The GuardOne clinical trial: A first-in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease

被引：0
|
作者：
Jacobsen, Leslie [1 ]
Kerner, Julie A. [1 ]
Ciotti, Rachel [1 ]
Golipour, Azadeh [1 ]
Yang, Josie [1 ]
Pfeifer, Richard [1 ]
Khan, Aneal [2 ]
Mason, Chris [1 ]
机构：
[1] AVROBIO, Cambridge, MA USA
[2] Univ Calgary, Calgary, AB, Canada
来源：
MOLECULAR GENETICS AND METABOLISM | 2021年 / 132卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2020.12.112
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
106
引用
收藏
页码：S51 / S52
页数：2
相关论文
共 50 条
  • [41] An open-label, multicentre, dose-escalation, first-in-human phase I study to evaluate safety, tolerability and antineoplastic activity of OATD-02 (dual arginase 1 and arginase 2 inhibitor) in patients with selected advanced and/or metastatic solid tumors
    Dudek, M. A.
    Zaslona, Z.
    Blaszczyk, R.
    Grzybowski, M. M.
    Rejczak, T.
    Cabaj, A.
    Dera, P.
    Lisiecki, K.
    Iwanowski, P.
    Charitos, T.
    Fung, S.
    ANNALS OF ONCOLOGY, 2023, 34 : S495 - S495
  • [42] First-in-human, open-label, phase 1/2 study of the monoclonal antibody programmed cell death protein-1 (PD-1) inhibitor cetrelimab (JNJ-63723283) in patients with advanced cancers
    Felip, Enriqueta
    Moreno, Victor
    Morgensztern, Daniel
    Curigliano, Giuseppe
    Rutkowski, Piotr
    Trigo, Jose Manuel
    Calvo, Aitana
    Kowalski, Dariusz
    Cortinovis, Diego
    Plummer, Ruth
    Maio, Michele
    Ascierto, Paolo A.
    Vladimirov, Vladimir, I
    Cervantes, Andres
    Zudaire, Enrique
    Hazra, Anasuya
    T'jollyn, Huybrecht
    Bandyopadhyay, Nibedita
    Greger, James G.
    Attiyeh, Edward
    Xie, Hong
    Calvo, Emiliano
    CANCER CHEMOTHERAPY AND PHARMACOLOGY, 2022, 89 (04) : 499 - 514
  • [43] First-in-human, open-label, phase 1/2 study of the monoclonal antibody programmed cell death protein-1 (PD-1) inhibitor cetrelimab (JNJ-63723283) in patients with advanced cancers
    Enriqueta Felip
    Victor Moreno
    Daniel Morgensztern
    Giuseppe Curigliano
    Piotr Rutkowski
    José Manuel Trigo
    Aitana Calvo
    Dariusz Kowalski
    Diego Cortinovis
    Ruth Plummer
    Michele Maio
    Paolo A. Ascierto
    Vladimir I. Vladimirov
    Andres Cervantes
    Enrique Zudaire
    Anasuya Hazra
    Huybrecht T’jollyn
    Nibedita Bandyopadhyay
    James G. Greger
    Edward Attiyeh
    Hong Xie
    Emiliano Calvo
    Cancer Chemotherapy and Pharmacology, 2022, 89 : 499 - 514
  • [44] Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study
    Kohn, Donald B.
    Sevilla, Julian
    Rao, Gayatri
    Lopez, Maria Chitty
    Almarza, Elena
    Terrazas, Dayna
    Zubicaray, Josune
    Gonzalez-Vicent, Marta
    Chetty, Kritika
    O'Toole, Grainne
    Xu-Bayford, Jinhua
    Nicoletti, Eileen
    Fernandez, Augustine
    Kuo, Caroline Y.
    De Oliveira, Satiro N.
    Moore, Theodore B.
    Choi, Grace
    Zeini, Miriam
    Mesa-Nunez, Cristina
    Thrasher, Adrian J.
    Bueren, Juan A.
    Schwartz, Jonathan
    Booth, Claire
    MOLECULAR THERAPY, 2023, 31 (04) : 727 - 728
  • [45] A first-in-human, multicenter, phase 1/2, open-label study of XTX202, a masked and tumor-selective recombinant human interleukin-2 (IL-2) protein, in patients with advanced solid tumors
    McKean, Meredith
    Powderly, John D.
    Duncan, Meghan
    Norman, Teleen
    Patel, Ekta
    O'Neil, Jennifer
    Lee, George
    Luptakova, Katarina
    Kaufman, Howard E.
    JOURNAL OF CLINICAL ONCOLOGY, 2022, 40 (16)
  • [46] The First-in-Human Study of the Hydrogen Sulfate (Hyd-Sulfate) Capsule of the MEK1/2 Inhibitor AZD6244 (ARRY-142886): A Phase I Open-Label Multicenter Trial in Patients with Advanced Cancer
    Banerji, Udai
    Camidge, D. Ross
    Verheul, Henk M. W.
    Agarwal, Roshan
    Sarker, Debashis
    Kaye, Stan B.
    Desar, Ingrid M. E.
    Timmer-Bonte, Johanna N. H.
    Eckhardt, S. Gail
    Lewis, Karl D.
    Brown, Kathryn H.
    Cantarini, Mireille V.
    Morris, Clive
    George, Sarah M. A.
    Smith, Paul D.
    van Herpen, Carla M. L.
    CLINICAL CANCER RESEARCH, 2010, 16 (05) : 1613 - 1623
  • [47] Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1-2 study (vol 18, 1061, 2017)
    Perl, A. E.
    Altman, J. K.
    Cortes, J.
    LANCET ONCOLOGY, 2019, 20 (06): : E293 - E293
  • [48] Preliminary results of a phase 1/2, first-in-human, open-label, dose escalation study of ZL-1211 (anti-Claudin 18.2 mAb) in patients with unresectable or metastatic solid tumors
    Sharma, Sunil
    Starodub, Alexander
    Xu, Nong
    Chaudhry, Arvind
    Sun, Meili
    Pelster, Meredith
    Fu, Yinjia
    Zhang, Xinyu
    Huang, Zhao
    Liu, Wenyu
    Hsu, Karl
    JOURNAL OF CLINICAL ONCOLOGY, 2023, 41 (16)
  • [49] Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease
    Kanter, Julie
    Walters, Mark C.
    Hsieh, Matthew
    Thompson, Alexis A.
    Krishnamurti, Lakshmanan
    Kwiatkowski, Janet
    Kamble, Rammurti T.
    von Kalle, Christof
    Kuypers, Frans A.
    Cavazzana, Marina
    Leboulch, Philippe
    Sandler, Laura
    Soni, Sandeep
    Tisdale, John F.
    BLOOD, 2015, 126 (23)
  • [50] AN OPEN LABEL PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA
    Weinstein, David A.
    Ahmad, Ayesha
    Poma, Allen
    Crombez, Eric
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (03) : 333 - 333
12345