Claire Booth

Claire Booth is 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, and haematological and metabolic disorders. She is a Consultant Paediatric Immunologist at Great Ormond Street Hospital, which is a global centre of excellence and one of only two units in the UK commissioned for stem cell transplantation for primary immunodeficiency.

Her skill set encompasses pre-clinical laboratory research, translational studies, Phase I/II clinical trials, and the clinical management of patients with PID, including haematopoietic stem cell transplantation. She is 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 work, she has acquired extensive clinical trial-related operational and management experience, including regulatory frameworks, IMP manufacture, clinical pathways, orphan drug designation, industrial partnerships and commercialisation.

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

One of her clinical interests is haemophagocytic lymphohistiocytosis (HLH); she is focused on improving outcomes for HLH patients by developing new therapeutic strategies, including gene therapies. She also holds a position as Visiting Lecturer at Boston Children's Hospital/Dana-Farber Cancer Institute (Paediatric Haematology/Oncology).