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.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: https://op.europa.eu/en/web/eu-vocabularies/euroscivoc.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: https://op.europa.eu/en/web/eu-vocabularies/euroscivoc.
- engineering and technology mechanical engineering manufacturing engineering
- medical and health sciences medical biotechnology genetic engineering gene therapy
- medical and health sciences basic medicine pharmacology and pharmacy pharmaceutical drugs
- medical and health sciences clinical medicine transplantation
- medical and health sciences medical biotechnology cells technologies
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Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being
MAIN PROGRAMME
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H2020-EU.3.1.3. - Treating and managing disease
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Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
RIA - Research and Innovation action
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) H2020-PHC-2014-2015
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
97080 Wurzburg
Germany
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.