ГУДНАУ ДЖ. Роберт Алан (US),ГАМИЛЬТОН Мэтью Майкл (US),КОВАЛЬЧИК Агнешка (US),СИДДУРИ Ачютарао (US)
申请号：
RU2014132837
公开号：
RU2014132837A
申请日:
2013.01.24
申请国别(地区):
RU
年份:
2016
代理人:
摘要：
1. A compound of formula I: or a pharmaceutically acceptable salt or ester thereof where n is 1-24, and wherein R1 is selected from the group consisting of the following compounds: (1) a compound of the formula: (2) a compound of the formula: and (3) compounds of the formula: wherein Q is H or OH, R2 is selected from the group consisting of the following compounds: (1) a compound of the formula: (2) compounds of the formula: (3) compounds of the formula: and (4) compounds of the formula: wherein R3 is a conjugated moiety and is either a sulfur atom or a compound of the formula: to PEG. to S2. A compound according to claim 1, wherein R1 is a compound of the formula: .3. A compound according to claim 1, wherein R1 is a compound of the formula: .4. A compound according to claim 1, wherein R1 is a compound of the formula: wherein Q is H or OH. A compound according to claim 4, wherein Q is H. The compound of claim 4, where Q is OH. A compound according to claim 1, wherein R2 is a compound of the formula: .8. A compound according to claim 1, wherein R2 is a compound of the formula:. A compound according to claim 1, wherein R2 is a compound of the formula: .10. A compound according to claim 1, wherein R2 is a compound of the formula: wherein R3 is a mono- or double-stranded oligonucleotide, and X is either a sulfur atom or a compound of the formula: to PEG. to S11. A compound according to claim 10, wherein X is a sulfur atom. A compound according to any of the preceding claims. 10 or 11, wherein R3 is a siRNA molecule. A compound according to any of the preceding claims. 7-10, wherein R1 is a compound of the formula:. A compound according to any of the preceding claims. 7-10, wherein R1 is a compound of the formula:. A compound according to any of the preceding claims. 7-10, wherein R1 is a compound of the formula: where Q is H or OH. The compound according to claim 1, selected from the group consisting of the following compounds: (S) -3- {3- {2- [2- (2- {2- [3- (2,5-dioxo
