Projet GetRadi (Horizon Europe, appel à projets 2021)
Action : Doctoral Networks
Panel : Life Sciences
Statut du projet : SIGNED

More than 300 million people worldwide are suffering from more than 6000 rare diseases. Nearly all of these rare diseases are caused by a single inherited mutation and cannot be treated effectively. Repair of the defective gene by gene editing is the only possible curative therapy. However, only for very few rare diseases such gene editing therapy has reached the clinic. The “Gene therapy of Rare Diseases” (GetRadi) consortium aims now to contribute strongly to the establishment of more gene therapies for rare diseases. This will be accomplished by training of future leaders in gene therapy of rare diseases preforming ambitious research projects, with the following objectives: (i) Improving transfer of genome editing tools to target cells, (ii) improving gene editing efficiency, and (iii) improving safety of gene therapy. Strong participation of the pharmaceutical industry to research projects and training, development of novel in vitro and in vivo models for rare diseases to test gene therapies in relevant settings, and application of several unique genome editing tools developed by the applicants to the treatment of rare diseases are hallmarks of the network. GetRadi brings together strong industrial beneficiaries (AstraZeneca, Miltenyi Biotec) supervising 3 ESR, highly innovative academic beneficiaries supervising 7 ESR, 3 additional industrial partners, and 4 student enrolling universities. Professional outreach training with access to widely used social media channels will be provided by the partner “European Consortium for Communicating Gene and Cell Therapy Information”. Complementary unique expertise that is spread efficiently by network-wide training and secondments, excellent quality of supervision and intersectoral interactions, innovative transferable skill training, and efficient consortium management will result in an exceptional training of future leaders in gene therapy of rare diseases.

Mots clés : gene therapy, rare diseases, crispr genome editing

Participants :
- Institute of Molecular Genetics (Rép. Tchèque) — Partner, beneficiary, Recherche, subvention : 237038.4 €
- Université Charles de Prague (Rép. Tchèque) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Université d'Édimbourg (Royaume-Uni) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Université de Naples - Frédéric-II (Italie) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- AstraZeneca (Sweden) (Suède) — Partner, beneficiary, Organismes privés (hors enseignement), subvention : 587419.2 €
- Miltenyi Biotec (Germany) (Allemagne) — Partner, beneficiary, Organismes privés (hors enseignement), subvention : 260539.2 €
- Université de Copenhague (Danemark) — Coordinator, beneficiary, Enseignement supérieur ou secondaire, subvention : 905364.0 €
- Max Delbrück Center (Allemagne) — Partner, beneficiary, Recherche, subvention : 260539.2 €
- Hôpital universitaire de la Charité de Berlin (Allemagne) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €
- Magle chemoswed holding ab (Suède) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Synvolux therapeutics bv (Pays-Bas) — Partner, associated partner, Organismes privés (hors enseignement), subvention : 0.0 €
- Leiden University Medical Center (Pays-Bas) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 274370.4 €
- Université de Fribourg-en-Brisgau (Allemagne) — Partner, beneficiary, Enseignement supérieur ou secondaire, subvention : 260539.2 €
- Université de Fribourg-en-Brisgau (Allemagne) — Partner, associated partner, Enseignement supérieur ou secondaire, subvention : 0.0 €