Atypical Hemolytic Uremic Syndrome (aHUS)


Cascade Biotechnology INC | Complement Therapeutics; novel approach to CNS/PNS disease management using the innate complement system.


Renal Disease

Atypical Hemolytic Uremic Syndrome (aHUS)


  • The complement cascade provides an important line of defense against invasive pathogens. However, the complement system also causes kidney injury in a variety of different diseases. 

  • Complement activation is particularly important in the development of and C3 glomerulopathy. Complement inhibition is effective for treatment of aHUS, and complement inhibitors will likely be tested in other kidney diseases in the future. 

  • While the role of the complement system in the pathogenesis of many kidney diseases is well established, however, there is not a simple algorithm for identifying which patients should be treated with complement inhibitors or for how long complement inhibition should be continued. (Joshua M. Thurman (2015) Complement in Kidney Disease: Core Curriculum 2015 Am J Kidney Dis. 2015 Jan; 65(1): 156–168).

  • Complement cascade is involved in several renal diseases and in renal transplantation. The different components of the complement cascade might represent an optimal target for innovative therapies. 

  • Study of  complement involvement in renal diseases and transplantation has led to a reclassification of some renal diseases, moving from a histopathological to a physiopathological classification.

  • Renal diseases involve complement over activation and complement dysregulation.  

  •  Many targets such as C1, C3, C5a and C5aR are currently the subject of national or international trials. 

  • In addition, many molecules proved to be effective in vitro or in preclinical trials and are waiting to move to human trials in the future. (Maurizio Salvadori, Giuseppina Rosso, and  Elisabetta Bertoni (2015) Complement involvement in kidney diseases: From physiopathology to therapeutical targeting World J Nephrol. 2015 May 6; 4(2): 169–184).