冷冻电镜技术与基于质量源于设计理念的药品研发的若干思考

Implications of Cryo-Electron Microscopy and the Concept of Quality by Design in Drug Discovery

目前,以冷冻电镜为代表的药物靶点发现及作用机制的研究,在国外已经开始逐步进入大型制药企业,而我国对于冷冻电镜的应用目前更多局限在高校和科研院所。本文阐述了冷冻电镜技术在全球药物开发中的应用,探索了其在基于质量源于设计(QbD)理念的药品研发及提升药品质量方面的应用前景,以期促进我国产学研之间的转化,推进监管机构在药品高质量发展阶段更好地理解冷冻电镜技术在药物开发中的价值和意义。

Recently cryo-EM has become an import tool in pharmaceutical industry,particularly in structurebased drug discovery paradigm(target selection and lead discovery)and mechanism of action.However,its adoption by Chinese pharmaceutical companies is actually very slow.Aiming to foster industry-university research collaboration and to help regulators better understand the value of this cutting-edge cryo-EM technology in high-quality drug development,this study focuses on how cryo-EM has enabled structure-based insights for the drug discovery pipeline globally,and discusses the implication of cryo-EM and the concept of quality by design for improving drug quality.