Claire Booth – Cofounder
Claire Booth is a Professor of Gene Therapy and Paediatric Immunology and UCL Great Ormond Street Institute of Child Health in London and a Consultant Paediatric Immunologist at Great Ormond Street Hospital. She is a clinical academic working in the field of inborn errors of immunity (IEI) and gene therapy with a skill set encompassing pre-clinical laboratory research, translational studies, Phase I/II clinical trials, GMP manufacture, ATMP commercialisation and clinical management of patients with IEI, including hematopoietic stem cell transplantation. She is currently Principal Investigator of 7 early phase lentiviral clinical trials and co-investigator on several others. Her lab focuses on the development of gene therapy strategies for rare IEIs including gene editing techniques to correct haematopoietic stem cells and T cells. She has served on the Boards of the British and European societies for Gene and Cell therapy and chaired the International Committee of the American Society of Gene and Cell Therapy. Claire serves on the editorial board of several gene therapy journals and holds an honorary position at Boston Children’s Hospital and Harvard Medical School.
Arjan Lankester – Cofounder
Arjan Lankester is professor of Pediatrics and Stem Cell Transplantation at the Willem-Alexander Children’s Hospital in the Leiden University Medical Center in Leiden, The Netherlands. Since 2009 he is clinical director of the JACIE-accredited Pediatric Stem Cell Transplantation program which serves as national center for stem cell therapy in pediatric patients with non-malignant disorders. He is heading the LUMC expert center on inherited immune disorders and stem cell transplantation (SCT) which is full member of ERN-RITA. His primary research interest is to improve efficacy and safety of stem cell therapy with particular focus on optimizing conditioning regimens and immune reconstitution after SCT. He has conducted and coordinated many single and multicenter studies including investigator-initiated adoptive cellular therapy trials. He is PI of the ongoing RECOMB trial on lentiviral stem cell gene therapy in RAG1 severe combined immunodeficiency. He is the past chair of Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and European Society for Immune Deficiencies.
Fabio Candotti – Board member
Fabio Candotti is Full Professor of Medicine at the University of Lausanne and Head Physician in the Division of Immunology and Allergy of the Lausanne University Hospital, in Lausanne, Switzerland. From 1998 to 2014, Dr. Candotti was a Tenured Investigator and Head of the Disorders of Immunity Section of the National Human Genome Research Institute of NIH where he focused his clinical and research activities on primary immunodeficiencies (PIDs), with particular interest on adenosine deaminase (ADA) deficiency and the Wiskott-Aldrich syndrome. During this time, he contributed to design and implementation of five clinical gene therapy trials for ADA deficiency.
In 2014, Prof Candotti joined the University Hospital of Lausanne, where he heads the Clinical Unit on PIDs of the Division of Immunology and Allergy. He currently serves as President of the European Society for Immunodeficiency (ESID), Associate Editor of Pediatric Allergy and Immunology, and Specialty Chief Editor of the Primary Immunodeficiencies section of Frontiers.
Johan Prevot – Board member
Johan Prevot has worked in the healthcare sector since 2001 in the field of patient advocacy and health policy. He is the Executive Director of the International Patient Organisation for Primary Immunodeficiencies (IPOPI) which he joined in 2010. As such he is responsible for the implementation of IPOPI’s global activities, awareness and advocacy campaigns as well as the strengthening of IPOPI’s national member organisations network.
Johan Prevot is currently a Board member of several other organisations including the European Reference Network on Rare Primary Immunodeficiency, Autoinflammatory and Autoimmune diseases (ERN-RITA), the AGORA Foundation (Access to Gene Therapies for Rare Diseases) and the RECOMB research programme. He is also a Steering Committee Member of the Platform of Plasma Products Users (PLUS), and Screen4Rare (newborn screening multi stakeholder platform). Previously, he worked as Director of Health Policy Europe for the Plasma Protein Therapeutics Association (PPTA) until 2010 and served as a Board member of Health First Europe (HFE) until 2023.
Johan Prevot has throughout his career been an advocate for improving patient access to early diagnosis and treatment in the field of primary immunodeficiencies and other rare plasma related disorders. Access to diagnosis and treatment for primary immunodeficiencies and other rare plasma related disorders varies greatly from country to country and many people living with these conditions in developing countries still nowadays can not access their life enhancing and/or life saving therapies. He regularly represents patients’ interests in policy discussions relating to early diagnosis and access to rare diseases therapies. He has also been an advocate for promoting equitable access to newborn screening and played a key role in the launch of the International Neonatal Screening Day (INSD). He has and continues to work closely with other stakeholder organisations sharing common objectives and priorities.
Alessandro Aiuti – Board member
Alessandro Aiuti is M.D., Ph.D., Haematologist. He is Deputy Director of the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan and Head of the Unit on Pathogenesis and Therapy of Primary Immunodeficiencies; Professor of Pediatrics, Vita-Salute San Raffaele University in Milan, Italy: Head of the Pediatric Immunohematology Unit, San Raffaele Hospital, Milan. He is author of more than 280 peer reviewed publications and received several awards. His main research interests are in the field of pediatric hematology and immunology, particularly in the pathogenesis and treatment of primary immunodeficiency. Dr. Aiuti has pioneered the EU market approval of the first ex vivo gene therapy for a genetic disease and led the successful clinical application of gene therapy with hematopoietic stem/progenitor cell for the treatment of inherited diseases of the immune system, inborn errors of metabolism and other blood disorders.