Treating sickle cell disease with a pyruvate kinase r activating compound

Abstract

Compounds that activate pyruvate kinase R can be used for the treatment of sickle cell disease (SCD). Methods and compositions for the treatment of SCD are provided herein, including a therapeutic compound designated as Compound 1.

We claim: 
     
         1 . A compound 1-(5-((4-(difluoromethoxy)phenyl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2,2-dimethylpropan-1-one, or a pharmaceutically acceptable salt thereof. 
     
     
         2 . A compound 1-(5-((4-(difluoromethoxy)phenyl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2,2-dimethylpropan-1-one. 
     
     
         3 . A pharmaceutical composition comprising the compound of  claim 1 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier. 
     
     
         4 . A pharmaceutical composition comprising the compound of  claim 2  and a pharmaceutically acceptable carrier. 
     
     
         5 . A composition comprising the compound of  claim 2 , obtained by a process comprising treating intermediate compound 2-((4-(difluoromethoxy)phenyl)sulfonyl)-1,2,3,4,5,6-hexahydropyrrolo[3,4-c]pyrrole with 3-hydroxy-2,2-dimethylpropanoic acid in the presence of an amide coupling reagent to afford the compound of  claim 2 .