Next generation self-replicating RNA vectors for vaccines and immunotherapies

被引:23
作者
Aliahmad, Parinaz [1 ]
Miyake-Stoner, Shigeki J. [1 ]
Geall, Andrew J. [1 ]
Wang, Nathaniel S. [1 ]
机构
[1] Replicate Biosci Inc, San Diego, CA 92121 USA
关键词
SINDBIS-VIRUS; MESSENGER-RNA; LIPID NANOPARTICLES; EXPRESSION VECTORS; DELIVERY; TRANSLATION; IMMUNOGENICITY; IDENTIFICATION; RECOGNITION; RESISTANCE;
D O I
10.1038/s41417-022-00435-8
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
RNA technology has recently come to the forefront of innovative medicines and is being explored for a wide range of therapies, including prophylactic and therapeutic vaccines, biotherapeutic protein expression and gene therapy. In addition to conventional mRNA platforms now approved for prophylactic SARS-CoV2 vaccines, synthetic self-replicating RNA vaccines are currently being evaluated in the clinic for infectious disease and oncology. The prototypical srRNA vectors in clinical development are derived from alphaviruses, specifically Venezuelan Equine Encephalitis Virus (VEEV). While non-VEEV alphaviral strains have been explored as single cycle viral particles, their use as synthetic vectors largely remains under-utilized in clinical applications. Here we describe the potential commonalities and differences in synthetic alphaviral srRNA vectors in host cell interactions, immunogenicity, cellular delivery, and cargo expression. Thus, unlike the current thinking that VEEV-based srRNA is a one-size-fits-all platform, we argue that a new drug development approach leveraging panels of customizable, synthetic srRNA vectors will be required for clinical success.
引用
收藏
页码:785 / 793
页数:9
相关论文
共 92 条
  • [1] mRNA vaccines against H10N8 and H7N9 influenza viruses of pandemic potential are immunogenic and well tolerated in healthy adults in phase 1 randomized clinical trials
    A Feldman, Robert
    Fuhr, Rainard
    Smolenov, Igor
    Ribeiro, Amilcar
    Panther, Lori
    Watson, Mike
    Senn, Joseph J.
    Smith, Mike
    Almarsson, Orn
    Pujar, Hari S.
    Laska, Michael E.
    Thompson, James
    Zaks, Tal
    Ciaramella, Giuseppe
    [J]. VACCINE, 2019, 37 (25) : 3326 - 3334
  • [2] The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
    Akinc, Akin
    Maier, Martin A.
    Manoharan, Muthiah
    Fitzgerald, Kevin
    Jayaraman, Muthusamy
    Barros, Scott
    Ansell, Steven
    Du, Xinyao
    Hope, Michael J.
    Madden, Thomas D.
    Mui, Barbara L.
    Semple, Sean C.
    Tam, Ying K.
    Ciufolini, Marco
    Witzigmann, Dominik
    Kulkarni, Jayesh A.
    van der Meel, Roy
    Cullis, Pieter R.
    [J]. NATURE NANOTECHNOLOGY, 2019, 14 (12) : 1084 - 1087
  • [3] Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms
    Akinc, Akin
    Querbes, William
    De, Soma
    Qin, June
    Frank-Kamenetsky, Maria
    Jayaprakash, K. Narayanannair
    Jayaraman, Muthusamy
    Rajeev, Kallanthottathil G.
    Cantley, William L.
    Dorkin, J. Robert
    Butler, James S.
    Qin, LiuLiang
    Racie, Timothy
    Sprague, Andrew
    Fava, Eugenio
    Zeigerer, Anja
    Hope, Michael J.
    Zerial, Marino
    Sah, Dinah W. Y.
    Fitzgerald, Kevin
    Tracy, Mark A.
    Manoharan, Muthiah
    Koteliansky, Victor
    de Fougerolles, Antonin
    Maier, Martin A.
    [J]. MOLECULAR THERAPY, 2010, 18 (07) : 1357 - 1364
  • [4] Proof-of-concept of a low-dose unmodified mRNA-based rabies vaccine formulated with lipid nanoparticles in human volunteers: A phase 1 trial
    Aldrich, Cassandra
    Leroux-Roels, Isabel
    Huang, Katell Bidet
    Bica, Mihai Alexandru
    Loeliger, Edde
    Schoenborn-Kellenberger, Oliver
    Walz, Lisa
    Leroux-Roels, Geert
    von Sonnenburg, Frank
    Oostvogels, Lidia
    [J]. VACCINE, 2021, 39 (08) : 1310 - 1318
  • [5] Safety and Immunogenicity of SARS-CoV-2 mRNA-1273 Vaccine in Older Adults
    Anderson, E. J.
    Rouphael, N. G.
    Widge, A. T.
    Jackson, L. A.
    Roberts, P. C.
    Makhene, M.
    Chappell, J. D.
    Denison, M. R.
    Stevens, L. J.
    Pruijssers, A. J.
    McDermott, A. B.
    Flach, B.
    Lin, B. C.
    Doria-Rose, N. A.
    O'Dell, S.
    Schmidt, S. D.
    Corbett, K. S.
    Swanson, P. A., II
    Padilla, M.
    Neuzil, K. M.
    Bennett, H.
    Leav, B.
    Makowski, M.
    Albert, J.
    Cross, K.
    Edara, V. V.
    Floyd, K.
    Suthar, M. S.
    Martinez, D. R.
    Baric, R.
    Buchanan, W.
    Luke, C. J.
    Phadke, V. K.
    Rostad, C. A.
    Ledgerwood, J. E.
    Graham, B. S.
    Beigel, J. H.
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2020, 383 (25) : 2427 - 2438
  • [6] [Anonymous], 2020, US Securities and Exchange Commission Form 8-K
  • [7] [Anonymous], MODERNAS ZIKA VIRUS
  • [8] Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine
    Baden, Lindsey R.
    El Sahly, Hana M.
    Essink, Brandon
    Kotloff, Karen
    Frey, Sharon
    Novak, Rick
    Diemert, David
    Spector, Stephen A.
    Rouphael, Nadine
    Creech, C. Buddy
    McGettigan, John
    Khetan, Shishir
    Segall, Nathan
    Solis, Joel
    Brosz, Adam
    Fierro, Carlos
    Schwartz, Howard
    Neuzil, Kathleen
    Corey, Larry
    Gilbert, Peter
    Janes, Holly
    Follmann, Dean
    Marovich, Mary
    Mascola, John
    Polakowski, Laura
    Ledgerwood, Julie
    Graham, Barney S.
    Bennett, Hamilton
    Pajon, Rolando
    Knightly, Conor
    Leav, Brett
    Deng, Weiping
    Zhou, Honghong
    Han, Shu
    Ivarsson, Melanie
    Miller, Jacqueline
    Zaks, Tal
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2021, 384 (05) : 403 - 416
  • [9] A guide to large-scale RNA sample preparation
    Baronti, Lorenzo
    Karlsson, Hampus
    Marusic, Maja
    Petzold, Katja
    [J]. ANALYTICAL AND BIOANALYTICAL CHEMISTRY, 2018, 410 (14) : 3239 - 3252
  • [10] PKR acts early in infection to suppress Semliki Forest virus production and strongly enhances the type I interferon response
    Barry, Gerald
    Breakwell, Lucy
    Fragkoudis, Rennos
    Attarzadeh-Yazdi, Ghassem
    Rodriguez-Andres, Julio
    Kohl, Alain
    Fazakerley, John K.
    [J]. JOURNAL OF GENERAL VIROLOGY, 2009, 90 : 1382 - 1391