Skip to main content
EnglishEN
European Commission logo print header

The GEne Therapy INnovation Training Network

Project description

DEENESFRITPL

Pioneering innovations in gene therapy

Gene therapy, once a distant hope, is now a reality with three rAAV gene therapies approved in the EU and over 30 phase III clinical trials underway. However, its broader application is hindered by challenges such as non-automated manufacturing, limited scalability, and high production costs. With the support of the Marie Skłodowska-Curie Actions programme, the GET-IN project brings together a multidisciplinary doctoral network comprising seven academic and eight non-academic partners. With expertise in vectorology, genome editing, and biomanufacturing, GET-IN aims to train 10 doctoral candidates (DCs) to pioneer innovations in gene therapy. Research focuses on optimised bioprocessing, digital simulation, improved vectors, targeted delivery systems, and human organ-on-chip models, promising a transformative leap in the field.

Objective

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.

Fields of science

Keywords

Programme(s)

Topic(s)

Call for proposal

HORIZON-MSCA-2022-DN-01

See other projects for this call

Funding Scheme

HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral Networks

Coordinator

KATHOLIEKE UNIVERSITEIT LEUVEN
Net EU contribution
€ 700 320,00
Address
OUDE MARKT 13
3000 Leuven
Belgium

See on map
Region
Vlaams Gewest Prov. Vlaams-Brabant Arr. Leuven
Activity type
Higher or Secondary Education Establishments
Links
Total cost
No data

Participants (7)

Partners (7)

Share this page

Download

Last update: 11 July 2023

My Booklet

Permalink: https://cordis.europa.eu/project/id/101119880

European Union, 2024