{"id":6048,"date":"2024-05-06T19:39:45","date_gmt":"2024-05-06T19:39:45","guid":{"rendered":"https:\/\/www.sicklecelldisease.org\/?page_id=6048"},"modified":"2024-12-03T22:04:59","modified_gmt":"2024-12-03T22:04:59","slug":"agios","status":"publish","type":"page","link":"https:\/\/www.sicklecelldisease.org\/agios\/","title":{"rendered":"Agios"},"content":{"rendered":"

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Agios<\/strong>\u00a0is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company’s deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, MDS-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera.<\/p>\n

For more information, please visit the company\u2019s website at www.agios.com<\/a><\/strong>.<\/p>\n

Featured Clinical Trial<\/h3>\n

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)<\/strong><\/p>\n

This study is currently enrolling in the Phase 3 portion. For 52 weeks (1 year), participants will randomly receive one of the following two treatment options twice daily:<\/p>\n