Cationic lipids and transfection methods

• US 10,501,404 B1
• Filed: 07/30/2019
• Issued: 12/10/2019
• Est. Priority Date: 07/30/2019
• Status: Active Grant

• Current Assignee: Factor Bioscience Inc
• Original Assignee: Factor Bioscience Inc
• Inventors: Angel, Matthew, Kostas, Franklin, Rohde, Christopher
• Primary Examiner(s): Davis, Brian J
• Application Number:
  US16/526,621
• Time in Patent Office: 133 Days
• Field of Search: None
• CPC Class Codes: 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
• CPC Class Codes: A61K 9/127 Liposomes
• CPC Class Codes: A61K 9/1272 with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids with cholesterol as the only non-phosphatidyl surfactant A61K9/127; lipids as modifying agent A61K47/543
• CPC Class Codes: A61K 9/1617 Organic compounds, e.g. phospholipids, fats
• CPC Class Codes: A61K 9/5015 Organic compounds, e.g. fats, sugars
• CPC Class Codes: A61K 9/5123 Organic compounds, e.g. fats, sugars
• CPC Class Codes: C07C 215/14 the nitrogen atom of the amino group being further bound to hydrocarbon groups substituted by amino groups
• CPC Class Codes: C07C 215/24 and acyclic

Abstract

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

Claims

1. A compound of Formula (I)

2. The compound of claim 1, wherein n is 2-15.

3. The compound of claim 1, wherein n is 2-12.

4. The compound of claim 1, wherein the compound is:

5. The compound of claim 1, wherein the compound is:

6. The compound of claim 1, wherein the compound is:

7. The compound of claim 1, wherein the compound is:

8. The compound of claim 1, wherein the compound is:

9. The compound of claim 1, wherein the compound is:

10. The compound of claim 1, wherein the compound is:

11. The compound of claim 1, wherein the compound is:

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

13. A lipid aggregate comprising the compound of claim 1.

14. The lipid aggregate of claim 13, wherein the lipid aggregate does not comprise one or more additional lipids or polymers.

15. The lipid aggregate of claim 13, further comprising a nucleic acid, selected from a DNA or RNA molecule.

16. A method for transfecting a cell with a nucleic acid, comprising contacting the cell with a complex of the nucleic acid and a compound of Formula (I):

17. The method of claim 16, wherein the compound is selected from: