Dr Austen Worth

Dr Austen Worth is a Paediatric Immunologist at Great Ormond Street Hospital (GOSH), and associate professor at UCL. His clinical work focuses on the diagnosis and management of severe immunodeficiency. This includes working up patients with inborn errors of immunity for curative therapies such as haematopoietic stem cell transplant, gene therapy and thymic transplant, as well as developing novel conservative approaches using targeted therapy with novel and re-purposed drugs. GOSH is one of two nationally commissioned services for the management of severe paediatric immunodeficiency and manages one of the largest cohorts of these patients in Europe.   Dr Worth’s doctoral and post-doctoral research at UCL focused on the molecular pathophysiology of Wiskott Aldrich Syndrome and dendritic cell motility defects. Subsequently his research interests have broadened to include thymic transplantation, EBV infection in immunodeficient hosts, XIAP deficiency, and novel immunedysregulatory disorders. He has over 100 publications in peer reviewed journals and is currently principal investigator on 5 multicentre clinical trials or studies.   He is currently the Associate Medical Director for Therapeutics at GOSH and since 2021 he has been Chair of the Drugs and Therapeutics Committee. He was clinical lead for the Immunology and Gene Therapy department at GOSH between 2016-21 and since 2024 he and is the clinical lead for the European Thymic Transplantation program.   He is an active member of ESID, on the scientific committee for the EBMT Inborn Errors Working Party, is on the steering committee for the British Society of Immunology Clinical Immunology Professionals Network (BSI-CIPN) and is on the UK Immunodeficiency (UKPID) Registry committee. He is extensively involved in the UK implementation of newborn screening for SCID, sitting on various committees and is an Immunology advisor for the Generation Study (newborn screening by whole genome sequencing). He is a member of several RCPCH clinical guidelines committees including the PANS / PANDAS clinical guideline group and the vaccination of immunodeficient children group, and is a member of European consensus guideline groups for SCID newborn screening and asplenia post chemotherapy. He has been an expert advisor to NICE on two technology appraisals. He has previous experience of working in health development in sub-Saharan Africa, and was Chair of the RCPCH VSO Fellowship committee for 4 years.

Primary Immunodeficiency Wiskott Aldrich Syndrome XIAP deficiency Inflammatory bowel disease associated with inborn errors of immunity Treatment by haematopoietic stem cell transplantation and thymic transplantation Haemophagocytic lymphohistiocytosis Immune dysregulatory disorders