Skip to main content

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

Objective

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, ADIENNE S.r.l. (Italy) has obtained orphan designation EU/3/08/571 for its Recombinant human minibody against Complement component C5 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 orphan 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”.

Call for proposal

FP7-HEALTH-2009-single-stage
See other projects for this call

Coordinator

ADIENNE S.r.l.
Address
Via Broseta 64/B
24128 Bergamo
Italy
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EU contribution
€ 975 372
Administrative Contact
Antonio Francesco Di Naro (Dr.)

Participants (3)

UNIVERSITA DEGLI STUDI DI TRIESTE
Italy
EU contribution
€ 206 400
Address
Piazzale Europa 1
34127 Trieste
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Francesco Tedesco (Prof.)
IMPERIAL COLLEGE OF SCIENCE TECHNOLOGY AND MEDICINE
United Kingdom
EU contribution
€ 235 663
Address
South Kensington Campus Exhibition Road
SW7 2AZ London
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Michael Robinson (Mr.)
CIT SAS
France
EU contribution
€ 387 000
Address
Miserey
27005 Evreux
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Stephane De Fortescu (Dr.)