Case ID: 94-075

Web Published: Dec 15, 2011


Haemophilia A Gene Therapy using a highly-expressing FVIII Sequence

Available for: Licensing


We have developed a gene therapy for Haemophilia A that utilises a novel promoter and a highly-expressed sequence for the clotting protein Factor VIII.

Building on our expertise in Haemophili B treatment with a Factor IX gene therapeutic, we are currently in pre-clinical development of our FVIII medicament. Planning is underway to take this technology into the clinc.

Market Opportunity

Haemophilia A is caused by a defiency 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 Heamophilia A are substantial.

Further Information

Please contact Chris Loryman, UCL Business PLC. T +44 (0)20 7679 9000 E

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