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
  • [1] 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
    Ridha, E.
    Jones, J. J.
    Abrahamsen, I.
    Golipour, A.
    Pfeifer, R.
    Khan, A.
    Mason, C.
    Escolar, D.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A80 - A80
  • [2] Preliminary Results from the Phase 1/2 GuardOne Trial: A First-in-Human Study of Ex Vivo Lentiviral Gene Therapy (AVR-RD-02) in People with Gaucher Disease Type 1
    Jacobsen, L. K.
    Trame, M. N.
    Golipour, A.
    Pfeifer, R.
    Khan, A.
    Mason, C.
    MOLECULAR THERAPY, 2021, 29 (04) : 340 - 340
  • [3] The Guard1 clinical trial - A first in-human, phase 1/2 study evaluating AVR-RD-02, a hematopoietic stem cell (HSC) gene therapy for Gaucher disease: Preliminary safety, pharmacodynamic and clinical efficacy results from the subjects observed for up to 24 months post-infusion
    Adera, Mathews
    Veselinovic, Milena
    Treohan, Anita
    Golipour, Azadeh
    Ridha, Essra
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 3 - 4
  • [4] Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial
    Palfi, Stephane
    Gurruchaga, Jean Marc
    Ralph, G. Scott
    Lepetit, Helene
    Lavisse, Sonia
    Buttery, Philip C.
    Watts, Colin
    Miskin, James
    Kelleher, Michelle
    Deeley, Sarah
    Iwamuro, Hirokazu
    Lefaucheur, Jean Pascal
    Thiriez, Claire
    Fenelon, Gilles
    Lucas, Cherry
    Brugieres, Pierre
    Gabriel, Inanna
    Abhay, Kou
    Drouot, Xavier
    Tani, Naoki
    Kas, Aurelie
    Ghaleh, Bijan
    Le Corvoisier, Philippe
    Dolphin, Patrice
    Breen, David P.
    Mason, Sarah
    Guzman, Natalie Valle
    Mazarakis, Nicholas D.
    Radcliffe, Pippa A.
    Harrop, Richard
    Kingsman, Susan M.
    Rascol, Olivier
    Naylor, Stuart
    Barker, Roger A.
    Hantraye, Philippe
    Remy, Philippe
    Cesaro, Pierre
    Mitrophanous, Kyriacos A.
    LANCET, 2014, 383 (9923): : 1138 - 1146
  • [5] Autologous Transplantation of P63+Lung Progenitor Cells for Chronic Obstructive Pulmonary Disease Therapy: An Open-label, Controlled, First-in-Human Trial
    Zhang, T.
    Wang, Y.
    Zhou, Y.
    Zhao, Y.
    Zuo, W.
    AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2024, 209
  • [6] AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: Overview of clinical data from phase 1 and phase 2 studies
    Thomas, Mark
    Nicholls, Kathleen
    Carnley, Benedict
    Trame, Mirjam
    Mason, Chris
    Tavakkoli, Fatemeh
    Keenan, Greg
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S106 - S106
  • [7] Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial), a First-in-Human Phase 1/2 Clinical Trial
    Jaski, Brian E.
    Jessup, Mariell L.
    Mancini, Donna M.
    Cappola, Thomas P.
    Pauly, Daniel F.
    Greenberg, Barry
    Borow, Kenneth
    Dittrich, Howard
    Zsebo, Krisztina M.
    Hajjar, Roger J.
    JOURNAL OF CARDIAC FAILURE, 2009, 15 (03) : 171 - 181
  • [8] Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
    Michaelides, Michel
    Laich, Yannik
    Wong, Sui Chien
    Oluonye, Ngozi
    Zaman, Serena
    Kumaran, Neruban
    Kalitzeos, Angelos
    Petrushkin, Harry
    Georgiou, Michalis
    Tailor, Vijay
    Pabst, Marc
    Staeubli, Kim
    Maimon-Mor, Roni O.
    Jones, Peter R.
    Scholte, Steven H.
    Georgiadis, Anastasios
    van der Spuy, Jacqueline
    Naylor, Stuart
    Forbes, Alexandria
    Dekker, Tessa M.
    Arulmuthu, Eugene R.
    Smith, Alexander J.
    Ali, Robin R.
    Bainbridge, James W. B.
    LANCET, 2025, 405 (10479): : 648 - 657
  • [9] CANaspire, a First-in-Human Phase 1/2 Controlled Open-Label Study of BBP-812, a Recombinant AAV9-hASPA Vector for the Treatment of Canavan Disease
    Eichler, F.
    Laforet, G.
    Andonian, H.
    Kinane, B.
    Kirby, K.
    Nagy, A.
    Shaywitz, A.
    Balser, J.
    Bley, A.
    ANNALS OF NEUROLOGY, 2022, 92 : S184 - S185
  • [10] A FIRST-IN-HUMAN, MULTICENTER, PHASE 1/2, OPEN-LABEL STUDY OF XTX101 IN PATIENTS WITH ADVANCED SOLID TUMORS
    Powderly, John
    Norman, Teleen
    Duncan, Meghan
    Huber, Martin
    O'Neil, Jennifer
    Patel, Ekta
    Vandross, Andrae
    JOURNAL FOR IMMUNOTHERAPY OF CANCER, 2021, 9 : A549 - A549
12345