系统综述类器官技术在药物筛选、安全性评价及药动学研究中的最新应用，并探讨技术标准化与临床转化面临的挑战。通过文献研究和案例分析，从药物筛选、安全性评价及药动学研究等方面，分别阐述不同类器官模型的应用场景，同时分析类器官的表征标准化体系建立的重要意义。类器官在药物筛选中优势明显，肿瘤类器官已成为抗肿瘤药物筛选的首选模型，在结直肠癌、肝癌和肺癌等药物筛选中发挥显著优势。在安全性评价方面，肝脏、心脏、肾脏和大脑等类器官可有效模拟药物导致的毒性。药动学研究中，肠道、肝脏类器官可以助力研究药物代谢机制。但类器官培养易受多种因素影响，存在培养成本高、技术要求高、标准化体系尚不完善等问题，且其与体内真实器官仍有差异。类器官技术虽有局限，但已展现出高效性和准确性，为药物研发提供有力支持，随着技术的不断进步，其有望推动药物研发的高质量发展。

To systematically review the latest applications of organoid technology in drug screening, safety evaluation and pharmacokinetic studies, and discuss challenges in technical standardization and clinical translation. Through literature research and case analysis, the application scenarios of different organ models were explained from the aspects of drug screening, safety evaluation and pharmacokinetic studies, and the importance of establishing a standardized system for organoid characterization was analyzed. Organoids demonstrate distinct advantages in drug screening. Tumor organoids have become the preferred model for anti-cancer drug screening, exhibiting remarkable utility in drug screening such as colorectal cancer, liver cancer and lung cancer. In safety evaluation, organoids of the liver, heart, kidney and brain can effectively simulate the toxicity caused by drugs. In pharmacokinetic studies, intestinal and liver organoids can help study drug metabolism mechanisms. However, organoid culture is susceptible to various factors, and there are problems such as high cultivation costs, high technical requirements, and incomplete standardization system, and it is still different from the real organs in the body. Despite current limitations, organoid technology has proven to be a highly efficient and accurate tool for drug development. With the continuous advancement of technology, it is expected to promote the high-quality development of drug research and development.

R965.2

国家重点研发计划项目（2022YFC2409702）