Description du projet
Des innovations pionnières en thérapie génique
La thérapie génique, qui n’était autrefois qu’un vague espoir, est aujourd’hui une réalité: trois thérapies géniques rAAV ont été approuvées dans l’UE et plus de 30 essais cliniques de phase III sont en cours. Son application plus large est toutefois entravée par divers défis tels qu’une fabrication non automatisée, son extensibilité limitée et les coûts de production élevés. Le projet GET-IN, soutenu par le programme Actions Marie Skłodowska-Curie, rassemble un réseau doctoral pluridisciplinaire de sept partenaires universitaires et huit partenaires non universitaires. S’appuyant sur son expertise en vectorologie, en édition du génome et en bioproduction, GET-IN entend former 10 doctorants qui seront des pionniers de l’innovation en thérapie génique. La recherche se concentre sur l’optimisation des bioprocédés, la simulation numérique, l’amélioration des vecteurs, les systèmes d’administration ciblés et les modèles d’organes humains sur puce, ce qui promet une formidable avancée dans le domaine.
Objectif
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.
Champ scientifique
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- social scienceseconomics and businessbusiness and managementinnovation management
- social scienceseconomics and businessbusiness and managemententrepreneurship
- engineering and technologyother engineering and technologiesmicrotechnologyorgan on a chip
- natural sciencesbiological sciencesgeneticsgenomes
Mots‑clés
Programme(s)
- HORIZON.1.2 - Marie Skłodowska-Curie Actions (MSCA) Main Programme
Régime de financement
HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral NetworksCoordinateur
3000 Leuven
Belgique