UniQure announced that it had received $100m from this first commercial sale milestone, on top of a $450m upfront payment. Once administered, the body will then be able to produce factor IX and prevent severe bleeds.Īlready a big player in haemophilia treatments, CSL licensed exclusive global rights to Hemgenix from gene therapy developers UniQure back in 2021. This protein helps to form blood clots and stop bleeding, with haemophilia B arising when the body is partially or completely unable to produce it. Hemgenix works by dosing a patient with an engineered adeno-associated virus (AAV), containing the gene responsible for producing a protein called factor IX. In a 20 June press release, CSL Behring’s North America general manager and senior vice-president Bob Lojewski said that the company was “thrilled” by this latest milestone and looked forward to more patients benefitting from the therapy.
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