# US 10,766,852 B2 · Ionizable cationic lipids

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- Number: US 10,766,852 B2
- Document ID: US10766852B2
- Status: Granted
- Kind: B2
- Application number: 16/100,853
- Filed: 2018-08-10
- Published: 2019-06-20
- Granted: 2020-09-08
- Earliest claimed: 2012-03-29
- Assignees: Translate Bio, Inc.
- Applicants: Translate Bio, Inc.
- Inventors: Frank DeRosa; Braydon Charles Guild; Michael Heartlein
- Web page: https://patentgrep.com/patent/US10766852B2

## CPC

- C07C 211/21: Monoamines
- A61K 9/1271: Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers (liposomes as conjugates {A61K47/6911})
- 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 9/5015: {Organic compounds, e.g. fats, sugars}
- A61K 38/1816: {Erythropoietin [EPO]}
- A61K 38/47: acting on glycosyl compounds (3.2), e.g. cellulases, lactases
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C12N 15/85: for animal cells
- C12Y 302/01023: Beta-galactosidase (3.2.1.23), i.e. exo-(1-->4)-beta-D-galactanase
- 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)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C12Y: ENZYMES

## Abstract

Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.

## Claims

### Claim 1

A lipid nanoparticle comprising:
mRNA; and
a cationic lipid having the following structure

### Claim 2 (depends on 1)

The lipid nanoparticle of claim 1, further comprising one or more lipids selected from the group consisting of a cationic lipid, a neutral lipid, a PEG-modified lipid, a non-cationic lipid and a helper lipid.

### Claim 3 (depends on 1)

The lipid nanoparticle of claim 1, further comprising C14-DMG-PEG2000, DOPE and cholesterol.

### Claim 4 (depends on 1)

A method of treating disease in a subject, wherein the method comprises administering an effective amount of the lipid nanoparticle of claim 1 to the subject.

### Claim 5 (depends on 1)

A method of transfecting one or more target cells with a polynucleotide, wherein the method comprises contacting the one or more target cells with the lipid nanoparticle of claim 1 such that the one or more target cells are transfected with the polynucleotide.

### Claim 6 (depends on 1)

The lipid nanoparticle of claim 1, wherein the mRNA encodes an enzyme or a protein.

### Claim 7 (depends on 6)

The lipid nanoparticle of claim 6, wherein the mRNA encodes the protein or enzyme selected from the group consisting of human growth hormone, erythropoietin, al-antitrypsin, acid alpha glucosidase, arylsulfatase A, carboxypeptidase N, α-galactosidase A, alpha-L-iduronidase, iduronate-2-sulfatase, iduronate sulfatase, N-acetylglucosamine-1-phosphate transferase, N-acetylglucosaminidase, alpha-glucosaminide acetyltransferase, N-acetylglucosamine 6-sulfatase, N-acetylgalactosamine-4-sulfatase, beta-glucosidase, galactose-6-sulfate sulfatase, beta-galactosidase, beta-glucuronidase, glucocerebrosidase, heparan sulfamidase, heparin-N-sulfatase, lysosomal acid lipase, hyaluronidase, galactocerebrosidase, ornithine transcarbamylase (OTC), carbamoyl-phosphate synthetase 1 (CPS1), argininosuccinate synthetase (ASS1), argininosuccinate lyase (ASL), arginase 1 (ARG1), cystic fibrosis transmembrane conductance regulator (CFTR), survival motor neuron (SMN), Factor VIII, Factor IX and low density lipoprotein receptors (LDLR).

### Claim 8 (depends on 1)

The lipid nanoparticle of claim 1, further comprising one or more helper lipids, non-cationic lipids, and PEG-modified lipid components.

### Claim 9 (depends on 1)

The lipid nanoparticle of claim 1, further comprising DOPE, cholesterol, and/or DMG-PEG2000.

### Claim 10

A lipid nanoparticle comprising:
mRNA; and
a cationic lipid having the structure,

### Claim 11 (depends on 10)

The lipid nanoparticle of claim 10, further comprising one or more lipids selected from the group consisting of a cationic lipid, a neutral lipid, a PEG-modified lipid, a non-cationic lipid and a helper lipid.

### Claim 12 (depends on 10)

The lipid nanoparticle of claim 10, further comprising C14-DMG-PEG2000, DOPE and cholesterol.

### Claim 13 (depends on 10)

A method of treating disease in a subject, wherein the method comprises administering an effective amount of the lipid nanoparticle of claim 10 to the subject.

### Claim 14 (depends on 10)

A method of transfecting one or more target cells with a polynucleotide, wherein the method comprises contacting the one or more target cells with the lipid nanoparticle of claim 10 such that the one or more target cells are transfected with the polynucleotide.

### Claim 15 (depends on 10)

The lipid nanoparticle of claim 10, wherein the mRNA encodes an enzyme or a protein.

### Claim 16 (depends on 15)

The lipid nanoparticle of claim 15, wherein the mRNA encodes the protein or enzyme selected from the group consisting of human growth hormone, erythropoietin, al-antitrypsin, acid alpha glucosidase, arylsulfatase A, carboxypeptidase N, α-galactosidase A, alpha-L-iduronidase, iduronate-2-sulfatase, iduronate sulfatase, N-acetylglucosamine-1-phosphate transferase, N-acetylglucosaminidase, alpha-glucosaminide acetyltransferase, N-acetylglucosamine 6-sulfatase, N-acetylgalactosamine-4-sulfatase, beta-glucosidase, galactose-6-sulfate sulfatase, beta-galactosidase, beta-glucuronidase, glucocerebrosidase, heparan sulfamidase, heparin-N-sulfatase, lysosomal acid lipase, hyaluronidase, galactocerebrosidase, ornithine transcarbamylase (OTC), carbamoyl-phosphate synthetase 1 (CPS1), argininosuccinate synthetase (ASS1), argininosuccinate lyase (ASL), arginase 1 (ARG1), cystic fibrosis transmembrane conductance regulator (CFTR), survival motor neuron (SMN), Factor VIII, Factor IX and low density lipoprotein receptors (LDLR).

### Claim 17 (depends on 10)

The lipid nanoparticle of claim 10, further comprising one or more helper lipids, non-cationic lipids, and PEG-modified lipid components.

### Claim 18 (depends on 10)

The lipid nanoparticle of claim 10, further comprising DOPE, cholesterol, and/or DMG-PEG2000.