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

The vast majority of rare diseases are characterized by genetic origin, often severe in pathology and with limited or no treatment options. This also includes the paediatric disorders cystic fibrosis (CF) and cystinosis. Both diseases usually manifest in the first year of life. In recent year, great progress has been made in CF research allowing a swift transfer of novel drugs from bench to bedside. This is fueled by the application of advanced in vitro models, viz. organoids, in phenotyping patients and personalize treatment options. In ORGESTRA, we propose to use stem cell-derived organoids to train 13 Doctoral Candidates (DCs) in developing personalized disease models for the lung and kidney diseases, CF and cystinosis, with significant unmet clinical needs. Research will aim to identify pathogenetic mechanisms and druggable targets, drug mode of action for effective and innovative treatment strategies, classify the pathway to clinic and address ethical and regulatory issues to foster acceptance and implementation of the organoid models. Eventually, a living technologies toolset will be developed for a swift transfer from bench to bedside. ORGESTRA will provide an international, intersectoral and interdisciplinary training by creating a new expert framework of 14 partners from 7 countries, combining molecular biologists, engineers, pharmacologists, clinicians, epidemiologists and ethicists, giving each DC unique, joint-doctoral training and tailored experience in industry and/or patient organisations, complemented with a rich workshop programme. The ORGESTRA objectives contribute perfectly to the Horizon Europe goals of the societal challenge on improving the health and well-being of the EU citizens.

Mots clés : precision medicine, cystic fibrosis, cystinosis, clinical trial design, organoids, personalised disease model, regulatory issues, public patient interactions, patient perspective

Participants :
- Université d'Utrecht (Pays-Bas) — Coordinator, beneficiary, Enseignement supérieur ou secondaire, subvention : 548740.8 €
- Danaher (Germany) (Allemagne) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Université de Lisbonne (Portugal) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- KU Leuven (Belgique) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 787860.0 €
- Université Paris Cité (France) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 565387.2 €
- Cystinosis ireland (Irlande) — Partner, associated partner, Recherche, subvention : 0.0 €
- AstraZeneca (Sweden) (Suède) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- University Medical Center Utrecht (Pays-Bas) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 548740.8 €
- Fciencias.id - associacao para a investigacao e desenvolvimento de ciencias (Portugal) — Partner, beneficiary, Recherche, subvention : 486806.4 €
- Instituto de Biologia Experimental e Tecnológica (Portugal) — Partner, beneficiary, Recherche, subvention : 243403.2 €
- Mercurna bv (Pays-Bas) — Partner, beneficiary, Organismes privés (hors enseignement), subvention : 274370.4 €
- Ceratium bv (Pays-Bas) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Nederlandse CF Registratie (Pays-Bas) — Partner, associated partner, Autres, subvention : 0.0 €
- Université nouvelle de Lisbonne (Portugal) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €