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US 11,896,672 B2Granted
Targeted nucleic acid conjugate compositions
AssigneeArbutus Biopharma Corporation
InventorsJames Heyes, Richard J. Holland, Alan D. Martin, Mark Wood
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Abstract
The invention provides conjugates that comprise a targeting moiety, a nucleic acid, and optional linking groups as well as synthetic intermediates and synthetic methods useful for preparing the conjugates. The conjugates are useful to target therapeutic nucleic acids to the liver and to treat liver diseases including hepatitis (e.g. hepatitis B and hepatitis D).
Claims11
The compound:
- or a salt thereof, wherein Q is -L1-R1;
- L1 is a divalent, branched or unbranched, saturated or unsaturated, hydrocarbon chain, having from 1 to 14 carbon atoms, wherein one or more of the carbon atoms in the hydrocarbon chain is optionally replaced with —NRX—C(═O)—, or —C(═O)—NRX—, and wherein RX is hydrogen or (C1-C6)alkyl, and wherein the hydrocarbon chain, is optionally substituted with oxo(═O);
- or
- L1 is selected from the group consisting of:
- R1 has the following formula:
- wherein:
- B1 is CH;
- B2 is selected from the group consisting of:
- B3 is selected from the group consisting of:
- T1 is a branched or unbranched, saturated or unsaturated, hydrocarbon chain, having from 1 to 20 carbon atoms, wherein one or more of the carbon atoms in the hydrocarbon chain is optionally replaced by —NRX—C(═O)—, or —C(═O)—NRX—, and wherein RX is hydrogen or (C1-C6)alkyl, and wherein the hydrocarbon chain, is optionally substituted with oxo (═O), that is covalently bonded to B1;
- T2 is a branched or unbranched, saturated or unsaturated, hydrocarbon chain, having from 1 to 20 carbon atoms, wherein one or more of the carbon atoms in the hydrocarbon chain is optionally replaced by —NRX—C(═O)—, or —C(═O)—NRX—, and wherein RX is hydrogen or (C1-C6)alkyl, and wherein the hydrocarbon chain, is optionally substituted with oxo (═O), that is covalently bonded to B1;
- each of T3, T4, T5, and T6 is independently selected from the group consisting of:
- n=1, 2, or 3;
- each saccharide is independently selected from the group consisting of:
- R2 is a siRNA substituent;
- and
- R′ is C1-9 alkyl, C2-9 alkenyl or C2-9 alkynyl;
- wherein the C1-9 alkyl, C2-9 alkenyl or C2-9 alkynyl are optionally substituted with halo or hydroxyl.
15 more elements
Dependencies are inferred from the claim text.