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Company Agios Pharmaceuticals Inc.
DescriptionOral small molecule activator of pyruvate kinase R
Molecular Target Pyruvate kinase R
Mechanism of ActionPyruvate kinase R activator
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase II
Standard Indication Metabolic (unspecified)
Indication DetailsTreat pyruvate kinase deficiency
Regulatory Designation U.S. - Orphan Drug (Treat pyruvate kinase deficiency)

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