Objetivo 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”. Ámbito científico medical and health sciencesclinical medicineangiologyvascular diseasesmedical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsnatural sciencesbiological sciencesbiochemistrybiomoleculesproteinsnatural sciencesbiological sciencesgeneticsmutation Programa(s) FP7-HEALTH - Specific Programme "Cooperation": Health Tema(s) HEALTH-2009-2.4.4-2 - Preclinical development of substances with a clear potential as orphan drugs Convocatoria de propuestas FP7-HEALTH-2009-single-stage Consulte otros proyectos de esta convocatoria Régimen de financiación CP-FP - Small or medium-scale focused research project Coordinador ADIENNE S.r.l. Aportación de la UE € 975 372,00 Dirección Via Broseta 64/B 24128 Bergamo Italia Ver en el mapa Tipo de actividad Private for-profit entities (excluding Higher or Secondary Education Establishments) Contacto administrativo Antonio Francesco Di Naro (Dr.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos Participantes (3) Ordenar alfabéticamente Ordenar por aportación de la UE Ampliar todo Contraer todo UNIVERSITA DEGLI STUDI DI TRIESTE Italia Aportación de la UE € 206 400,00 Dirección PIAZZALE EUROPA 1 34127 Trieste Ver en el mapa Región Nord-Est Friuli-Venezia Giulia Trieste Tipo de actividad Higher or Secondary Education Establishments Contacto administrativo Francesco Tedesco (Prof.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos IMPERIAL COLLEGE OF SCIENCE TECHNOLOGY AND MEDICINE Reino Unido Aportación de la UE € 235 663,00 Dirección SOUTH KENSINGTON CAMPUS EXHIBITION ROAD SW7 2AZ LONDON Ver en el mapa Región London Inner London — West Westminster Tipo de actividad Higher or Secondary Education Establishments Contacto administrativo Michael Robinson (Mr.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos CIT SAS Francia Aportación de la UE € 387 000,00 Dirección MISEREY 27005 EVREUX Ver en el mapa Tipo de actividad Private for-profit entities (excluding Higher or Secondary Education Establishments) Contacto administrativo Stephane De Fortescu (Dr.) Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Coste total Sin datos