外泌体作为生物药物的监管:外泌体开发和生产过程中面临的监管挑战。

IF 3.1 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL
Chun-Kai Wang, Teng-Huang Tsai, Chung-Hsi Lee
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引用次数: 0

摘要

随着医疗技术的进步,细胞外囊泡又称外泌体,因其潜在的治疗用途而受到广泛关注。然而,由于其独特的细胞内作用机制,各国对外囊泡的监管情况复杂多样。制造技术的多样性使其标准化面临挑战,从而导致监管环境支离破碎。目前全球对外泌体的监管框架大致可分为两种策略:一种是阐明外泌体的组成成分,另一种是研究外泌体分泌的生理反响。将外泌体用作治疗剂时,其管理方法应与生物制药类似。与生物制剂类似,外泌体也经过分析,以确定其颗粒大小和蛋白质成分。基于外泌体的治疗剂应在了解其分子组成和结构并证明其药代动力学和疗效后获得临床批准。然而,对于监管机构来说,证明外泌体的药代动力学和疗效具有挑战性。本文回顾了外泌体的技术特点,分析了各国监管法律的发展趋势,并讨论了临床应用的化学、制造和控制要求。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Regulation of exosomes as biologic medicines: Regulatory challenges faced in exosome development and manufacturing processes

With advances in medical technology, extracellular vesicles, also known as exosomes, are gaining widespread attention because of their potential therapeutic applications. However, their regulatory landscape is complex and varies across countries because of their unique intracellular mechanisms of action. The diversity of manufacturing techniques renders their standardization challenging, leading to a fragmented regulatory landscape. The current global regulatory framework of exosomes can be broadly classified into two strategies: one involves elucidating constituent components within exosomes and the other involves examining the physiological repercussions of their secretion. When using exosomes as therapeutic agents, they should be governed similarly to biological medicinal products. Similar to biologics, exosomes have been analyzed to determine their particle size and protein composition. An exosome-based therapeutic agent should be clinically approved after understanding its molecular composition and structure and demonstrating its pharmacokinetics and therapeutic efficacy. However, demonstrating the pharmacokinetics and therapeutic efficacy of exosomes is challenging for regulatory agencies. This article reviews the technical characteristics of exosomes, analyzes the trends in regulatory laws in various countries, and discusses the chemistry, manufacturing, and control requirements of clinical applications.

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来源期刊
Cts-Clinical and Translational Science
Cts-Clinical and Translational Science 医学-医学:研究与实验
CiteScore
6.70
自引率
2.60%
发文量
234
审稿时长
6-12 weeks
期刊介绍: Clinical and Translational Science (CTS), an official journal of the American Society for Clinical Pharmacology and Therapeutics, highlights original translational medicine research that helps bridge laboratory discoveries with the diagnosis and treatment of human disease. Translational medicine is a multi-faceted discipline with a focus on translational therapeutics. In a broad sense, translational medicine bridges across the discovery, development, regulation, and utilization spectrum. Research may appear as Full Articles, Brief Reports, Commentaries, Phase Forwards (clinical trials), Reviews, or Tutorials. CTS also includes invited didactic content that covers the connections between clinical pharmacology and translational medicine. Best-in-class methodologies and best practices are also welcomed as Tutorials. These additional features provide context for research articles and facilitate understanding for a wide array of individuals interested in clinical and translational science. CTS welcomes high quality, scientifically sound, original manuscripts focused on clinical pharmacology and translational science, including animal, in vitro, in silico, and clinical studies supporting the breadth of drug discovery, development, regulation and clinical use of both traditional drugs and innovative modalities.
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