Descripción del proyecto
Innovaciones pioneras en terapia génica
La terapia génica, antaño una esperanza lejana, es ahora una realidad gracias a las tres terapias génicas con virus adenoasociados recombinantes (rAAV, por sus siglas en inglés) aprobadas en la Unión Europea y más de treinta ensayos clínicos de fase III en curso. Sin embargo, su aplicación más amplia se ve obstaculizada por problemas como la fabricación no automatizada, la escalabilidad limitada y los elevados costes de producción. El equipo del el proyecto GET-IN, que cuenta con el apoyo de las Acciones Marie Skłodowska-Curie, reúne una red doctoral multidisciplinaria formada por siete socios universitarios y ocho de otros ámbitos. Con experiencia en vectorología, edición genómica y biofabricación, el equipo de GET-IN pretende formar a diez doctorandos para que sean pioneros en innovaciones en terapia génica. La investigación se centra en el bioprocesamiento optimizado, la simulación digital, los vectores mejorados, los sistemas de administración selectiva y los modelos de órganos humanos en un chip, lo que promete un salto transformador en este campo.
Objetivo
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.
Ámbito científico
- 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
Palabras clave
Programa(s)
- HORIZON.1.2 - Marie Skłodowska-Curie Actions (MSCA) Main Programme
Régimen de financiación
HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral NetworksCoordinador
3000 Leuven
Bélgica