# US 10,501,404 B1 · Cationic lipids and transfection methods

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- Number: US 10,501,404 B1
- Document ID: US10501404B1
- Status: Granted
- Kind: B1
- Application number: 16/526,621
- Filed: 2019-07-30
- Granted: 2019-12-10
- Earliest claimed: 2019-07-30
- Assignees: Factor Bioscience Inc.
- Applicants: Factor Bioscience Inc.
- Inventors: Matthew Angel; Franklin Kostas; Christopher Rohde
- Web page: https://patentgrep.com/patent/US10501404B1

## CPC

- C07C 215/14: the nitrogen atom of the amino group being further bound to hydrocarbon groups substituted by amino groups
- C07C 215/24: and acyclic
- 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 9/127: Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- 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 9/1617: {Organic compounds, e.g. phospholipids, fats}
- A61K 9/5015: {Organic compounds, e.g. fats, sugars}
- A61K 9/5123: {Organic compounds, e.g. fats, sugars}
- C07C: ACYCLIC OR CARBOCYCLIC COMPOUNDS
- 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)

## Abstract

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

## Claims

### Claim 1

A compound of Formula (I)
wherein n is 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, or 15.

### Claim 2 (depends on 1)

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

### Claim 3 (depends on 1)

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

### Claim 4 (depends on 1)

The compound of claim 1, wherein the compound is:

### Claim 5 (depends on 1)

The compound of claim 1, wherein the compound is:

### Claim 6 (depends on 1)

The compound of claim 1, wherein the compound is:

### Claim 7 (depends on 1)

The compound of claim 1, wherein the compound is:

### Claim 8 (depends on 1)

The compound of claim 1, wherein the compound is:

### Claim 9 (depends on 1)

The compound of claim 1, wherein the compound is:

### Claim 10 (depends on 1)

The compound of claim 1, wherein the compound is:

### Claim 11 (depends on 1)

The compound of claim 1, wherein the compound is:

### Claim 12 (depends on 1)

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

### Claim 13 (depends on 1)

A lipid aggregate comprising the compound of claim 1.

### Claim 14 (depends on 13)

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

### Claim 15 (depends on 13)

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

### Claim 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):
wherein n is 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, or 15.

### Claim 17 (depends on 16)

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