Gene Therapy in Haemophilia: Updates from Clinical Trials and Insights to Future Technologies

Abstract

At the International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress, held 9th–13th July, multiple oral and poster presentations were dedicated to gene therapy as a treatment for haemophilia A or B. These included updates from clinical trials of adeno-associated virus (AAV)-based gene therapy products and guidance on the real-world monitoring of patients with haemophilia who have received gene therapy, both in the short- and long-term. The unmet needs and challenges associated with gene therapy were also discussed, and several preclinical studies that aimed to refine AAV-based strategies were presented. Finally, there were a number of presentations providing an insight into the ongoing research into alternative gene therapy strategies, including the use of non-viral gene transfer, gene editing strategies, and nanobodies.