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Before its acquisition, Achillion was a clinical-stage biopharmaceutical company developing oral small molecule inhibitors of complement factor D for alternative pathway-mediated rare diseases; its lead drug danicopan (ACH-4471) was in phase 2 for paroxysmal nocturnal hemoglobinuria and C3 glomerulopathy/immune complex membranoproliferative glomerulonephritis, with additional factor D inhibitors and antiviral programs in earlier stages.[1][2][7]
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