The Gene Therapy CoE at the Center of Portugal

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.