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US 2024/0366518 A1Published application
Compounds, compositions, and methods of using thereof
AssigneeElevateBio Technologies, Inc.
InventorsJessica Deterling, Sean Essex, Lorena Lerner, Qi-Ying Hu, Christophe Quéva
Life of this patent
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Abstract
The present disclosure includes, among other things, lipids, compositions, and methods useful for delivering a polynucleotide or oligonucleotide, e.g., viral genome.
Claims30
A compound of Formula (I):
- or a pharmaceutically acceptable salt or solvate thereof, wherein:
- A is —N(CH2RN1)(CH2RN2) or a 4-7-membered heterocyclyl ring containing at least one N, wherein the 4-7-membered heterocyclyl ring is optionally substituted with 0-6 R3;
- each X is independently —O—, —N(R1)—, or —N(R2)—;
- R1 is selected from the group consisting of optionally substituted C1-C31 aliphatic and steroidyl;
- R2 is selected from the group consisting of optionally substituted C1-C31 aliphatic and steroidyl;
- R3 is optionally substituted C1-C6 aliphatic;
- RN1 and RN2 are each independently hydrogen, hydroxy-C1-C6 alkyl, C2-C6 alkenyl, or a C3-C7 cycloalkyl;
- L1 is selected from the group consisting of an optionally substituted C1-C20 alkylene chain and a bivalent optionally substituted C2-C20 alkenylene chain;
- L2 is selected from the group consisting of an optionally substituted C1-C20 alkylene chain and a bivalent optionally substituted C2-C20 alkenylene chain;
- and
- L3 is a bond, an optionally substituted C1-C6 alkylene chain, or a bivalent optionally substituted C3-C7 cycloalkylene;
- and
- with the proviso that when A is —N(CH3)(CH3) and X is O, L3 is not an C1-C6 alkylene chain.
10 more elements
A compound of Formula (A-a), Formula (A-b), Formula (A-c), or Formula (A-d):
- or a pharmaceutically acceptable salt thereof, wherein:
- n is an integer between 10 to 200, inclusive of all endpoints;
- RP1 is C5-C25 alkyl or C5-C25 alkenyl;
- and
- RP2 is hydrogen or —CH3.
2 more elements
A lipid nanoparticle (LNP) comprising an ionizable lipid, wherein the ionizable lipid is a compound of Formula (I):
- or a pharmaceutically acceptable salt or solvate thereof, wherein:
- A is —N(CH2RN1)(CH2RN2) or a 4-7-membered heterocyclyl ring containing at least one N, wherein the 4-7-membered heterocyclyl ring is optionally substituted with 0-6 R3;
- each X is independently —O—, —N(R1)—, or —N(R2)—;
- R1 is selected from the group consisting of optionally substituted C1-C31 aliphatic and steroidyl;
- R2 is selected from the group consisting of optionally substituted C1-C31 aliphatic and steroidyl;
- R3 is optionally substituted C1-C6 aliphatic;
- RN1 and RN2 are each independently hydrogen, hydroxy-C1-C6 alkyl, C2-C6 alkenyl, or a C3-C7 cycloalkyl;
- L1 is selected from the group consisting of an optionally substituted C1-C20 alkylene chain and a bivalent optionally substituted C2-C20 alkenylene chain;
- L2 is selected from the group consisting of an optionally substituted C1-C20 alkylene chain and a bivalent optionally substituted C2-C20 alkenylene chain;
- and
- L3 is a bond, an optionally substituted C1-C6 alkylene chain, or a bivalent optionally substituted C3-C7 cycloalkylene;
- and
- with the proviso that when A is —N(CH3)(CH3) and X is O, L3 is not an C1-C6 alkylene chain.
10 more elements
A lipid nanoparticle (LNP) comprising a polyethyleneglycol (PEG)-lipid, wherein the PEG-lipid is a compound of Formula (A-a), Formula (A-b), Formula (A-c), or Formula (A-d):
- or a pharmaceutically acceptable salt thereof, wherein:
- n is an integer between 10 to 200, inclusive of all endpoints;
- RP1 is C5-C25 alkyl or C5-C25 alkenyl;
- and
- RP2 is hydrogen or —CH3.
2 more elements
Dependencies are inferred from the claim text.