Complement-targeted therapy for autoimmune diseases

Abstract

Abstract The success and safety seen in treating complement-mediated hemolysis conditions has sparked the development of targeted therapies for rare autoimmune diseases, with expansion to more common autoimmune conditions. Various classes of drugs, including small molecules, peptides, monoclonal antibodies, and small interfering RNA (siRNA), are undergoing development to specifically address complement activity. A dual approach targeting both complement and other immune components may be required for autoimmune diseases characterized by inflammation and complex pathogenic mechanisms. siRNA, which suppresses complement production, is emerging as a potent therapeutic tool. Combining a complement-blocking siRNA drug with a treatment that reduces autoantibodies could prove clinically feasible and impactful in managing these conditions.