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CORDIS

Gene therapy Enhancement through Novel Engineering in Biomanufacturing On Sustainable Technologies

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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.

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HORIZON-IA - HORIZON Innovation Actions

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Aufforderung zur Vorschlagseinreichung

(öffnet in neuem Fenster) HORIZON-HLTH-2025-01

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