发明申请
- 专利标题: CARBOHYDRATE CONJUGATES AS DELIVERY AGENTS FOR OLIGONUCLEOTIDES
- 专利标题(中): 碳氢化合物作为交付剂的交付代理
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申请号: US13326203申请日: 2011-12-14
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公开(公告)号: US20120136042A1公开(公告)日: 2012-05-31
- 发明人: Muthiah MANOHARAN , Kallanthottathil G. RAJEEV , Narayanannair K. JAYAPRAKASH , Martin MAIER
- 申请人: Muthiah MANOHARAN , Kallanthottathil G. RAJEEV , Narayanannair K. JAYAPRAKASH , Martin MAIER
- 申请人地址: US MA CAMBRIDGE
- 专利权人: ALNYLAM PHARMACEUTICALS, INC
- 当前专利权人: ALNYLAM PHARMACEUTICALS, INC
- 当前专利权人地址: US MA CAMBRIDGE
- 主分类号: A61K31/713
- IPC分类号: A61K31/713 ; C07K2/00 ; C12N5/071 ; C07H21/02
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; and Z′, Z″, Z′″ and Z″″ are each independently for each occurrence O or S.
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