Haemophilia A Gene Therapy

Haemophilia A Gene Therapy using a highly-expressing FVIII Sequence

Available For: Licensing

Summary:

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 c.loryman@uclb.com