Ateneo - UPO

The course provides a comprehensive overview of the regulatory aspects of Advanced Therapy Medicinal Products (ATMPs) and the scientific foundations of gene therapy, somatic cell therapy, and tissue engineering. Within the context of tissue engineering, special attention is given to the various types of scaffolds used, the biomaterials they are composed of, and the technologies employed for their production. Additionally, the course delves into the formulation and technological aspects of ATMPs, as well as industrial production in accordance with Good Manufacturing Practice (GMP) guidelines, including examples of production processes for gene therapy, somatic cell therapy, and tissue engineering. Finally, an overview of the entire drug development process for ATMPs is provided, spanning from production to clinical trials and through to marketing authorization.

• Daan J. A. Crommelin, Robert D. Sindelar, Bernd Meibohm. Pharmaceutical biotechnology. Ed. Springer Nature; 5° edizione
• Material distributed during the lessons

Throughout the course, students will gain a strong understanding of the theoretical principles related to the design, formulation, industrial production in compliance with GMP guidelines, and control of ATMPs.

Fundamentals of pharmacology and pharmaceutical technology.

The lessons are supported by multimedia tools for the exposition of the didactic materials. As a method of innovative teaching, the use of Kahoot is being experimented in the classroom. Kahoot is an app that allows students to participate in a fun competition by answering 10-40 multiple-choice questions within a predetermined time, at the end of each topic. The use of Kahoot enables students to consolidate their understanding of the explained concepts, especially the more complex ones, grasp the relationships among various concepts, and comprehend the connections between different parts of the study material. For the teacher, the use of Kahoot stimulates active student interaction, facilitates the assessment of learning progress over time, and helps identify any topics that require review or further integration during the lessons.

It is not allowed to record, video-record and photograph the lessons.
Students with physical disabilities, Learning Disabilities or Special Education Needs can request specific services and tools via the Staff Sviluppo e Coordinamento Carriere e Servizi alle Studentesse e agli Studenti, consulting the University webpage: https://www.uniupo.it/en/services/services-students-physical-or-learning-disabilities
Students with disabilities, learning disabilities or special education needs, once they have contacted the University Staff, can refer to the tutor in charge of the course to define the examination modalities, concerning academic aspects.

The written exam consists of of short-answer open-ended questions intended to test the knowledge and ability to apply in an independent way the knowledge acquired. Moreover, the student’s communication skills are evaluated, particularly the use of specific language for the pharmaceutical and technological discipline. The exam takes place during the official dates, and the grade is expressed in thirtieths.

• Introductory concepts related to Advanced Therapy Medicinal Products (ATMPs).
• Regulatory framework of ATMPs.
• Gene therapy: viral and non-viral vectors, integrating and non-integrating vectors. In vivo and ex vivo gene therapy.
• Somatic cell therapy: autologous, allogeneic, and xenogeneic cells and tissues. Embryonic stem cells, mesenchymal stem cells, and induced pluripotent stem cells. Differentiated cells.
• Tissue engineering: repair and regeneration. Regenerative medicine. Biomaterials for regenerative medicine and tissue engineering. Biocompatibility and biodegradation. Types of scaffolds and technologies for their production.
• Production, formulation, and quality control for ATMPs. Examples of production processes for gene therapy, somatic cell therapy, and tissue engineering products.
• The drug development process for ATMPs: requirements for manufacturing, clinical trials, and marketing authorization of ATMPs.

By the end of the course, students will have acquired a thorough understanding of the regulation related to ATMPs. They will possess knowledge of the biological scientific foundations and the production process of gene therapy, somatic cell therapy, and tissue engineering medicinal products. Additionally, they will be able to identify the major challenges encountered during the development of ATMPs and will have developed the necessary skills to address them effectively.