# US 2025/0145582 A2 · Lipid compounds and lipid nanoparticle compositions

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- Number: US 2025/0145582 A2
- Document ID: US20250145582A2
- Status: Published application
- Kind: A2
- Application number: 18/675,317
- Filed: 2024-05-28
- Published: 2025-05-08
- Earliest claimed: 2022-12-28
- Assignees: Rinuagene Biotechnology Co., Ltd.; Rinuagene International Hk Limited
- Applicants: Rinuagene Biotechnology Co., Ltd.; Rinuagene International Hk Limited
- Inventors: Shan Cen; Kai Lv; Zhenlei Yu; Yijie Dong
- Web page: https://patentgrep.com/patent/US20250145582A2

## CPC

- C07D 317/12: with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, directly attached to ring carbon atoms
- A61K 9/5123: {Organic compounds, e.g. fats, sugars}
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- A61K 48/0033: {the non-active part being non-polymeric}
- A61P 3/00: Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00)
- A61P 3/10: for hyperglycaemia, e.g. antidiabetics
- A61P 9/00: Drugs for disorders of the cardiovascular system
- A61P 13/12: of the kidneys
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A61P 35/00: Antineoplastic agents
- A61P 37/02: Immunomodulators
- C07D 317/30: Radicals substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
- C07D 319/06: not condensed with other rings
- C07D 327/04: Five-membered rings
- C07D 339/06: having the hetero atoms in positions 1 and 3, e.g. cyclic dithiocarbonates
- C12N 15/88: using microencapsulation, e.g. using {amphiphile} liposome vesicle
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

The present application disclosed a compound of formula (I) and a salt and a stereoisomer thereof, wherein each variable is as defined in the specification. The present application also disclosed a nanoparticle composition comprising the compound or a salt or a stereoisomer thereof, and the use of the nanoparticle composition for delivering active agents.

## Claims

### Claim 1

A compound of formula (I), or a salt or a stereoisomer thereof,
wherein
R1 is
R1a and R1b are each independently —CH3;
R2 and R3 are each independently H;
R4 is C10, C11 or C12 linear alkyl;
R5 and R6 are identical and are selected from C6, C7 and C8 linear alkyl;
X and Y are each independently O;
X1 and X2 are independently selected from C═O and O, Y1 and Y2 are independently selected from C═O and O, with the proviso that X1 and Y1 are not both C═O or O, and X2 and Y2 are not both C═O or O;
m is 0, and n is 1;
o is 5 or 6;
p is 7 or 8, and
q is 2, 3 or 4.

### Claim 2

(canceled)

### Claim 3

(canceled)

### Claim 4 (depends on 1)

The compound according to claim 1, or a salt or a stereoisomer thereof, wherein o is 6; and/or p is 7.

### Claim at position 5

5-7. (canceled)

### Claim 8 (depends on 1)

The compound according to claim 1, or a salt or a stereoisomer thereof, the compound of formula (I) having the following structure of formula (IIA):
wherein;
R1a and R1b are each independently —CH3;
R2 and R3 are each independently H;
R4 is C10, C11 or C12 linear alkyl;
R5 and R6 are identical and are selected from C6, C7 and C8 linear alkyl;
X1 and X2 are both C═O, and Y1 and Y2 are both O; or X1 is O, Y1 is C═O, and X2 is C═O, Y2 is O;
o is 5 or 6;
p is 7; and
q is 2 or 3.

### Claim 9 (depends on 8)

The compound according to claim 8, or a salt or a stereoisomer thereof, the compound of formula (I) having a structure selected from:

### Claim 10

(canceled)

### Claim 11

(canceled)

### Claim 12 (depends on 1)

The compound according to claim 1, or a salt or a stereoisomer thereof, wherein q is 2.

### Claim 13 (depends on 1)

The compound according to claim 1, or a salt or a stereoisomer thereof, wherein X1 and X2 are both C═O, and Y1 and Y2 are both O; or X1 is O, Y1 is C═O, and X2 is C═O, Y2 is O; or, X1 is C═O, Y1 is O, and X2 is O, Y2 is C═O.

### Claim 14

(canceled)

### Claim 15

(canceled)

### Claim 16 (depends on 1)

The compound according to claim 1, or a salt or a stereoisomer thereof, wherein R4 is C10-C11 linear alkyl; and/or R5 and R6 are both C8 linear alkyl.

### Claim 17

(canceled)

### Claim 18 (depends on 1)

The compound according to claim 1, or a salt or a stereoisomer thereof, wherein
o is 5, R4 is C11 linear alkyl, p is 7, and R5 and R6 are both C8 linear alkyl, or
is 6, R4 is C10 linear alkyl, p is 7, and R5 and R6 are both C8 linear alkyl.

### Claim 19

A compound, or a salt or a stereoisomer thereof, wherein the compound is selected from

### Claim 20 (depends on 19)

The compound according to claim 19 or a salt or a stereoisomer thereof, wherein the compound is selected from:

### Claim at position 19

21-23. (canceled)

### Claim 24 (depends on 1)

A nanoparticle composition comprising a lipid component which comprises a compound according to claim 1, or a salt or a stereoisomer thereof; said nanoparticle composition is lipid nanoparticle (LNP).

### Claim 25 (depends on 24)

The nanoparticle composition according to claim 24, wherein said lipid component further comprises a phospholipid, a structural lipid and/or a PEG lipid;
wherein said phospholipid is optionally selected from one, two or more of the following compounds:
Dilauroyl lecithin (DLPC),
Dimyristoyl phosphatidylcholine (DMPC),
Dioleoyl lecithin (DOPC),
Dipalmitoyl phosphatidylcholine (DPPC),
Distearoyl phosphatidylcholine (DSPC),
Dioleoyl phosphatidylcholine (DUPC),
Palmitoyl oleoyl phosphatidylcholine (POPC),
1,2-Di-O-octadecyl-sn-glycero-3-phosphocholine (18:0 Diether PC),
1-Oleoyl-2-cholesteryldimethylsuccinoyl-sn-glycero-3-phosphocholine (OChemsPC),
1-Hexadecyl-sn-glycero-3-phosphocholine (C16 Lyso PC),
1,2-Divinyl-sn-glycero-3-phosphocholine,
1,2-Diarylacyl-sn-glycero-3-phosphocholine,
1,2-Dioleoyl-sn-glycero-3phosphorylethanolamine (DOPE),
1,2-Di-phytanoyl-sn-glycero-3-phosphoethanolamine (ME 16.0 PE),
1,2-Distearoyl-sn-glycero-3-phosphoethanolamine,
1,2-Diethenol-sn-glycero-3-phosphoethanolamine,
1,2-Divinyl-sn-glycero-3-phosphoethanolamine,
1,2-Diaryl-sn-glycero-3-phosphoethanolamine,
1,2-Dithiohexaenoate-sn-glycero-3-phosphoethanolamine,
1,2-dioleoyl-sn-glycero-3-phosphate-(1-glycerol) sodium salt (DOPG) or sphingomyelin,
said structural lipid is optionally selected from one, two or more of cholesterol, coprosterol, sitosterol, ergosterol and stigmasterol; and/or
said PEG lipid is optionally selected from one, two or more of PEG-modified phosphatidylethanolamine, PEG-modified phosphatidic acid, PEG-modified ceramide, PEG-modified dialkylamine, PEG-modified diacylglycerol or PEG-modified dialkylglycerol.

### Claim 26

(canceled)

### Claim 27 (depends on 24)

The nanoparticle composition according to claim 24, further comprising a therapeutic agent and/or a prophylactic agent, wherein said therapeutic and/or prophylactic agent is selected from vaccines, compounds capable of eliciting an immune response and nucleic acids,
wherein the nucleic acid is optionally RNA, and said RNA is selected from one, two or more of siRNA, aiRNA, miRNA, dsRNA, shRNA or mRNA.

### Claim 28

(canceled)

### Claim 29

(canceled)

### Claim 30 (depends on 27)

A pharmaceutical composition comprising the nanoparticle composition according to claim 27 and a pharmaceutically acceptable excipient.

### Claim 31 (depends on 27)

A method of delivering a therapeutic and/or prophylactic agent to a mammalian cell, comprising administering the nanoparticle composition according to claim 27 to a subject, wherein said administration comprises contacting the cell with said nanoparticle composition to deliver the therapeutic and/or prophylactic agent to the cell.

### Claim 32

(canceled)

### Claim 33 (depends on 27)

A method of treating a disease or condition in a mammal, comprising administering a therapeutically or prophylactically effective amount of a nanoparticle composition according to claim 27 to the mammal; wherein said disease or condition is selected from infectious diseases, cancer and proliferative diseases, genetic diseases, autoimmune diseases, diabetes, neurodegenerative diseases, cardiovascular diseases, renal vascular diseases and metabolic diseases, and wherein the treating consists of achieving partial or complete response, alleviation, amelioration, or remission of said disease or condition, inhibiting progression of said disease or condition, reducing severity of said disease or condition and/or reducing incidence of one or more symptoms or signs thereof.

### Claim 34

(canceled)

### Claim 35 (depends on 25)

The nanoparticle composition according to claim 25, wherein said lipid component further comprises a phospholipid, a structural lipid and a PEG lipid; wherein said phospholipid is DOPE or DSPC, said structural lipid is cholesterol, and said PEG lipid is selected from PEG-modified phosphatidylethanolamine, PEG-modified phosphatidic acid, PEG-modified ceramide, PEG-modified dialkylamine, PEG-modified diacylglycerol and PEG-modified dialkylglycerol.

### Claim 36 (depends on 1)

A method of preparation of a lipid nanoparticle composition, comprising using the compound according to claim 1 or a salt or a stereoisomer thereof.

### Claim 37 (depends on 27)

A method of delivering a therapeutic and/or prophylactic agent to a patient, comprising administering to a patient in need thereof the nanoparticle composition according to claim 27.

### Claim 38 (depends on 27)

A method of introducing a nucleic acid into a cell, comprising contacting said cell with a nanoparticle composition according to claim 27.