Factor VIII Gene Therapy – November, 2016


Elie Dolgin has a very thoughtful article on the status of hemophilia gene therapy in the October, 2016 issue of Nature Biotechnology (1).  This article should be read along with the review in Seminars in Thrombosis and Hemostasis by Giangrande (2) which has previously discussed on this site.  I would further direct the reader to a note by Antonio Regalado in MIT Technology Review (November/December, 2016) entitled “Gene-Therapy Cure Has Money-Back Guarantee.”  It would be nice to think that gene therapy is coming either through the gene augmentation approaches described by Dolgin or by actual repair of the defective gene such as may be possible with the use of designer nucleases such as CRISPR/cas 9.  Remaining to developed is a business strategy which will satisfy the various stake holders from investors to third-party payers.


1.  Dolgin, E., Early clinical data raise the bar for hemophilia gene therapies, Nature Biotechnology 14, 999-1001, 2016
2.  Giangrande, P., The future of hemophilia treatment: Longer-acting factor concentrates versus gene therapy, Sem.Thromb.Hemost. 42, 513-517, 2016