FIELD: medicine; pharmaceuticals.
SUBSTANCE: invention relates to a compound of general formula (I) or a pharmaceutically acceptable salt thereof, where each RX independently represents a group of formula (A1); each R1 independently represents a group of formula (Bl-a); each RB independently represents -NRE RF; each RC independently represents -C(=O)C1-6 alkyl; each RE independently represents C(=O)C1-6 alkyl; each RF is independently H; each R2 is independently selected from a group consisting of: -C(R6)2-; *-NR7C(=O)-; where * represents a point of attachment to the structure presented in the claims; R3 is selected from a group given in the claim; -L3 is –C(R6)2-; R4 is selected from a group consisting of: -C(R6)2-; *-NR7C(=O)-; - R5 is selected from the group given in the claim, where Pg is C6-10 aryl substituted with 1–5 substituents, each of which is independently selected from the group given in the claim, where Pg2 is selected from the group consisting of 4-monomethoxytrityl (MMTR); 4,4-dimethoxytrityl (DMTR); and triphenylmethyl (trityl); each R6 is independently selected from a group consisting of: H; each R7 is independently selected from a group consisting of: H; each of a and b is an independently selected integer from 1 to 10; each of c and d is an independently selected integer from 1 to 10. Invention also relates to a conjugated compound, use of the conjugated compound, a method for detecting or localizing RNA in the liver, a pharmaceutical composition and use of the pharmaceutical composition.
EFFECT: novel compounds which contain one or more asialoglycoprotein receptor (ASGPR) ligand fragments and which can additionally contain an oligonucleotide used for targeted delivery of oligonucleotides to liver cells.
16 cl, 5 tbl, 18 ex
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Authors
Dates
2024-05-28—Published
2021-01-20—Filed