Agios Pharmaceuticals, Inc.
Agios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes used for the treatment of hemolytic anemias in adults with PK deficiency. The company's PYRUKYND product also used for the treatment of sickle cell disease that is in phase 3 clinical trial; for the treatment of PK deficiency in pediatric patients; and for the treatment of adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia. In addition, it develops tebapivat, a PK activator for the treatment of lower-risk myelodysplastic syndrome and hemolytic anemias; AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria (PKU); and AG-236, an siRNA in-licensed from Alnylam, targeting the transmembrane serine protease 6 (TMPRSS6) gene for the treatment of polycythemia vera. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.
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X4 Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases of the immune system. It offers XOLREMDI, an orally bioavailable selective antagonist of chemokine receptor C-X-C chemokine receptor type 4 (CXCR4) for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome. The company also develops mavorixafor that is in Phase 3 4WARD clinical trial to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor with or without stable doses of G-CSF in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications; and with Norgine to develop, manufacture, and commercialize mavorixafor in Europe, Australia, and New Zealand. The company is headquartered in Boston, Massachusetts.
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