Cell, Gene and RNA Therapy

Course goals

After this course, students should have a good overview of the current GCT landscape, the role these innovative therapies currently play in clinical practice and may play in the future, and the role of the pharmacist in the GCT product lifecycle. More specifically, this course addresses the following learning objectives:

• To differentiate cell therapies, gene therapies, tissue engineered products and RNA-based therapies.
• To evaluate the role each of these therapies can play in treating diseases.
• To evaluate the risks that are associated with each of these therapies.
• To weigh these benefits and risks, also taking into account the ethical and patient perspectives.
• To contrast the different phases of development GCTs have to go through and the important gatekeepers in these phases.
• To formulate and defend a standpoint about patient access to GCTs.

Content

Gene and cell therapies (GCT), in Europe also grouped as “advanced therapy medicinal products,” are currently among the most innovative medicines that are being developed. GCTs consist of living human cells and/or genetic constructs that can be targeted to specific defects in the human body in a highly personalised manner. Hereby, they can for example provide new effective treatment options for various types of cancer and previously poorly or untreatable diseases – some of which now even seem curable.

The aim of the course FA-MA224 Gene and Cell Therapies is to get acquainted with GCTs and the different stages of their development and use, also known as their “product lifecycle.” After an initial overview of their current applications in clinical practice and the role of the pharmacist (week 1), the course explores the biological and pharmacological fundamentals of GCTs (week 2), their manufacturing and quality assurance (week 3), the first-in-human study of GCTs along with ethical considerations thereof (week 4), and finally marketing authorisation and patient access (week 5). Throughout the course, several typical CGTs will be revisited to explain these aspects.

Throughout the course, lectures will be given by various experts from the GCT field, varying from (hospital) pharmacists and physicians to assessors involved in the assessment of first-in-human studies, marketing authorisation and reimbursement. These lectures will be supported by workshops (“werkcolleges”) in week 1 and 2 and flanked by weekly group assignments in week 3, 4 and 5.