Projet GET-IN (Horizon Europe, appel à projets 2022)
Action : Doctoral Networks
Panel : Life Sciences
Statut du projet : SIGNED

Gene therapy has transitioned from a distant hope to reality. To date 3 rAAV gene therapies are approved in the EU, >30 phase III clinical trials ongoing, and exciting developments in therapeutic gene editing in the pipeline. However, fundamental limitations in the bioprocessing of gene therapy vectors limit broader application. Manufacturing is not automated, with an open process environment, limited scalability and robustness, and inefficient downstream processing, resulting in huge footprint and exceedingly high cost of goods. The field requires scalable manufacturing technology with modular design to produce high doses for large patient groups at a fraction of cost. Improved delivery approaches with increased specificity and efficacy at lower doses are needed to overcome emerging safety concerns observed in clinical trials. Prediction of therapeutic efficacy in man is challenging due to a species barrier, underlining the need for humanized models to reduce attrition rates in the development pipeline.  To overcome these challenges, innovation driven by multidisciplinary approaches is direly needed. GET-IN is a doctoral network of 7 academic and 8 non-academic partners, with expertise in vectorology, genome editing, process engineering, biomanufacturing and innovative humanized models. Together, they provide an excellent training framework for 10 Doctoral Candidates (DCs) who will be the future innovators in the gene therapy field. Research in GET-IN will investigate disruptive innovations including optimised bioprocessing, digital simulation, novel and improved vectors and genome editors, targeted delivery systems, and human organ-on-chip models for more relevant safety and efficacy evaluation. The joint training programme will emphasise responsible, cooperative research and innovation, creativity, and entrepreneurship to maximize the career potential of the DCs.

Mots clés : genome editing, raav, virus like particles, viral vector production, producer cell lines, process optimization, digital twin, crispr cas9, targeted delivery, humanized organ on chip models

Participants :
- Antleron (Belgique) — Partner, beneficiary, Organismes privés (hors enseignement), subvention : 218850.0 €
- Université Aristote de Thessalonique (Grèce) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 360147.6 €
- Miltenyi Biotec (Germany) (Allemagne) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Institut national de la santé et de la recherche médicale (France) — Partner, beneficiary, Recherche, subvention : 282693.6 €
- Université Eberhard Karl de Tübingen (Allemagne) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 260539.2 €
- Process Systems Enterprise (United Kingdom) (Royaume-Uni) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Université de Trente (Italie) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 259437.6 €
- KU Leuven (Belgique) — Coordinator, beneficiary, Enseignement supérieur ou secondaire, subvention : 700320.0 €
- Université Côte d'Azur (France) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Mary ann liebert, inc. (Etats-Unis) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Université de Leyde (Pays-Bas) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Alia therapeutics (Italie) — Partner, beneficiary, Organismes privés (hors enseignement), subvention : 259437.6 €
- European Infrastructure for Translational Medicine (Pays-Bas) — Partner, associated partner, Recherche, subvention : 0.0 €
- UCB Pharma (Belgium) (Belgique) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Mimetas (Netherlands) (Pays-Bas) — Partner, beneficiary, Organismes privés (hors enseignement), subvention : 274370.4 €