公开(公告)号：US20230016929A1

公开(公告)日：2023-01-19

申请号：US17774477

申请日：2020-11-06

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Jayaprakash K. NAIR ,  Martin A. MAIER ,  Juan C. SALINAS ,  Shigeo MATSUDA ,  Alexander V. KEL'IN ,  Scott P. LENTINI ,  Guo HE ,  Michelle H. JUNG ,  Justin M. PIERSON ,  Muthiah MANOHARAN ,  Dale C. GUENTHER ,  Ivan ZLATEV ,  Christopher S. THEILE ,  Vasant R. JADHAV ,  Stuart MILSTEIN ,  Maja JANAS ,  Dhrubajyoti DATTA

IPC分类号： A61K31/713 ,  A61K48/00 ,  C12N15/113 ,  A61P25/28

摘要： One aspect of the present invention relates to a compound comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic monomers, containing one or more lipophilic moieties, conjugated to one or more positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic monomer-conjugated compound.