US2011313144A1PendingUtilityA1
iRNA Agents Targeting CCR5 Expressing Cells And Uses Thereof
Est. expiryNov 21, 2026(~0.4 yrs left)· nominal 20-yr term from priority
C07D 211/96C07D 401/12C07D 405/12C07D 211/24C07D 471/10C07D 309/14C07D 401/14C07H 21/00C07D 407/12C07D 409/12C07D 211/58
53
PatentIndex Score
0
Cited by
0
References
0
Claims
Abstract
The invention relates to iRNA agents that preferably include a modification that targets CC chemokine receptor 5 (CCR5). The invention also relates to methods of making and using such modified iRNA agents.
Claims
exact text as granted — not AI-modified1 . A compound having structure:
wherein,
A is H, a hydroxyl protecting group, a phosphate group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, a nucleotide, or an oligonucleotide;
B is H, a hydroxyl protecting group, a phosphate group, an activated phosphate group, an activated phosphate group, a phosphoramidite, a solid support, a nucleotide, or an oligonucleotide;
Z is a CCR5-binding ligand;
Q is —(CH 2 ) n NH—, —C(O)(CH 2 ) n NH—, —(CH 2 ) n ONH—, —C(O)(CH 2 ) n ONH—, —C(O)—(CH 2 ) n —, —(CH 2 ) n NHNH 2 —, —C(O)(CH 2 ) n NHNH 2 —, —C(O)—O—, —(CH 2 ) n —C(O)—, —C(O)—NH—, —C(O)—, —C(O)—(CH 2 ) n —C(O)—, —C(O)—(CH 2 ) n —C(O)O—, —C(O)—(CH 2 ) n —NH—C(O)—, —(CH 2 ) n —C(O)O—, —C(O)(CH 2 ) n NHC(O)(CH 2 ) m —, —C(O)(CH 2 ) n NHC(O)(CH 2 ) m NH—, —(CH 2 ) n —NH—C(O)—, or —(CH 2 ) n —,
n is 1-20; and
m is 1-6.
2 . The compound of claim 1 , wherein A is an activated phosphite group, a phosphoramidite, or a solid support.
3 . The compound of claim 1 , wherein B is a hydroxyl protecting group.
4 . The compound of claim 1 , wherein Q is —C(O)(CH 2 ) n NHC(O)(CH 2 ) m NH—, —C(O)(CH 2 ) n NHC(O)(CH 2 ) m —, —C(O)(CH 2 ) n NH—, or.
5 . The compound of claim 4 , wherein Q is —C(O)(CH 2 ) n NHC(O)(CH 2 ) m NH—.
6 . The compound of claim 1 , wherein the CCR5-binding ligand is a CCR5 antagonist.
7 . The compound of claim 6 , wherein the CCR5 antagonist is an antagonist listed in column 1 of Table 1.
8 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
9 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
10 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
11 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
12 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
13 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
14 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
15 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
16 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
17 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
18 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
19 . The compound of claim 7 , wherein the CCR5 antagonist has the following structure
20 . The compound of claim 1 , wherein A is an oligonucleotide.
21 . The compound of claim 20 , wherein the oligonucleotide is double stranded.
22 . The compound of claim 1 , wherein B is an oligonucleotide.
23 . The compound of claim 22 , wherein the oligonucleotide is double stranded.
24 . The compound of claim 1 , wherein A is an oligonucleotide and B is an oligonucleotide.
25 . The compound of claim 24 , wherein oligonucleotide is double stranded.Cited by (0)
No later patents cite this yet.
References (0)
No backward citations on record.