Advances and innovations in haemophilia treatment

New therapies in development for haemophilia — including gene therapies, proteins with extended half-lives and inhibitors of natural anticoagulants — look poised to change the management of this disease. In this Review, Peters and Harris discuss recent progress, including the hurdles that still need to be overcome in order to translate these advances into routine clinical use. Haemophilia is a rare disease for which the approved therapeutic options have remained virtually unchanged for 50 years. In the past decade, however, there has been an explosion of innovation in the treatment options that are either in development or have been approved for haemophilia, including engineered clotting factors and an extensive pipeline of new approaches and modalities. Several of these new modalities, especially gene therapy, demonstrate proof of principle in haemophilia but could have broader applications. These advances, in combination with better diagnostics, are now enabling clinicians to improve the standard of care for people with haemophilia. The different mechanisms of action and modifications used in these therapies have implications for their safe and efficacious use, which must be balanced with their therapeutic utility. This Review focuses on the biological aspects of the most advanced and innovative approaches for haemophilia treatment and considers their future use.