Type A haemophilia, the idea that made gene therapy more effective

The principle is the same: to provide what is missing. But the difference is substantial: in one case what is missing comes from the outside, it must always be available and taken on at regular intervals. In the other it is enough – or more cautiously it would be enough – a single administration to ensure a continuous endogenous production, through gene therapy.

Let’s talk about coagulation factor VIII, absent in type A haemophilia: a study, just published in the New England Journal of Medicine, shows that it is possible to ensure its production for more than two years in some haemophilia patients, reducing the risk of bleeding.