Gene therapy company Spark Therapeutics has published data showing its one-time treatment can almost entirely eliminate bleeds in patients with haemophilia B.
A double trial success from Roche sees it eyeing a combination therapy in lucrative first-line lung cancer, and wider use of its just-approved haemophilia drug, Hemlibra.
The received wisdom is that a generation of new products in haemophilia A, led by Roche’s emicizumab and potentially curative gene therapies, will bring about a transformation of treatment