Andrea Ballabio obtained his M.D. degree at the University of Naples, Italy, where he completed his residency in Pediatrics. After working as post-doctoral fellow at the Institute of Genetics and Biophysics in Naples and at Guy’s hospital in London UK, in 1989 he moved to the Department of Molecular and Human Genetics of Baylor College of Medicine in Houston TX USA, where he became Associate Professor and Co-director of the Human Genome Center. At the end of 1994 he moved back to Italy to become the founder and director of the Telethon Institute of Genetics and Medicine (TIGEM). He is also Professor of Medical Genetics at the Faculty of Medicine of the University of Naples “Federico II” and Visiting Professor at both Baylor College of Medicine in Houston, Texas, and at the University of Oxford, UK. He was two-time recipient of the Advanced European Research Council (ERC) 5 year-grant, in 2010 and in 2016. He is the winner of the 2016 Louis-Jeantet prize for Medicine. He is also Co-Founder of CASMA Therapeutics, an autophagy-focused company based in Boston MA. The focus of Andrea Ballabio’s research has been on the elucidation of the mechanisms underlying genetic diseases. The contributions of his laboratory have spanned a wide range of biological processes and human diseases. Recently, his group discovered that lysosomal function and autophagy are subject to a global transcriptional regulation by the master gene TFEB, which is involved in a lysosome-to-nucleus signaling pathway. Induction lysosomal biogenesis and autophagy via TFEB has proven to be a potent tool to promote cellular clearance in lysosomal storage disorders and neurodegenerative diseases. He has authored over 320 publications in international peer-reviewed journals.