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

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.

Field of science

  • /social sciences/sociology/industrial relations/automation
  • /natural sciences/biological sciences/genetics and heredity/mutation
  • /engineering and technology/environmental biotechnology/bioremediation/bioreactor
  • /medical and health sciences/medical biotechnology/genetic engineering/gene therapy

Call for proposal

H2020-PHC-2015-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

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

Participation ended

Germany
EU contribution
€ 179 845,34
Address
Kettelerstrasse 30
64646 Heppenheim
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
UNIVERSITA DEGLI STUDI DEL PIEMONTE ORIENTALE AMEDEO AVOGADRO
Italy
EU contribution
€ 1 400 000
Address
Duomo 6
13100 Vercelli
Activity type
Higher or Secondary Education Establishments
LOUGHBOROUGH UNIVERSITY
United Kingdom
EU contribution
€ 983 280
Address
Ashby Road
LE11 3TU Loughborough
Activity type
Higher or Secondary Education Establishments
SERNOVA CORP.
Canada
EU contribution
€ 944 177,50
Address
700 Collip Circle Suite 114
N6G4X8 London Ontario
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
GABO:MI GESELLSCHAFT FUR ABLAUFORGANISATION:MILLIARIUM MBH & CO KG

Participation ended

Germany
EU contribution
€ 34 237
Address
Oskar Von Miller Ring 29
80333 Muenchen
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
ARTTIC
France
EU contribution
€ 150 000
Address
Rue Du Dessous Des Berges 58A
75013 Paris
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
GRUNEWALD VERWALTUNGS GMBH
Germany
EU contribution
€ 158 342,16
Address
Spessartstrasse 2
64646 Heppenheim
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)