PRATH

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

Coordinatore	ADIENNE S.r.l.    more  Organization address address: Via Broseta 64/B city: Bergamo postcode: 24128 contact info Titolo: Dr. Nome: Antonio Francesco Cognome: Di Naro Email: send email Telefono: 39035402872 Fax: 39035258672
Nazionalità Coordinatore	Italy [IT]
Sito del progetto	http://www.prath.eu/
Totale costo	2˙528˙472 €
EC contributo	1˙804˙435 €
Programma	FP7-HEALTH Specific Programme "Cooperation": Health
Code Call	FP7-HEALTH-2009-single-stage
Funding Scheme	CP-FP
Anno di inizio	2010
Periodo (anno-mese-giorno)	2010-05-01   -   2012-04-30

 Partecipanti

#	participant	country	role	EC contrib. [€]
1	ADIENNE S.r.l.  Organization address address: Via Broseta 64/B city: Bergamo postcode: 24128 contact info Titolo: Dr. Nome: Antonio Francesco Cognome: Di Naro Email: send email Telefono: 39035402872 Fax: 39035258672	IT (Bergamo)	coordinator	975˙372.00
2	CIT SAS  Organization address address: MISEREY city: EVREUX postcode: 27005 contact info Titolo: Dr. Nome: Stephane Cognome: De Fortescu Email: send email Telefono: 33232292626 Fax: 33232678705	FR (EVREUX)	participant	387˙000.00
3	IMPERIAL COLLEGE OF SCIENCE, TECHNOLOGY AND MEDICINE  Organization address address: SOUTH KENSINGTON CAMPUS EXHIBITION ROAD city: LONDON postcode: SW7 2AZ contact info Titolo: Mr. Nome: Michael Cognome: Robinson Email: send email Telefono: +44 (0) 208 383 8339 Fax: +4 (0) 208 383 2208	UK (LONDON)	participant	235˙663.00
4	UNIVERSITA DEGLI STUDI DI TRIESTE  Organization address address: PIAZZALE EUROPA 1 city: TRIESTE postcode: 34127 contact info Titolo: Prof. Nome: Francesco Cognome: Tedesco Email: send email Telefono: 0039 040 5584037 Fax: -0039 040 5584023	IT (TRIESTE)	participant	206˙400.00

Mappa

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 Obiettivo del progetto (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”.'

Introduzione (Teaser)

During pathogen invasion our body recruits immune cells and antibodies to fight the infection, a response that is also aided by a collection of proteins known as the complement system. Excessive activation of the Complement System leads to disease and pharmaceutical efforts to prevent this are urgently required.