A Debate on the Use of Gene Therapy in Patients with Haemophilia

Abstract

GENE therapy is an innovative approach to treating haemophilia A and haemophilia B, with the potential to increase quality of life, promote prophylaxis, and even achieve curative factor levels in some cases. Despite early success in recent clinical trials, gene therapy for treating haemophilia is a relatively new area of research, and the long-term safety and efficacy are yet to be determined. Additionally, the current high price limits access for most patients. The suitability, safety, and accessibility of gene therapy for patients with haemophilia were discussed during a highly engaging debate session at the European Haematology Association (EHA) Congress 2024, titled ‘Haemophilia: Gene Therapy Access for Patients?’.