发明名称 CARBOHYDRATE CONJUGATES AS DELIVERY AGENTS FOR OLIGONUCLEOTIDES
摘要 The present invention provides iRNA agents comprising at least one subunit of the formula (I):;wherein:A and B are each independently for each occurrence O, N(RN) or S;X and Y are each independently for each occurrence H, OH, a hydroxyl protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, —P(Z′)(Z″)O-nucleoside, —P(Z′)(Z″)O-oligonucleotide, a lipid, a PEG, a steroid, a lipophile, a polymer, —P(Z′)(Z″)O-Linker-OP(Z′″)(Z″″)O-oligonucleotide, a nucleotide, an oligonucleotide, —P(Z′)(Z″)-formula(I), —P(Z′)(Z″)— or -Linker-R;R is LG, -Linker-LG, or has the structure shown below:;LG is independently for each occurrence a carbohydrate, e.g., monosaccharide, disaccharide, trisaccharide, tetrasaccharide, oligosaccharide, polysaccharide;RN is independently for each occurrence H, methyl, ethyl, propyl, isopropyl, butyl, or benzyl; andZ′, Z″, Z′″ and Z″″ are each independently for each occurrence O or S.
申请公布号 US2016051691(A1) 申请公布日期 2016.02.25
申请号 US201514835272 申请日期 2015.08.25
申请人 Alnylam Pharmaceuticals, Inc. 发明人 MANOHARAN Muthiah;RAJEEV Kallanthottathil G.;NAIR Jayaprakash;MAIER Martin
分类号 A61K47/48;A61K31/713;A61K31/7088 主分类号 A61K47/48
代理机构 代理人
主权项
地址 Cambridge MA US