J Haem Pract 2015; 2(2):26-28. doi: 10.17225/jhp00058

Authors: Amit Nathwani

Professor Amit Nathwani
Katharine Dormandy Haemophilia Centre & Thrombosis Unit, Ground floor, Royal Free Hospital, Pond Street, London NW3 2QG, UK. Email:


AAV-mediated gene therapy has the potential to be paradigm shifting as a treatment for haemophilia. A single administration of AAV vector can result in safe and consistent long-term expression of transgene (>5 years), reduction in spontaneous bleeding episodes, reduction in clotting factor usage and an improvement in quality of life. There is huge commercial interest in this approach, with the expectation that an AAV gene therapy product for haemophilia B will be licensed by 2020.


The author would like to thank the patients of the Royal Free Hospital. Their bravery permitted us to perform a proof-of-concept, first-in-man study without any expectation of benefit. In addition, thanks to the staff of UCL/Royal Free Hospital (particularly Edward Tuddenham), and of the St Jude Children’s Research Hospital (particularly Andrew Davidoff).




  1. Morris J. Patient advocacy helps patients weigh up gene therapy trial risk/benefits. J Haem Pract 2015; 2(1): 6-8. doi: 10.17225/jhp.00040. Avaialble at
  2. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365(25): 2357-65. doi: 10.1056/NEJMoa1108046.

The Journal of Haemophilia Practice is published by Haemnet.

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