Granted Patent B2

US 12,296,053 · App. 18/611,561 · Granted May 13, 2025

Inventors: Cory Dane Sago (Cambridge, MA); Gregory Lawrence Hamilton (Cambridge, MA); Neeraj Narendra Patwardhan (Cambridge, MA)

Assignee: Beam Therapeutics Inc.

A61K9/5015A61K39/385A61K48/0033C07C271/20C07D295/13C12N15/113

Abstract

Claims (49)

1. A compound of Formula IX-B-i:

or a pharmaceutically acceptable salt thereof, wherein:

each L 2 and L 2′ is independently a bivalent saturated, straight or branched C 1-12 hydrocarbon chain;

R 1 is optionally substituted C 1-20 aliphatic;

each R 1′ is independently optionally substituted C 1-20 aliphatic;

Y 2 is a bivalent saturated, straight or branched C 1-6 hydrocarbon chain;

Y 3 is optionally substituted C 1-20 aliphatic; and

—X 2 -X 3 is

2. The compound of claim 1 , wherein L 2 is a bivalent saturated, straight or branched C 4-8 hydrocarbon chain and/or L 2′ is a bivalent saturated, straight or branched C 1-6 hydrocarbon chain.

3. The compound of claim 2 , wherein

L 2 is —(CH 2 ) 6 — or —(CH 2 ) 7 — and L 2′ is —(CH 2 ) 2 -;

or

L 2 is —(CH 2 ) 4 — or —(CH 2 ) 5 — and L 2′ is —(CH 2 ) 2 —.

4. The compound of claim 1 , wherein R 1 is optionally substituted C 12-20 aliphatic and/or each R 1′ is independently optionally substituted C 7-9 aliphatic.

5. The compound of claim 1 , wherein R 1 is

and/or

each R 1′ is independently

6. The compound of claim 1 , wherein L 2 is —(CH 2 ) 6 — and R 1 is

7. The compound of claim 1 , wherein Y 2 is —CH 2 — or —(CH 2 ) 2 — and/or Y 3 is optionally substituted C 4-8 aliphatic.

8. A compound selected from the group consisting of:

or a pharmaceutically acceptable salt thereof.

9. A lipid nanoparticle (LNP) preparation comprising an ionizable lipid that is a compound according to claim 1 , or a pharmaceutically acceptable salt thereof.

10. The lipid nanoparticle (LNP) preparation of claim 9 , wherein the LNP preparation further comprises:

a therapeutic and/or prophylactic agent;

a phospholipid;

a cholesterol; and

a conjugate-linker lipid.

11. The LNP preparation of claim 10 , wherein the therapeutic and/or prophylactic agent comprises one or more nucleic acids.

12. A pharmaceutical composition comprising a LNP preparation of claim 10 and a pharmaceutically acceptable excipient.

13. The compound of claim 1 , wherein —X 2 -X 3 is

14. The compound of claim 1 , wherein —X 2 -X 3 is

15. The compound of claim 8 , wherein the compound is selected from the group consisting of:

or a pharmaceutically acceptable salt thereof.

16. A compound having a structure that is:

or a pharmaceutically acceptable salt thereof.

17. A compound having a structure that is:

or a pharmaceutically acceptable salt thereof.

18. A compound having a structure that is:

or a pharmaceutically acceptable salt thereof.

19. A compound having a structure that is:

or a pharmaceutically acceptable salt thereof.

20. A compound having a structure that is:

or a pharmaceutically acceptable salt thereof.

21. A lipid nanoparticle (LNP) preparation comprising an ionizable lipid that is a compound according to claim 15 or a pharmaceutically acceptable salt thereof.

22. A lipid nanoparticle (LNP) preparation comprising an ionizable lipid that is a compound according to claim 16 or a pharmaceutically acceptable salt thereof.

23. A lipid nanoparticle (LNP) preparation comprising an ionizable lipid that is a compound according to claim 17 or a pharmaceutically acceptable salt thereof.

24. A lipid nanoparticle (LNP) preparation comprising an ionizable lipid that is a compound according to claim 18 or a pharmaceutically acceptable salt thereof.

25. A lipid nanoparticle (LNP) preparation comprising an ionizable lipid that is a compound according to claim 19 or a pharmaceutically acceptable salt thereof.

26. A lipid nanoparticle (LNP) preparation comprising an ionizable lipid that is a compound according to claim 20 or a pharmaceutically acceptable salt thereof.

Assignments (3)

Continuity (3)

References Cited (138)