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News & Trends - Biotechnology

UniQure’s gene therapy potential in haemophilia B

Health Industry Hub | December 13, 2019 |

uniQure, a leading gene therapy company announced updated clinical data on the three patients treated in uniQure’s ongoing Phase IIb study of etranacogene dezaparvovec, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.

In addition, uniQure presented up to 4 years of follow-up data on the 10 patients in the Phase I/II trial of AMT-060, its first-generation gene therapy for the treatment of hemophilia B. These clinical data are being presented this weekend in poster presentations at the 61st Annual Meeting of the American Society of Hematology (ASH), taking place in Orlando, Florida.

Key Outcomes

  • Sustained FIX Activity at Therapeutic Levels Up to 50% of Normal at One Year After Administration of Etranacogene Dezaparvovec in Phase IIb Study
  • Zero Bleeds and No Requirement for Immunosuppression in One Year Following Dosing with Etranacogene Dezaparvovec
  • Long-term Clinical Benefit and Tolerability of AMT-060 Maintained in All Patients Through up to 4 Years of Follow-Up

“We are very pleased with these latest results, and continue to believe that etranacogene dezaparvovec has the potential to be the first- and best-in-class gene therapy for patients with hemophilia B,” stated Robert Gut, M.D., Ph.D., chief medical officer of uniQure. “We remain focused on dosing all patients in our ongoing, fully-enrolled HOPE-B pivotal trial, and expect to announce top-line data on our primary endpoint of Factor IX activity by the end of 2020.”

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