Cure for hemophilia B steps closer to becoming reality

23 June 2021

Dutch gene therapy specialist uniQure (Nasdaq: QURE) has announced positive 52-week clinical data from the Phase III HOPE-B trial of etranacogene dezaparvovec.

The company is testing the investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of people with severe and moderately severe hemophilia B.

The result underscores a turnaround in fortunes for the program, after the US regulator lifted a clinical hold on the trial, imposed in December, due to an incidence of hepatocellular carcinoma (HCC).

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