Targeting the underlying clonal B cell lymphoproliferative disorder and controlling the complement-mediated hemolysis remain as the most critical therapy challenge in cold agglutinin disease (CAD). With our extensive experience in CAD therapy development, Protheragen is in a unique and ideal position to help you navigate from CAD therapy research to commercialization.
Cold agglutinin disease (CAD) is an uncommon autoimmune ailment distinguished by the existence of antibodies known as cold agglutinins that attach to red blood cells and cause their early destruction. These antibodies are effective at lower temperatures, usually under normal body temperature, leading to problems such as hemolytic anemia, tiredness, pale skin, and poor blood flow to the limbs.
Fig.1 Cold agglutinin disease (CAD) is an acquired autoimmune hemolytic anemia (AIHA). (Climent F, et al., 2022)
The pathologic features of cold agglutinin disease (CAD) are caused by pathogenic IgM autoantibodies which attach to erythrocyte membrane antigens (mostly I/i system) at subnormal body temperature and activate the classical complement pathway by C1q binding. This causes C3b opsonization of red blood cells (RBCs), which brings about intravascular hemolysis as well as extravascular hemolysis.
Fig.2 Complement-mediated hemolysis in cold agglutinin disease (CAD). (Berentsen S, et al., 2022)
| Drug Names | Mechanism of Action | Targets | NCT Number | Phase |
|---|---|---|---|---|
| Pegcetacoplan | Inhibits complement-mediated hemolysis by preventing C3 cleavage and downstream activation. | C3 protein | NCT05096403 | Phase III |
| Sutimlimab | Monoclonal antibody that blocks C1s protease activity, preventing classical pathway initiation. | C1s protease | NCT05132127 | Approved |
| Iptacopan | Small molecule inhibitor of factor B, preventing alternative pathway amplification. | Factor B | NCT05086744 | Phase II |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Protheragen is a specialized service provider driving innovation in diagnostics and therapeutics for cold agglutinin disease (CAD). Our expertise spans biomarker-driven diagnostic development, including cutting-edge in vitro diagnostic (IVD) kits, and the design of targeted therapies that disrupt CAD pathogenesis. Leveraging advanced disease models and preclinical research, we rigorously validate each solution to ensure translational relevance.
Animal Model Development Services
At Protheragen, we are committed to validating and optimizing therapies for cold agglutinin disease (CAD) through preclinical studies including pharmacodynamics (PD), pharmacokinetics (PK) and toxicology to ensure their successful regulatory approval. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)