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一个通过设计的质量方法开发人类多能干细胞衍生细胞治疗产品

Yidi Gan¹, Yichao Wang¹, Shuyan Wang¹

¹Zephyrm Biotechnologies, Beijing, China.

Advances in experimental medicine and biology

|October 24, 2025

概括

这项研究引入了设计质量 (QbD) 战略,用于开发人类多能干细胞 (hPSC) 衍生疗法. 它通过风险评估和流程优化来确保细胞疗法的安全性,有效性和质量.

科学领域:

细胞生物学细胞生物学
发育生物学是发展生物学.
生物技术是生物技术.

背景情况:

人类多能干细胞 (hPSCs) 能够在临床应用中分化各种功能性细胞类型.
许多临床研究正在进行中,一些细胞疗法产品处于后期开发阶段.
确保这些细胞疗法的安全性,有效性和质量是一个重大挑战.

研究的目的:

为细胞疗法提出基于设计质量 (QbD) 的产品开发策略.
作为一个案例研究,利用PSC衍生的中细胞样 (MSC样) 干细胞治疗.
为生产高质量,安全和有效的细胞疗法提供结构化的框架.

主要方法:

根据ICH指南定义了质量目标产品配置文件 (QTPP).
应用风险评估工具 (例如风险排名和过),以确定关键质量属性 (CQA).
优化关键工艺参数 (CPPs) 以提高制造稳定性,重点关注可行性,纯度和无菌加工.

主要成果:

确定了关键的CQAs和优化的CPP,以稳定制造PSC衍生的MSC类疗法.
证明了分析方法验证,原材料控制和生命周期管理的重要性.
为开发高质量的细胞疗法产品建立了一个结构化的框架.

结论:

整合QbD原则对于PSC衍生细胞疗法的成功开发至关重要.
这种方法提高了制造的稳定性,并确保了产品的质量,安全性和有效性.
该战略为未来基于细胞的治疗开发和监管合规提供了有价值的先例.

关键词:

控制战略控制策略关键过程参数 (CPP) 是指关键过程参数.关键质量属性 (CQA) 是指关键的质量属性.人类多能干细胞 (hPSCs)生命周期管理生命周期管理类似于介质细胞层细胞 (MSC-like) 的介质细胞层细胞.过程开发过程开发质量目标产品配置文件 (QTPP)通过设计的质量 (QbD)风险评估风险评估风险评估

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