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US 2024/0190808 A1Published application
Lipid nanoparticle compositions for delivering circular polynucleotides
AssigneeOrna Therapeutics, Inc.
InventorsAllen T. HORHOTA, Junghoon YANG, Kevin J. KAUFFMAN, Thomas BARNES, Robert Alexander WESSELHOEFT, Amy M. BECKER, Gregory MOTZ
Life of this patent
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Abstract
Disclosed herein are novel lipids that can be used in combination with other lipid components, such as helper lipids, structural lipids, and cholesterols, to form lipid nanoparticles for delivery of therapeutic agents, such as nucleic acids (e.g., circular polynucleotides), both in vitro and in vivo.
Claims31
1-107. (canceled)
An ionizable lipid represented by Formula (13*):
- or a pharmaceutically acceptable salt thereof, wherein:
- n* is an integer from 1 to 7;
- Ra is hydrogen;
- Rb is hydrogen or C1-C6 alkyl;
- R1 and R2 are each independently unsubstituted, linear, or branched C6-C30 alkyl or —(CH2)qOC(O)(CH2)rCH(R8)(R9), wherein at least one of R1 and R2 is —(CH2)qOC(O)(CH2)rCH(R8)(R9),
- wherein:
- q is an integer from 0 to 12,
- r is 0,
- R8 is H or R10, and
- R9 and R10 are independently unsubstituted linear C1-C12 alkyl or unsubstituted linear C2-C12 alkenyl.
7 more elements
An ionizable lipid represented by Formula (13):
- or a pharmaceutically acceptable salt thereof, wherein:
- n is 1, 2, 3, or 4;
- Ra is hydrogen;
- R1 and R2 are each independently —(CH2)qOC(O)(CH2)rCH(R8)(R9),
- wherein:
- q is 3, 4, 5, or 6,
- r is 0,
- R8 is R10, and
- R9 and R10 are independently unsubstituted linear C4-C8 alkyl.
6 more elements
Dependencies are inferred from the claim text.