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. 2024 Dec 2:27:10-16.
doi: 10.1016/j.csbj.2024.11.051. eCollection 2025.

CTGCT, Centre of Excellence for the Technologies of Gene and Cell Therapy: Collaborative translation of scientific discoveries into advanced treatments for neurological rare genetic diseases and cancer

Darja Marolt Presen  1 , Duško Lainšček  1   2 , Jane Kinghorn  3 , Zsolt Sebestyen  4 , Jurgen Kuball  4   5 , Leila Amini  6   7 , Petra Reinke  6   7 , Anke Fuchs  8 , Roman Jerala  1   2 , Mojca Benčina  1   2
Affiliations

CTGCT, Centre of Excellence for the Technologies of Gene and Cell Therapy: Collaborative translation of scientific discoveries into advanced treatments for neurological rare genetic diseases and cancer

Darja Marolt Presen et al. Comput Struct Biotechnol J. .

Abstract

The emerging field of precision medicine relies on scientific breakthroughs to understand disease mechanisms and develop cutting-edge technologies to overcome underlying genetic and functional aberrations. The establishment of the Centre of Excellence for the Technologies of Gene and Cell Therapy (CTGCT) at the National Institute of Chemistry (NIC) in Ljubljana represents a significant step forward, as it is the first centre of its kind in Slovenia. The CTGCT is poised to spearhead advances in cancer immunotherapy and personalised therapies for neurological and other rare genetic diseases. The centre's overarching mission is to extend beyond the NIC's scientific excellence in basic research and bring new therapeutic solutions toward clinical application. The CTGCT aims to develop a broad pipeline of biomedical tools, including innovative synthetic biology tools, gene editing and splicing technologies, RNA-based technologies, immune regulation engineering and novel viral and non-viral delivery systems. The CTGCT is supported by partner institutions from the UK, the Netherlands and Germany, which already have academic good manufacturing practice (GMP) facilities for the manufacture of advanced therapy medicinal products (ATMPs) and is committed to active collaboration with clinicians and patient organizations at all stages of development to improve access to gene and cell therapies (GCTs) for patients. The Centre also seeks to collaborate with national and international academic and industrial partners, and the newly established GMP facilities will address a critical bottleneck in the translation of GCTs from research to practice. Finally, CTGCT's translational research and technology transfer units will ensure the impactful dissemination of research and innovation activities in Slovenia, throughout the Western Balkans and Eastern Europe region, and beyond. With its comprehensive approach and forward-looking vision, the CTGCT will drive transformative advances in gene and cell therapies for the benefit of patients on a global scale.

Keywords: Advanced therapy medicinal products; Bioscience entrepreneurship; Cancer immunotherapy; Cell therapy; Gene therapy; Good manufacturing practice; Neurological diseases; Synthetic biology; Translational medicine.

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Conflict of interest statement

The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Jurgen Kuball reports a relationship with Gadeta BV that includes: co-founder and shareholder. Jurgen Kuball reports a relationship with Gadeta BV, Novartis and Miltenyi Biotec that includes funding grants. Jurgen Kuball and Zsolt Sebestyen are inventors on patents with γδ TCR-related topics. Petra Reinke reports a relationship with UK Medical Research Council that includes board membership. Petra Reinke reports funding of HORIZON 2020 EU Project ReSHAPE, HORIZON-HLTH_2021/RIA EU Project geneTIGA, AiF TcellMed-M1. Other authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

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Graphical abstract
Fig. 1
Fig. 1
Vertical integration of technologies and translational expertise of the CTGCT consortium.
See this image and copyright information in PMC

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