Objective
AAV-based gene therapies are promising strategies for patients with life-threatening genetic disorders by restoring gene function in a
long-term effect reversing disease state. The domain of gene therapy is still facing development challenges at the source of extremely
high costs of these drugs on the market today, representing several million euros/dollars per dose, which still highly limits patient
access to these therapies.
This project aims at developing novel tools for second generation processes enabling a significant impact on the development and
monitoring of gene therapy manufacturing processes. The new tools should help to raise productivity by a factor of 100 compared to
first generation processes that enabled current clinical trials.
The ambition of the project relies on combining different expertise to provide innovative process and analytical technologies for the
major critical parameters that influence the production of viral particles and eventually of AAV vectors, previously identified by the
consortium.
A part of the project will be dedicated to the improvement of the upstream part of the process. Development of next generation
solutions to monitor and improve cell culture conditions will allow a deep characterization of cell culture parameters with the
objective of being able to control and to adapt the parameters during the process.
Another major challenge in AAV based gene therapy manufacturing is the purification steps to ensure product quality and safety.
Optimizing the purification steps of the DSP will be also a big improvement in the field, with major outputs such as time and cost
reduction.
Eventually, data collected from all monitored parameters for the DSP will feed a digital twin for comprehensive modeling to predict
and optimize bioprocess performance, better monitor operations and facilitate decision making.
Fields of science (EuroSciVoc)
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
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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HORIZON.2.1 - Health
MAIN PROGRAMME
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HORIZON.2.1.5 - Tools, Technologies and Digital Solutions for Health and Care, including personalised medicine
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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.
HORIZON-IA - HORIZON Innovation Actions
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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) HORIZON-HLTH-2025-01
See all projects funded under this callCoordinator
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.
91002 Evry
France
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.