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US 2023/0322689 A1Published application
Ionizable lipid compounds and lipid nanoparticle compositions
AssigneeSunVax mRNA Therapeutics Inc.
InventorsLibin ZHANG, Yingzhong LI
Life of this patent
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Abstract
Novel ionizable lipids, compositions, and methods of using the novel ionizable lipids and compositions are disclosed. Lipid nanoparticle compositions include a novel ionizable lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticle compositions further including biologically active agents such as mRNA or DNA are useful in the delivery of biologically active agents to mammalian cells or organs.
Claims30
A compound selected from Formulae I to VII, wherein Formula I is
- or a salt or isomer thereof, wherein
- each m and n are independently an integer from 0-10;
- each R1 and R2 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R3, R4 and R5 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24
- each X is independently CH2, NH, O, or S;
- each Y is independently CH2, NH, O, or S;
- each Z is independently CH or N;
- and
- each L is independently CH2, NH, O, or S;
- wherein Formula II is
- or a salt or isomer thereof, wherein
- each n is independently an integer from 0-10;
- each R1 and R2 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R3, R4 and R5 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each X is independently selected from CH, or N;
- each Y is independently selected from CH2, NH, O, or S;
- and
- each Z is independently selected from CH2, NH, O, or S;
- wherein Formula III is
- or a salt or isomer thereof, wherein
- each n is independently an integer from 0-10;
- each R1, R1′, R2 and R3 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R9 is independently selected from
- wherein each R10, R10′, R10″, R11, R11′, R11″, and R12 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- R3″, R4″, and R5″ are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- wherein Formula IV is
- or a salt or isomer thereof, wherein
- each R1, R2, R3, R4 and R5 is independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R6, R7, R8 and R9 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each X is independently selected from CH, N;
- each Y is independently selected from CH2, NH, O, or S;
- each Z is independently selected from CH or N;
- wherein Formula V is
- or a salt or isomer thereof, wherein
- each R1, R2, R3, R4, R5 and R6 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R3′, R4′ and R5′ are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- wherein Formula VI is
- or a salt or isomer thereof, wherein
- each n is independently an integer from 0-10;
- each R1 and R2 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R3, R4 and R5 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each Y is independently selected from CH or N;
- each Z is independently selected from CH2, NH, O, or S;
- and
- wherein Formula VII is
- or a salt or isomer thereof, wherein
- each m and n are independently an integer from 0-10;
- each R1 and R2 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R3, R4 and R5 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each X is independently selected from CH2, NH, O, or S;
- each Y is independently selected from CH, N;
- each Z is independently selected from CH2, NH, O, or S;
- and
- each E is independently selected from CH2, NH, O, or S.
66 more elements
A compound selected from Formulae I-1, II-1, III-1, VI-1, and VII-1, wherein Formula I-1 is
- or a salt or isomer thereof, wherein
- each R1 and R2 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- each a is independently an integer selected from 0-24;
- each b and c are independently an integer selected from 0-24;
- each R3, R4 and R5 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- wherein Formula II-1 is
- or a salt or isomer thereof, wherein
- each R1 and R2 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R3, R4 and R5 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- wherein Formula III-1 is
- or a salt or isomer thereof, wherein
- each R1, R1′, R2, R3, R4, R5, R6, R7 and R8 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- R3″, R4″, and R5″ are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- or wherein each R4, R5, R6, R7 and R8 are independently:
- wherein R1′″, R1″″, R2′″ and R3′″ are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- each a, b, and c are independently an integer selected from 0-24;
- each X is independently CH, N, O or S;
- each Y is independently CH, N, O or S
- each Z is independently CH, N, O or S
- each F is independently CH, N, O or S and
- each E is independently CH, N, O or S;
- wherein Formula VI-1 is
- or a salt or isomer thereof, wherein
- each n is independently an integer from 0-10;
- each R1 and R2 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R3, R4 and R5 is independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- wherein Formula VII-1 is
- wherein each R1 and R2 is independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R3, R4 and R5 are independently selected from C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24.
35 more elements
Dependencies are inferred from the claim text.