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Cold agglutinin disease

Disease ID:1253
Name:Cold agglutinin disease
Associated with:0 target
1 immuno-relevant ligand
Synonyms
autoimmune hemolytic anemia | chronic cold agglutinin disease | cold agglutinin syndrome
Description
Cold agglutinin disease is an autoimmune hemolytic anemia caused by autoantibodies that destroy erythrocytes via activation of the C1-complex of the classical complement pathway. The disease causes chronic anemia, severe fatigue and potentially fatal thrombotic events.
Database Links
Orphanet: ORPHA56425

Targets

No target related data available for Cold agglutinin disease

Ligands

Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
TNT009
Immuno Disease Comments: TNT009 is a first-in-class C1-complex-targeting mAb, that is hoped to provide an effective treatment for CAD and other complement-mediated disorders. It is a Phase 3 clinical candidate for CAD (see NCT03347396) and Phase 1 for other disorders (see NCT02502903 and NCT03275454). TNT009 is an FDA and EMA orphan drug for the treatment of hemolysis in patients with primary CAD.
Clinical Use: Click here to link to ClinicalTrials.gov's list of current TNT009 (and BIVV009) trials. | View clinical data
Bioactivity Comments: Preclinical details of TNT009 in vitro activity are published in [3], and results from the first-in-human Phase 1 trial are reported in [2]. In kidney transplant patients, TNT009 (BIVV009) effectively blocks alloantibody-triggered complement pathway activation, but this fails to translate to therapeutic benefit in the short term [1]. Further studies are needed to fully evaluate the therapeutic value of complement pathway blockade in transplant patients. | View biological activity

References

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1. Eskandary F, Jilma B, Mühlbacher J, Wahrmann M, Regele H, Kozakowski N, Firbas C, Panicker S, Parry GC, Gilbert JC et al.. (2018) Anti-C1s monoclonal antibody BIVV009 in late antibody-mediated kidney allograft rejection-results from a first-in-patient phase 1 trial. Am. J. Transplant., 18 (4): 916-926. [PMID:28980446]

2. Mühlbacher J, Jilma B, Wahrmann M, Bartko J, Eskandary F, Schörgenhofer C, Schwameis M, Parry GC, Gilbert JC, Panicker S et al.. (2017) Blockade of HLA Antibody-Triggered Classical Complement Activation in Sera From Subjects Dosed With the Anti-C1s Monoclonal Antibody TNT009-Results from a Randomized First-in-Human Phase 1 Trial. Transplantation, 101 (10): 2410-2418. [PMID:28926521]

3. Wahrmann M, Mühlbacher J, Marinova L, Regele H, Huttary N, Eskandary F, Cohen G, Fischer GF, Parry GC, Gilbert JC et al.. (2017) Effect of the Anti-C1s Humanized Antibody TNT009 and Its Parental Mouse Variant TNT003 on HLA Antibody-Induced Complement Activation-A Preclinical In Vitro Study. Am. J. Transplant., 17 (9): 2300-2311. [PMID:28251805]