内容目录1、小核酸药物概述—引领生物医药变革..............................................................51.1向阳花木易为春，小核酸药物优势显著...................................................51.2小核酸药物的发展现状...............................................................................72、小核酸药物的作用机理与研发趋势..................................................................92.1ASO与siRNA药物作用机理........................................................................92.2ASO与siRNA的管线研发现状....................................................................102.3小核酸药物化学修饰发展趋势...................................................................122.4小核酸药物递送系统发展概况...................................................................142.4.1阳离子脂质体(LNP)递送系统............................................................152.4.2GalNAc技术.......................................................................................153、反义寡核苷酸（ASO）已上市及潜力药物分析...............................................163.1Fomivirsen—开创ASO药物之先河................................................................163.2Mipomersen—降脂领域第一款ASO药物...................................................183.3Eteplisen—争议中前行的ASO药物.........................................................203.4Nusinersen—第一款重磅炸弹...................................................................223.5Inotersen—商业化上落于下风.................................................................243.6Volanesoren—静待继续开花结果...................................................................273.7Golodirsen与Viltolarsen—后续值得关注...........................................293.8ASO成功的关键因素....................................................................................304、siRNA药物—每一部分都匠心独具...................................................................314.1Patisiran-基于脂质体LNP,实现siRNA药物零的突破..........................314.1.1核酸的化学修饰................................................................................314.1.2脂质体LNP递送系统........................................................................334.1.3临床疗效............................................................................................354.1.4专利情况............................................................................................364.1.5Patisiran商业化成功.....................................................................374.2Givosiran—GalNAc大放光彩....................................................................374.2.1GalNAc递送系统...............................................................................384.2.2临床疗效............................................................................................394.2.3专利情况............................................................................................404.3Inclisiran——进军慢病领域...................................................................414.3.1Inclisiran作用机理.......................................................................414.3.2Inclisiran临床疗效.......................................................................424.3.3Inclisiran意义重大.......................................................................434.4siRNA发展总结............................................................................................435、他山之石——国外小核酸领域头部企业.........................................................435.1Alnylam——行业坚守者.............................................................................435.2Ionis——小核酸行业的先驱.....................................................................465.3Sarepta——深耕DMD领域.........................................................................475.4Arrowhead-专注主动靶向递送...................................................................495.5WhiteOakGroup——肿瘤领域的新型脂质体递送系统.........................506、投资建议..............................................................................................................516.1苏州瑞博.......................................................................................................516.2海昶生物.......................................................................................................526.3圣诺制药.......................................................................................................536.4中美瑞康.......................................................................................................54

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