Therapeutic targeting of the complement system in ocular disease

Sonika Rathi, Rafiq Hasan, Marius Ueffing, Simon J Clark

Research output: Contribution to journalReview articlepeer-review


The complement system is involved in the pathogenesis of several ocular diseases, providing a rationale for the investigation of complement-targeting therapeutics for these conditions. Dry age-related macular degeneration, as characterised by geographic atrophy (GA), is currently the most active area of research for complement-targeting therapeutics, with a complement C3 inhibitor approved in the United States earlier this year marking the first approved therapy for GA. This review discusses the role of complement in ocular disease, provides an overview of the complement-targeting agents currently under development for ocular conditions, and reflects on the lessons that can be learned from the preclinical investigations and clinical trials conducted in this field to date.

Original languageEnglish
Pages (from-to)103757
JournalDrug discovery today
Issue number11
Publication statusPublished - Nov 2023


  • Humans
  • Macular Degeneration/drug therapy
  • Eye
  • Geographic Atrophy/drug therapy


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