UniQure announces positive 52-week clinical data from Phase III HOPE-B pivotal trial of investigational adeno-associated virus five-based gene therapy, eranacogene dezaparvovec, in patients with haemophilia B

Study (n=54) showed that a single dose significantly reduced annualised rate of bleeding requiring treatment by 80% from 3.39 at baseline to 0.68 bleeding episodes per year (p<0.0001). This gene therapy has orphan drug status in EU; submission to FDA is planned for early 2022.

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Biospace Inc.