Obiettivo The main objective of the HemAcure project is to develop and refine the tools and technologies for a novel ex vivo prepared cell based therapy to treat the bleeding disease haemophilia A (caused by genetic deficiency in clotting factor VIII (FVIII)) that should ultimately lead to improved quality of life of the patients. The concept is a further development of our approach, established during the FP7 ReLiver project led by Medicyte. From the very beginning, we balance two important goals, maximizing the product’s efficacy and safety profile on one side and minimizing production cost on the other by enhancing the product’s manufacturability. HemAcure relates to the work program as we focus on the refinement of all steps and tools of our ex vivo gene therapy approach. These steps involve 1) isolation and culture of cells from patients’ blood, 2) manipulation of patient cells to repair its genetic defect by ex vivo introducing the correct genetic copy of FVIII (mutations in this gene lead to hemophilia A), 3) automation of cell expansion in a novel and passage-less scalable bioreactor, 4) continuously monitoring of cells during and after expansion with respect to their safety profile and functionality, 5) cell implantation into a worldwide unique medical device for targeted delivery of therapeutic Factor VIII and 6) proof-of-concept and safety studies in appropriate haemophilia A animal models. The aim of adapting the proof-of-concept to GMP requires a risk based approach, by means of a clear understanding of the whole process from design to production of the therapeutic cells and a systematic way to identify and prevent risks that are not acceptable for the patient. All steps will be designed and conducted according to European GMP-regulations to ensure that the product will fully comply to the requirements for quality of the European authorities. Campo scientifico engineering and technologymechanical engineeringmanufacturing engineeringmedical and health sciencesmedical biotechnologygenetic engineeringgene therapymedical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsmedical and health sciencesclinical medicinetransplantationmedical and health sciencesmedical biotechnologycells technologies Programma(i) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Argomento(i) PHC-16-2015 - Tools and technologies for advanced therapies Invito a presentare proposte H2020-PHC-2014-2015 Vedi altri progetti per questo bando Bando secondario H2020-PHC-2015-two-stage Meccanismo di finanziamento RIA - Research and Innovation action Coordinatore UNIVERSITAETSKLINIKUM WUERZBURG - KLINIKUM DER BAYERISCHEN JULIUS-MAXIMILIANS-UNIVERSITAT Contribution nette de l'UE € 1 714 513,00 Indirizzo JOSEF-SCHNEIDER-STRASSE 2 97080 Wurzburg Germania Mostra sulla mappa Regione Bayern Unterfranken Würzburg, Kreisfreie Stadt Tipo di attività Higher or Secondary Education Establishments Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 1 714 513,00 Partecipanti (7) Classifica in ordine alfabetico Classifica per Contributo netto dell'UE Espandi tutto Riduci tutto GRUNEWALD JURGEN Partecipazione conclusa Germania Contribution nette de l'UE € 179 845,34 Indirizzo Mostra sulla mappa Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Collegamenti Contatta l’organizzazione Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 179 845,34 UNIVERSITA DEGLI STUDI DEL PIEMONTE ORIENTALE AMEDEO AVOGADRO Italia Contribution nette de l'UE € 1 400 000,00 Indirizzo DUOMO 6 13100 Vercelli Mostra sulla mappa Regione Nord-Ovest Piemonte Vercelli Tipo di attività Higher or Secondary Education Establishments Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 1 400 000,00 LOUGHBOROUGH UNIVERSITY Regno Unito Contribution nette de l'UE € 983 280,00 Indirizzo ASHBY ROAD LE11 3TU Loughborough Mostra sulla mappa Regione East Midlands (England) Leicestershire, Rutland and Northamptonshire Leicestershire CC and Rutland Tipo di attività Higher or Secondary Education Establishments Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 983 280,00 SERNOVA CORP. Canada Contribution nette de l'UE € 944 177,50 Indirizzo 700 COLLIP CIRCLE SUITE 114 N6G4X8 LONDON ONTARIO Mostra sulla mappa PMI L’organizzazione si è definita una PMI (piccola e media impresa) al momento della firma dell’accordo di sovvenzione. Sì Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Collegamenti Contatta l’organizzazione Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 944 177,50 GABO:MI GESELLSCHAFT FUR ABLAUFORGANISATION:MILLIARIUM MBH & CO KG Partecipazione conclusa Germania Contribution nette de l'UE € 34 237,00 Indirizzo OSKAR VON MILLER RING 29 80333 Muenchen Mostra sulla mappa PMI L’organizzazione si è definita una PMI (piccola e media impresa) al momento della firma dell’accordo di sovvenzione. Sì Regione Bayern Oberbayern München, Kreisfreie Stadt Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 34 237,00 ARTTIC Francia Contribution nette de l'UE € 150 000,00 Indirizzo 39 RUE DES MATHURINS 75008 Paris Mostra sulla mappa Regione Ile-de-France Ile-de-France Paris Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 150 000,00 GRUNEWALD VERWALTUNGS GMBH Germania Contribution nette de l'UE € 158 342,16 Indirizzo SPESSARTSTRASSE 2 64646 HEPPENHEIM Mostra sulla mappa Regione Hessen Darmstadt Bergstraße Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Collegamenti Contatta l’organizzazione Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 158 342,16