Gene therapy boosts factor IX activity and reduces bleeding events in patients with haemophilia B, study finds

A single treatment with a vector carrying an engineered factor IX transgene achieved a sustained increase in the clotting factor’s coagulant activity in patients with haemophilia B and reduced bleeding events, results from a small open label trial showed.1 Haemophilia B results from a deficiency or dysfunction of coagulation factor IX, which leads to recurrent spontaneous haemarthrosis. It is currently treated with exogenous factor IX that is infused one to three times each week to prevent bleeding or given on demand when bleeding occurs. But this causes peaks and troughs of coagulant activity …