申请人:Alnylam Pharmaceuticals, Inc.
公开号:EP3808846A1
公开(公告)日:2021-04-21
The present invention relates to a double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene, comprising a sense strand and an antisense strand, with a duplex region of 17-21 nucleotide pairs in length, wherein the dsRNA agent is represented by formula (I):
wherein:
B1 is 2'-OMe or 2'-F, n1 is 8, T1 is 2'F, n2 is 3, B2 is 2'-OMe, n3 is 7, n4 is 0, B3 is 2'-OMe, n5 is 3, B1' is 2'-OMe or 2'-F, q1 is 9, Tl' is 2'-F, q2 is 1, B2' is 2'-OMe or 2'-F, q3 is 4, q4 is 0, B3' is 2'-OMe or 2'-F, q5 is 7, T3' is 2'-F, q6 is 1, B4' is 2' OMe, and q7 is 1.
本发明涉及一种能够抑制靶基因表达的双链 RNA(dsRNA)制剂,包括有义链和反义链,双链区长度为 17-21 个核苷酸对,其中 dsRNA 制剂由式(I)表示:
其中
B1是2'-OMe或2'-F,n1是8,T1是2'F,n2是3,B2是2'-OMe,n3是7,n4是0,B3是2'-OMe,n5是3,B1'是2'-OMe或2'-F,q1是9、Tl' 是 2'-F,q2 是 1,B2' 是 2'-OMe 或 2'-F,q3 是 4,q4 是 0,B3' 是 2'-OMe 或 2'-F,q5 是 7,T3' 是 2'-F,q6 是 1,B4' 是 2' OMe,q7 是 1。