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RNA Vaccines pp 203–222Cite as

The European Regulatory Environment of RNA-Based Vaccines

Part of the Methods in Molecular Biology book series (MIMB,volume 1499)

Abstract

A variety of different mRNA-based drugs are currently in development. This became possible, since major breakthroughs in RNA research during the last decades allowed impressive improvements of translation, stability and delivery of mRNA. This article focuses on antigen-encoding RNA-based vaccines that are either directed against tumors or pathogens. mRNA-encoded vaccines are developed both for preventive or therapeutic purposes. Most mRNA-based vaccines are directly administered to patients. Alternatively, primary autologous cells from cancer patients are modified ex vivo by the use of mRNA and then are adoptively transferred to patients. In the EU no regulatory guidelines presently exist that specifically address mRNA-based vaccines. The existing regulatory framework, however, clearly defines that mRNA-based vaccines in most cases have to be centrally approved. Interestingly, depending on whether RNA-based vaccines are directed against tumors or infectious disease, they are formally considered gene therapy products or not, respectively. Besides an overview on the current clinical use of mRNA vaccines in various therapeutic areas a detailed discussion of the current regulatory situation is provided and regulatory perspectives are discussed.

Keywords

  • mRNA
  • Vaccines
  • Anticancer vaccination
  • Vaccination against infectious disease
  • Preventive and therapeutic approaches
  • Regulatory framework in the EU
  • Advanced therapy medicinal products (ATMP)
  • Genetically modified medicinal products

* Thomas Hinz and Kajo Kallen contributed equally to this manuscript.

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Acknowledgements

This chapter was issued as a joint effort by the Regulatory Research Group (RRG). The RRG was founded within the Association for Cancer Immunotherapy (CIMT) in 2008 as an independent group of experts focusing on regulatory aspects of drug development. CIMT was founded in 2002 by physicians and researchers from different fields of clinical and theoretical medicine as an independent nonprofit organization (www.cimt.eu).

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Author notes

    Authors and Affiliations

    1. Section for Therapeutic Vaccines, Division for Immunology, Paul-Ehrlich-Institut, Paul-Ehrlich-Str. 51-59, 63225, Langen, Germany

      Thomas Hinz

    2. Kallen Consulting, Köln/Frechen, Germany

      Kajo Kallen

    3. R&D Oncology, Glaxo Smith Kline, Stevenage, UK

      Cedrik M. Britten

    4. URPhyM, NARILIS, University of Namur, Namur, Belgium

      Bruno Flamion

    5. Granzer, Regulatory Consulting & Services, Munich, Germany

      Ulrich Granzer

    6. Glaxo Smith Kline, Collegeville, PA, USA

      Axel Hoos

    7. Association for Cancer Immunotherapy, Mainz, Germany

      Christoph Huber & Sebastian Kreiter

    8. GHSU Cancer Center, Augusta, GA, USA

      Samir Khleif

    9. Department of Immunology, Institute for Cell Biology, University of Tübingen, Tübingen, Germany

      Hans-Georg Rammensee

    10. German Cancer Consortium, DKFZ Partner Site, Tübingen, Germany

      Hans-Georg Rammensee

    11. TRON – Translational Oncology at the University Medical Center, Johannes Gutenberg University, Mainz, Germany

      Ugur Sahin

    12. Biopharmaceutical New Technologies (BioNTech) Corporation, Mainz, Germany

      Ugur Sahin

    13. Immatics Biotechnologies GmbH, Tübingen, Germany

      Harpreet Singh-Jasuja

    14. CI3, Cluster for individualized Immune Intervention, Mainz, Germany

      Özlem Türeci

    15. Institute for Experimental Infection Research, Twincore, Centre for Experimental and Clinical Infection Research a joint venture between the Hannover Medical School and the Helmholtz Centre for Infection Research, Feodor-Lynen-Str. 7-9, 30625, Hannover, Germany

      Ulrich Kalinke

    16. Research Center for Immunotherapy (FZI), Mainz, Germany

      Ugur Sahin

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    1. Thomas Hinz
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    Correspondence to Thomas Hinz or Ulrich Kalinke .

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    1. Boehringer Ingelheim Pharma GmbH & Co. KG, Ingelheim am Rhein, Germany

      Thomas Kramps

    2. Boehringer Ingelheim GmbH, Ingelheim am Rhein, Germany

      Knut Elbers

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    Hinz, T. et al. (2017). The European Regulatory Environment of RNA-Based Vaccines. In: Kramps, T., Elbers, K. (eds) RNA Vaccines. Methods in Molecular Biology, vol 1499. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-6481-9_13

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