Jérôme Ausseil is Professor in medical biochemistry and molecular biology at the Toulouse Health department of the University Paul Sabatier (UT3) and clinical biochemist at the Toulouse University Hospital. He is involved in the diagnosis of hereditary metabolic diseases and more particularly lysosomal storage disorders. He is also responsible of the regional newborn screening center laboratory (région Occitanie). Since 2020, he co-leads a research team (Team 5, Infinity, InsermU1291/CNRS5051/UT3) working on the pathophysiology of inflammatory and neuro-inflammatory disorders, and on the design and the development of new biotherapies and their implementation at the preclinical and clinical steps. J. Ausseil’s group is currently studying the functional and molecular status of microglia in Sanfilippo syndrome (Mucopolysaccharidosis III) and the production, composition and role of extracellular vesicles produced by microglia during neuro-inflammation. Few years ago, J. Ausseil was involved in the first phase I / II intracerebral gene therapy clinical trial on 4 Sanfilippo B children, the results of which were very promising. His group has recently started a new preclinical study on murine/canine and non-human primate models using a new vector serotype able to cross the blood brain barrier and the corresponding clinical trial for this neuropediatric disease is planned for the next few months.