Projet ENTRY-DM (Horizon Europe, appel à projets 2023)
Action : Doctoral Networks
Panel : Life Sciences
Statut du projet : SIGNED

Rare diseases, though individually infrequent, collectively pose important challenges for patients, clinicians and researchers in terms of diagnostics, healthcare and treatment. Myotonic Dystrophy (DM), the most predominant inherited muscular dystrophy in adults, impacts 60,000-70,000 individuals in Europe. Its complex genetic and clinical variability result in a lack of robust genotype-phenotype correlations, which complicates the understanding of tissue-specific disease mechanisms, the development of effective therapies and the stratification of patients into well-defined clinical groups for clinical trial purposes. In recent years, Antisense Oligonucleotides (ASO) have emerged as promising therapeutics, notably in neuromuscular disorders. However, previous clinical trials in DM have failed due the reduced efficacy and bioavailability of the ASO tested. ENTRY-DM aims to train 14 DCs in translational research, through the combination of basic and clinical competencies across multiple disciplines, as well as strong soft and transferable skills. Our interdisciplinary network seeks to enhance scientific and technological knowledge, spanning from disease mechanisms, ASO design and delivery strategies, to clinical trial preparedness, using innovative multidisciplinary approaches and best practices. The consortium includes experts in DM research, bioengineering of model systems for preclinical drug screening, ASO chemistry, as well as clinical and neuropsychological assessment. Close collaborations with multi-sectoral partners will address the challenges in technology transfer, providing high-quality bench-to-bedside training to the next generation of researchers. Through these efforts, ENTRY-DM will catalyze ASO therapeutic development towards upcoming impeding clinical trials, establishing solid foundations for future clinical applications, increased investment and entrepreneurship ventures in the field of DM and other related diseases.

Mots clés : myotonic dystrophy, neuromuscular disease, oligonucleotide therapy, in vitro 3d disease models, disease biomarkers, neuropsychological assessment of decision making

Participants :
- Biobam bioinformatics sl (Espagne) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Université Radboud de Nimègue (Pays-Bas) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Université d'Oxford (Royaume-Uni) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Université de Valence (Espagne) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 503942.4 €
- Université de Barcelone (Espagne) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Institute for Bioengineering of Catalonia (Espagne) — Partner, beneficiary, Recherche, subvention : 251971.2 €
- Genartis srl (Italie) — Partner, beneficiary, Organismes privés (hors enseignement), subvention : 259437.6 €
- Sanofi (United States) (Etats-Unis) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Centre d' etude des cellules souches (France) — Partner, beneficiary, Recherche, subvention : 282693.6 €
- Arthex biotech sl (Espagne) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Association européenne contre les dystrophies myotoniques (France) — Partner, associated partner, Autres, subvention : 0.0 €
- Radboud University Medical Centre (Pays-Bas) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 548740.8 €
- Université de Göteborg (Suède) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Université Évry Paris-Saclay (France) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Myotonic dystrophy foundation (Etats-Unis) — Partner, associated partner, Autres, subvention : 0.0 €
- Institut national de la santé et de la recherche médicale (France) — Coordinator, beneficiary, Recherche, subvention : 565387.2 €
- Centre de recherche en myologie (France) — Partner, associated partner, Recherche, subvention : 0.0 €
- Sorbonne Université (France) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Université Paris Cité (France) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 282693.6 €
- Université du Commonwealth de Virginie (Etats-Unis) — Partner, associated partner, Organismes publics (hors enseignement & recherche), subvention : 0.0 €
- Ecrin european clinical research infrastructure network (France) — Partner, associated partner, Recherche, subvention : 0.0 €
- Conseil supérieur de la recherche scientifique (Espagne) — Partner, beneficiary, Recherche, subvention : 251971.2 €
- Université de Rome « La Sapienza » (Italie) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- European Infrastructure for Translational Medicine (Pays-Bas) — Partner, associated partner, Recherche, subvention : 0.0 €
- Université de Rome « Tor Vergata » (Italie) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 259437.6 €
- Université de Vérone (Italie) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Université Louis-et-Maximilien de Munich (Allemagne) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 260539.2 €
- Université Adam-Mickiewicz de Poznań (Pologne) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 226512.0 €
- State University System of Florida (Etats-Unis) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €