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Peptide inhibitors of C3 activation as a novel strategy of complement inhibition for the treatment of paroxysmal nocturnal hemoglobinuria

2014; Elsevier BV; Volume: 123; Issue: 13 Linguagem: Inglês

10.1182/blood-2013-11-536573

1528-0020

Antonio M. Risitano, Daniel Ricklin, Yijun Huang, Edimara S. Reis, Hui Chen, Patrizia Ricci, Zhuoer Lin, Caterina Pascariello, Maddalena Raia, Michela Sica, Luigi Del Vecchio, Fabrizio Pane, Florea Lupu, Rosario Notaro, Ranillo R.G. Resuello, Robert A. DeAngelis, John D. Lambris,

Renal Diseases and Glomerulopathies

Key Points Peptidic C3 inhibitors of the compstatin family (Cp40) efficiently prevent hemolysis and opsonization of PNH erythrocytes in vitro. Pharmacokinetic studies show that sustained therapeutic concentrations can be achieved with both Cp40 and its PEGylated derivative, PEG-Cp40.