The Autonomous University of Barcelona is one of the major public universities in Spain. The broad range of research activities includes biotechnology, biomedicine, and animal health.
As part of UAB, the Centre of Animal Biotechnology and Gene Therapy (CBATEG) is specialised in studying the pathophysiological causes of diabetes mellitus using transgenic animal models and in developing gene therapy approaches for this disease by in-vivo gene- tic manipulation of tissues using adeno-associated (AAV) viral vectors.
In recent years, CBATEG researchers have also used their knowledge of gene transfer technologies to develop gene therapies for severe inherited metabolic disorders such as mucopolysaccharidosis (MPS). Within the frame of a public-private partnership with ESTEVE Pharmaceuticals, three gene therapy medicinal products have received the orphan drug designation by the European and North American authorities (EMA and FDA). The first clinical trial started in 2018, with a gene therapy for the treatment of MPSIIIA patients.
Moreover, CBATEG researchers coordinate the ERASMUS+ Knowledge Alliance consortium PATHBIO, a project that will gather information, data and expertise to support teaching in mouse pathobiology and different aspects of mouse phenotyping.
The CBATEG has also established a core of platforms that offer metabolic, morphological and pathological mouse phenotypic analyses to internal and external users.