Marolt Presen Darja, Lainšček Duško, Kinghorn Jane, Sebestyen Zsolt, Kuball Jurgen, Amini Leila, Reinke Petra, Fuchs Anke, Jerala Roman, Benčina Mojca
Centre for the Technologies of Gene and Cell Therapy, National Institute of Chemistry, Hajdrihova 19, SI1000 Ljubljana, Slovenia.
Department for Synthetic Biology and Immunology, National Institute of Chemistry, Hajdrihova 19, SI1000 Ljubljana, Slovenia.
Comput Struct Biotechnol J. 2024 Dec 2;27:10-16. doi: 10.1016/j.csbj.2024.11.051. eCollection 2025.
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.
精准医学这一新兴领域依赖科学突破来理解疾病机制，并开发前沿技术以克服潜在的基因和功能异常。位于卢布尔雅那的国家化学研究所（NIC）设立基因与细胞治疗技术卓越中心（CTGCT）是向前迈出的重要一步，因为它是斯洛文尼亚首个此类中心。CTGCT有望引领癌症免疫疗法以及针对神经和其他罕见遗传病的个性化疗法取得进展。该中心的总体使命是超越NIC在基础研究方面的卓越科学成就，为临床应用带来新的治疗方案。CTGCT旨在开发广泛的生物医学工具产品线，包括创新的合成生物学工具、基因编辑和剪接技术、基于RNA的技术、免疫调节工程以及新型病毒和非病毒递送系统。CTGCT得到了来自英国、荷兰和德国的合作机构的支持，这些机构已经拥有用于生产先进治疗药品（ATMPs）的学术良好生产规范（GMP）设施，并致力于在开发的各个阶段与临床医生和患者组织积极合作，以改善患者获得基因和细胞疗法（GCTs）的机会。该中心还寻求与国内和国际学术及产业伙伴合作，新设立的GMP设施将解决GCTs从研究转化为实践过程中的一个关键瓶颈。最后，CTGCT的转化研究和技术转移部门将确保在斯洛文尼亚、整个西巴尔干和东欧地区以及其他地区有效传播研究和创新活动。凭借其全面的方法和前瞻性的愿景，CTGCT将推动基因和细胞疗法取得变革性进展，造福全球患者。
