siRNA药物研发进展及趋势  

Recent progress and trends in research and development of siRNA drug

作　　者：张霖 ZHANG Lin(State Key Laboratory of Innovative Immunotherapy,Central Research Institute,Shanghai Pharmaceuticals Holding Co.,Ltd.,Shanghai 201203,China)

机构地区：[1]上海医药集团股份有限公司中央研究院创新免疫治疗全国重点实验室,上海201203

出　　处：《上海医药》2025年第2期85-89,共5页Shanghai Medical & Pharmaceutical Journal

摘　　要：小干扰RNA(siRNA)是基于RNA干扰(RNAi)技术通过沉默疾病相关基因的表达最终达到治疗效果的一种新型治疗手段,具有治疗效率高、特异性强等特点。自2018年首款siRNA药物上市以来,已成为最受关注的小RNA药物之一,被视为制药领域一次新技术革命。目前siRNA药物的研发正处于快速发展期,吸引众多制药企业纷纷加入赛道。本文简要概述siRNA药物的作用机制、优势及面临的挑战、关键技术突破、在研情况及趋势等,希望为制药企业相关研发人员或项目管理人员提供参考。Small interfering RNA(siRNA)is a new therapeutic method based on RNA interference(RNAi)technology that finally achieves the therapeutic effect by silencing the expression of disease-related genes,which has the characteristics of high therapeutic efficacy and strong specificity.Since the first siRNA drug was approved in 2018,it has become one of the most concerned small RNA drugs and is regarded as another new technological revolution in the pharmaceutical field.At present,siRNA drugs are in a period of rapid development,attracting many pharmaceutical companies to join the track.This review briefly summarizes the mechanism of action,advantage and challenges,key technological breakthroughs,research status and trends of siRNA drugs,hoping to provide references for relevant R&D personnel or project managers in pharmaceutical enterprises.

关 键 词：小RNA 小干扰RNA(siRNA) RNA干扰(RNAI) 化学修饰 递送系统 

分 类 号：R97[医药卫生—药品]