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HemAcure SIGNED

Application of combined gene and cell therapy within an implantable therapeutic device for the treatment of severe hemophilia A

Total Cost €

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EC-Contrib. €

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Partnership

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Project "HemAcure" data sheet

The following table provides information about the project.

Coordinator
UNIVERSITAETSKLINIKUM WUERZBURG - KLINIKUM DER BAYERISCHEN JULIUS-MAXIMILIANS-UNIVERSITAT 

Organization address
address: JOSEF-SCHNEIDER-STRASSE 2
city: WURZBURG
postcode: 97080
website: www.uk-wuerzburg.de

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Germany [DE]
 Project website https://www.hemacure.eu/
 Total cost 5˙564˙395 €
 EC max contribution 5˙564˙395 € (100%)
 Programme 1. H2020-EU.3.1.3. (Treating and managing disease)
 Code Call H2020-PHC-2015-two-stage
 Funding Scheme RIA
 Starting year 2015
 Duration (year-month-day) from 2015-11-01   to  2018-10-31

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITAETSKLINIKUM WUERZBURG - KLINIKUM DER BAYERISCHEN JULIUS-MAXIMILIANS-UNIVERSITAT DE (WURZBURG) coordinator 1˙714˙513.00
2    UNIVERSITA DEGLI STUDI DEL PIEMONTE ORIENTALE AMEDEO AVOGADRO IT (VERCELLI) participant 1˙400˙000.00
3    LOUGHBOROUGH UNIVERSITY UK (LOUGHBOROUGH) participant 983˙280.00
4    SERNOVA CORP. CA (LONDON ONTARIO) participant 944˙177.00
5    GRUNEWALD JURGEN DE (HEPPENHEIM) participant 179˙845.00
6    GRUNEWALD VERWALTUNGS GMBH DE (HEPPENHEIM) participant 158˙342.00
7    ARTTIC FR (PARIS) participant 150˙000.00
8    GABO:MI GESELLSCHAFT FUR ABLAUFORGANISATION:MILLIARIUM MBH & CO KG DE (MUENCHEN) participant 34˙237.00

Map

 Project objective

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.

 Deliverables

List of deliverables.
Final dissemination activity on project results Other 2019-08-30 14:16:29
Exploitation activity (as above) Documents, reports 2019-08-30 14:16:29
Launch of website, Twitter, Facebook, LinkedIn etc. Websites, patent fillings, videos etc. 2019-08-30 14:16:29
Individualized press release for every partner Documents, reports 2019-08-30 14:16:29

Take a look to the deliverables list in detail:  detailed list of HemAcure deliverables.

 Publications

year authors and title journal last update
List of publications.
2017 Patrick Bittorf, Martin Zierau, Ralf Sanzenbacher, Heike Walles, Joris Braspenning
Combined ATMPs Containing Genetically Modified Cells
published pages: 168-174, ISSN: , DOI:
Pharmazeutische Medizin 2019-08-30
2017 Chiara Borsotti, Simone Merlin, Cristina Olgasi, Thorsten Bergmann, Delfina M. Mazzuca, Alexandra Stolzing, Martin Zierau, Philip M. Toleikis, Joris Braspenning and Antonia Follenzi
Application of combined gene and cell therapy within an implantabletherapeutic device for the treatment of severe haemophilia A
published pages: s483, ISSN: 1723-2007, DOI:
Blood Transfusion Vol.15, Supplement No. 3, Septe 2019-08-30
2018 Miguel Rodrigues
\"\"\"Use of BOECs for therapeutic applications\"\"\"
published pages: , ISSN: , DOI:
2019-08-30
2017 Cristina Olgasi, Simone Merlin, Thorsten Bergmann, Chiara Borsotti, Delfina M. Mazzuca, Alexandra Stolzing, Martin Zierau, Philip M. Toleikis, Joris Braspenning and Antonia Follenzi
Application of combined gene and cell therapy within an implantabletherapeutic device for the treatment of severe haemophilia A (poster presentation at ESGCT congress, Berlin, 2017)
published pages: pp A92, ISSN: 1043-0342, DOI:
Human Gene Therapy Vol.28, No. 12, 1st December 20 2019-08-30
2019 Patrick Bittorf
Genetically modified cells for treatment of heamophilia A
published pages: , ISSN: , DOI:
2019-08-30
2018 C Olgasi, S Merlin, C Borsotti, T Bergmann, D M Mazzucca, A Stolzing, M Zierau, P M Toleikis, J Braspenning, A Follenzi  
Application of combined gene and cell therapy within an implantable therapeutic device for the treatment of severe haemophilia A
published pages: , ISSN: , DOI:
ESGCT abstract book 2018 16th-19th October 2018 2019-08-30

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The information about "HEMACURE" are provided by the European Opendata Portal: CORDIS opendata.

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