Claire Booth, MBSS, PhD

At-Large Director (2024-2027)

I am a clinical academic working in the field of primary immunodeficiency (PID) and gene therapy, leading gene therapy clinical trials at Great Ormond Street Hospital treating patients with immune deficiencies, haematological and metabolic disorders. I possess a skill set encompassing pre-clinical laboratory research, translational studies, phase I/II clinical trials and clinical management of patients with PID, including hematopoietic stem cell transplantation. I am currently Principal Investigator of phase I/II lentiviral clinical trials of haematopoietic stem cell gene therapy for ADA-SCID, X-SCID and Fanconi anaemia and co-investigator on numerous others. Through this aspect of my work I have acquired extensive clinical trial related operational and management experience incorporating regulatory framework, IMP manufacture, clinical pathways, orphan drug designation, industrial partnerships and commercialisation.

My own pre-clinical research focuses on the development of gene therapy strategies to treat X-linked lymphoproliferative diseases (XLP-1 and XIAP deficiency) using a conventional lentiviral mediated corrective strategy alongside exploration of novel gene editing techniques to facilitate targeted gene addition in both haematopoietic stem cells and T cells.

I am a consultant paediatric immunologist at Great Ormond Street Hospital which is a global centre of excellence and one of only two units commissioned for stem cell transplantation for primary immunodeficiency in the U.K. One of my clinical interests is haemophagocytic lymphohistiocytosis (HLH) and I am focused on improving outcome for HLH patients by developing new therapeutic strategies, including gene therapies. I also hold a position as visiting lecturer, Boston Children’s Hospital/ Dana Farber Cancer Institute, (pediatric hematology/oncology).