THEILE, CHRISTOPHER,MILSTEIN, STUART,SCHLEGEL, MARK K.,JANAS, MAJA,JADHAV, VASANT R.,FOSTER, DONALD,MANOHARAN, MUTHIAH,RAJEEV, KALLANTHOTTATHIL G.,JAYARAMAN, MUTHUSAMY,KEL'IN, ALEXANDER V.,MATSUDA, SH,KELIN, ALEXANDER V.,MATSUDA, SHI
申请号：
CA3044598
公开号：
CA3044598A1
申请日:
2017.11.22
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region； the dsRNA comprises at least four 2'-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
