Agios Therapeutics

Agios Pharmaceuticals is a commercial-stage biopharmaceutical company pioneering therapies in the field of cellular metabolism. The company's primary focus is on developing small molecule medicines for genetically defined rare diseases, particularly hemolytic anemias. Its lead product, Pyrukynd (mitapivat), is the first approved disease-modifying therapy for adults with pyruvate kinase deficiency. Agios is also advancing its pipeline into other indications such as thalassemia, sickle cell disease, and phenylketonuria. Following the sale of its oncology portfolio to Servier in 2021, the company has pivoted its strategy to focus exclusively on rare diseases.