Dr Gonzalez-Aseguinolaza’s scientific career has always been associated with the development of treatments for orphan diseases. She began working on the development of genetic vaccines against parasitic infections like malaria at the Centro de Investigaciones Biológicas in Madrid, Spain and the University of New York, USA. Then she moved to the field of rare diseases. Dr Gonzalez-Aseguinolaza has been working on hepatic rare diseases for the last 10 years focusing on the development of the characterization of animal models, the development of new therapeutic strategies and new gene delivery vectors. Her main achievement in the field was the coordination of a European consortium created to perform the first AAV-gene therapy clinical trial for a liver-inherited metabolic disease, Acute intermittent porphyria. Since 2014, Dr Gonzalez-Aseguinolaza is the head of the Gene Therapy Program at CIMA and the coordinator of the area of advanced therapies at the Institute of Biomedical of Navarra, IdiSNA. Dr. Gonzalez-Aseguinolaza earned her PhD in Science from Universidad Autonoma de Madrid.