MANOHARAN Muthiah,RAJEEV Kallanthottathil G.,NAIR Jayaprakash,MAIER Martin
申请号:
US201715817473
公开号:
US2018326070(A1)
申请日:
2017.11.20
申请国别(地区):
美国
年份:
2018
代理人:
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 ind