Haemophilia care has progressed through successive therapeutic revolutions, from plasma-derived and recombinant factor replacement to extended half-life products and non-factor therapies. The introduction of gene therapy, with currently approved approaches using adeno-associated viral vectors to deliver functional copies of the factor VIII or IX gene into hepatocytes, now represents the next paradigm shift: a one-time, irreversible intervention designed to restore endogenous factor production. Although early results are promising, the experience of transfusion transmitted infections reminds the haemophilia community that innovation must be matched by vigilance, transparency, and accountability.
The Lancet Haematology published a clinical update in Oncology on 10 Mar 2026.
The item focuses on Building safeguards for gene therapy in haemophilia.
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