What's in a word? Defining "gene therapy medicines".

IF 4.6 2区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Molecular Therapy-Methods & Clinical Development Pub Date : 2024-10-21 eCollection Date: 2024-12-12 DOI:10.1016/j.omtm.2024.101348

Maren von Fritschen, Ewa Janosz, Constanze Blume, Ulrike Jägle, Karen Keating, Christian K Schneider

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Abstract

Gene therapy medicinal products (GTMPs) have emerged as a transformative class of medicines. Defining what a certain class of medicines encompasses, and what it does not, is key, with ample implications and consequential regulatory requirements. In April 2023, the European Commission proposed new pharmaceutical legislation safeguarding the public health within the European Union with a new, broader definition of GTMP, including genome editing medicines and nucleic acids of either source, regulating, replacing, or adding a genetic sequence that mediates its effect by transcription or translation. This definition is all-encompassing for any "genetic" intervention and is agnostic to mechanism of action, duration of action, location of action, and associated risk. Here, we take this as a paradigm to discuss how terminology and definitions are more than just words and can have meaningful regulatory, scientific, and public health implications.

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词中有什么？定义 "基因治疗药物"。

基因治疗药物产品 (GTMP) 已成为一类变革性药物。界定某类药品包括哪些内容以及不包括哪些内容是关键所在，并会产生广泛影响和相应的监管要求。2023 年 4 月，欧盟委员会提出了保障欧盟公众健康的新药品立法，对 GTMP 进行了新的、更广泛的定义，包括基因组编辑药品和核酸，无论其来源如何，均可调节、替换或增加基因序列，通过转录或翻译发挥其作用。这一定义涵盖了所有 "基因 "干预措施，并且与作用机制、作用持续时间、作用位置和相关风险无关。在此，我们将以此为范例，讨论术语和定义不仅仅是文字，还可能对监管、科学和公共卫生产生有意义的影响。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Molecular Therapy-Methods & Clinical Development Biochemistry, Genetics and Molecular Biology-Molecular Biology

CiteScore

9.90

自引率

4.30%

发文量

163

审稿时长

12 weeks

期刊介绍： The aim of Molecular Therapy—Methods & Clinical Development is to build upon the success of Molecular Therapy in publishing important peer-reviewed methods and procedures, as well as translational advances in the broad array of fields under the molecular therapy umbrella. Topics of particular interest within the journal''s scope include: Gene vector engineering and production, Methods for targeted genome editing and engineering, Methods and technology development for cell reprogramming and directed differentiation of pluripotent cells, Methods for gene and cell vector delivery, Development of biomaterials and nanoparticles for applications in gene and cell therapy and regenerative medicine, Analysis of gene and cell vector biodistribution and tracking, Pharmacology/toxicology studies of new and next-generation vectors, Methods for cell isolation, engineering, culture, expansion, and transplantation, Cell processing, storage, and banking for therapeutic application, Preclinical and QC/QA assay development, Translational and clinical scale-up and Good Manufacturing procedures and process development, Clinical protocol development, Computational and bioinformatic methods for analysis, modeling, or visualization of biological data, Negotiating the regulatory approval process and obtaining such approval for clinical trials.