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AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years
Highlights From the 62nd ASH Annual Meeting and Exposition

AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years

Frank W.G. Leebeek, MD, PhD1, Karina Meijer, MD, PhD2, Michiel Coppens, MD3, Peter Kampmann, MD4, Robert Klamroth, MD, PhD5, Roger Schutgens, MD, PhD, MSc6, Giancarlo Castaman, MD7, Erhard Seifried, MD, PhD8, Joachim Schwaeble9, Halvard Bönig1,10, Eileen K K Sawyer, PhD11, and Wolfgang A. Miesbach, MD12

1Erasmus University Medical Center, Rotterdam, Netherlands

2Dept. of Hematology, University of Groningen, University Medical Center Groningen, Groningen, Netherlands

3Amsterdam University Medical Center, University of Amsterdam, Amsterdam, Netherlands

4Rigshospitalet, Copenhagen, Denmark

5Department for Internal Medicine, Vascular Medicine and Haemostaseology, Vivantes Klinikum im Friedrichshain, Berlin, Germany

6Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands

7Azienda Ospedaliera Universitaria Careggi, Florence, Italy

8Institute Frankfurt, German Red Cross Blood Service Baden Wurttemberg-Hessen, Frankfurt, Germany

9Institute Frankfurt, German Red Cross Blood Service Baden-Wurttemberg-Hessen, Frankfurt, Germany

10University of Washington School of Medicine, Seattle, WA

11uniQure Inc., Lexington, MA

12Universitatsklinikum Frankfurt, Frankfurt, Germany

Key Data Points

PHASE I/II STUDY DESIGN

Data Points

This figure summarizes the study design for the phase 1/2 study of AMT-060. The study was open-label with dose escalation. Cohort 1 (n = 5) received a single infusion at a dose of 5x1012 gc/kg and cohort 2 (n = 5) received a single infusion at a dose of 2x1013 gc/kg. Duration of follow-up is 5 years for cohort 1 and 4.5 years for cohort 2.

Image

During 5 years (cohort 1) and 4.5 years (cohort 2) of follow-up, mean endogenous FIX activity was 5.2% (95% CI 2.0–8.4) in cohort 1 and 7.4% (95% CI 4.2–10.6) in cohort 2. FIX activity was dose dependent and stable during the follow-up period for both cohorts. Values in parentheses represent mean FIX activity over time. Only values at least 10 days after last FIX concentrate administration are included.

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