Born in Barcelona in 1966. PhD in Genetics at Hospital Duran i Reynals and Universitat de Barcelona, 1994. National Award in Human Genetics by the Spanish Association of Human Genetics in 1995. HHMI postdoctoral fellow on Gene Therapy for Cystic Fibrosis, at the Internal Medicine Dept, University of Iowa, USA (1994-1997). EMBO fellow in Genethon III (Evry, France) (1997-1999) on Development of viral vectors for Gene Therapy. Postdoctoral fellow on Gene Therapy (2000-2001) at the Universitat Autònoma de Barcelona (UAB). ICREA Research Professor at the UAB since 2001. Director of the Vector Production Unit, a technological platform to produce viral vectors, since 2004. Assistant Professor of the Biochemistry and Molecular Biology Dept. at the UAB since 2005. Co-founder of two spin-off companies in biotechnology, NanoTherapix in 2009, and Kogenix Therapeutics in 2016. He has published more than 70 papers and generated 7 patents in viral vectors.
Medicine still has many challenges to solve specially on complex diseases where a large number of both, genetic and environmental factors, are involved. Among them, autoimmune disorders have attracted attention because there are no effective curative treatments for them. Some of these autoimmune diseases affect the central nervous system, which in addition, is difficult to access and to manipulate with classical pharmacological treatments. To address these problems we have focused our research interests in three main objectives: (1) gene therapy strategies for autoimmune diseases; (2) gene therapy strategies for diseases affecting the nervous system, such as neuromuscular and neurodegenerative disorders, and (3) development of more efficient and less immunogenic viral vectors.
– Pagès G, Giménez-Llort L, García-Lareu B, Ariza L, Navarro M, Ruberte J, Casas C, Chillón M & Bosch A 2019, ‘Intrathecal AAVrh10 corrects Biochemical and Histological Hallmarks of Mucopolysaccharidosis VII Mice and Improves Behavior and Survival’, Human Molecular Genetics: 1;28(21). pp: 3610-3624