Report of the European Medicines Agency Conference on RNA-Based Medicines.

IF 4 2区医学 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY Nucleic acid therapeutics Pub Date : 2024-02-01 Epub Date: 2024-01-04 DOI:10.1089/nat.2023.0021

Falk Ehmann, Andreas Kuhn, Anna Maria Gerdina Pasmooij, Anthony Humphreys, Arjon Van Hengel, Brian Dooley, Brigitte Anliker, Camilla Svensson, Daniel Capaldi, David Henshall, Emer Cooke, Haiyan Zhou, Hilde Bastaerts, Jeske Smink, Joop Van Gerven, Leonor Enes, Lubomir Nechev, Marcel Hoefnagel, Mariëtte Driessens, Michael Wenger, Oriane Blanquie, Pawel Widomski, Ralf Herold, René Thürmer, Sol Ruiz, Steffen Thirstrup, Susan Goody, Tal Zaks, Valentina Cordò, Annemieke M Aartsma-Rus

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Abstract

RNA-based medicines have potential to treat a large variety of diseases, and research in the field is very dynamic. Proactively, The European Medicines Agency (EMA) organized a virtual conference on February 2, 2023 to promote the development of RNA-based medicines. The initiative addresses the goal of the EMA Regulatory Science Strategy to 2025 to "catalyse the integration of science and technology in medicines development." The conference focused on RNA technologies (excluding RNA vaccines) and involved different stakeholders, including representatives from academia, industry, regulatory authorities, and patient organizations. The conference comprised presentations and discussion sessions conducted by panels of subject matter experts. In this meeting report, we summarize the presentations and recap the main themes of the panel discussions.

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欧洲药品管理局关于基于 RNA 的药物会议的报告。

基于 RNA 的药物具有治疗多种疾病的潜力，该领域的研究非常活跃。欧洲药品管理局（EMA）于 2023 年 2 月 2 日积极组织了一次虚拟会议，以促进 RNA 类药物的开发。这一举措旨在实现欧洲药品管理局 2025 年监管科学战略的目标，即 "促进科学与技术在药品开发中的融合"。会议重点讨论了 RNA 技术（不包括 RNA 疫苗），来自学术界、工业界、监管机构和患者组织的代表等不同利益相关方参加了会议。会议包括专题专家小组的发言和讨论环节。在本会议报告中，我们总结了演讲内容，并回顾了小组讨论的主题。

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来源期刊

Nucleic acid therapeutics BIOCHEMISTRY & MOLECULAR BIOLOGY-CHEMISTRY, MEDICINAL

CiteScore

7.60

自引率

7.50%

发文量

审稿时长

>12 weeks

期刊介绍： Nucleic Acid Therapeutics is the leading journal in its field focusing on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. The Journal examines many new approaches for using nucleic acids as therapeutic agents or in modifying nucleic acids for therapeutic purposes including: oligonucleotides, gene modification, aptamers, RNA nanoparticles, and ribozymes.