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US 12,180,138 B2Granted
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.
Claims28
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)qC(O)(CH2)rCH(R8)(R9), wherein one of R1 and R2 is an unsubstituted, linear or branched C6-C30 alkyl,
- wherein:
- q is an integer from 0 to 12,
- r is 0,
- R8 is 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 is unsubstituted linear C6-C30 alkyl;
- R2 is —(CH2)qC(O)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 C2-C8 alkyl.
7 more elements
Dependencies are inferred from the claim text.