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HEDoctoral network2023–2027

GET-IN · The GEne Therapy INnovation Training Network

Horizon Europe — Marie Skłodowska-Curie Actions

Duration
2023-09-01 → 2027-08-31
EU contribution
€2,615,796
Participants
15
Scheme
HORIZON-TMA-MSCA-DN

Lines connect the coordinator with its partners.

Results in brief

The GEne Therapy INnovation Training Network

Gene therapy has evolved from concept to clinical reality, with several rAAV-based therapies approved and many phase III trials ongoing. CRISPR-based editing is also progressing, offering hope for previously untreatable disorders. Yet, widespread adoption remains constrained by inefficient, costly, and largely manual vector manufacturing, and by limitations in delivery specificity and cross-species predictivity. Addressing these bottlenecks requires scalable, safe, and precise platforms. The MSCA GET-IN (Gene Therapy Innovation Network) unites seven academic and eight non-academic partners with expertise in viral vector technology, genome editing, process engineering, biomanufacturing, and humanized organ-on-chip (OoC) models. Ten Doctoral Candidates (DCs) are trained as the next generation of innovators. Objectives GET-IN aims to drive disruptive innovation across the gene therapy value chain by: Optimizing bioprocesses for scalable, modular, and cost-effective vector manufacturing. Developing improved vectors and genome editing tools that are safer and more widely applicable. Creating targeted delivery systems for higher tissue specificity and efficacy. Establishing human OoC models for predictive, ethical safety and efficacy testing. These goals aim to lower production costs, enhance outcomes, and accelerate translation from bench to clinic.

Data: CORDIS, © European Union

Project 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.

Original text from CORDIS.

Participants

  • KATHOLIEKE UNIVERSITEIT LEUVEN · LeuvenCoordinatorBelgium
  • ALIA THERAPEUTICS · MILANOItaly
  • ANTLERON · LouvainBelgium
  • ARISTOTELIO PANEPISTIMIO THESSALONIKIS · THESSALONIKIGreece
  • EATRIS ERIC · AmsterdamNetherlands
  • EBERHARD KARLS UNIVERSITAET TUEBINGEN · TuebingenGermany
  • INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE · ParisFrance
  • MILTENYI BIOTEC GMBH · Bergish GladbachGermany
  • MIMETAS BV · LeidenNetherlands
  • Mary Ann Liebert, Inc. · New RochelleUnited States
  • SIEMENS PROCESS SYSTEMS ENGINEERING LIMITED · LONDONUnited Kingdom
  • UCB PHARMA SA · Bruxelles / BrusselBelgium
  • UNIVERSITA DEGLI STUDI DI TRENTO · TrentoItaly
  • UNIVERSITE COTE D'AZUR · NiceFrance
  • UNIVERSITEIT LEIDEN · LeidenNetherlands

Links

Data: CORDIS, © European Union