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细胞和基因疗法:为以患者为中心的护理转变治疗范式

Sojeong Yi¹, Chia-Yung Wu², Avery McIntosh³

¹Alnylam Pharmaceuticals, Inc, Cambridge, Massachusetts, USA.

Clinical and translational science

|December 5, 2025

概括

细胞和基因疗法提供潜在的治疗方法,但面临成本,获取和临床不确定性方面的挑战. 本摘要涵盖了基因疗法和T细胞免疫疗法的进展,重点是以患者为中心的护理和优化发展.

科学领域:

药理学和治疗学药理学和治疗学
生物技术是生物技术.
遗传学遗传学是一个

背景情况:

细胞和基因疗法 (CGTs) 正在彻底改变医学,为难以治疗的疾病提供潜在的治疗方法.
仍然存在重大挑战,包括优化疗效,安全,患者可访问性和管理高成本,特别是在罕见疾病中.

研究的目的:

总结2025年ASCPT CGT卫星会议的关键讨论,以改变以患者为中心的护理治疗模式.
从科学,临床和患者的角度提供对基因疗法和T细胞免疫疗法的见解.

主要方法:

从ASCPT 2025 CGT卫星会议的演讲和讨论的回顾.
对基因疗法平台,对血友病的腺相关病毒 (AAV) 基因疗法,临床药理学和基于模型的药物开发 (MIDD) 的信息的综合.
对T细胞免疫疗法的分析,包括CAR T细胞疗法,全原CAR T和MIDD策略.

主要成果:

基因治疗的进步包括'平台化'以简化开发和从基于AAV的血友病疗法中吸取经验教训.
像CAR T疗法这样的T细胞免疫疗法显示出希望,重点是动力学,疗效,安全性以及对自身免疫性疾病的新兴全基因方法.
基于模型的药物开发 (MIDD) 对于优化治疗设计和基因和T细胞疗法的临床结果至关重要.

结论:

CGT具有巨大的治疗潜力,但克服发展,成本和可访问性方面的挑战对于以患者为中心的护理至关重要.
持续研究和应用平台化和MIDD等策略对于推进CGT的有效性和安全性至关重要.
会议强调了需要综合科学,临床和患者的观点,以充分实现CGT的变革力量.

关键词:

基于细胞和组织的疗法.基因编辑基因编辑基因疗法的基因治疗.免疫疗法免疫疗法模式信息化药物开发模型

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