Gene therapy treatment of haemophilia

Published on 16 December 2011

The recent publication by UCL (Professor Amit Nathwani), St Judes Children’s hospital and the Royal Free Hospital in the New England Journal of Medicine describes how a single infusion of this Factor IX treatment consistently leads to long-term expression of the missing protein. This missing protein is found at therapeutic levels, without acute or long-lasting toxicity in patients with severe haemophilia B.


The gene-therapy approach, even with the associated risk of transient hepatic dysfunction, has the potential to convert the severe bleeding phenotype into a mild form of the disease or to reverse it entirely. Physicians have experienced hope that follow-up studies will fully define the benefits, risks and optimize dosing.


UCLB, working with the team lead by Professor Nathwani is developing a gene therapy for Hemophilia A which utilises a novel promoter and a highly-expressed sequence for the clotting protein Factor VIII.


Haemophilia A is caused by a deficiency of the clotting factor “Factor VIII”. This is the most frequent clotting disorder, and has an incidence of approximately 1 in 5,000–10,000 male births. The direct (clotting factor therapeutics) and indirect (in-patient treatment) costs of treating haemophilia A are substantial.


Building on expertise in haemophilia B treatment with a Factor IX gene therapeutic, the UCLB technology is currently in pre-clinical development. Planning is underway to take this technology into the clinic within the next few years.



New England Journal of Medicine

UCL’s press release

Heamophilia A technology on UCLB technology directory

For further information, please contact Chris Loryman, Senior Business Manager: Email,, Telephone 020 7679 9000

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