Gene therapy and CAR-T cell therapy represent cutting-edge therapeutic strategies that are opening new opportunities for the treatment of highly debilitating diseases, including cancer, neurodegenerative disorders, and genetic diseases, for which standardized therapeutic approaches are often still unavailable. The development and characterization of these advanced therapies require a highly multidisciplinary framework involving biomedical engineers, biologists, and clinicians, as well as the integration of state-of-the-art technologies such as advanced microscopy techniques, engineered animal models, artificial intelligence tools, and bioinformatics approaches to support their validation and clinical translation. The aim of this course is to provide students with foundational knowledge of gene and cell therapies as a basis for the implementation of bioengineering approaches to their development, validation, and translational application. To this end, the course combines theoretical lectures on the design, development, and validation of advanced therapies with sessions focused on their practical clinical application, including regulatory, manufacturing, and commercialization aspects. The overall goal is to foster the training of biomedical engineering professionals with expertise in a rapidly evolving field characterized by significant therapeutic impact and strong potential for future industrial and commercial development.

By the end of the course, students will be able to: Describe the fundamental principles of advanced therapies, including gene and cell therapies, and their role in the treatment of cancer, neurodegenerative disorders, and genetic diseases. Explain the biological, cellular, genetic, and immunological mechanisms underlying advanced therapeutic approaches. Discuss the main classes of cell-based therapies, including CAR-T and alternative cellular immunotherapies, together with their clinical applications and current limitations. Understand the technologies enabling the development, formulation, and delivery of gene therapies, including vector engineering and delivery platforms. Evaluate the role of experimental models and advanced preclinical validation strategies in the development and translational assessment of advanced therapies. Apply knowledge of advanced microscopy, sensing technologies, and bioengineering tools to the validation and characterization of gene and cell therapies. Recognize the main regulatory, manufacturing, and translational challenges associated with the clinical implementation and commercialization of advanced therapies. Critically analyze clinical case studies and translational success stories in the field of advanced therapeutic technologies. Develop a multidisciplinary perspective integrating engineering, biological, and clinical approaches for the design and validation of next-generation biomedical therapies.

Basic knowledge of chemical bioengineering Basic Knowledge of polymeric materials science and engineering

Introduction to gene and cell therapies Fundamental concepts of immunology for advanced therapeutic applications Cell factories for the development and clinical implementation of CAR-T cell therapies Clinical applications of CAR-T therapies: current achievements, limitations, and future perspectives Technologies for the formulation and delivery of gene therapies Clinical applications of gene therapy: current achievements, challenges, and future directions Validation methodologies for gene and cell therapies, including advanced microscopy and sensing technologies Case studies and success stories on the translation of (bio)technologies for advanced therapies from research to market

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60 hours of classroom lectures aimed at developing knowledge and understanding of the biological, engineering, technological, and clinical principles underlying advanced therapies, with particular focus on gene and cell therapies, enabling technologies, preclinical validation strategies, and translational applications.

“The era of gene therapy: From preclinical development to clinical application” 10.1016/j.drudis.2021.03.021 "Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States" https://doi.org/10.3389/fphar.2019.00921 "Therapeutic in vivo delivery of gene editing agents" https://doi.org/10.1016/j.cell.2022.03.045

Slides;

Modalita di esame: Prova scritta in aula tramite PC con l'utilizzo della piattaforma di ateneo;

Exam: Computer-based written test in class using POLITO platform;

... The assessment consists of a written examination designed to evaluate the students’ understanding of the theoretical foundations, enabling technologies, translational aspects, and clinical applications of advanced therapies discussed during the course. The written exam includes: 15 multiple-choice questions, each with 4 answer options, covering all course topics, including: introduction to advanced therapies, gene and cell therapies, basic immunology concepts, genetic and cellular engineering approaches, CAR-T and alternative cellular therapies, technologies for gene therapy formulation and delivery, enabling technologies and experimental models, advanced preclinical validation methods, regulatory and translational aspects, clinical applications in oncology, neurodegenerative disorders, and genetic diseases. Each question is graded as follows: +2 points for each correct answer, 0 points for unanswered questions, −0.2 points for incorrect answers. One open-ended question, to be selected by the student from three proposed topics, aimed at assessing the ability to critically discuss and integrate concepts addressed during the course. The open-ended question is graded up to 3 points. The final evaluation is based on the overall score obtained in the written examination.

Gli studenti e le studentesse con disabilita o con Disturbi Specifici di Apprendimento (DSA), oltre alla segnalazione tramite procedura informatizzata, sono invitati a comunicare anche direttamente al/la docente titolare dell'insegnamento, con un preavviso non inferiore ad una settimana dall'avvio della sessione d'esame, gli strumenti compensativi concordati con l'Unita Special Needs, al fine di permettere al/la docente la declinazione piu idonea in riferimento alla specifica tipologia di esame.