uniQure Reports 52-Week Clinical Data of Etranacogene Dezaparvovec in P-III HOPE-B Trial for Hemophilia B

Shots:

• The P- III HOPE-B gene therapy trial involves assessing etranacogene dezaparvovec in 54 patients with severe and moderately hemophilia B
• The results showed that the patients achieved sustained increases in FIX activity @ 1 yr. post-infusion with a mean FIX activity of 41.5% compared to 39.0% @ 26-wks of follow-up, reduced the ABR by 80% from 3.39 at baseline to 0.68 bleeds & 85% from 1.16 at baseline to 0.18 bleeds/yr from single-dose @ 1 yr. & generally well-tolerated with no treatment-related AEs
• uniQure to conduct 1EPs analysis of ABR @78 wks. after the administration and expects to complete at the end of Q3’21 while UniQure & CSL Behring expects to submit the BLA in Q1’22

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