Agios制药

Agios Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company. The Company's therapeutic areas of focus are cancer and rare genetic metabolic disorders, which are a group of over 600 rare genetic diseases caused by mutations, or defects, of single metabolic genes. The Company's cancer product candidates are enasidenib and ivosidenib (AG-120), which target mutated isocitrate dehydrogenase 2 (IDH2) and isocitrate dehydrogenase 1 (IDH1), respectively, and AG-881, which targets both mutated IDH1 and mutated IDH2. These mutations are found in a range of hematological malignancies and solid tumors. The lead product candidate in its rare genetic metabolic disorder (RGD), programs, AG-348, targets pyruvate kinase-R for the treatment of pyruvate kinase deficiency. Pyruvate kinase deficiency is a rare disorder that often results in severe hemolytic anemia due to inherited mutations in the pyruvate kinase enzyme within red blood cells.