Applying complement therapeutics to rare diseases

Clinical Immunology : the Official Journal of the Clinical Immunology Society
Edimara S ReisJohn D Lambris

Abstract

Around 350 million people worldwide suffer from rare diseases. These may have a genetic, infectious, or autoimmune basis, and several include an inflammatory component. Launching of effective treatments can be very challenging when there is a low disease prevalence and limited scientific insights into the disease mechanisms. As a key trigger of inflammatory processes, complement has been associated with a variety of diseases and has become an attractive therapeutic target for conditions involving inflammation. In view of the clinical experience acquired with drugs licensed for the treatment of rare diseases such as hereditary angioedema and paroxysmal nocturnal hemoglobinuria, growing evidence supports the safety and efficacy of complement therapeutics in restoring immune balance and preventing aggravation of clinical outcomes. This review provides an overview of the candidates currently in the pharmaceutical pipeline with potential to treat orphan diseases and discusses the molecular mechanisms triggered by complement involved with the disease pathogenesis.

References

Jun 1, 1990·Proceedings of the National Academy of Sciences of the United States of America·R J FalkJ C Jennette
Apr 1, 1996·Protein Science : a Publication of the Protein Society·J E Volanakis, S V Narayana
May 17, 2000·The Journal of Clinical Investigation·W ZhouS H Sacks
Jul 18, 2001·The Journal of Clinical Investigation·D H BiesmaD Roos
Oct 17, 2001·Developmental and Comparative Immunology·I K ZarkadisJ D Lambris
May 4, 2002·Clinical Immunology : the Official Journal of the Clinical Immunology Society·Guohui RenRichard J Quigg
Jul 18, 2002·Pancreatology : Official Journal of the International Association of Pancreatology (IAP) ... [et Al.]·T Keck
Mar 21, 2003·The Journal of Immunology : Official Journal of the American Association of Immunologists·Bart de VriesWim A Buurman
Aug 9, 2003·The New England Journal of Medicine·Kristian Stengaard-PedersenJens C Jensenius
May 26, 2004·Molecular Immunology·Michael C Carroll
Oct 8, 2005·The Journal of Allergy and Clinical Immunology·Martijn B A van DoornJan Nuijens
Dec 13, 2005·Clinical Immunology : the Official Journal of the Clinical Immunology Society·David D Kim, Wen-Chao Song
Feb 2, 2006·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Conrad A FarrarSteven H Sacks
Nov 4, 2006·Expert Review of Cardiovascular Therapy·Jennifer S LiPage A W Anderson
Jul 3, 2007·British Journal of Pharmacology·P N MonkD P Fairlie
Aug 7, 2007·The Journal of Immunology : Official Journal of the American Association of Immunologists·Dirk SpitzerDennis E Hourcade
Dec 7, 2007·Muscle & Nerve·David GrobMurali Pagala
Nov 4, 2008·American Journal of Transplantation : Official Journal of the American Society of Transplantation and the American Society of Transplant Surgeons·J E LockeR A Montgomery
Dec 17, 2008·Journal of the American Society of Nephrology : JASN·Adrian SchreiberRalph Kettritz
May 15, 2009·Scandinavian Journal of Immunology·P J Lachmann, R A G Smith
May 30, 2009·Molecular Immunology·Andreas KlosJörg Köhl
Dec 1, 2009·Clinical Immunology : the Official Journal of the Clinical Immunology Society·Zsuzsanna KelemenLilian Varga
Dec 22, 2009·Nature Reviews. Microbiology·Ludger Johannes, Winfried Römer
Feb 4, 2010·Nature Reviews. Rheumatology·Marinos C Dalakas
Jan 1, 2008·Expert Review of Clinical Immunology·Linda L KusnerJindrich Soltys
Jul 20, 2010·Nature Reviews. Neurology·Sven Jarius, Brigitte Wildemann
Jul 30, 2010·Journal of Neurology, Neurosurgery, and Psychiatry·N YukiP J Späth
Aug 20, 2010·Nature Immunology·Daniel RicklinJohn D Lambris
Nov 1, 2008·Therapeutic Advances in Neurological Disorders·Marinos C Dalakas
May 10, 2011·Molecular Immunology·Trent M WoodruffFrancesco Tedesco
Jun 23, 2011·Journal of the Peripheral Nervous System : JPNS·Amanda M FitzpatrickHugh J Willison
Aug 13, 2011·American Journal of Transplantation : Official Journal of the American Society of Transplantation and the American Society of Transplant Surgeons·S Chen SongG Chen
Sep 29, 2011·American Journal of Transplantation : Official Journal of the American Society of Transplantation and the American Society of Transplant Surgeons·M D StegallJ M Gloor

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Citations

Mar 30, 2016·Neuromuscular Disorders : NMD·Anne K BraczynskiMichel Mittelbronn
Mar 29, 2016·Nature Structural & Molecular Biology·Matthijs M JoreSusan M Lea
May 24, 2016·Nature Reviews. Nephrology·Daniel RicklinJohn D Lambris
Jun 21, 2016·Seminars in Immunology·Daniel Ricklin, John D Lambris
Oct 27, 2016·Immunological Reviews·Daniel RicklinJohn D Lambris
Oct 27, 2016·Clinical and Experimental Immunology·C WehlingM Kirschfink
May 31, 2018·Diagnosis·Thomas LungLorenz Risch
Jun 28, 2016·Scientific Reports·Marina MorigiGiuseppe Remuzzi
Apr 27, 2019·Postgraduate Medical Journal·Héctor Miguel Ramos-Zaldívar
Mar 5, 2017·Rheumatology International·Savino SciasciaMaria Dall'Era
Sep 27, 2018·Current Protein & Peptide Science·Jean-Étienne R L Morlighem, Gandhi Radis-Baptista
Dec 25, 2019·Proceedings of the National Academy of Sciences of the United States of America·Martin P ReichhardtSusan M Lea
Aug 28, 2020·Current Rheumatology Reports·Lauren N Smith, Julie J Paik
Sep 15, 2020·Frontiers in Immunology·Anne K MühligJun Oh
Nov 30, 2016·Frontiers in Cellular and Infection Microbiology·Caroline M MarcosAna M Fusco-Almeida
Jan 23, 2018·Annals of the New York Academy of Sciences·Linda L KusnerHenry J Kaminski
Jun 3, 2016·Inflammation and Regeneration·Takahiko Horiuchi, Hiroshi Tsukamoto
Jan 10, 2017·Frontiers in Immunology·Tatiana Rodrigues FragaAngela Silva Barbosa
Aug 14, 2020·Journal of Molecular Modeling·Didier DevaursLydia E Kavraki
Aug 19, 2016·Molecular Cancer Research : MCR·Stephanie K DoernerJoseph H Nadeau
Jun 30, 2016·Immunobiology·Dimitrios C MastellosJohn D Lambris
Jul 24, 2018·Seminars in Hematology·Dimitrios C MastellosJohn D Lambris
Apr 19, 2017·Trends in Immunology·Dimitrios C MastellosJohn D Lambris
Mar 16, 2021·Frontiers in Immunology·Marcin Okrój, Elena Volokhina
Jun 21, 2021·Clinical Immunology : the Official Journal of the Clinical Immunology Society·Dimitrios C MastellosArvind Sahu
Oct 5, 2017·Journal of Medicinal Chemistry·Abishek IyerDavid P Fairlie

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