Granted Patent B2

US 12,721,909 · App. 18/660,467 · Granted Sep 1, 2026

Inventors: Blair Leavitt (Vancouver, CA); Pieter Cullis (Vancouver, CA); Terri Petkau (Vancouver, CA); Austin Hill (Vancouver, CA); Pamela Wagner (Vancouver, CA); Jayesh Kulkarni (Vancouver, CA)

Assignee: The University of British Columbia

A61K48/0091A61K9/1272A61K9/1277A61K31/7105A61K38/465A61K47/10A61K47/18A61K47/186A61K47/24A61K47/28

Abstract

Claims (30)

1 . A method of transfection, the method comprising administering to a patient a composition comprising a water-based solution which comprises:

(A) a transfection competent vesicle (TCV) comprising lipid components forming the TCV; and

(B) a cargo entrapped in the TCV,

wherein:

(a) the lipid components consist of: (i) 1,2-Dioleyloxy-3-dimethylamino-propane (DODMA) at 20-30 mol %; (ii) 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE) at 20-30 mol %; (iii) 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC) at 10 mol %; and (iv) cholesterol at 40 mol %; and

(b) the cargo comprises a Cas protein and a guide RNA,

optionally wherein the patient comprises a human.

2 . The method according to claim 1 , wherein the administering comprises delivering the composition to the brain of the patient.

3 . The method according to claim 1 , wherein:

(i) the Cas protein comprises Cas9, Cas10, Cas11, Cas12, or Cpf1; and/or

(ii) the cargo further comprises an additional nucleic acid, optionally a single-stranded DNA.

4 . A method of transfection, the method comprising transfecting a target cell with a composition comprising a water-based solution which comprises:

(A) a transfection competent vesicle (TCV) comprising lipid components forming the TCV; and

(B) a cargo entrapped in the TCV,

wherein:

(a) the lipid components consist of: (i) 1,2-Dioleyloxy-3-dimethylamino-propane (DODMA) at 20-30 mol %; (ii) 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE) at 20-30 mol %;

(iii) 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC) at 10 mol %; and (iv) cholesterol at 40 mol %; and

(b) the cargo comprises a Cas protein and a guide RNA,

wherein the target cell is a mammalian cell, optionally a human cell.

5 . The method according to claim 4 , wherein:

(i) the Cas protein comprises Cas9, Cas10, Cas11, Cas12, or Cpf1; and/or

(ii) the cargo further comprises an additional nucleic acid, optionally a single-stranded DNA.

6 . A method for treating Huntington's disease in a patient, the method comprising administering to the brain of the patient a therapeutically effective amount of a composition comprising a water-based solution which comprises:

(A) a transfection competent vesicle (TCV) comprising lipid components forming the TCV; and

(B) a cargo entrapped in the TCV,

wherein:

(a) the lipid components consist of: (i) 1,2-Dioleyloxy-3-dimethylamino-propane (DODMA) at 20-30 mol %; (ii) 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE) at 20-30 mol %; (iii) 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC) at 10 mol %; and (iv) cholesterol at 40 mol %; and

(b) the cargo comprises a ribonucleoprotein (RNP) targeting the huntingtin gene, wherein the RNP comprises a Cas9 protein and a guide RNA,

optionally wherein the patient comprises a human.

7 . The method according to claim 6 , wherein the cargo further comprises an additional nucleic acid, optionally a single-stranded DNA.

Continuity (3)

References Cited (318)