破茧 2026·mRNA 与小核酸药物行业研究白皮书

前言..................................................................................................................................................4第一章破茧2026·mRNA与小核酸药物行业研究总览............................................................6第二章破茧2026·mRNA疫苗与药物行业研究......................................................................112.1mRNA的结构优化与递送系统构建..............................................................................122.2mRNA疫苗......................................................................................................................162.2.1 mRNA抗感染疫苗（新冠疫苗/流感疫苗等）..................................................192.2.2 mRNA肿瘤疫苗...................................................................................................212.3 mRNA药物......................................................................................................................232.3.1 mRNA细胞疗法...................................................................................................242.3.2 mRNA蛋白替代疗法...........................................................................................252.3.3 mRNA编码细胞因子疗法...................................................................................272.3.4 mRNA编码治疗性抗体.......................................................................................282.4获批上市的mRNA疗法................................................................................................292.5国内mRNA疫苗与药物代表性企业............................................................................312.5.1 mRNA个性化肿瘤疫苗代表性企业研发进展...................................................322.5.2通用型mRNA肿瘤疫苗代表性企业研发进展.................................................332.5.3国内mRNA抗感染疫苗代表性企业研发进展.................................................352.5.4国内获批IND盘点...............................................................................................36第三章破茧2026·小核酸药物行业研究..................................................................................423.1 siRNA...............................................................................................................................453.1.1罕见病已成体系攻克...........................................................................................483.1.2代谢性疾病稳扩市场............................................................................................523.1.3心血管适应症拓展蓝海.......................................................................................543.2 ASO...................................................................................................................................573.2.1罕见病已成体系攻克............................................................................................613.2.2乙肝小核酸有望全球首批....................................................................................633.2.3肿瘤与血液疾病现拓展潜力................................................................................653.3 miRNA..............................................................................................................................653.4其他小核酸疗法..............................................................................................................673.4.1核酸适配体(Aptamer)...........................................................................................673.4.2小激活RNA(saRNA)............................................................................................683.5获批上市的小核酸药物...................................................................................................71第四章破茧2026·核酸疗法递送技术行业研究.......................................................................74 4.1经典的LNP递送.............................................................................................................774.1.1 LNP........................................................................................................................774.1.2 mRNA-LNP...........................................................................................................794.2肝靶向 ...............................................................................................................................824.2.1小核酸-GalNAc....................................................................................................844.2.2小核酸-LNP..........................................................................................................854.3肝外递送...........................................................................................................................874.3.1 AOC.......................................................................................................................894.3.2增强靶向递送LNPs..............................................................................................924.4获批上市的核酸药物及其所用递送载体一览...............................................................93第五章破茧2026·核酸疗法企业融资、交易行业研究...........................................................955.1核酸企业融资、IPO........................................................................................................955.1.1国内小核酸企业融资盘点