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HEDoctoral network2024–2027

ORGESTRA · Organoid technologies for disease modeling, drug discovery and development for rare diseases

Horizon Europe — Marie Skłodowska-Curie Actions

Duration
2024-01-01 → 2027-12-31
EU contribution
€3,455,309
Participants
14
Scheme
HORIZON-TMA-MSCA-DN-JD

Lines connect the coordinator with its partners.

Results in brief

Organoid technologies for disease modeling, drug discovery and development for rare diseases

ORGESTRA is a Joint Doctoral Network training thirteen PhD researchers in organoid technologies for disease modelling, drug discovery and clinical development for rare diseases including cystic fibrosis and cystinosis. An innovative training programme has been developed to provide unique international, intersectoral and interdisciplinary training experience for our researchers. We are developing both healthy and diseased organoid models that phenotypically correspond to affected organs. The developed advanced disease models will be used to identify druggable targets, through screening of existing drugs for the ability to correct diseased phenotypes (candidates for drug repurposing) and using gene-based strategies. In addition, we will develop scalable bioprocesses for improved production and cryopreservation of tubuloids and/or kidney organoids in cGMP compatible conditions using 3D cell culture approaches and stirred-tank bioreactor technology. Our work on cystic fibrosis (CF) and primary ciliary dyskinesia models aims to develop large scale, cheap and animal free protocols for airway cell expansion and modulation, and image-based functional and molecular readouts (subcellular) through integration of automation and AI image analysis and high-content imaging. The efficiency of different therapeutic strategies, including mRNA delivery, viral gene integration, site-specific gene editing and DNA repair will be compared in the different models developed by the project. To maximise impact our work also includes proposing innovative clinical trial designs using novel outcomes under clinical and trial success relevance and linked to patient’s perspectives.

Data: CORDIS, © European Union

Project objective

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.

Original text from CORDIS.

Participants

  • UNIVERSITEIT UTRECHT · UtrechtCoordinatorNetherlands
  • ASTRAZENECA AB · SodertaeljeSweden
  • CERATIUM BV · AmsterdamNetherlands
  • Cystinosis Ireland · DublinIreland
  • FACULDADE DE CIENCIAS DA UNIVERSIDADE DE LISBOA · LisboaPortugal
  • FCIENCIAS.ID - ASSOCIACAO PARA A INVESTIGACAO E DESENVOLVIMENTO DE CIENCIAS · LisbonPortugal
  • INSTITUTO DE BIOLOGIA EXPERIMENTAL E TECNOLOGICA · OeirasPortugal
  • KATHOLIEKE UNIVERSITEIT LEUVEN · LeuvenBelgium
  • LEICA MICROSYSTEMS CMS GMBH · WetzlarGermany
  • MERCURNA BV · NIJMEGENNetherlands
  • Nederlandse Cystic Fibrosis Stichting · BaarnNetherlands
  • UNIVERSIDADE NOVA DE LISBOA · LisboaPortugal
  • UNIVERSITAIR MEDISCH CENTRUM UTRECHT · UtrechtNetherlands
  • UNIVERSITE PARIS CITE · ParisFrance

Links

Data: CORDIS, © European Union