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

Pipe, S. W., Recht, M., Key, N. S., Leebeek, F. W. G., Castaman, G., Lattimore, S. U., … Miesbach, W. A. (2020). 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. Blood, 136. https://doi.org/10.1182/BLOOD-2020-143560