Gene therapy for haemophilia - progress at last

Series Name Department of Genetics and Genome Biology
Speaker Professor Edward Tuddenham, Department of Haematology, UCL, London
Type Lectures & Talks
When 11 Oct 2017, 02:00PM - 03:00PM
Venue Bennett Link Lower Ground Floor LT
Open To University staff and students
For Bookings Contact Lisa Witherington 252 3659

Gene therapy provides hope of a cure for patients with haemophilia by establishing continuous endogenous expression of factor VIII or factor IX following transfer of a functional gene copy to replace the haemophilic patient’s own defective gene. Haemophilia may be considered a ‘‘low-hanging fruit’’ for gene therapy because a small increment in blood factor levels (≥ 2% of normal) significantly improves the bleeding tendency from severe to moderate, eliminating most spontaneous bleeds.

After decades of research, the first trial to provide clear evidence of efficiency after gene transfer in patients with haemophilia B using adeno-associated virus vectors was reported by the speaker’s group in 2011. This has been followed by unprecedented activity in this area, with the commencement of seven new early-phase trials involving >55 patients with haemophilia A or haemophilia B. These studies have, in large part, generated promising clinical data that lay a strong foundation for gene therapy to move forward rapidly to market authorization.