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.
Open AGIO dashboard ↗Ocugen, Inc. Common Stock
Ocugen, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel gene and cell therapies, biologic, and vaccines that improve patients' health. The company's pipeline product includes OCU400, a novel gene therapy product candidate restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases, currently under Phase 3 trials for the treatment of retinitis pigmentosa and Phase 1/2 trials for the treatment of leber congenital amaurosis; OCU410, a gene therapy under phase 1/2 for the treatment of dry age-related macular degeneration (AMD); and OCU410ST, a gene therapy under phase 1/2 for the treatment of Stargardt disease. It is involved in the development of OCU200, a novel fusion protein for the treatment of diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration; and NeoCart, an autologous chondrocyte-derived neocartilage, currently under Phase 3 studies indicated for the repair of knee cartilage injuries in adult. In addition, the company is developing OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. It has collaboration agreements with National Institute of Allergy and Infectious Diseases for early clinical studies for the OCU500 program; and a strategic partnership with CanSino Biologics Inc. for manufacturing its modifier gene therapy pipeline product candidates. The company was founded in 2013 and is headquartered in Malvern, Pennsylvania.
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