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Preclinical study of Recombinant human Anti-C5 for the Treatment of atypical HUS

Coordinator: Antonio Francesco DI NARO
Project Number: 242273
EC contribution: € 1,804,436.00
Project website: http://www.prath.eu/

The complement system is implicated in the onset, maintenance and amplification of tissue damage in several inflammatory clinical conditions. Haemolytic uraemic syndrome (HUS) is a systemic disease characterized by damage to endothelial cells and erythrocytes, thrombocytopenia, micro thrombosis and kidney failure. During the last few years it has become evident that familial atypical HUS is strongly associated with mutations in proteins needed either for activation or regulation of the alternative pathway of complement. For this reason Recombinant human minibody against complement component C5 has obtained the EU orphan designation (EU/3/08/571) for the treatment of atypical Haemolytic Uraemic Syndrome (aHUS) associated with an inherited abnormality of the complement system in September 2008. This molecule has been already characterized by in vitro and in vivo experiments and a complete preclinical study is the next step for the development of this potential drug useful for the treatment of aHUS..
Preclinical studies of this EU designated orphan medicinal product has been design by ADIENNE s.r.l. and will be perform in collaboration with the University of Trieste (which has isolated and characterized the recombinant human antibody against C5), the Imperial Collage of London, ADIENNE Spain SL and CIT-Safety and Health Research Laboratories. In this two years project we plan to perform pharmacological, pharmacokinetic and toxicological studies, a pre-clinical evaluation of the therapeutic efficacy of the anti-C5 antibody in mouse models of aHUS and characterization of GMP-grade anti-C5 antibody production.
Data collected in this project will be sufficient for a complete “Preclinical development of substances with a clear potential as orphan drugs” as proposed in topic “Rare diseases - HEALTH-2009-2.4.4-2”.

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