in vitro diagnostic medical devices portfolio and some therapeutic files. Later he was assistant to veterinary therapeutic projects and more recently health software and bacteriology related ones.
Pr Rebecca DEPREZ-POULAIN holds a BS in Chemistry (Juniata College, PA, USA 1995), an engineering degree and an MSc in Organic Chemistry (HEI and Université de Lille, 1996), and a PhD in Pharmacy (Université de Lille, 2009). She completed her PhD under the supervision of Pr Tartar (1996-1999) in combinatorial chemistry applied to drug discovery within the company CEREP.SA. After a postdoctoral fellowship at the Pasteur Institute of Lille, she was appointed Senior Lecturer in General Chemistry in 2001 and is, since 2010, Professor of Medicinal Chemistry and Drug Discovery at the Faculty of Pharmacy of Lille. Her research expertise focuses on metalloprotease inhibition for therapeutic uses. She is currently coordinating the European project CAPSTONE-ETN (www.capstone-etn.eu) dedicated to ERAP modulation in immuno-oncology and autoimmune diseases. She has published more than 70 articles in international peer-reviewed journals, including J. Med. Chem, Nat. Comm, Nat. Med. and is the inventor of several patents. Prof. Deprez-Poulain was a member of the Institut Universitaire de France (2015-2020) and has been the recipient of several awards including the SCT/Servier Research Encouragement Award (2009) and the Michel Delalande Award in Pharmacochemistry from the Académie Nationale de Pharmacie (2020). She is currently the president of the French Society of Therapeutic Chemistry.
Pr Laurent STORME is a neonatal resuscitator, Head of the Neonatology Department at the Jeanne de Flandre Hospital at the University Hospital of Lille, member of the ULR 2694 METRICS multidisciplinary research team in Public Health at the University of Lille and Coordinator of the Reference Center for Rare Disease of Diaphragmatic Cup Hernia, with a recognized expertise in neonatal resuscitation and long-term medical follow-up of children with this rare disease
Alnylam is one of the few companies that have been able to transform this scientific approach into a therapeutic reality for patients. Alnylam's ambition remains to find drugs that meet unmet therapeutic needs and have a high public health impact. The understanding and mastery of RNAi technology has enabled the company to create and improve its own technological platform, which is the driving force behind its research.
With a broad portfolio of approved drugs in development, Alnylam is participating in the paradigm shift in the management of rare and prevalent diseases with subcutaneous injections every 3 to 6 months.
Dr. Adbellah TEBANI is a senior lecturer at the University and a hospital practitioner with international experience in care, research and teaching. He is passionate about all innovations at the intersection of medicine, artificial intelligence and digital health, a convergence that is essential for the emergence of precision medicine. Dr. Tebani has developed and implemented omic strategies for the diagnosis of Inherited Metabolic Diseases using advanced analytical technologies and machine learning techniques. His current activity includes clinical practice, laboratory management, R&D supervision and training. He co-leads a multidisciplinary team tasked with implementing precision medicine by leveraging multimodal biomedical data via computational modeling, machine learning and innovative intuitive data visualization strategies. He is co-founder of the international symposium "Pathways to Precision Medicine" on Precision Medicine.
Pr Alexandre BELOT is a pediatrician and researcher in immunology at the Hospices Civils de Lyon. He is the director of the RAISE-Lyon national reference center and is interested in rare pediatric onset inflammatory diseases. He co-leads the FAI2R rare diseases health network and pilots the steering committee for pediatric multisystemic inflammatory syndromes, which were identified in children in 2020 and correspond to a post-infectious inflammatory disease. His research work at the International Center for Infectious Diseases Research is focused on genetic and immunological explorations of early-onset inflammatory diseases, in particular juvenile lupus.
Dr Isabelle TALON was born in 1977 and is originally from Besançon. She is a pediatric surgeon at the Strasbourg University Hospital since 2007, joined the biomaterials and bioengineering research team in 2017 and created the DIAPID project in 2019. It is therefore a very young but solid and promising project due to its collaborative aspect. No less than 5 laboratories of excellence work hand in hand around the DIAPID innovation, 4 in Strasbourg (Inserm UMR1121, CNRS CAMB, ICube, ICPEES) and Metrics in Lille.
Anne DUBURCQ is in charge of the Public Health/Epidemiology division of CEMKA. Her activity is mainly focused on the evaluation of actions, programs and health policies, as well as on the evaluation of experiments (innovation in health with Article 51 in particular), innovative devices and care paths. Part of his activity also concerns the design and implementation of pharmaco-epidemiological observational studies and epidemiological studies or evaluations in the field of occupational health. Nearly 25 years spent at CEMKA have allowed her to acquire a solid experience, to implement diversified and complementary methodologies, both quantitative and qualitative or organizational, and to work for all types of actors. Today, she has conducted more than 200 studies in these different fields. Before joining CEMKA (EVAL) in 1994, Anne DUBURCQ worked as a statistician and study manager at INSERM (U170), devoted to epidemiological and statistical research on the environment and health. She is a graduate of ENSAE (Ecole Nationale de la Statistique et de l'Administration Economique) and holds a DEA in Public Health (University of Paris XI, Kremlin-Bicêtre) and the ECORISQUE diploma in the field of environmental risks (training coordinated by the Institut National de Veille Sanitaire, the Ecole Nationale de Santé Publique and EDF) She is also a member of the Association des Epidémiologistes de Langue Française (ADELF), the Société Française de Santé Publique (SFSP) and the Société Française d'Evaluation (SFE).
HUMAN AND SOCIAL SCIENCES AT THE SERVICE OF RARE DISEASES
Dr Barbara LE DRIANT is a Doctor in Psychology at the University of Picardie Jules Verne (Amiens). Her research activity is in developmental psychology in an integrative perspective. Her work focuses more specifically on the study of early parent-child interactions and the construction of attachment bonds with both neuro-typical populations and children in vulnerable or handicapped situations (children born prematurely, children with hearing impairments or autism spectrum disorders). She studies the specificity of the developmental processes implemented in such situations in order to understand the developmental trajectories in a dynamic and non-linear approach of development. Dr. Barbara LE DRIANT participates in the SURGENEA project, financed by the FMR, and in the Federative Hospital-University project 2020 - 2025 "1000 days for health: taking care before treating."
Pr Caroline DESOMBRE is a professor of social psychology (PSITEC laboratory, University of Lille). She is also Deputy Director in charge of research and development at the Institut National Supérieur du Professorat et de l'Education. Her work focuses on the obstacles to the inclusion of students with special educational needs in school and the effect of stereotypes on the behavior of individuals.
Dr. Sophie FANTONI-QUINTON is a doctor of medicine, doctor of law, PUPH in medicine and occupational health (CHRU Lille) specialized in professional integration and job retention in the face of chronic diseases and disabilities and Ministerial Advisor in 2021 in charge of occupational health issues at the Ministry of Labor.
Mr Jean Denys WAELES has been leading innovation activities in the Hauts-de-France region for Bpifrance since 2021. With an annual budget of more than €70M, Jean-Denys Waeles manages and leads a team of 6 engineers in charge of innovation financing within the Lille Regional Office. Finally, he is in charge of representing and promoting Bpifrance to institutions and partners (Banks, State, Region, Investment and Seed Funds, Investment Committee).
Ms. Brigitte COURTOIS is the head of the Research and Innovation Department of the Lille University Hospital, one of the largest structures of its kind in France with nearly 350 employees, some twenty research units (of which the University Hospital is co-supervisor), and a multi-sector CIC. It participates in the definition of the research and innovation strategy of the University Hospital, pilots the main structuring projects and is involved in the coordination of relations with the University of Lille, the EPST and EURASANTE. At the same time, she is the Director of the G4 Health Cooperation Group which associates the University Hospital Centres of Amiens, Caen, Lille and Rouen on the major themes of the HU missions. In particular, she contributed to the establishment of the first G4 University Hospital Federations and laid the foundations for a future inter-regional hub for massive health data. As a hospital director for 24 years, Brigitte COURTOIS has acquired a solid experience of the health world, both in general hospitals and in the specific HU environment.
Mr. François-Xavier Denimal is a Doctor of Pharmacy, majoring in industry, with a Master's degree in Business Administration and a Certificate of Specialized Studies in Bio-statistics. He has 25 years of experience in sales and marketing management positions in healthcare companies specialized in medical devices and/or in vitro diagnostic medical devices. He has been 3 times pharmacist in charge of primary and secondary pharmaceutical establishments. He started at SATT Nord in January 2013 as the sole business developer (all technology areas), then managed the medical devices and in vitro diagnostic medical devices portfolio and some therapeutic files. Later he was assistant to veterinary therapeutic projects and more recently health software and bacteriology related ones.
Pr Rebecca DEPREZ-POULAIN holds a BS in Chemistry (Juniata College, PA, USA 1995), an engineering degree and an MSc in Organic Chemistry (HEI and Université de Lille, 1996), and a PhD in Pharmacy (Université de Lille, 2009). She completed her PhD under the supervision of Pr Tartar (1996-1999) in combinatorial chemistry applied to drug discovery within the company CEREP.SA. After a postdoctoral fellowship at the Pasteur Institute of Lille, she was appointed Senior Lecturer in General Chemistry in 2001 and is, since 2010, Professor of Medicinal Chemistry and Drug Discovery at the Faculty of Pharmacy of Lille. Her research expertise focuses on metalloprotease inhibition for therapeutic uses. She is currently coordinating the European project CAPSTONE-ETN (www.capstone-etn.eu) dedicated to ERAP modulation in immuno-oncology and autoimmune diseases. She has published more than 70 articles in international peer-reviewed journals, including J. Med. Chem, Nat. Comm, Nat. Med. and is the inventor of several patents. Prof. Deprez-Poulain was a member of the Institut Universitaire de France (2015-2020) and has been the recipient of several awards including the SCT/Servier Research Encouragement Award (2009) and the Michel Delalande Award in Pharmacochemistry from the Académie Nationale de Pharmacie (2020). She is currently the president of the French Society of Therapeutic Chemistry.
Pr Laurent STORME is a neonatal resuscitator, Head of the Neonatology Department at the Jeanne de Flandre Hospital at the University Hospital of Lille, member of the ULR 2694 METRICS multidisciplinary research team in Public Health at the University of Lille and Coordinator of the Reference Center for Rare Disease of Diaphragmatic Cup Hernia, with a recognized expertise in neonatal resuscitation and long-term medical follow-up of children with this rare disease
Alnylam is one of the few companies that have been able to transform this scientific approach into a therapeutic reality for patients. Alnylam's ambition remains to find drugs that meet unmet therapeutic needs and have a high public health impact. The understanding and mastery of RNAi technology has enabled the company to create and improve its own technological platform, which is the driving force behind its research.
With a broad portfolio of approved drugs in development, Alnylam is participating in the paradigm shift in the management of rare and prevalent diseases with subcutaneous injections every 3 to 6 months.
Dr. Adbellah TEBANI is a senior lecturer at the University and a hospital practitioner with international experience in care, research and teaching. He is passionate about all innovations at the intersection of medicine, artificial intelligence and digital health, a convergence that is essential for the emergence of precision medicine. Dr. Tebani has developed and implemented omic strategies for the diagnosis of Inherited Metabolic Diseases using advanced analytical technologies and machine learning techniques. His current activity includes clinical practice, laboratory management, R&D supervision and training. He co-leads a multidisciplinary team tasked with implementing precision medicine by leveraging multimodal biomedical data via computational modeling, machine learning and innovative intuitive data visualization strategies. He is co-founder of the international symposium "Pathways to Precision Medicine" on Precision Medicine.
Pr Alexandre BELOT is a pediatrician and researcher in immunology at the Hospices Civils de Lyon. He is the director of the RAISE-Lyon national reference center and is interested in rare pediatric onset inflammatory diseases. He co-leads the FAI2R rare diseases health network and pilots the steering committee for pediatric multisystemic inflammatory syndromes, which were identified in children in 2020 and correspond to a post-infectious inflammatory disease. His research work at the International Center for Infectious Diseases Research is focused on genetic and immunological explorations of early-onset inflammatory diseases, in particular juvenile lupus.
Dr Isabelle TALON was born in 1977 and is originally from Besançon. She is a pediatric surgeon at the Strasbourg University Hospital since 2007, joined the biomaterials and bioengineering research team in 2017 and created the DIAPID project in 2019. It is therefore a very young but solid and promising project due to its collaborative aspect. No less than 5 laboratories of excellence work hand in hand around the DIAPID innovation, 4 in Strasbourg (Inserm UMR1121, CNRS CAMB, ICube, ICPEES) and Metrics in Lille.
Anne DUBURCQ is in charge of the Public Health/Epidemiology division of CEMKA. Her activity is mainly focused on the evaluation of actions, programs and health policies, as well as on the evaluation of experiments (innovation in health with Article 51 in particular), innovative devices and care paths. Part of his activity also concerns the design and implementation of pharmaco-epidemiological observational studies and epidemiological studies or evaluations in the field of occupational health. Nearly 25 years spent at CEMKA have allowed her to acquire a solid experience, to implement diversified and complementary methodologies, both quantitative and qualitative or organizational, and to work for all types of actors. Today, she has conducted more than 200 studies in these different fields. Before joining CEMKA (EVAL) in 1994, Anne DUBURCQ worked as a statistician and study manager at INSERM (U170), devoted to epidemiological and statistical research on the environment and health. She is a graduate of ENSAE (Ecole Nationale de la Statistique et de l'Administration Economique) and holds a DEA in Public Health (University of Paris XI, Kremlin-Bicêtre) and the ECORISQUE diploma in the field of environmental risks (training coordinated by the Institut National de Veille Sanitaire, the Ecole Nationale de Santé Publique and EDF) She is also a member of the Association des Epidémiologistes de Langue Française (ADELF), the Société Française de Santé Publique (SFSP) and the Société Française d'Evaluation (SFE).
ROUND TABLE "WHAT ETHICS OF INNOVATION FOR RARE DISEASES?"
Pr Alain LOUTE is a philosopher by training, and is now professor of clinical ethics at the Faculty of Medicine and Dentistry of the Catholic University of Louvain (Belgium). He is affiliated with the Institute for Health and Society Research of the same university. He is a member of the French Society of Digital Health. His research focuses on the ethics of digital health.
Dr. Laurent PASQUIER is a clinical geneticist at the University Hospital of Rennes, France, specializing in malformative syndromes and neurodevelopmental disorders at all ages of life. More recently, he has focused his research activity on the sociological, legal and ethical issues related to the possibility of numerous genetic tests on the scale of an entire population.
Pr Caroline DESOMBRE is a professor of social psychology (PSITEC laboratory, University of Lille). She is also Deputy Director in charge of research and development at the Institut National Supérieur du Professorat et de l'Education. Her work focuses on the obstacles to the inclusion of students with special educational needs in school and the effect of stereotypes on the behavior of individuals.
Dr. Sophie FANTONI-QUINTON is a doctor of medicine, doctor of law, PUPH in medicine and occupational health (CHRU Lille) specialized in professional integration and job retention in the face of chronic diseases and disabilities and Ministerial Advisor in 2021 in charge of occupational health issues at the Ministry of Labor.
Mr Jean Denys WAELES has been leading innovation activities in the Hauts-de-France region for Bpifrance since 2021. With an annual budget of more than €70M, Jean-Denys Waeles manages and leads a team of 6 engineers in charge of innovation financing within the Lille Regional Office. Finally, he is in charge of representing and promoting Bpifrance to institutions and partners (Banks, State, Region, Investment and Seed Funds, Investment Committee).
Ms. Brigitte COURTOIS is the head of the Research and Innovation Department of the Lille University Hospital, one of the largest structures of its kind in France with nearly 350 employees, some twenty research units (of which the University Hospital is co-supervisor), and a multi-sector CIC. It participates in the definition of the research and innovation strategy of the University Hospital, pilots the main structuring projects and is involved in the coordination of relations with the University of Lille, the EPST and EURASANTE. At the same time, she is the Director of the G4 Health Cooperation Group which associates the University Hospital Centres of Amiens, Caen, Lille and Rouen on the major themes of the HU missions. In particular, she contributed to the establishment of the first G4 University Hospital Federations and laid the foundations for a future inter-regional hub for massive health data. As a hospital director for 24 years, Brigitte COURTOIS has acquired a solid experience of the health world, both in general hospitals and in the specific HU environment.
Mr. François-Xavier Denimal is a Doctor of Pharmacy, majoring in industry, with a Master's degree in Business Administration and a Certificate of Specialized Studies in Bio-statistics. He has 25 years of experience in sales and marketing management positions in healthcare companies specialized in medical devices and/or in vitro diagnostic medical devices. He has been 3 times pharmacist in charge of primary and secondary pharmaceutical establishments. He started at SATT Nord in January 2013 as the sole business developer (all technology areas), then managed the medical devices and in vitro diagnostic medical devices portfolio and some therapeutic files. Later he was assistant to veterinary therapeutic projects and more recently health software and bacteriology related ones.
Pr Rebecca DEPREZ-POULAIN holds a BS in Chemistry (Juniata College, PA, USA 1995), an engineering degree and an MSc in Organic Chemistry (HEI and Université de Lille, 1996), and a PhD in Pharmacy (Université de Lille, 2009). She completed her PhD under the supervision of Pr Tartar (1996-1999) in combinatorial chemistry applied to drug discovery within the company CEREP.SA. After a postdoctoral fellowship at the Pasteur Institute of Lille, she was appointed Senior Lecturer in General Chemistry in 2001 and is, since 2010, Professor of Medicinal Chemistry and Drug Discovery at the Faculty of Pharmacy of Lille. Her research expertise focuses on metalloprotease inhibition for therapeutic uses. She is currently coordinating the European project CAPSTONE-ETN (www.capstone-etn.eu) dedicated to ERAP modulation in immuno-oncology and autoimmune diseases. She has published more than 70 articles in international peer-reviewed journals, including J. Med. Chem, Nat. Comm, Nat. Med. and is the inventor of several patents. Prof. Deprez-Poulain was a member of the Institut Universitaire de France (2015-2020) and has been the recipient of several awards including the SCT/Servier Research Encouragement Award (2009) and the Michel Delalande Award in Pharmacochemistry from the Académie Nationale de Pharmacie (2020). She is currently the president of the French Society of Therapeutic Chemistry.
Pr Laurent STORME is a neonatal resuscitator, Head of the Neonatology Department at the Jeanne de Flandre Hospital at the University Hospital of Lille, member of the ULR 2694 METRICS multidisciplinary research team in Public Health at the University of Lille and Coordinator of the Reference Center for Rare Disease of Diaphragmatic Cup Hernia, with a recognized expertise in neonatal resuscitation and long-term medical follow-up of children with this rare disease
Alnylam is one of the few companies that have been able to transform this scientific approach into a therapeutic reality for patients. Alnylam's ambition remains to find drugs that meet unmet therapeutic needs and have a high public health impact. The understanding and mastery of RNAi technology has enabled the company to create and improve its own technological platform, which is the driving force behind its research.
With a broad portfolio of approved drugs in development, Alnylam is participating in the paradigm shift in the management of rare and prevalent diseases with subcutaneous injections every 3 to 6 months.
Dr. Adbellah TEBANI is a senior lecturer at the University and a hospital practitioner with international experience in care, research and teaching. He is passionate about all innovations at the intersection of medicine, artificial intelligence and digital health, a convergence that is essential for the emergence of precision medicine. Dr. Tebani has developed and implemented omic strategies for the diagnosis of Inherited Metabolic Diseases using advanced analytical technologies and machine learning techniques. His current activity includes clinical practice, laboratory management, R&D supervision and training. He co-leads a multidisciplinary team tasked with implementing precision medicine by leveraging multimodal biomedical data via computational modeling, machine learning and innovative intuitive data visualization strategies. He is co-founder of the international symposium "Pathways to Precision Medicine" on Precision Medicine.
Pr Alexandre BELOT is a pediatrician and researcher in immunology at the Hospices Civils de Lyon. He is the director of the RAISE-Lyon national reference center and is interested in rare pediatric onset inflammatory diseases. He co-leads the FAI2R rare diseases health network and pilots the steering committee for pediatric multisystemic inflammatory syndromes, which were identified in children in 2020 and correspond to a post-infectious inflammatory disease. His research work at the International Center for Infectious Diseases Research is focused on genetic and immunological explorations of early-onset inflammatory diseases, in particular juvenile lupus.
Dr Isabelle TALON was born in 1977 and is originally from Besançon. She is a pediatric surgeon at the Strasbourg University Hospital since 2007, joined the biomaterials and bioengineering research team in 2017 and created the DIAPID project in 2019. It is therefore a very young but solid and promising project due to its collaborative aspect. No less than 5 laboratories of excellence work hand in hand around the DIAPID innovation, 4 in Strasbourg (Inserm UMR1121, CNRS CAMB, ICube, ICPEES) and Metrics in Lille.
Anne DUBURCQ is in charge of the Public Health/Epidemiology division of CEMKA. Her activity is mainly focused on the evaluation of actions, programs and health policies, as well as on the evaluation of experiments (innovation in health with Article 51 in particular), innovative devices and care paths. Part of his activity also concerns the design and implementation of pharmaco-epidemiological observational studies and epidemiological studies or evaluations in the field of occupational health. Nearly 25 years spent at CEMKA have allowed her to acquire a solid experience, to implement diversified and complementary methodologies, both quantitative and qualitative or organizational, and to work for all types of actors. Today, she has conducted more than 200 studies in these different fields. Before joining CEMKA (EVAL) in 1994, Anne DUBURCQ worked as a statistician and study manager at INSERM (U170), devoted to epidemiological and statistical research on the environment and health. She is a graduate of ENSAE (Ecole Nationale de la Statistique et de l'Administration Economique) and holds a DEA in Public Health (University of Paris XI, Kremlin-Bicêtre) and the ECORISQUE diploma in the field of environmental risks (training coordinated by the Institut National de Veille Sanitaire, the Ecole Nationale de Santé Publique and EDF) She is also a member of the Association des Epidémiologistes de Langue Française (ADELF), the Société Française de Santé Publique (SFSP) and the Société Française d'Evaluation (SFE).