GeneT: The Gene Therapy CoE at the Center of Portugal

本项目的主要目标是建立一个基因治疗研究和创新中心，该中心位于葡萄牙中部地区的卓越中心（CoE）-GeneT。该卓越中心将利用科英布拉大学（UC）现有的研发和创新能力-Red Biotechnology的创新潜力和知识转移的区域（和国家）枢纽-并作为基因治疗创新和制造的科学和商业卓越的（国际）国家推动者。基因治疗是治疗严重人类疾病的最新革命，在卫生部门具有最高的治疗和经济潜力。在治疗一些神经系统、眼科、肝脏、肿瘤和血液系统疾病方面已经取得了非凡的成果，具有突出的临床和经济影响。加州大学，特别是其神经科学和细胞生物学中心，已证明在该领域的特定领域的科学卓越性，以及在这个高附加值领域努力的无与伦比的生态系统，其中包括科英布拉大学医院和生物技术园BIOCANT，被公认为葡萄牙生物技术产业的中心。为了实现其目标，该项目将与谢菲尔德大学合作，该大学是基因治疗创新和制造中心的所在地，该中心是英国致力于推进新基因治疗临床开发的先驱中心，以及东芬兰大学，该大学拥有芬兰国家病毒载体实验室，具有病毒载体GMP生产能力和规模扩大（基因治疗开发的关键）。这两个顶级的欧洲机构在基础，转化和创新层面上是该领域的国际参考。因此，基因技术将成为基因治疗研究与创新领域的一盏明灯，对降低死亡率和痛苦产生巨大影响，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.