First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Wolfgang Miesbach ,Nigel S Key,Guy Young,Frank W.G Leebeek,Karina Meijer,Richard S Lemons,Allison P Wheeler,V Colletta ,Stephanie Verweij,Miguel A Escobar,Michael Recht,Robert Gut,Adam Kotowski,Michiel Coppens,Shelley E Crary ,Naghmana Bajma,Susan Lattimore ,Michael Wang,Cédric Hermans ,Steven W Pipe,Annette Von Drygalski,Niamh O’connell ,Kathelijne Peerlinck,Emily Symington,Nathan Visweshwar,Robert Klamroth,Jan Astermark,Paul Van Der Valk,Peter Kampmann,Esteban Gomez,John Pasi,Doris Quon,Eileen K Sawyer ,Giancarlo Castaman,Rashid Kazmi,Rebecca Kruse‐Jarres

doi:10.1182/blood-2020-143560

Abstract

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

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