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Last Updated: December 23, 2024

Claims for Patent: 10,632,114


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Summary for Patent: 10,632,114
Title:Pyruvate kinase activators for use in therapy
Abstract: Described herein are methods for using compounds that activate pyruvate kinase.
Inventor(s): Su; Shin-San Michael (Boston, MA), Dang; Lenny (Boston, MA)
Assignee: Agios Pharmaceuticals, Inc. (Cambridge, MA)
Application Number:15/965,088
Patent Claims: 1. A method for treating sickle cell anemia in a subject comprising administering to the subject a therapeutically effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the compound is: ##STR00019##

2. A method for treating thalassemia in a subject comprising administering to the subject a therapeutically effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the compound is: ##STR00020##

3. The method of claim 2 wherein the thalassemia is beta-thalassemia.

4. A method for increasing the lifetime of red blood cells (RBCs) in need thereof comprising contacting blood with a therapeutically effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the compound is: ##STR00021##

5. The method of claim 4 wherein the compound, pharmaceutically acceptable salt thereof or pharmaceutically acceptable composition is administered to a subject in need thereof.

6. The method of claim 4 wherein the compound, pharmaceutically acceptable salt thereof or pharmaceutically acceptable composition is added directly to whole blood or packed red blood cells extracorporeally.

7. A method for regulating the levels of 2,3-diphosphoglycerate (2,3-DPG) levels in blood in need thereof comprising contacting blood with an effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the compound is: ##STR00022##

8. The method of claim 7 wherein the compound, pharmaceutically acceptable salt thereof or pharmaceutically acceptable composition is administered to a subject in need thereof.

9. The method of claim 7 wherein the compound, pharmaceutically acceptable salt thereof or pharmaceutically acceptable composition is added directly to whole blood or packed red blood cells extracorporeally.

10. A method for treating hemolytic anemia in a subject comprising administering to the subject a therapeutically effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the hemolytic anemia is selected from congenital anemia and hereditary spherocytosis and wherein the compound is: ##STR00023##

11. The method of claim 10 wherein the hemolytic anemia is congenital anemia.

12. The method of claim 10 wherein the hemolytic anemia is hereditary spherocytosis.

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