This article reviews presentations that took place at the 64th American Society of Hematology (ASH) Annual Meeting, between 10th–13th December 2022, in New Orleans, Louisiana, USA. Exploring gene therapy for the treatment of haemophilia A and B by highlighting key updates from clinical trials and pre-clinical data, this congress review discusses recombinant adeno-associated virus (rAAV)-based gene therapy products and non-viral gene therapy strategies in haemophilia treatment, and details the translation from research into clinical practice.
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