Targeted Gene Insertion of Vectorized Monoclonal Antibodies in Non-Human Primates Overcomes AAV Genome Silencing in the Liver and Supports High, Sustained In Vivo Expression of Functional Antibodies

被引：0
|
作者：
Sattler, Rachel [1 ]
LoMastro, Anthony [1 ]
Ezemba, Juanita [1 ]
Sterlacci, Tara [1 ]
Koehler-Stec, Ellen-Marie [1 ]
Parveen, Asma [1 ]
Ivanova, Elena [1 ]
Pascal, Kristen [1 ]
Sabin, Leah [1 ]
Kyratsous, Christos [1 ]
机构：
[1] Regeneron Pharmaceut Inc, 777 Old Saw Mill River Rd, Tarrytown, NY 10591 USA
来源：
MOLECULAR THERAPY | 2024年 / 32卷 / 04期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
197
引用
收藏
页码：106 / 107
页数：2
相关论文
共 12 条
  • [1] In Vivo Immunoprophylaxis in Non-Human Primates Following Administration of Synthetic DNA-Monoclonal Antibodies (dMAbs)
    Patel, Ami
    Wise, Megan C.
    Esquivel, Rianne
    Elliott, Sarah T. C.
    Ramos, Stephanie
    Stoltz, Regina
    Park, Daniel H.
    Boyer, Jean
    Muthumani, Kar
    Broderick, Kate E.
    Humeau, Laurent
    Weiner, David B.
    MOLECULAR THERAPY, 2019, 27 (04) : 333 - 333
  • [2] Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
    Gray, S. J.
    Kalburgi, S. Nagabhushan
    McCown, T. J.
    Samulski, R. Jude
    GENE THERAPY, 2013, 20 (04) : 450 - 459
  • [3] Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
    S J Gray
    S Nagabhushan Kalburgi
    T J McCown
    R Jude Samulski
    Gene Therapy, 2013, 20 : 450 - 459
  • [4] Erratum: Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
    S J Gray
    S Nagabhushan Kalburgi
    T J McCown
    R Jude Samulski
    Gene Therapy, 2013, 20 (4) : 465 - 465
  • [5] Repeated Infusion of AAV2-hAADC Into the Thalamus of Non-Human Primates: Effect of Anti-AAV2 Antibodies on Transgene Expression
    Samaranch, Lluis
    Hadaczek, Piotr
    Kells, Adrian P.
    Bringas, John
    Stockinger, Diane
    San Sebastian, Waldy
    Macayan, Michael
    Samineni, Sridhar
    Pivirotto, Philip
    Forsayeth, John
    Bankiewicz, Krystof
    MOLECULAR THERAPY, 2015, 23 : S257 - S258
  • [6] In vivo delivery of engineered synthetic DNA-encoded SARS-CoV-2 monoclonal antibodies for pre-exposure prophylaxis in non-human primates
    Patel, Ami
    Rosenke, Kyle
    Parzych, Elizabeth M.
    Feldmann, Friederike
    Bharti, Suman
    Griffin, Amanda J.
    Schouest, Blake
    Lewis, Matt
    Choi, Jihae
    Chokkalingam, Neethu
    Machado, Viviane
    Smith, Brian J.
    Frase, Drew
    Ali, Ali R.
    Lovaglio, Jamie
    Nguyen, Brian
    Hanley, Patrick W.
    Walker, Susanne N.
    Gary, Ebony N.
    Kulkarni, Abhijeet
    Generotti, Allison
    Francica, Joseph R.
    Rosenthal, Kim
    Kulp, Daniel W.
    Esser, Mark T.
    Smith, Trevor R. F.
    Shaia, Carl
    Weiner, David B.
    Feldmann, Heinz
    EMERGING MICROBES & INFECTIONS, 2024, 13 (01)
  • [7] Global CNS Gene Delivery in Non-Human Primates by Intra-CSF Administration: Evasion of Anti-AAV Neutralizing Antibodies but Not a Transgene-Specific CTL Response
    Gray, Steven J.
    Kalburgi, Sahana Nagabhushan
    McCown, Thomas J.
    Samulski, R. Jude
    MOLECULAR THERAPY, 2013, 21 : S46 - S46
  • [8] A Targeted AAV Gene Therapy Product Candidate, 4D-310, for the Treatment of Fabry Disease: Intravenous Biodistribution, Transgene Expression and Safety in Non-Human Primates
    Whittlesey, Kevin
    Brooks, Gabriel
    Szymanski, Paul
    Beliakoff, Ghezal
    Nye, Julie
    Sepulveda, Jessica
    Croze, Roxanne
    Schmitt, Chris
    Bickta, Janelle
    Barglow, Katy
    Kotterman, Melissa
    Francis, Peter
    Schaffer, David
    Kirn, David
    MOLECULAR THERAPY, 2021, 29 (04) : 115 - 115
  • [9] Safety and efficacy of pre-treatment with imlifidase prior to AAV-based gene therapy in non-human primates with pre-existing anti-AAVrh74 antibodies
    Potter, R.
    Khan, S.
    Snedeker, J.
    Adegboye, K.
    Haile, A.
    Sayanjali, B.
    Pukos, N.
    Cochran, K.
    Ahner, J.
    Su, T.
    Uzcategui, N.
    Stenberg, Y.
    Freiburghaus, C.
    Winstedt, L.
    Rodino-Klapac, L.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S101 - S101
  • [10] C3 Complement Inhibitor Limits Transaminitis and Immune Response Following Liver Targeted Adeno-Associated Virus (AAV) Gene Transfer (GT) in Non-Human Primates (NHPs)
    Kropf, Elizabeth
    Smith, Corinne J.
    Kamal, Ali L.
    Markusic, David M.
    Majowicz, Anna
    Kuranda, Klaudia
    MOLECULAR THERAPY, 2024, 32 (04) : 190 - 191
12