The Istituto Superiore di Sanità (ISS) is the leading technical and scientific body of the Italian National Health Service. Its activities include research, clinical trials, control, and training. The ISS promotes scientific research in a wide variety of fields of special relevance for public health, and in particular basic, translational and clinical research in major disease areas, including cancer, neurological, infectious and rare diseases. ISS is a scientific institute where regulatory sciences go together with experimental sciences, in particular for the field of advanced therapies. For more than 20 years ISS scientists have served as experts to the Italian competent authority AIFA (Italian Medicine Agency) for authorizing clinical studies in Italy, as well as experts participating in EMA (European Medicine Agency) working groups, which generated guidelines for gene therapy and biotechnology.