# US 10,500,158 B2 · Cationic lipid

> Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt

- Number: US 10,500,158 B2
- Document ID: US10500158B2
- Status: Granted
- Kind: B2
- Application number: 15/322,271
- Filed: 2015-06-30
- Published: 2017-08-10
- Granted: 2019-12-10
- Earliest claimed: 2014-06-30
- Assignees: Kyowa Hakko Kirin Co., Ltd.
- Applicants: Kyowa Hakko Kirin Co., Ltd.
- Inventors: Shintaro Hosoe; Tomoyuki Naoi
- Web page: https://patentgrep.com/patent/US10500158B2

## CPC

- A61K 9/1272: comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers (lipids as modifying agents {A61K47/543})
- A61K 9/0019: {Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00)}
- A61K 31/7105: Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
- A61K 31/713: Double-stranded nucleic acids or oligonucleotides
- A61K 47/541: {Organic ions forming an ion pair complex with the pharmacologically or therapeutically active agent}
- A61K 47/545: {Heterocyclic compounds (A61K47/558 takes precedence)}
- A61P 1/16: for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
- A61P 7/00: Drugs for disorders of the blood or the extracellular fluid
- A61P 11/00: Drugs for disorders of the respiratory system
- A61P 13/12: of the kidneys
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C07D 205/04: having no double bonds between ring members or between ring members and non-ring members
- C07D 207/06: with radicals, containing only hydrogen and carbon atoms, attached to ring carbon atoms
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/315: Phosphorothioates
- C12N 2310/344: Position-specific modifications, e.g. on every purine, at the 3'-end
- C12N 2320/32: Special delivery means, e.g. tissue-specific
- 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
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- 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 invention provides a cationic lipid represented by the formula (I) wherein R1 and R2 each represents alkyl having 8 to 24 carbon atoms or the like; R3 represents a hydrogen atom, alkyl having 1 to 3 carbon atoms, the formula (A) wherein R4 and R5 each represents a hydrogen atom or the like, and n3 represents an integer from 2 to 6, or the formula (B) wherein R6 and R7 each represents a hydrogen atom or the like, and n4 represents an integer from 1 to 6; n1 represents an integer from 0 to 4; and n2 represents an integer from 1 to 4, provided that the case where n1 is 0 and n2 is 1 is excluded, and a composition which contains the cationic lipid and a nucleic acid, or the like.

## Claims

### Claim 1

A cationic lipid of the formula (I″):
wherein R1′ and R2′ are, the same or different, linear, alkenyl, having 12 to 24 carbon atoms;
R3′ is alkyl having 1 to 3 carbon atoms.

### Claim 2 (depends on 1)

The cationic lipid according to claim 1, wherein R1′ and R2′ are, the same or different, selected from the group consisting of (Z)-tetradec-9-enyl, (Z)-hexadec-9-enyl, (Z)-octadec-6-enyl, (Z)-octadec-9-enyl, (E)-octadec-9-enyl, (Z)-octadec-11-enyl, (9Z,12Z)-octadeca-9,12-dienyl, (9Z,12Z,15Z)-octadeca-9,12,15-trienyl, (Z)-icos-11-enyl, (11Z,14Z)-icosa-11,14-dienyl, and (Z)-docos-13-enyl.

### Claim 3 (depends on 1)

The cationic lipid according to claim 1, wherein R1′ and R2′ are, the same or different, selected from the group consisting of (Z)-hexadec-9-enyl, (Z)-octadec-9-enyl, (9Z,12Z)-octadeca-9,12-dienyl, and (11Z,14Z)-icosa-11,14-dienyl.

### Claim 4 (depends on 1)

The cationic lipid according to claim 1, wherein R1′ and R2′ are the same.

### Claim 5 (depends on 1)

A composition comprising the cationic lipid according to claim 1 and a nucleic acid.

### Claim 6 (depends on 5)

The composition according to claim 5, wherein the cationic lipid and the nucleic acid form a complex, or the cationic lipid combined with a neutral lipid and/or a polymer and the nucleic acid form a complex.

### Claim 7 (depends on 5)

The composition according to claim 5, wherein the cationic lipid and the nucleic acid form a complex, or the cationic lipid combined with a neutral lipid and/or a polymer and the nucleic acid form a complex, and the composition contains a lipid membrane with which the complex is enclosed.

### Claim 8 (depends on 5)

The composition according to claim 5, wherein the nucleic acid is a nucleic acid having a silencing effect on a target gene through the use of RNA interference (RNAi).

### Claim 9 (depends on 8)

The composition according to claim 8, wherein the target gene is a gene expressed in the liver, the lung, the kidney, or the spleen.

### Claim 10 (depends on 9)

A therapeutic agent for a disease related to the liver, the lung, the kidney, or the spleen, comprising a composition according to claim 9.

### Claim 11 (depends on 10)

The therapeutic agent for a disease related to the liver, the lung, the kidney, or the spleen according to claim 10, wherein the therapeutic agent is for intravenous administration.

### Claim 12 (depends on 8)

A medicament for use in the treatment of a disease, comprising a composition according to claim 8.

### Claim 13 (depends on 12)

The medicament according to claim 12, wherein the medicament is for intravenous administration.