IP Library › Granted Patent US 10,526,284
Granted Patent B2
US 10,526,284 · App. 15/849,573 · Granted Jan 7, 2020

Ionizable cationic lipid for RNA delivery

Inventors: Joseph E. Payne (San Diego, CA); Padmanabh Chivukula (San Diego, CA); Priya Karmali (San Diego, CA); Steven P. Tanis (Carlsbad, CA)
Assignee: ARCTURUS THERAPEUTICS, INC.
C07C333/04C07C271/22
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Quick Facts
Patent No.
US 10,526,284
App. No.
15/849,573
Granted
Jan 7, 2020
Kind
B2
Abstract

What is described is a compound of formula I consisting of a compound in which R 1 is a branched chain alkyl consisting of 10 to 31 carbons; R 2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons, or a branched chain alkyl consisting of 10 to 31 carbons; L 1 and L 2 are the same or different, each a linear alkane of 1 to 20 carbons or a linear alkene of 2 to 20 carbons; X 1 is S or O; R 3 is a linear or branched alkylene consisting of 1 to 6 carbons; and R 4 and R 5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

Claims (17)

1. A compound selected from the group consisting of

2. The compound of claim 1 , wherein the compound is

3. The compound of claim 1 , wherein the compound is

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. A pharmaceutical composition comprising a compound of claim 1 .

11. The pharmaceutical composition of claim 10 , comprising the compound in a lipid nanoparticle.

12. The pharmaceutical composition of claim 11 , wherein the lipid nanoparticle comprises an mRNA, a cationic lipid, a phospholipid, and a conjugated lipid.

13. The pharmaceutical composition of claim 11 , wherein the lipid nanoparticle encapsulates an mRNA.

14. The pharmaceutical composition of claim 11 , further comprising an mRNA encoding a biologically active protein.

15. The pharmaceutical composition of claim 11 , further comprising an RNA comprising a nucleotide sequence homologous to a mRNA in a target cell.

16. A method of treating a genetic abnormality, comprising administering the pharmaceutical composition of claim 10 , wherein genetic abnormality is in the liver of a subject; wherein the administration is intravenous injection.

17. The method of claim 16 , wherein the pharmaceutical composition is administered at least twice.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 23, 2018
From: PAYNE, JOSEPH E.; CHIVUKULA, PADMANABH; KARMALI, PRIYA; TANIS, STEVEN P.
To: ARCTURUS THERAPEUTICS, INC.
Reel/Frame 044700/0614 →
Continuity (1)
Related Publication 20180170866A1 · Jun 21, 2018
Cited By (11)
US 50,681 US 12,286,395 US 12,311,033 US 12,351,834 US 12,398,379 US 12,460,231 US 12,527,881 US 12,545,912 US 12,637,422 US 12,703,858 US 12,723,023