Cationic lipids and transfection methods

Patent metadata

Field	Value
Publication number	US-12344572-B2
Application number	US-202016920556-A
Country	US
Kind code	B2
Filing date	Jul 3, 2020
Priority date	Jul 3, 2019
Publication date	Jul 1, 2025
Grant date	Jul 1, 2025

Abstract

The present invention relates in part to novel cationic lipids and their use, e.g., in delivering nucleic acids to cells.

First claim

What is claimed is:

1. A compound of Formula (V)

wherein
R 21 , R 23 , and R 24 are independently selected from hydrogen, C 1-6 alkyl, C 1-6 haloalkyl, C 1-6 hydroxyalkyl, C 1-6 alkoxyalkyl, C 5-10 aryl, 5- to 10-membered heteroaryl, or C 3-6 cycloalkyl;
n is an integer from 1 to 20;
m is 6, 7, 8, 9, or 10;
p is 1, 2, 3, or 4;
q is 1, 2, 3, 4, or 5; and
r is 1, 2, 3, 4, 5, or 6.

2. The compound of claim 1 , wherein R 21 , R 23 , and R 24 are hydrogen.

3. The compound of claim 1 , wherein n is 16, 17, 18, 19, or 20.

4. The compound of claim 3 , wherein:
q is 4;
p is 1; and
r is 1.

5. The compound of claim 3 , wherein m is 8.

6. A pharmaceutical composition comprising a compound of claim 1 and a pharmaceutically acceptable carrier or excipient.

7. A lipid aggregate comprising a compound of claim 1 .

8. The lipid aggregate of claim 7 , wherein the lipid aggregate does not comprise one or more additional lipids or polymers.

9. The lipid aggregate of claim 7 further comprising a nucleic acid, selected from a DNA molecule or an RNA molecule.

10. The compound of claim 1 , wherein n is 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, or 15.

11. The compound of claim 1 , wherein n is 4.

Assignees

• Factor Bioscience Inc

Inventors

• Angel Matthew
• Kostas Franklin
• Rohde Christopher

Classifications

• A61K9/10

  Dispersions; Emulsions · CPC title

• A61K9/0021

  Intradermal administration, e.g. through microneedle arrays or needleless injectors · CPC title

• A61K47/543 Primary

  Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine · CPC title

• A61K31/7088

  Compounds having three or more nucleosides or nucleotides · CPC title

• A61K9/1272

  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}) · CPC title