创新药物研发的多维度突破：从分子设计到临床实践

doi:10.3969/j.issn.1006-7795.2026.03.001

首都医科大学学报

创新药物研发的多维度突破：从分子设计到临床实践

王瑶琪^1,2#,霍然^1,2#,张爽^1,3,孙琪^1,2,张筱宜^4,5*△,崔纯莹^1,2*△,张强⁶

1.首都医科大学药学院药剂学系，北京 100069；2.首都医科大学基础-临床联合实验室（儿童肿瘤发病机制与创新药物研究实验室），北京 100069；3.首都医科大学临床-基础合作平台（牙周黏膜免疫分子机制及精准调控策略研究联合实验室），北京 100069；4.首都医科大学药学院药物化学系，北京 100069；5.首都医科大学临床-基础合作平台（骨肉瘤耐药与精准治疗基础研究与应用联合实验室），北京 100069；6.北京大学药学院药剂学系，北京 100091

收稿日期:2026-02-13 修回日期:2026-03-19 出版日期:2026-06-21 发布日期:2026-06-25
通讯作者: 张筱宜,崔纯莹 E-mail:zhangdd@ccmu.edu.cn；ccy@ccmu.edu.cn
基金资助:
国家自然科学基金项目（82304390，82404529，82304389），首都医科大学临床-基础合作平台培育项目（JLPYPT2025010，JLPYPT2025012）。

Multidimensional breakthroughs in innovative drug development: from molecular design to clinical practice

Wang Yaoqi^1,2#, Huo Ran^1,2#, Zhang Shuang^1,3, Sun Qi^1,2, Zhang Xiaoyi^4,5*△, Cui Chunying^1,2*△, Zhang Qiang⁶

1. Department of Pharmaceutics, School of Pharmaceutical Sciences, Capital Medical University, Beijing 100069, China；2. Laboratory for Clinical Medicine (Laboratory of Pediatric Tumor Pathogenesis and Innovative Drug Research), Capital Medical University, Beijing 100069, China；3. Collaboration Platform for Clinical Medicine (Joint Laboratory for Molecular Mechanisms of Periodontal Mucosal Immunity and Precision Modulation Strategies), Capital Medical University, Beijing 100069, China；4. Department of Medicinal Chemistry, School of Pharmaceutical Sciences, Capital Medical University, Beijing 100069, China; 5. Collaboration Platform for Clinical Medicine (Joint Laboratory for Basic Research and Application of Osteosarcoma Drug Resistance and Precision Therapy), Capital Medical University, Beijing 100069, China；6. Department of Pharmaceutics, School of Pharmaceutical Sciences, Peking University, Beijing 100091, China

Received:2026-02-13 Revised:2026-03-19 Online:2026-06-21 Published:2026-06-25
Supported by:
This study was supported by National Natural Science Foundation of China (82304390, 82404529, 82304389), Capital Medical University Collaboration Platform for Clinical Medicine (JLPYPT2025010, JLPYPT2025012).

摘要/Abstract

摘要： 传统药物研发面临周期长、成本高、临床成功率低及单一靶点易耐药等结构性瓶颈。随着多学科交叉融合与技术革新，创新药物研发正经历从靶点发现、药物设计到临床应用的全链条变革。本文系统阐述多组学整合与人工智能驱动的靶点精准挖掘、智能药物设计与先进递释系统革新及精准医学引领的个体化治疗三大核心突破方向，深度整合纳米技术、材料科学、合成生物学等前沿领域成果，解析“从分子机制到临床价值落地”的闭环路径，进一步驱动药物创新，助力医药创新革新。结合国家生物医药产业战略需求，提出以“临床价值为核心、技术创新为支撑、多学科融合为路径”的研发新范式，为我国实现高水平医药科技自立自强、筑牢生物医药产业发展命脉提供理论参考与实践借鉴。

关键词: 创新药物研发, 多组学技术, 人工智能, 精准医学, 个体化治疗, 先进药物递释系统

Abstract: Conventional drug discovery and development is constrained by structural bottlenecks, including protracted timelines, high costs, low clinical success rates, and the rapid emergence of resistance associated with single-target strategies. With accelerating interdisciplinary convergence and continuous technological innovation, innovative drug R&D is undergoing a comprehensive, end-to-end transformation spanning target discovery, drug design, and clinical translation. This review systematically summarizes three major frontiers: (i) multi-omics integration and artificial intelligence (AI)-enabled precision target identification; (ii) intelligent drug design coupled with innovations in advanced drug delivery and controlled-release systems; and (iii) precision-medicine-driven individualized therapy. By integrating advances from nanotechnology, materials science, synthetic biology, and related disciplines, we elucidate a closed-loop pathway that translates molecular mechanisms into actionable clinical value, thereby further catalyzing drug innovation and supporting transformative progress in biomedicine. In alignment with national strategic priorities for the biopharmaceutical sector, we propose a new R&D paradigm centered on clinical value, supported by technological innovation, and advanced through multidisciplinary integration, providing both theoretical insights and practical reference for achieving high-level scientific and technological self-reliance and strengthening the foundation of biopharmaceutical industry development.

Key words: innovative drug development, multi-omics technologies, artificial intelligence, precision medicine, individualized therapy, advanced drug delivery systems

中图分类号:

R9
TP18

王瑶琪, 霍然, 张爽, 孙琪, 张筱宜, 崔纯莹, 张强. 创新药物研发的多维度突破：从分子设计到临床实践[J]. 首都医科大学学报, doi: 10.3969/j.issn.1006-7795.2026.03.001.

Wang Yaoqi, Huo Ran, Zhang Shuang, Sun Qi, Zhang Xiaoyi, Cui Chunying, Zhang Qiang. Multidimensional breakthroughs in innovative drug development: from molecular design to clinical practice[J]. Journal of Capital Medical University, doi: 10.3969/j.issn.1006-7795.2026.03.001.

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创新药物研发的多维度突破：从分子设计到临床实践

Multidimensional breakthroughs in innovative drug development: from molecular design to clinical practice

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