IP Library › Granted Patent US 12,251,470
Granted Patent B2
US 12,251,470 · App. 17/291,942 · Granted Mar 18, 2025

Lipid nanoparticle formulations

Inventors: James Heyes (Vancouver, CA); Adam Judge (Bainbridge Island, WA); Kieu Mong Lam (Richmond, CA); Lorne Ralph Palmer (Vancouver, CA); Petra Schreiner (Burnaby, CA)
Assignee: ARBUTUS BIOPHARMA CORPORATION
A61K9/1272A61K9/5123A61K31/713C12N15/113B82Y5/00C12N2310/14C12N2320/30
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Quick Facts
Patent No.
US 12,251,470
App. No.
17/291,942
Granted
Mar 18, 2025
Kind
B2
Abstract

The invention provides certain specific lipid nanoparticles comprising: (a) one or more nucleic acid molecules; (b) cholesterol; (c) DSPC; (d) PEG-C-DMA; and (e) a cationic lipid; and pharmaceutical compositions comprising the lipid nanoparticles. The lipid nanoparticles and pharmaceutical compositions are particularly useful for delivering a nucleic acid such as siRNA or mRNA to a patient (e.g. a human) or to a cell.

Claims (24)

1. A lipid nanoparticle comprising:

(a) one or more nucleic acid molecules, wherein the nucleic acid molecules are mRNA molecules;

(b) cholesterol;

(c) DSPC;

(d) PEG-C-DMA; and

(e) a cationic lipid of formula:

or a salt thereof, wherein the molar percentage of total lipid for PEG-C-DMA, cationic lipid, cholesterol, and DSPC are as follows:

PEG-C-DMA: 1.485-1.515;

cationic lipid: 49.5-50.5;

cholesterol: 38.115-38.885; and

DSPC: 9.9-10.1.

2. The lipid nanoparticle as described in claim 1 , having a (total lipid):(nucleic acid) weight ratio that is greater than about 17.

3. The lipid nanoparticle as described in claim 1 , having a (total lipid):(nucleic acid) weight ratio that is greater than about 18.

4. The lipid nanoparticle as described in claim 1 , having a (total lipid):(nucleic acid) weight ratio that is greater than about 19.

5. The lipid nanoparticle as described in claim 1 , having a (total lipid):(nucleic acid) weight ratio that is from about 22 to about 25.

6. A pharmaceutical composition comprising a lipid nanoparticle as described in claim 1 , and a pharmaceutically acceptable carrier.

7. The pharmaceutical composition of claim 6 that is formulated for subcutaneous administration.

8. A method for delivering a nucleic acid to a cell comprising contacting the cell with a lipid nanoparticle as described in claim 1 .

9. A method for treating a disease characterized by a genetic defect that results in a deficiency of a functional protein, the method comprising: administering to a subject having the disease, a lipid nanoparticle as described in claim 1 , wherein the nucleic acid molecule is an mRNA that encodes the functional protein or a protein having the same biological activity as the functional protein.

10. The lipid nanoparticle of claim 1 , wherein the molar percentage of total lipid for PEG-C-DMA, cationic lipid, cholesterol, and DSPC are as follows:

PEG-C-DMA: 1.5;

cationic lipid: 50.0;

cholesterol: 38.5; and

DSPC: 10.0.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 2, 2021
From: HEYES, JAMES; JUDGE, ADAM; LAM, KIEU MONG; PALMER, LORNE RALPH; SCHREINER, PETRA
To: ARBUTUS BIOPHARMA CORPORATION
Reel/Frame 056412/0571 →
Continuity (2)
Provisional Application 62758088 · Nov 9, 2018
Related Publication 20220000778A1 · Jan 6, 2022
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