CATIONIC LIPID

• US 20170197903A1
• Filed: 08/06/2015
• Published: 07/13/2017
• Est. Priority Date: 08/07/2014
• Status: Active Application

• Current Assignee: Takeda Pharmaceutical Company Limited
• Original Assignee: Takeda Pharmaceutical Company Limited
• Inventors: Hoashi, Yasutaka
• Granted Patent: US 10,252,974 B2
• Time in Patent Office: Days
• CPC Class Codes: A61K 31/7105 Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
• CPC Class Codes: A61K 31/713 Double-stranded nucleic acids or oligonucleotides
• CPC Class Codes: A61K 47/18 Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
• CPC Class Codes: A61K 48/00 Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
• CPC Class Codes: A61K 9/08 Solutions composition of solutions A61K47/00
• CPC Class Codes: A61K 9/107 Emulsions ; Emulsion preconcentrates; Micelles composition of emulsions A61K47/00
• CPC Class Codes: A61K 9/127 Liposomes
• CPC Class Codes: A61P 1/16 for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
• CPC Class Codes: A61P 35/00 Antineoplastic agents
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
• CPC Class Codes: A61P 7/00 Drugs for disorders of the blood or the extracellular fluid
• CPC Class Codes: C07C 229/12 to carbon atoms of acyclic carbon skeletons
• CPC Class Codes: C07C 69/34 Esters of acyclic saturated polycarboxylic acids having an esterified carboxyl group bound to an acyclic carbon atom
• CPC Class Codes: C11C 3/003 by esterification of fatty acids with alcohols C11C3/02 takes precedence
• CPC Class Codes: C12N 15/113 Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing when used in plants C12N15/8218
• CPC Class Codes: C12N 2310/14 interfering N.A.
• CPC Class Codes: C12N 2320/32 Special delivery means, e.g. tissue-specific

Abstract

The present invention provides a technology which enables introduction of an active ingredient (e.g. nucleic acids) into various cells with a high efficiency, and compounds used therefor. The present invention provides a compound represented by the formula:

[wherein, each symbol is as defined in the present description] or a salt thereof.

Claims

1. A compound represented by the formula:

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

R¹, R², R³, R⁴ and R⁵ are a methyl group;

Z⁻

is a halide ion;

X is an ethylene group, trimethylene group or tetramethylene group;

Y^A, Y^B and Y^C are a methine group;

L^A, L^B and L^C are, each independently, a bond or methylene group; and

R^A1, R^A2, R^B1, R^B2, R^C1 and R^C2 are, each independently, a butyl group, pentyl group or hexyl group.

6. A lipid particle containing the compound according to claim 1 or a salt thereof.

7. A composition containing an active ingredient and the compound according to claim 1 or a salt thereof.

8. The composition according to claim 7, wherein the active ingredient is a nucleic acid.

9. The composition according to claim 8, wherein the nucleic acid is siRNA or mRNA.

3. A compound selected from the group consisting of:

3-((5-(dimethylamino)pentanoyl)oxy)-2,2-bis(((2-pentylheptanoyl) oxy)methyl)propyl 2-pentylheptanoate or a salt thereof and

3-((5-(dimethylamino)pentanoyl)oxy)-2,2-bis(((3-pentyloctanoyl) oxy)methyl)propyl 3-pentyloctanoate or a salt thereof.

4. (canceled)

5. A salt of N,N,N-trimethyl-5-oxo-5-(3-((3-pentyloctanoyl)oxy)-2,2-bis(((3-pentyloctanoyl)oxy)methyl)propoxy)pentane-1-aminium and a negative ion.