Dataset Information

Complement-targeted therapy for autoimmune diseases.

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.

SUBMITTER: Chu CQ

PROVIDER: S-EPMC10808840 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature

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Complement-targeted therapy for autoimmune diseases.

Chu Cong-Qiu CQ

Medical review (2021) 20231201 6

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 aut ...[more]

PMID: 38282797

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Project description:The complement system is a powerful effector arm of innate immunity that typically confers protection from microbial intruders and accumulating debris. In many clinical situations, however, the defensive functions of complement can turn against host cells and induce or exacerbate immune, inflammatory, and degenerative conditions. Although the value of inhibiting complement in a therapeutic context has long been recognized, bringing complement-targeted drugs into clinical use has proved challenging. This important milestone was finally reached a decade ago, yet the clinical availability of complement inhibitors has remained limited. Still, the positive long-term experience with complement drugs and their proven effectiveness in various diseases has reinvigorated interest and confidence in this approach. Indeed, a broad variety of clinical candidates that act at almost any level of the complement activation cascade are currently in clinical development, with several of them being evaluated in phase 2 and phase 3 trials. With antibody-related drugs dominating the panel of clinical candidates, the emergence of novel small-molecule, peptide, protein, and oligonucleotide-based inhibitors offers new options for drug targeting and administration. Whereas all the currently approved and many of the proposed indications for complement-targeted inhibitors belong to the rare disease spectrum, these drugs are increasingly being evaluated for more prevalent conditions. Fortunately, the growing experience from preclinical and clinical use of therapeutic complement inhibitors has enabled a more evidence-based assessment of suitable targets and rewarding indications as well as related technical and safety considerations. This review highlights recent concepts and developments in complement-targeted drug discovery, provides an overview of current and emerging treatment options, and discusses the new milestones ahead on the way to the next generation of clinically available complement therapeutics.

Dataset Information

Complement-targeted therapy for autoimmune diseases.

Publications

Complement-targeted therapy for autoimmune diseases.

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