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In this prospective cohort study, a single intravenous dose of scAAV2/8-LP1-hFIXco gene therapy in men with severe hemophilia B led to sustained factor IX expression and significantly reduced bleeding ...

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The first haemophilia B patient in Germany has been treated with the one-time gene therapy HEMGENIX at the Hemophilia Treatment Center at Hannover Medical School, CSL Behring Germany has announced.

CSL Behring Germany today announced that the first haemophilia B patient in Germany was treated with the gene therapy HEMGENIX® (etranacogene dezaparvovec) at the Hemophilia Treatment Center at ...

Primary Source New England Journal of Medicine Source Reference: Reiss UM, et al "Sustained clinical benefit of AAV gene therapy in severe hemophilia B" N Engl J Med 2025; DOI: 10.1056/NEJMoa2414783.