Targeting ligand and use thereof
AssigneeChengdu Xinzhenghe Pharmaceutical Technology Co. Ltd
InventorsChong LI, Ke YIN, Qian LI, Haiping MA
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Abstract
Some embodiments of the present disclosure relate to a nucleic acid delivery compound, a liver-targeting nucleic acid ligand, and the use thereof, which are suitable for the nucleic acid drug delivery. Specifically, a compound as represented by formula (Z-1), and compounds as represented by formula (I) and (II). An oligonucleotide conjugate provided in the embodiments of the present disclosure can improve the binding efficiency of a targeting moiety to a cell or a cell receptor, and increase the effect of RNA interference.
Claims18
A cleavable compound, as shown in formula (Z-1), or a pharmaceutically acceptable salt thereof:
- wherein R1 is O, S, NR3, or CR3R4, and R3 and R4 are independent hydrogen, halogen, substituted or unsubstituted aliphatic group, substituted or unsubstituted aryl group, substituted or unsubstituted heteroaryl group, substituted or unsubstituted heterocyclic group, or substituted or unsubstituted cycloalkyl group;
- wherein R2 is —O—, —S—, —NH—, —CH2—, —C(O)—, —OC(O)—, —C(O)O—, —NHC(O)—, —C(O)NH—,
- —CH2NH—, —CH2O—, —NH—C(O)—CH2—, —C(O)—CH2—NH—, or —NH(CO)NH—, wherein the —CH2— is optionally replaced with a substituent group selected from the group consisting of halogen, alkyl, alkoxy, and alkylamino;
- each of a and b are the same or different, and is independently an integer from 0 to 20.
Dependencies are inferred from the claim text.