细胞和基因治疗（CGT）的分水岭年：2024年CGT批准的回顾。

IF 10.1 1区医学 Q1 ONCOLOGY

Cancer letters Pub Date : 2025-08-11 DOI:10.1016/j.canlet.2025.217980

Aiwen Jian , Guo Zhao , Yuning Wang , Shuhang Wang, Ning Li

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引用次数: 0

摘要

2024年是人类疾病治疗取得突破性进展的关键一年，同时精准医学，特别是细胞和基因疗法（cgt）也在迅速发展，美国FDA、中国国家药物局、欧盟EMA和日本PMDA批准了13种新型cgt，这是前所未有的。2024年也是许多“第一”的一年：第一个CRISPR疗法，第一个mri引导的颅内AAV传递基因疗法，第一个肿瘤浸润淋巴细胞疗法，开启了基因编辑和细胞技术创新临床转化的全新篇章。cgt通过利用细胞或遗传材料来治疗或预防疾病，为以前难治性疾病提供治疗潜力，代表了精密医学中一种新兴的转化模式。尽管cgt的发展蓬勃发展，但对2024年批准的全面分析仍然缺乏。本文采用四轴比较框架分析了中国和美国2024年批准的cgt，将机制创新与逐步完善的监管进展结合起来。原始数据摘自官方机构数据库、关键试验和生产报告。这项工作进一步描述了机制多样性如何与调节敏捷性融合，从而重新定义治疗发展。

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Watershed year of cell and gene therapy (CGT): A review of 2024 CGT approvals

The year 2024 is a pivotal year for therapeutic breakthroughs in human diseases, alongside with an uprising growth in precision medicine, especially in cell and gene therapies (CGTs), marked by an unprecedented approval number of 13 novel CGTs authorized by the U.S. FDA, China's NMPA, E.U. EMA, and Japan's PMDA. 2024 is also a year of many firsts: the first CRISPR therapy, the first MRI-guided intracranial AAV delivery gene therapy, and the first tumor infiltration lymphocyte therapy, opening a whole new chapter of clinical translation of innovations in gene-editing and cell technologies. CGTs represent an emerging translational modality in precision medicine through utilization of cellular or genetic materials to treat or prevent disease, offering curative potential for previously refractory diseases. Despite thriving in CGTs' development, comprehensive analyses of 2024 approvals remain absent. This review employs a quadruple axle comparative framework to analyze 2024-approved CGTs in China and USA, integrating mechanistic innovation with gradually improving regulatory advancements. Raw data was extracted from official agency databases, pivotal trials, and manufacturing reports. This work further delineates how mechanistic diversity converges with regulatory agility to redefine therapeutic development.

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来源期刊

Cancer letters 医学-肿瘤学

CiteScore

17.70

自引率

2.10%

发文量

427

审稿时长

15 days

期刊介绍： Cancer Letters is a reputable international journal that serves as a platform for significant and original contributions in cancer research. The journal welcomes both full-length articles and Mini Reviews in the wide-ranging field of basic and translational oncology. Furthermore, it frequently presents Special Issues that shed light on current and topical areas in cancer research. Cancer Letters is highly interested in various fundamental aspects that can cater to a diverse readership. These areas include the molecular genetics and cell biology of cancer, radiation biology, molecular pathology, hormones and cancer, viral oncology, metastasis, and chemoprevention. The journal actively focuses on experimental therapeutics, particularly the advancement of targeted therapies for personalized cancer medicine, such as metronomic chemotherapy. By publishing groundbreaking research and promoting advancements in cancer treatments, Cancer Letters aims to actively contribute to the fight against cancer and the improvement of patient outcomes.