Haemophilias, genetic disorders that cause uncontrolled bleeding, affect ~400,000 individuals globally. Both haemophilia A and B arise from deficiency in a specific protein required for normal blood clotting. In both cases, the standard treatment is intravenous injection of the missing clotting factor, but this is not fully effective and about a third of patients develop inhibitory antibodies to this.

Now Jim Huntington’s group at the Cambridge Institute for Medical Research have devised a potential new therapeutic approach, one that aims to allow more time for blood clot formation by inhibiting the anticoagulant enzyme activated protein C (APC) (Blood 2016). In collaboration with Trevor Baglin at Addenbrooke’s Hospital, they engineered a protein that selectively inhibits APC, and thereby rescues thrombin generation and blood clot formation in vitro. It is effective in mouse models of haemophilia B and also on blood samples from haemophilia patients.

Their goal is to develop this agent into a once-fortnightly subcutaneous treatment for all haemophilia sufferers, including those in the developing world who currently have no access to treatment. To achieve this, Jim and Trevor have founded the company ApcinteX, with help from Cambridge Enterprise and funding from Medicxi.