Course Outline: European Advanced Cell Therapy Manufacturing & Regulatory

Course 1: Cell and Gene Therapy Manufacturing Methods 

Provides the theoretical basis to understand conditions for ATMP production, with an emphasis on the importance of culture media, equipment, facility design, etc. Basic knowledge of cell identity, sterility, virus safety and detection of replicant competent virus will be discussed among quality control in manufacturing cell and gene therapy products.

Course 2: Quality Assurance

Provides the knowledge of guaranteeing that all the processes are organized and carried out with the objective of ensuring that medicinal products are of the quality required for their intended use (clinical grade). 

Course 3: Product Development Pathways

Helps students to develop their abilities and show a roadmap to bring basic laboratory results to the bedside. It reviews intellectual property and industry right management in advanced therapies adopting a worldwide perspective. Finally, some speculation is provided on next generation ATMPs (iPSC? transdifferentiated cells? targeted mutation replacement?) and future perspectives in advanced therapies will be summarized so that the student may foresee alternatives that might impact their product development strategy in the not-so-distant future.

Course 4: GMP Compliance

Describes Good Manufacturing Practice (GMP) as applied to ATMPs. While the generic GMP regulations is common knowledge that is easily accessible to anyone, we expect this section to explain this to the students in a very clear manner by making use of specific examples. 

Course 5: IMPD Writing

Summarizes the structure of an investigational medicinal product dossier (IMPD). The IMPD is required to start a clinical trial. During this course, it is described as applied to ATMPs using specific examples not only related to this purpose but also to the difficulties usually encountered by independent researchers.

Course 6: Biosafety 

Examines biosafety issues related to donor selection, cell and tissue manipulation as considered specifically under transplant and pharmaceutical legislation. Regulatory requirements for gene therapy products (GMO) and pharmacovigilance are also discussed.