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Claire Booth

Claire Booth

Professor of Gene Therapy and Paediatric Immunology, UCL
Claire is Professor of paediatric immunology and gene therapy at UCL Great Ormond Street Hospital Institute of Child Health in London and leads the clinical stem cell gene therapy programme at Great Ormond Street Hospital. She is a clinical academic leading an expanding number of gene therapy clinical trials for patients with immune deficiencies, haematological and metabolic disorders. Her research is focused on developing novel therapies for immune system disorders using innovative gene modification techniques. She has extensive experience of translating, leading, and delivering first in human clinical trials and the commercialisation pathway. As an attending physician she oversees the clinical management of patients with immune deficiencies, including hematopoietic stem cell transplantation and maintains a strong interest in HLH disorders.
Claire is an elected board member of the European and American Societies of Gene and Cell Therapy, serves on the editorial board of several journals and grant review committees and holds an honorary position at Boston Children’s Hospital and Harvard Medical School.
She is also the co-founder of the AGORA initiative (Access to Gene therapies fOr Rare disease) which brings together European academic developers and patient organisations, aiming to facilitate access to effective gene therapies for treatment of patients with ultra-rare diseases.

No commercial incentive to develop gene therapy – hospitals will try to fill the gap

Jun 13, 2024 06:12 am UTC| Insights & Views Business

Individually, rare diseases are rare, but there are so many rare diseases that over 400 million people are affected worldwide. Together, rare diseases arent so rare. Only 5% of rare diseases have an approved treatment...

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