中国药物评价2025，Vol.42Issue(6)：409-415,7.
体内嵌合抗原受体T细胞(in vivo CAR-T)研究进展及非临床研究一般考虑
Research Progress and General considerations on Nonclinical Evaluation of In Vivo Chimeric Antigen Receptor(CAR)-T Cell Therapy
周宇 1张旻 1闫莉萍1
作者信息
- 1. 国家药品监督管理局药品审评中心,北京 100022
- 折叠
摘要
Abstract
Chimeric antigen receptor(CAR)-T cell therapy has demonstrated significant therapeutic potential in hematologic malig-nancies(particularly B-cell malignancies).However,its broad application faces considerable challenges,including complex ex vivo man-ufacturing processes and high production costs.In recent years,with rapid advancements in RNA therapeutics and targeted delivery sys-tems,in vivo generation of chimeric antigen receptor T cells(in vivo CAR-T)has emerged as an innovative strategy.This approach utili-zes viral vectors or lipid nanoparticles(LNPs)as targeted delivery systems to directly introduce genetic material encoding the CAR into patients,enabling in vivo T cell engineering.This strategy fundamentally eliminates the need for cumbersome ex vivo cell manipulation steps and traditional chemotherapy-based preconditioning regimens.This article systematically reviews the technological progress and non-clinical research considerations for in vivo CAR-T.As in vivo CAR-T combines attributes of both gene therapy and cell therapy,involves diverse delivery vectors,and exhibits varied types and complex mechanisms,its nonclinical studies can be guided by risk-based,case-by-case principles.Within the framework of existing relevant technical guidelines,scientifically sound nonclinical studies should be rationally designed and conducted to generate robust experimental data supporting clinical trials and marketing approval.关键词
体内嵌合抗原受体T细胞(in vivo CAR-T)/非临床研究/病毒载体/LNP载体Key words
In vivo CAR-T/Non-clinical research/Viral vector/LNP vector分类
医药卫生引用本文复制引用
周宇,张旻,闫莉萍..体内嵌合抗原受体T细胞(in vivo CAR-T)研究进展及非临床研究一般考虑[J].中国药物评价,2025,42(6):409-415,7.
