By the end of the course, the student will acquire: 1. Knowledge of recent developments in the emerging field of advanced therapies, with particular reference to therapeutic approaches that have recently completed the approval process and are currently used in clinical practice; 2. Knowledge on the pathway a new advanced therapy must follow to progress from the bench to the bedside, with particular focus on regulatory aspects, validation and access procedures, manufacturing processes (e.g., scale-up, sterilization, storage procedures for advanced therapies), and market authorization; 3. Knowledge of the contribution of artificial intelligence methods to the development of new advanced therapies; 4. Ability to apply the knowledge and methods acquired during the course to address emerging and frontier challenges related to human health; 5. Ability to conduct literature and market surveys and to effectively communicate data and results.

Knowledge of artificial intelligence applications in medicine Knowledge of personalized medicine approaches Knowledge of bionanotechnologies Basic knowledge of material science Basic knowledge of tissue engineering and regenerative medicine principles

- Introduction to advanced biomedical technologies: advanced therapies - The pathway enabling new advanced therapies to move from the bench to the bedside (regulatory aspects, validation and access procedures, manufacturing processes, and market authorization) - Advanced therapies (e.g., gene therapy products with particular focus on in vivo therapies, tissue engineering products, combined advanced therapies): classification, definitions, examples of already approved therapies and therapies currently at the development stage - Multi omics approaches as drivers for advanced therapies development - Artificial intelligence in the development of advanced therapies: definitions, methodologies, examples, contribution to personalized medicine During the in-class exercises, students will work in groups to conduct an in-depth analysis of case studies, with particular focus on the pathway driving a new therapy from the scientific idea to the approved and commercially available product (through literature review, market research, and analysis of clinical trial results). Furthermore, students will evaluate the potential contribution of artificial intelligence across the different stages of development, including target identification, patient stratification, clinical trial design, manufacturing optimization, and post-marketing monitoring.

The course is composed by lectures (42 h) and in-class exercises (18 h). The lectures will be delivered in the classroom (or in the virtual classroom if necessary). For in-class exercises the students will be divided into groups of 3-4 components each.

Reviews and research papers

Lecture slides;

Exam: Compulsory oral exam;

The final examination will consist of a group presentation followed by an individual discussion on the course topics. GROUP PRESENTATION (maximum 15 points) Students will be divided into groups and asked to present an advanced therapy, focusing on the pathway that has led from the initial scientific idea to the development of an approved and commercially available product. The presentation may be prepared using PowerPoint or other software, according to the students’ preferences. Each group will have a maximum of 20 minutes to present its work, and all group members are expected to contribute to the presentation. The required structure and guidelines will be communicated during the introductory lecture to the in-class exercises. The presentation will be evaluated based on: - The clarity and effectiveness of the presentation. - The students’ understanding of the scientific and technological aspects underlying the advanced therapy under investigation. - The students’ ability to critically analyze the advantages and limitations of the selected case study, as well as the outstanding questions and challenges that remain to be addressed. INDIVIDUAL DISCUSSION (maximum 18 points, approx. 15 min per student) The presentation will be followed by an individual discussion aimed at assessing the students’ knowledge of the course topics. Each student will be asked two questions covering: - The definition, classification, and development pipeline of advanced therapies, including regulatory aspects, validation and access procedures, manufacturing processes, and marketing authorization. - The contribution of emerging tools and approaches, such as multiomics analysis and artificial intelligence, to the development of advanced therapies. The evaluation will take into account: - The correctness of the answers. - The relevance of the information provided. - The ability to respond clearly, accurately, and logically, adequately supporting the arguments presented. Final grade: the grade obtained in the individual discussion will be added to the grade awarded for the group presentation. Grades above 30.5 will be recorded as 30 cum laude (30 e lode).

In addition to the message sent by the online system, students with disabilities or Specific Learning Disorders (SLD) are invited to directly inform the professor in charge of the course about the special arrangements for the exam that have been agreed with the Special Needs Unit. The professor has to be informed at least one week before the beginning of the examination session in order to provide students with the most suitable arrangements for each specific type of exam.