Fabio Candotti

Fabio Candotti is a full professor at the University of Lausanne (UNIL) in the Faculty of Biology and Medicine and serves as head physician in the Department of Immunology and Allergology at the Lausanne University Hospital (CHUV). A world-renowned expert in immunodeficiency, pediatrics, and gene therapy, he has held academic and research leadership roles in both the United States and Europe, including a long tenure at the National Institutes of Health (NIH).

• Born: 1962, Italian nationality
• Education: MD, University of Brescia (1987); specialization in pediatrics (University of Pavia) and immunology/allergology (Brescia)
• Postdoctoral training: National Institutes of Health (NIH), USA (1992)
• Academic appointments: Associate Professor at UNIL (2014), promoted to Full Professor (2022)
• Prior roles: Senior Investigator and Head of Immune Diseases Section, NHGRI/NIH; Head, NIH Gene Therapy Interest Group

His research and clinical work focus on primary immunodeficiencies, particularly adenosine deaminase deficiency (ADA) and Wiskott-Aldrich syndrome (WAS). He investigates the molecular and cellular mechanisms underlying immune dysregulation, autoimmunity, and metabolic defects in these disorders. His work has revealed how high adenosine levels and DNA repair deficiencies contribute to inflammatory, fibrogenic, and oncogenic processes in ADA-deficient patients. Using murine models, he has advanced understanding of autoimmune complications in WAS. A major focus of his work is the development and application of gene therapy strategies, including viral vector-based tools and transgenesis, for curative treatments.

The trends in his recent publications highlight sustained leadership in translational immunology and gene therapy, with recurring themes in immune reconstitution, metabolic consequences of immunodeficiency, long-term patient outcomes, and innovative therapeutic design. His work bridges basic science and clinical application, emphasizing durable correction of immune function and quality of life.

He has received multiple scientific awards, including:

• National Institutes of Health Award of Merit (1999, 2006)
• National Human Genome Research Institute Service Award (2002)
• US Government Service Award (2003, 2008)
• Best Docs in Northern Virginia (2009)

Candotti is actively involved in medical education, having taught at Metropolitan Washington D.C. Medical Genetics since 2004 and contributed to numerous fellowship and residency training programs in immunology and hematology. He is an adjunct member of the Graduate Faculty at the University of Medicine and Dentistry of New Jersey. He also contributes to the scientific community through editorial roles in journals such as Clinical and Translational Science, The Open Gene Therapy Journal, Frontiers in Primary Immunodeficiencies, and Journal of Clinical Immunology. He is a member of the Italian Society of Pediatric Allergology and Immunology, the American Society for Gene Therapy, and the European Society for Immunodeficiencies.

His work is supported by long-term patient cohorts and clinical studies aimed at developing new drugs and gene-based therapies. He leads a research team focused on advancing gene therapy for monogenic immune disorders, with a vision of translating laboratory discoveries into safe and effective clinical applications.