Projektbeschreibung
Zukunftsweisende Innovationen in der Gentherapie
Die Gentherapie, einst eine ferne Hoffnung, ist heute Realität: Drei rAAV-Gentherapien sind in der EU zugelassen und über 30 klinische Studien der Phase III laufen. Eine breitere Anwendung wird jedoch durch Probleme wie die nicht automatisierte Fertigung, die begrenzte Skalierbarkeit und die hohen Produktionskosten behindert. Mit Unterstützung der Marie-Skłodowska-Curie-Maßnahmen wird über das Projekt GET-IN ein multidisziplinäres Doktorandennetzwerk aus sieben akademischen und acht nichtakademischen Partnern zusammengebracht. Mit seinem Fachwissen in den Bereichen Vektorologie, Genomeditierung und Bioproduktion wird GET-IN 10 Promovierende ausbilden, die Innovationen in der Gentherapie vorantreiben sollen. Die Forschung konzentriert sich auf optimierte Bioprozesse, digitale Simulationen, verbesserte Vektoren, gezielte Abgabesysteme und menschliche Organ-on-Chip-Modelle und verspricht einen transformativen Sprung in diesem Bereich.
Ziel
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.
Wissenschaftliches Gebiet
- 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
Schlüsselbegriffe
Programm/Programme
- HORIZON.1.2 - Marie Skłodowska-Curie Actions (MSCA) Main Programme
Aufforderung zur Vorschlagseinreichung
Andere Projekte für diesen Aufruf anzeigenFinanzierungsplan
HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral NetworksKoordinator
3000 Leuven
Belgien