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Selected References

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Selected references that support Apellis’ science and areas of focus


Autoimmune Hemolytic Anemia (AIHA): Cold Agglutinin and Warm Antibodies
Berentsen, S., 2015. Role of complement in autoimmune hemolytic anemia. Transfusion Medicine and Hemotherapy, 42(5), pp.303-310.
Wouters, D. and Zeerleder, S., 2015. Complement inhibitors to treat IgM-mediated autoimmune hemolysis. Haematologica, 100(11), pp.1388-1395.

Age-related Macular Degeneration (AMD)
Anderson, D.H., Mullins, R.F., Hageman, G.S. and Johnson, L.V., 2002. A role for local inflammation in the formation of drusen in the aging eye. American Journal of Ophthalmology, 134(3), pp.411-431.
Anderson, D.H., Radeke, M.J., Gallo, N.B., Chapin, E.A., Johnson, P.T., Curletti, C.R., Hancox, L.S., Hu, J., Ebright, J.N., Malek, G., Hauser, M.A., Rickman, C.., Bok, D., Hageman, G.S. and Johnson, L.V., 2010. The pivotal role of the complement system in aging and age-related macular degeneration: hypothesis re-visited. Progress in Retinal and Eye Research, 29(2), pp.95-112.
Boyer, D.S., Schmidt-Erfurth, U., van Lookeren Campagne, M., Henry, E.C. and Brittain, C., 2017. The pathophysiology of geographic atrophy secondary to age-related macular degeneration and the complement pathway as a therapeutic target. Retina (Philadelphia, Pa.), 37(5), p.819-835.
Chakravarthy, U., Bailey, C.C., Johnston, R.L., McKibbin, M., Khan, R.S., Mahmood, S., Downey, L., Dhingra, N., Brand, C., Brittain, C.J., Willis, J.R., Rabhi, S., Muthutantri, A. and Cantrell, R.A., 2018. Characterizing disease burden and progression of geographic atrophy secondary to age-related macular degeneration. Ophthalmology, 125(6), pp.842-849.
Friedman, D.S., O’Colmain, B.J., Munoz, B., Tomany, S.C., McCarty, C., De Jong, P.T., Nemesure, B., Mitchell, P. and Kempen, J., 2004. Prevalence of age-related macular degeneration in the United States. Archives of Ophthalmology, 122(4), pp.564-572.
Holz, F.G., Strauss, E.C., Schmitz-Valckenberg, S. and van Lookeren Campagne, M., 2014. Geographic atrophy: clinical features and potential therapeutic approaches. Ophthalmology, 121(5), pp.1079-1091.
Scholl, H.P., Issa, P.C., Walier, M., Janzer, S., Pollok-Kopp, B., Börncke, F., Fritsche, L.G., Chong, N.V., Fimmers, R., Wienker, T., Holz, F.G., Weber, B.H.F. and Oppermann, M., 2008. Systemic complement activation in age-related macular degeneration. PloS One, 3(7), p.e2593.
Sunness, J.S., Gonzalez-Baron, J., Applegate, C.A., Bressler, N.M., Tian, Y., Hawkins, B., Barron, Y. and Bergman, A., 1999. Enlargement of atrophy and visual acuity loss in the geographic atrophy form of age-related macular degeneration. Ophthalmology, 106(9), pp.1768-1779.

C3 Glomerulopathy (C3G)

Servais, A., Frémeaux-Bacchi, V., Lequintrec, M., Salomon, R., Blouin, J., Knebelmann, B., Grünfeld, J.P., Lesavre, P., Noël, L.H. and Fakhouri, F., 2007. Primary glomerulonephritis with isolated C3 deposits: a new entity which shares common genetic risk factors with haemolytic uraemic syndrome. Journal of Medical Genetics, 44(3), pp.193-199.
Servais, A., Noël, L.H., Roumenina, L.T., Le Quintrec, M., Ngo, S., Dragon-Durey, M.A., Macher, M.A., Zuber, J., Karras, A., Provot, F., Moulin, B., Grünfeld, J.P., Niaudet, P., Lesavre, P. and Frémeaux-Bacchi, V., 2012. Acquired and genetic complement abnormalities play a critical role in dense deposit disease and other C3 glomerulopathies. Kidney International, 82(4), pp.454-464.
Zipfel, P.F., Skerka, C., Chen, Q., Wiech, T., Goodship, T., Johnson, S., Fremeaux-Bacchi, V., Nester, C., de Córdoba, S.R., Noris, M., Pickering, M. and Smith, R., 2015. The role of complement in C3 glomerulopathy. Molecular Immunology, 67(1), pp.21-30.

Complement C3 and Complement Inhibitors

Delanghe, J.R., Speeckaert, R. and Speeckaert, M.M., 2014. Complement C3 and its polymorphism: biological and clinical consequences. Pathology, 46(1), pp.1-10.
Holers, V.M., 2008. The spectrum of complement alternative pathway‐mediated diseases. Immunological Reviews, 223(1), pp.300-316.
Mastellos, D.C., Yancopoulou, D., Kokkinos, P., Huber‐Lang, M., Hajishengallis, G., Biglarnia, A.R., Lupu, F., Nilsson, B., Risitano, A.M., Ricklin, D. and Lambris, J.D., 2015. Compstatin: a C3‐targeted complement inhibitor reaching its prime for bedside intervention. European Journal of Clinical Investigation, 45(4), pp.423-440.
Morgan, B.P. and Harris, C.L., 2015. Complement, a target for therapy in inflammatory and degenerative diseases. Nature Reviews Drug Discovery, 14(12), p.857-877.
Ricklin, D. and Lambris, J.D., 2007. Complement-targeted therapeutics. Nature Biotechnology, 25(11), p.1265-1275.
Ricklin, D. and Lambris, J.D., 2008. Compstatin: a complement inhibitor on its way to clinical application. In Current Topics in Complement II (pp. 262-281). Springer, New York, NY.
Risitano, A.M., 2015. Current and future pharmacologic complement inhibitors. Hematology/Oncology Clinics, 29(3), pp.561-582.

Paroxysmal Nocturnal Hemoglobinuria (PNH)

DeZern, A.E. and Brodsky, R.A., 2015. Paroxysmal nocturnal hemoglobinuria: a complement-mediated hemolytic anemia. Hematology/Oncology Clinics, 29(3), pp.479-494.
Griffin, M., Hillmen, P., Munir, T., Richards, S., Arnold, L., Riley, K. and Hill, A., 2018. Significant hemolysis is not required for thrombosis in paroxysmal nocturnal hemoglobinuria. Haematologica, pp.haematol-2018.
Hill, A., Rother, R.P., Arnold, L., Kelly, R., Cullen, M.J., Richards, S.J. and Hillmen, P., 2010. Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization. Haematologica, 95(4): 567–573.
Risitano, A.M., Notaro, R., Marando, L., Serio, B., Ranaldi, D., Seneca, E., Ricci, P., Alfinito, F., Camera, A., Gianfaldoni, G., Amendola, A., Boschetti, C., Di Bona, E., Fratellanza, G., Barbano, F., Rodeghiero, F., Zanella, A., Iori, P., Selleri, C., Luzzatto, L. and Rotoli, B., 2009. Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood, 113(17), pp.4094-4100.
Risitano, A.M., Ricklin, D., Huang, Y., Reis, E.S., Chen, H., Ricci, P., Lin, Z., Pascariello, C., Raia, M., Sica, M., Del Vecchio, L., Pane, F., Lupu, F., Notaro, R., Resuello, R.R.G., DeAngelis, R.A. and Lambris, J.D., 2014. Peptide inhibitors of C3 activation as a novel strategy of complement inhibition for the treatment of paroxysmal nocturnal hemoglobinuria. Blood, 123(13): pp.2094–2101.
Risitano, A.M., 2015. Dissecting complement blockade for clinic use. Blood, 125(5), pp.742-744.
Sica, M., Rondelli, T., Ricci, P., De Angioletti, M., Risitano, A.M. and Notaro, R., 2017. Eculizumab treatment: stochastic occurrence of C3 binding to individual PNH erythrocytes. Journal of Hematology & Oncology, 10(1), p.126.
Thurman, J.M., 2014. New anti-complement drugs: not so far away. Blood, 123(13), pp.1975-1976.


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