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细胞和基因疗法:设计可提取物和可化物研究和进行安全性评估的挑战

Adeyma Arroyo¹, Petra Booij², Getachew Woldemariam³

¹Genentech, Inc., a member of the Roche Group, South San Francisco, CA 94080, USA.

Journal of pharmaceutical sciences

|January 4, 2024

概括

细胞和基因疗法 (C>) 需要对可提取物和可浸物 (E&L) 进行严格的物质风险评估. 本研究审查了指导方针,并提出了测试策略,以确保C>产品的安全性和有效性.

科学领域:

生物技术是生物技术.
制药制造业制药制造业制药制造业
监管科学监管科学

背景情况:

细胞和基因疗法 (C>) 是一个快速增长的治疗领域,有许多已批准的产品和正在进行的临床试验.
汽车及汽车用品的扩张需要对制造和储存材料进行严格的评估,特别是对于可提取物和可浸物 (E&L).
建立材料资格的正式业务流程对于降低C>开发中的风险至关重要.

研究的目的:

提供现有的指导方针和出版物相关的E&L风险评估的概述,对于ex vivo C>产品.
确定C>材料的生物相容性和可提取性条件中的差距和需要标准化的领域.
介绍适合C>制造和储存的实用漏性测试策略和安全评估考虑.

主要方法:

关于C>中的E&L风险评估的当前指南和出版物的文献综述.
对生物相容性和可提取物测试条件的标准化需求的分析.
为C>.开发拟议的漏性测试策略和安全评估方法.

主要成果:

确定了适用于ex vivo C>的E&L风险评估的关键准则和出版物.
突出显著的差距和生物相容性和可提取性测试的标准化需求,具体针对C>.
概述了C>中有机和无机化合物的相关漏性测试策略和安全评估考虑因素.

结论:

对材料资格和E&L风险评估的结构化方法对于C>发展至关重要.
测试方法和条件的标准化是必要的,以确保C>产品的安全性和有效性.
拟议的战略为在复杂的C>制造和储存环境中管理E&L风险提供了一个框架.

关键词:

生物相容性生物相容性细胞和基因疗法细胞和基因疗法.可以提取的可提取物.液性食品可以液化.材料制造业的材料制造业监管生物相容性监管生物相容性风险评估风险评估风险评估安全性 (毒理学) 评估储存储存储存储存

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