The European Regulatory Environment of RNA-Based Vaccines
The European Regulatory Environment of RNA-Based Vaccines
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DOI：
10.1007/978-1-4939-6481-9_13
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发表时间：
2017-01-01
期刊：
影响因子：
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通讯作者：
Kalinke, Ulrich
中科院分区：
文献类型：
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作者：
Hinz, Thomas;Kallen, Kajo;Kalinke, Ulrich
目前正在开发各种不同的基于mRNA的药物。这成为可能，因为在过去几十年中，RNA研究的重大突破使mRNA的翻译，稳定性和递送得到了令人印象深刻的改善。这篇文章主要关注基于RNA的抗原编码疫苗，这些疫苗可以直接针对肿瘤或病原体。mRNA编码的疫苗被开发用于预防或治疗目的。大多数基于mRNA的疫苗直接给予患者。或者，通过使用mRNA离体修饰来自癌症患者的原代自体细胞，然后过继转移至患者。在欧盟，目前没有专门针对基于mRNA的疫苗的监管指南。然而，现有的监管框架明确规定，在大多数情况下，基于mRNA的疫苗必须得到集中批准。有趣的是，根据基于RNA的疫苗是针对肿瘤还是感染性疾病，它们分别被正式认为是基因治疗产品。除了对mRNA疫苗在各个治疗领域的当前临床使用的概述之外，还提供了对当前监管情况的详细讨论，并讨论了监管前景。
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.