Gene therapy treatment of haemophilia

16 December 2011 | UCLB News

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 Haemophilia 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