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.

血友病是一种罕见的疾病，其批准的治疗选择实际上已维持50年不变。然而，在过去的十年中，无论是在开发中还是已被批准用于血友病的治疗方案中，都有创新的爆炸式增长，包括工程化凝血因子和大量新方法和新方法。这些新方法中的几种，特别是基因治疗，证明了血友病的原理证明，但可能具有更广泛的应用。这些进步与更好的诊断方法相结合，现在使临床医生能够改善对血友病患者的护理标准。这些疗法中使用的不同作用机制和修饰对其安全和有效使用产生影响，必须与它们的治疗效用相平衡。