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

Steven W. Pipe; Michael Recht; Nigel S. Key; Frank W.G. Leebeek; Giancarlo Castaman; Susan U. Lattimore; Paul van der Valk; Kathelijne Peerlinck; Michiel Coppens; Niamh O'Connell; John Pasi; Peter Kampmann; Karina Meijer; Annette von Drygalski; Guy Young; Cedric Hermans; Jan Astermark; Robert Klamroth; Richard S. Lemons; Nathan Visweshwar; Shelley Crary; Rashid Kazmi; Emily Symington; Miguel A. Escobar; Esteban Gomez; Rebecca Kruse-Jarres; Adam Kotowski; Doris Quon; Michael Wang; Allison P. Wheeler; Eileen K. Sawyer; Stephanie Verweij; Valerie Colletta; Naghmana Bajma; Robert Gut; Wolfgang A. Miesbach

doi:10.1182/BLOOD-2020-143560

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

Steven W. Pipe, Michael Recht, Nigel S. Key, Frank W.G. Leebeek, Giancarlo Castaman, Susan U. Lattimore, Paul van der Valk, Kathelijne Peerlinck, Michiel Coppens, Niamh O'Connell, John Pasi, Peter Kampmann, Karina Meijer, Annette von Drygalski, Guy Young, Cedric Hermans, Jan Astermark, Robert Klamroth, Richard S. Lemons, Nathan VisweshwarShelley Crary, Rashid Kazmi, Emily Symington, Miguel A. Escobar, Esteban Gomez, Rebecca Kruse-Jarres, Adam Kotowski, Doris Quon, Michael Wang, Allison P. Wheeler, Eileen K. Sawyer, Stephanie Verweij, Valerie Colletta, Naghmana Bajma, Robert Gut, Wolfgang A. Miesbach

Hematology

Research output: Contribution to journal › Article › Academic › peer-review

18 Citations (Scopus)

Original language	English
Journal	Blood
Volume	136
DOIs	https://doi.org/10.1182/BLOOD-2020-143560
Publication status	Published - 19 Nov 2020

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10.1182/BLOOD-2020-143560

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Pipe, S. W., Recht, M., Key, N. S., Leebeek, F. W. G., Castaman, G., Lattimore, S. U., van der Valk, P., Peerlinck, K., Coppens, M., O'Connell, N., Pasi, J., Kampmann, P., Meijer, K., von Drygalski, A., Young, G., Hermans, C., Astermark, J., Klamroth, R., Lemons, R. S., ... 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

@article{7ccda7499fa7422faf28017e52e8b36d,

title = "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",

author = "Pipe, {Steven W.} and Michael Recht and Key, {Nigel S.} and Leebeek, {Frank W.G.} and Giancarlo Castaman and Lattimore, {Susan U.} and {van der Valk}, Paul and Kathelijne Peerlinck and Michiel Coppens and Niamh O'Connell and John Pasi and Peter Kampmann and Karina Meijer and {von Drygalski}, Annette and Guy Young and Cedric Hermans and Jan Astermark and Robert Klamroth and Lemons, {Richard S.} and Nathan Visweshwar and Shelley Crary and Rashid Kazmi and Emily Symington and Escobar, {Miguel A.} and Esteban Gomez and Rebecca Kruse-Jarres and Adam Kotowski and Doris Quon and Michael Wang and Wheeler, {Allison P.} and Sawyer, {Eileen K.} and Stephanie Verweij and Valerie Colletta and Naghmana Bajma and Robert Gut and Miesbach, {Wolfgang A.}",

year = "2020",

month = nov,

day = "19",

doi = "10.1182/BLOOD-2020-143560",

language = "English",

volume = "136",

journal = "Blood",

issn = "0006-4971",

publisher = "Elsevier",

}

Pipe, SW, Recht, M, Key, NS, Leebeek, FWG, Castaman, G, Lattimore, SU, van der Valk, P, Peerlinck, K, Coppens, M, O'Connell, N, Pasi, J, Kampmann, P, Meijer, K, von Drygalski, A, Young, G, Hermans, C, Astermark, J, Klamroth, R, Lemons, RS, Visweshwar, N, Crary, S, Kazmi, R, Symington, E, Escobar, MA, Gomez, E, Kruse-Jarres, R, Kotowski, A, Quon, D, Wang, M, Wheeler, AP, Sawyer, EK, Verweij, S, Colletta, V, Bajma, N, Gut, R & Miesbach, WA 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, vol. 136. https://doi.org/10.1182/BLOOD-2020-143560

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, Steven W.; Recht, Michael; Key, Nigel S. et al.
In: Blood, Vol. 136, 19.11.2020.

Research output: Contribution to journal › Article › Academic › peer-review

TY - JOUR

T1 - First data from the phase 3 HOPE-B gene therapy trial

T2 - 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

AU - Pipe, Steven W.

AU - Recht, Michael

AU - Key, Nigel S.

AU - Leebeek, Frank W.G.

AU - Castaman, Giancarlo

AU - Lattimore, Susan U.

AU - van der Valk, Paul

AU - Peerlinck, Kathelijne

AU - Coppens, Michiel

AU - O'Connell, Niamh

AU - Pasi, John

AU - Kampmann, Peter

AU - Meijer, Karina

AU - von Drygalski, Annette

AU - Young, Guy

AU - Hermans, Cedric

AU - Astermark, Jan

AU - Klamroth, Robert

AU - Lemons, Richard S.

AU - Visweshwar, Nathan

AU - Crary, Shelley

AU - Kazmi, Rashid

AU - Symington, Emily

AU - Escobar, Miguel A.

AU - Gomez, Esteban

AU - Kruse-Jarres, Rebecca

AU - Kotowski, Adam

AU - Quon, Doris

AU - Wang, Michael

AU - Wheeler, Allison P.

AU - Sawyer, Eileen K.

AU - Verweij, Stephanie

AU - Colletta, Valerie

AU - Bajma, Naghmana

AU - Gut, Robert

AU - Miesbach, Wolfgang A.

PY - 2020/11/19

Y1 - 2020/11/19

UR - http://www.scopus.com/inward/record.url?scp=85097772491&partnerID=8YFLogxK

U2 - 10.1182/BLOOD-2020-143560

DO - 10.1182/BLOOD-2020-143560

M3 - Article

AN - SCOPUS:85097772491

SN - 0006-4971

VL - 136

JO - Blood

JF - Blood

ER -

Pipe SW, Recht M, Key NS, Leebeek FWG, Castaman G, Lattimore SU et al. 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. 2020 Nov 19;136. doi: 10.1182/BLOOD-2020-143560

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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