Mind-blowing' data raise excitement around early stage hemophilia A candidate

Striking results from hemophilia A trials carried out at research institutes in London have been described as potentially “transformational” by the lead investigator.

Professor John Pasi said: "It's ground-breaking because the option to think about normalizing levels in patients with severe hemophilia is absolutely mind-blowing."

The 18-month data from the Phase I/II trial, published in the New England Journal of Medicine, show sustained factor VIII levels, within the normal range, for most patients with severe hemophilia A.