The European regulatory environment of rna-based vaccines

Thomas Hinz, Kajo Kallen, Cedrik M. Britten, Bruno Flamion, Ulrich Granzer, Axel Hoos, Christoph Huber, Samir N. Khleif, Sebastian Kreiter, Hans Georg Rammensee, Ugur Sahin, Harpreet Singh-Jasuja, Özlem Türeci, Ulrich Kalinke

Research output: Chapter in Book/Report/Conference proceedingChapter

5 Citations (Scopus)

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.

Original languageEnglish (US)
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Pages203-222
Number of pages20
DOIs
StatePublished - Jan 1 2017

Publication series

NameMethods in Molecular Biology
Volume1499
ISSN (Print)1064-3745

Fingerprint

Vaccines
RNA
Messenger RNA
Neoplasms
RNA Stability
Genetic Therapy
Communicable Diseases
Guidelines
Antigens
Therapeutics
Research
Pharmaceutical Preparations

Keywords

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

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics

Cite this

Hinz, T., Kallen, K., Britten, C. M., Flamion, B., Granzer, U., Hoos, A., ... Kalinke, U. (2017). The European regulatory environment of rna-based vaccines. In Methods in Molecular Biology (pp. 203-222). (Methods in Molecular Biology; Vol. 1499). Humana Press Inc.. https://doi.org/10.1007/978-1-4939-6481-9_13

The European regulatory environment of rna-based vaccines. / Hinz, Thomas; Kallen, Kajo; Britten, Cedrik M.; Flamion, Bruno; Granzer, Ulrich; Hoos, Axel; Huber, Christoph; Khleif, Samir N.; Kreiter, Sebastian; Rammensee, Hans Georg; Sahin, Ugur; Singh-Jasuja, Harpreet; Türeci, Özlem; Kalinke, Ulrich.

Methods in Molecular Biology. Humana Press Inc., 2017. p. 203-222 (Methods in Molecular Biology; Vol. 1499).

Research output: Chapter in Book/Report/Conference proceedingChapter

Hinz, T, Kallen, K, Britten, CM, Flamion, B, Granzer, U, Hoos, A, Huber, C, Khleif, SN, Kreiter, S, Rammensee, HG, Sahin, U, Singh-Jasuja, H, Türeci, Ö & Kalinke, U 2017, The European regulatory environment of rna-based vaccines. in Methods in Molecular Biology. Methods in Molecular Biology, vol. 1499, Humana Press Inc., pp. 203-222. https://doi.org/10.1007/978-1-4939-6481-9_13
Hinz T, Kallen K, Britten CM, Flamion B, Granzer U, Hoos A et al. The European regulatory environment of rna-based vaccines. In Methods in Molecular Biology. Humana Press Inc. 2017. p. 203-222. (Methods in Molecular Biology). https://doi.org/10.1007/978-1-4939-6481-9_13
Hinz, Thomas ; Kallen, Kajo ; Britten, Cedrik M. ; Flamion, Bruno ; Granzer, Ulrich ; Hoos, Axel ; Huber, Christoph ; Khleif, Samir N. ; Kreiter, Sebastian ; Rammensee, Hans Georg ; Sahin, Ugur ; Singh-Jasuja, Harpreet ; Türeci, Özlem ; Kalinke, Ulrich. / The European regulatory environment of rna-based vaccines. Methods in Molecular Biology. Humana Press Inc., 2017. pp. 203-222 (Methods in Molecular Biology).
@inbook{2ab3304dc1024aca89c6db0f12fda694,
title = "The European regulatory environment of rna-based vaccines",
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 = "Advanced therapy medicinal products (ATMP), Anticancer vaccination, Genetically modified medicinal products, Preventive and therapeutic approaches, Regulatory framework in the EU, Vaccination against infectious disease, Vaccines, mRNA",
author = "Thomas Hinz and Kajo Kallen and Britten, {Cedrik M.} and Bruno Flamion and Ulrich Granzer and Axel Hoos and Christoph Huber and Khleif, {Samir N.} and Sebastian Kreiter and Rammensee, {Hans Georg} and Ugur Sahin and Harpreet Singh-Jasuja and {\"O}zlem T{\"u}reci and Ulrich Kalinke",
year = "2017",
month = "1",
day = "1",
doi = "10.1007/978-1-4939-6481-9_13",
language = "English (US)",
series = "Methods in Molecular Biology",
publisher = "Humana Press Inc.",
pages = "203--222",
booktitle = "Methods in Molecular Biology",

}

TY - CHAP

T1 - The European regulatory environment of rna-based vaccines

AU - Hinz, Thomas

AU - Kallen, Kajo

AU - Britten, Cedrik M.

AU - Flamion, Bruno

AU - Granzer, Ulrich

AU - Hoos, Axel

AU - Huber, Christoph

AU - Khleif, Samir N.

AU - Kreiter, Sebastian

AU - Rammensee, Hans Georg

AU - Sahin, Ugur

AU - Singh-Jasuja, Harpreet

AU - Türeci, Özlem

AU - Kalinke, Ulrich

PY - 2017/1/1

Y1 - 2017/1/1

N2 - 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.

AB - 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.

KW - Advanced therapy medicinal products (ATMP)

KW - Anticancer vaccination

KW - Genetically modified medicinal products

KW - Preventive and therapeutic approaches

KW - Regulatory framework in the EU

KW - Vaccination against infectious disease

KW - Vaccines

KW - mRNA

UR - http://www.scopus.com/inward/record.url?scp=85007163158&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85007163158&partnerID=8YFLogxK

U2 - 10.1007/978-1-4939-6481-9_13

DO - 10.1007/978-1-4939-6481-9_13

M3 - Chapter

C2 - 27987152

AN - SCOPUS:85007163158

T3 - Methods in Molecular Biology

SP - 203

EP - 222

BT - Methods in Molecular Biology

PB - Humana Press Inc.

ER -