GeneT: The Gene Therapy CoE at the Center of Portugal

本项目的主要目标是创建一个基因治疗研究和创新中心，其根基是位于葡萄牙Centro(中心)地区的卓越中心(COE)--Genet。该COE将利用科英布拉大学(UC)在该领域已有的研发能力--红色生物技术创新潜力和知识转移的地区性(和全国性)支点--并在(国际)全国范围内推动基因治疗创新和制造领域的科学和商业卓越。基因疗法是治疗人类严重疾病的最新革命，在卫生部门具有最高的治疗和经济潜力。在一些神经、眼科、肝脏、肿瘤和血液病的治疗方面已经取得了非凡的成果，具有显著的临床和经济影响。加州大学，特别是其神经科学和细胞生物学中心，在该领域的特定领域证明了卓越的科学成就，并拥有无与伦比的生态系统，努力在这个高附加值领域努力，其中包括科英布拉大学医院和被公认为葡萄牙生物技术产业中心的生物技术园Biocant。为了实现其目标，该项目将与谢菲尔德大学和东芬兰大学合作，谢菲尔德大学是基因治疗创新和制造中心的所在地，谢菲尔德大学是致力于推进新基因治疗临床开发的先驱中心，东芬兰大学拥有Finish国家病毒载体实验室，具有病毒载体GMP的生产能力和规模(基因治疗开发的关键)。这两个欧洲顶尖机构在基础、翻译和创新层面都是该领域的国际参考。Genet将因此成为基因治疗研发领域卓越的灯塔，在降低死亡率和痛苦方面产生巨大影响，同时为葡萄牙和欧盟的经济复苏和增长做出贡献。

The main goal of the present project is the creation of a Gene Therapy research & innovation hub rooted in a Center of Excellence (CoE) located in the CENTRO (center) region of Portugal – the GeneT. This CoE will capitalize on the already existing RD&I capacity in the field at the University of Coimbra (UC) – the regional (and national) pivot of the innovation potential and knowledge transfer in Red Biotechnology – and act as an (inter)national enabler of scientific and business excellence in Gene Therapy innovation and manufacturing. Gene Therapy is the latest revolution in the treatment of severe human diseases with the highest curative and economic potential in the health sector. Extraordinary results have already been achieved in the treatment of a few neurological, ophthalmic, hepatic, oncological and hematological disorders with outstanding clinical and economic impacts. UC, notably its Center for Neuroscience and Cell Biology, has proven scientific excellence in specific areas of the field, and a matchless ecosystem to strive in this high added-value sector, which encompasses the Coimbra University Hospitals and the Biotechnology Park BIOCANT, recognized as the epicenter of the biotech industry in Portugal. To achieve its goal, the project will team-up with the University of Sheffield, home of the Gene Therapy Innovation and Manufacturing Centre, a UK pioneer hub dedicated to advancing the clinical development of new genetic treatments, and the University of Eastern Finland, which hosts the Finish National Virus Vector Laboratory with viral vector GMP production capacity and scale-up (key to gene therapy development). These two top European institutions are international references in the field at fundamental, translational and innovation levels. GeneT will thus become a beacon of excellent in Gene Therapy R&I, with enormous impact in the decrease of mortality and suffering, while contributing to the economic recovery and growth of Portugal and the EU.