小核酸有望成为小分子和抗体后的第三大类药物。小核酸药物指ASO(反义寡核苷酸)、siRNA(小干扰 RNA)、miRNA(微小RNA)等短链核酸,通过与mRNA相互作用调节基因表达,具备靶标广、特异性强、研发效率高和给药间隔长等独特优势,极有潜力成为可产生大量产品的新技术路径。罕见病商业化成熟,慢性病长效优势显现。目前,共有11 款ASO、7 款siRNA 和1款Aptamer(适配体)上市销售。...
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