P13M1 Exploring the fundamentals of Advanced Therapy Medicinal Products (ATMPs): Specificities, Challenges, and Applications

This activity introduces the three main categories of Advanced Therapy Medicinal Products (ATMPs): gene therapy, cell therapy, and tissue engineering. Learners explore the scientific principles, mechanisms of action, in vivo/ex vivo approaches, and key challenges associated with each type. The goal is to build foundational knowledge and understand how these innovative treatments apply to rare or serious diseases.

This activity explores the regulatory framework applicable to Advanced Therapy Medicinal Products (ATMPs) in Europe and internationally, highlighting the specific requirements related to their innovative nature, including Good Manufacturing Practices (GMP), regulations on Genetically Modified Organisms (GMOs), and the role of competent authorities such as the European Medicines Agency (EMA) or the Food and Drug Administration (FDA). This activity emphasizes the importance of regulatory compliance in the development and commercialization of these treatments.

This activity covers the key steps in ATMP development, from fundamental research to clinical trials. It addresses risk-based approaches, the CMC (Chemistry, Manufacturing and Controls) strategy, and preclinical phases (toxicology, biodistribution, efficacy). The goal is to understand the complex pathway from lab to market authorization.

This activity draws on concrete examples of marketed Advanced Therapy Medicinal Products (ATMPs) to introduce key principles of industrial production, including process types by therapeutic modality, critical manufacturing steps, and technical constraints (storage, vectors, cells). It also addresses technological maturity and the challenges of scale-up, grounding the learning in real-world industrial contexts.