Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B
Highlights From the 14th Annual Congress of EAHAD

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial

Annette von Drygalski, MD, PharmD1, Adam Giermasz, MD, PhD2, Giancarlo Castaman, MD3, Nigel S. Key, MBChB4, Susan U. Lattimore, RN5, Frank W.G. Leebeek, MD6, Wolfgang A. Miesbach, MD, PhD7, Michael Recht, MD, PhD5, Esteban Gomez, MD8, Robert Gut, MD, PhD9, and Steven W. Pipe, MD10

1University of California San Diego, La Jolla, CA, USA

2University of California Davis, Sacramento, CA, USA

3Azienda Ospedaliera Universitaria Careggi, Florence, Italy

4University of North Carolina, Chapel Hill, USA

5Oregon Health and Science University, Portland, USA

6Erasmus University Medical Center, Rotterdam, The Netherlands

7University Hospital Frankfurt, Germany

8Phoenix Children's Hospital, Phoenix, USA

9uniQure Inc, Lexington, MA, USA

10University of Michigan, Ann Arbor, MI, USA

Key Data Points

FIX Activity After AAV5-Padua hFIX Vector Infusion

After infusion of 2x1013 gc/kg, the primary endpoint of FIX activity ≥ 5% at 6 weeks post-infusion was achieved in all 3 participants. The mean FIX activity at 2 years was 44.2%, and FIX activity was stable from year 1 to year 2.

Bleeds After AAV5-Padua hFIX Vector Infusion

Over 2 years, 2 of 3 participants had no bleeds or use of FIX concentrate. The third participant used a total of 2 infusions of FIX replacement therapy (1 suspected and 1 confirmed bleed) on separate occasions (excluding surgery). All participants remain prophylaxis-free 2 years after infusion.

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