Agios is the pioneering leader in PK activation and dedicated to developing transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class PK activator for adults with PK deficiency, a lifelong, debilitating hemolytic anemia. Building on its scientific expertise in classical hematology and cellular metabolism, Agios is advancing a robust pipeline of investigational medicines in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, MDS-associated anemia and phenylketonuria.