Achillion Pharmaceuticals is a biopharmaceutical company. The company is engaged in the discovery and development of small molecule drug therapies for immune system disorders. The company is advancing orally administered small molecules from its platform that target complement factor D, an essential protein of the alternative pathway. The alternative pathway plays a role in a number of disease conditions, including therapeutic areas of nephrology, hematology, ophthalmology and neurology. Initially Co. is targeting C3 glomerulopathy, and immune complex membranoproliferative glomerulonephritis, two related rare diseases affecting the kidney, and paroxysmal nocturnal hemoglobinuria, a blood disorder.
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