CORDIS
EU research results

CORDIS

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

Project information

Grant agreement ID: 667421

Status

Closed project

  • Start date

    1 November 2015

  • End date

    31 October 2018

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 5 564 395

  • EU contribution

    € 5 564 395

Coordinated by:

UNIVERSITAETSKLINIKUM WUERZBURG - KLINIKUM DER BAYERISCHEN JULIUS-MAXIMILIANS-UNIVERSITAT

Germany

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.

Coordinator

UNIVERSITAETSKLINIKUM WUERZBURG - KLINIKUM DER BAYERISCHEN JULIUS-MAXIMILIANS-UNIVERSITAT

Address

Josef-Schneider-Strasse 2
97080 Wurzburg

Germany

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 1 714 513

Participants (7)

GRUNEWALD JURGEN

Germany

EU Contribution

€ 179 845,34

UNIVERSITA DEGLI STUDI DEL PIEMONTE ORIENTALE AMEDEO AVOGADRO

Italy

EU Contribution

€ 1 400 000

LOUGHBOROUGH UNIVERSITY

United Kingdom

EU Contribution

€ 983 280

SERNOVA CORP.

Canada

EU Contribution

€ 944 177,50

GABO:MI GESELLSCHAFT FUR ABLAUFORGANISATION:MILLIARIUM MBH & CO KG

Germany

EU Contribution

€ 34 237

ARTTIC

France

EU Contribution

€ 150 000

GRUNEWALD VERWALTUNGS GMBH

Germany

EU Contribution

€ 158 342,16

Project information

Grant agreement ID: 667421

Status

Closed project

  • Start date

    1 November 2015

  • End date

    31 October 2018

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 5 564 395

  • EU contribution

    € 5 564 395

Coordinated by:

UNIVERSITAETSKLINIKUM WUERZBURG - KLINIKUM DER BAYERISCHEN JULIUS-MAXIMILIANS-UNIVERSITAT

Germany