Descrizione del progetto
Innovazioni pionieristiche nella terapia genica
La terapia genica, un tempo una speranza lontana, è ora una realtà alla luce delle tre terapie geniche rAAV approvate nell’UE e degli oltre 30 studi clinici di fase III attualmente in corso. Tuttavia, la diffusione della sua applicazione è ostacolata da problemi quali produzione non automatizzata, scalabilità limitata ed elevati costi di produzione. Con il sostegno del programma di azioni Marie Skłodowska-Curie, il progetto GET-IN riunisce una rete di dottorato multidisciplinare composta da sette partner accademici e otto non accademici. Forte delle sue competenze in vettoriologia, editing genomico e biomanifattura, GET-IN mira a formare 10 dottorandi affinché diventino pionieri dell’innovazione nel campo della terapia genica. La ricerca si concentra su bioprocessi ottimizzati, simulazione digitale, vettori migliorati, sistemi di somministrazione mirati e modelli di organi umani su chip, promettendo di far compiere un salto di qualità al settore.
Obiettivo
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.
Campo scientifico
- 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
Parole chiave
Programma(i)
- HORIZON.1.2 - Marie Skłodowska-Curie Actions (MSCA) Main Programme
Meccanismo di finanziamento
HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral NetworksCoordinatore
3000 Leuven
Belgio