US2020289502A1PendingUtilityA1

Pyruvate kinase activators for use in therapy

72
Assignee: AGIOS PHARMACEUTICALS INCPriority: May 3, 2011Filed: Mar 17, 2020Published: Sep 17, 2020
Est. expiryMay 3, 2031(~4.8 yrs left)· nominal 20-yr term from priority
Inventors:Shin-San Michael SuLenny Dang
A61K 31/497A61P 7/06C07D 271/12A61K 31/4965C07D 307/14A61K 31/535A61K 31/496C07D 241/04C07D 333/24C07D 215/14C07D 241/20C07D 405/12C07D 213/81C07D 401/12A61K 31/4406C07D 235/06C07D 417/12A61K 31/47C07D 295/192A61K 31/415C07D 205/04C07C 307/10C07C 13/04A61K 31/495A61P 7/00
72
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Claims

Abstract

Described herein are methods for using compounds that activate pyruvate kinase.

Claims

exact text as granted — not AI-modified
1 - 30 . (canceled) 
     
     
         31 . A method of treating sickle cell anemia in a subject in need thereof by administering to the subject an effective amount of a pyruvate kinase activator. 
     
     
         32 . The method of  claim 31 , wherein the pyruvate kinase activator is a small molecule. 
     
     
         33 . The method of  claim 31 , wherein the pyruvate kinase activator reduces intramolecular polymerization of hemoglobin. 
     
     
         34 . The method of  claim 31 , wherein the pyruvate kinase activator activates wild type pyruvate kinase R. 
     
     
         35 . The method of  claim 31 , wherein the pyruvate kinase activator activates a mutant pyruvate kinase R. 
     
     
         36 . The method of  claim 35 , wherein the mutant pyruvate kinase R is selected from the group consisted of G332S, G364D, T384M, G37E, R479H, R479K, R486W, R532W, R510Q, and R490W. 
     
     
         37 . The method of  claim 31 , wherein the pyruvate kinase activator is orally administered to the subject. 
     
     
         38 . The method of  claim 31 , wherein the pyruvate kinase activator is orally administered to the subject with a pharmaceutically acceptable carrier. 
     
     
         39 . A method of reducing the level of 2,3-DPG in a subject in need thereof by administering to the subject an effective amount of a pyruvate kinase activator. 
     
     
         40 . The method of  claim 39  wherein the pyruvate kinase activator is a small molecule. 
     
     
         41 . The method of  claim 40 , wherein the subject has sickle cell anemia. 
     
     
         42 . The method of  claim 40 , wherein the subject has hemolytic anemia. 
     
     
         43 . The method of  claim 40 , wherein the subject has pyruvate kinase deficiency. 
     
     
         44 . The method of  claim 40 , wherein the subject has thalassemia. 
     
     
         45 . The method of  claim 40 , wherein the pyruvate kinase activator activates wild type pyruvate kinase R. 
     
     
         46 . The method of  claim 40 , wherein the pyruvate kinase activator activates a mutant pyruvate kinase R. 
     
     
         47 . The method of  claim 46 , wherein the mutant pyruvate kinase R is selected from the group consisting of G332S, G364D, T384M, G37E, R479H, R479K, R486W, R532W, R510Q, and R490W. 
     
     
         48 . The method of  claim 40 , wherein the pyruvate kinase activator is orally administered to the subject. 
     
     
         49 . The method of  claim 40 , wherein the pyruvate kinase activator is orally administered to the subject with a pharmaceutically acceptable carrier. 
     
     
         50 . A method of treating sickle cell anemia in a subject in need thereof by orally administering to the subject an effective amount of a small molecule pyruvate kinase activator, wherein the pyruvate kinase activator activates wild type or mutant pyruvate kinase R.

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