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Complement-targeted therapeutics.

Daniel Ricklin1, John D Lambris

  • 1Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA.

Nature Biotechnology
|November 9, 2007
PubMed
Summary
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The complement system, crucial for immunity, can harm host cells, leading to diseases. Targeting it therapeutically, like with eculizumab, offers a promising drug development strategy.

Area of Science:

  • Immunology
  • Pharmacology
  • Drug Discovery

Background:

  • The complement system is vital for innate and adaptive immunity.
  • Dysregulation of complement contributes to various diseases.
  • Targeting complement is a key strategy in drug discovery.

Purpose of the Study:

  • To review the role of the complement system in disease.
  • To discuss the challenges and breakthroughs in developing complement-targeted therapies.
  • To highlight the significance of eculizumab approval.

Main Methods:

  • Literature review of complement system function.
  • Analysis of therapeutic strategies targeting complement.
  • Examination of drug development successes and failures.

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Main Results:

  • The complement system plays a dual role in host defense and pathology.
  • Developing and marketing complement-targeted drugs has faced significant hurdles.
  • The FDA approval of eculizumab (Soliris) marked a breakthrough.

Conclusions:

  • The complement system is a validated therapeutic target.
  • Eculizumab's approval may pave the way for other complement inhibitors.
  • Further research and development in complement modulation are warranted.