Objectif 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. Champ scientifique 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 Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Thème(s) PHC-16-2015 - Tools and technologies for advanced therapies Appel à propositions H2020-PHC-2014-2015 Voir d’autres projets de cet appel Sous appel H2020-PHC-2015-two-stage Régime de financement RIA - Research and Innovation action Coordinateur UNIVERSITAETSKLINIKUM WUERZBURG - KLINIKUM DER BAYERISCHEN JULIUS-MAXIMILIANS-UNIVERSITAT Contribution nette de l'UE € 1 714 513,00 Adresse JOSEF-SCHNEIDER-STRASSE 2 97080 Wurzburg Allemagne Voir sur la carte Région Bayern Unterfranken Würzburg, Kreisfreie Stadt Type d’activité Higher or Secondary Education Establishments Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 1 714 513,00 Participants (7) Trier par ordre alphabétique Trier par contribution nette de l'UE Tout développer Tout réduire GRUNEWALD JURGEN Participation terminée Allemagne Contribution nette de l'UE € 179 845,34 Adresse Voir sur la carte Type d’activité Private for-profit entities (excluding Higher or Secondary Education Establishments) Liens Contacter l’organisation Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 179 845,34 UNIVERSITA DEGLI STUDI DEL PIEMONTE ORIENTALE AMEDEO AVOGADRO Italie Contribution nette de l'UE € 1 400 000,00 Adresse DUOMO 6 13100 Vercelli Voir sur la carte Région Nord-Ovest Piemonte Vercelli Type d’activité Higher or Secondary Education Establishments Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 1 400 000,00 LOUGHBOROUGH UNIVERSITY Royaume-Uni Contribution nette de l'UE € 983 280,00 Adresse ASHBY ROAD LE11 3TU Loughborough Voir sur la carte Région East Midlands (England) Leicestershire, Rutland and Northamptonshire Leicestershire CC and Rutland Type d’activité Higher or Secondary Education Establishments Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 983 280,00 SERNOVA CORP. Canada Contribution nette de l'UE € 944 177,50 Adresse 700 COLLIP CIRCLE SUITE 114 N6G4X8 LONDON ONTARIO Voir sur la carte PME L’entreprise s’est définie comme une PME (petite et moyenne entreprise) au moment de la signature de la convention de subvention. Oui Type d’activité Private for-profit entities (excluding Higher or Secondary Education Establishments) Liens Contacter l’organisation Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 944 177,50 GABO:MI GESELLSCHAFT FUR ABLAUFORGANISATION:MILLIARIUM MBH & CO KG Participation terminée Allemagne Contribution nette de l'UE € 34 237,00 Adresse OSKAR VON MILLER RING 29 80333 Muenchen Voir sur la carte PME L’entreprise s’est définie comme une PME (petite et moyenne entreprise) au moment de la signature de la convention de subvention. Oui Région Bayern Oberbayern München, Kreisfreie Stadt Type d’activité Private for-profit entities (excluding Higher or Secondary Education Establishments) Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 34 237,00 ARTTIC France Contribution nette de l'UE € 150 000,00 Adresse 39 RUE DES MATHURINS 75008 Paris Voir sur la carte Région Ile-de-France Ile-de-France Paris Type d’activité Private for-profit entities (excluding Higher or Secondary Education Establishments) Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 150 000,00 GRUNEWALD VERWALTUNGS GMBH Allemagne Contribution nette de l'UE € 158 342,16 Adresse SPESSARTSTRASSE 2 64646 HEPPENHEIM Voir sur la carte Région Hessen Darmstadt Bergstraße Type d’activité Private for-profit entities (excluding Higher or Secondary Education Establishments) Liens Contacter l’organisation Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 158 342,16