发明名称 |
OLIGONUCLEOTIDES COMPRISING A C5-MODIFIED PYRIMIDINE |
摘要 |
One aspect of the present invention relates to a double-stranded oligonucleotide comprising at least one ligand. In certain embodiments, a ligand is bound to only one of the two oligonucleotide strands comprising the double-stranded oligonucleotide. In certain embodiments, both of the oligonucleotide strands of the double-stranded oligonucleotide independently comprise a bound ligand. In certain embodiments, the oligonucleotide strands comprise at least one modified sugar moiety. In certain embodiments, a phosphate linkage in one or both of the strands of the oligonucleotide has been replaced with a phosphorothioate or phosphorodithioate linkage. In a preferred embodiment, the ligand is cholesterol or 5.beta.-cholanic acid. Another aspect of the present invention relates to a single-stranded oligonucleotide comprising at least one ligand. In certain embodiments, the oligonucleotide comprises at least one modified sugar moiety. In certain embodiments, a phosphate linkage of the oligonucleotide has been replaced with a phosphorothioate or phosphorodithioate linkage. In a preferred embodiment, the ligand is cholesterol or 5.beta.-cholanic acid. The ligand improves the pharmacokinetic properties of the oligonucleotide. |
申请公布号 |
CA2562151(C) |
申请公布日期 |
2016.09.06 |
申请号 |
CA20052562151 |
申请日期 |
2005.04.29 |
申请人 |
ALNYLAM PHARMACEUTICALS, INC. |
发明人 |
MANOHARAN, MUTHIAH;RAJEEV, KALLANTHOTTATHIL G. |
分类号 |
C07H21/00;A61K31/7052;A61K31/7064;A61K31/7076;A61K48/00;A61P25/28;A61P29/00;A61P31/00;A61P33/00;A61P35/00;C07H21/02;C12N15/11;C12N15/113 |
主分类号 |
C07H21/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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