Granted Patent B2

US 12,268,753 · App. 17/579,150 · Granted Apr 8, 2025

Inventors: Mina Fawzy Shehata (Spartanburg, SC); Neeraj Narendra Patwardhan (Medford, MA); Gregory Lawrence Hamilton (Atlanta, GA); Cory Dane Sago (Boston, MA); Milloni Balwantkumar Chhabra (Dunwoody, GA)

Assignee: Beam Therapeutics Inc.

A61K47/6929A61K31/7105A61K31/711A61P1/16C07C219/16C07D211/22

Abstract

Claims (28)

1. A compound of Formula (IIIA):

or a pharmaceutically acceptable salt thereof, wherein:

L 2 is a covalent bond, an optionally substituted bivalent saturated or unsaturated, straight or branched C 1 -C 12 hydrocarbon chain, or

Cy A is an optionally substituted ring selected from phenylene and 3- to 7-membered saturated or partially unsaturated carbocyclene;

each m is independently 0, 1, or 2;

R 1 is a saturated, branched C 3 -C 20 hydrocarbon chain;

each L 4 is independently a bivalent saturated or unsaturated, straight or branched C 1 -C 6 hydrocarbon chain;

each A 1 and A 2 is independently C 2 -C 20 alkenyl;

X 1 is a covalent bond, —O—, or —NR—;

—X 2 —X 3 is selected from:

2. The compound of claim 1 , wherein the compound of Formula (IIIA) is a compound of Formula (VI):

or a pharmaceutically acceptable salt thereof, wherein n is 1, 2, 3 or 4.

3. The compound of claim 1 , wherein the compound of Formula (IIIA) is a compound of Formula (VIA):

or a pharmaceutically acceptable salt thereof, wherein n is 1, 2, 3 or 4.

4. The compound of claim 1 , wherein R′ is selected from:

5. The compound of claim 1 , wherein each of A1 and A2 is independently selected from:

6. The compound of claim 1 , wherein the compound is selected from

or a pharmaceutically acceptable salt thereof.

7. A lipid nanoparticle (LNP) preparation comprising the compound according to claim 1 .

8. A pharmaceutical composition comprising the LNP preparation of claim 7 and a pharmaceutically acceptable excipient.

9. A compound having the following structure:

or a pharmaceutically acceptable salt thereof.

10. A lipid nanoparticle (LNP) preparation comprising the compound according to claim 9 .

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

12. A compound having the following structure:

or a pharmaceutically acceptable salt thereof.

13. A lipid nanoparticle (LNP) preparation comprising the compound according to claim 12 .

14. A pharmaceutical composition comprising the LNP preparation of claim 13 and a pharmaceutically acceptable excipient.

Assignments (3)

Continuity (2)

References Cited (150)