Dr Ivan Wall
Three-hour written examination (70%)
Coursework (workshops, written report and presentations) (30%)
course provides the biochemical engineering student with the necessary
knowledge to understand the translational challenges of moving from
promising scientific discoveries through to advanced therapeutic
products for use in routine clinical practice. A coordinated series of
lectures examines these challenges, from rigorously assessing robustness
of the science that underpins a therapy, to understanding the critical
pathways to clinic and considerations for commercialization of advanced
Following completion of the course, students will have an understanding of:
- Fundamental science underpinning several cell-based therapies
- Pathways required to take a ground-breaking scientific discovery from thelaboratory bench through the clinical, scalable manufacturing and commercialization route into routine clinical practice
- Regulation, clinical trials, intellectual property rights, ethics and reimbursement with respect to advanced therapies
- How to evaluate the potential of advanced medical technology with respect to clinical, bioprocessing and commercial opportunities
- Current technologies and critical decision making with respect to new/disruptive healthcare technologies.
Case studies/practicals: 20h
Students will gain a better understanding of the scientific, clinical and commercial interactions required to translate basic science into a routinely deployed advanced therapy. In particular, the course examines the requirements for cutting edge platform technologies that offer a paradigm shift in medical care and intervention of diseases that are becoming more problematic as the global population ages. The underlying requirement is to produce quality therapies that are safe, clinically effective and cost effective. Throughout the course, all the material is based on real world examples and data.
Major themes: Stem cells, tissue engineering, gene therapy.
Subthemes: Basic science research underpinning candidate cell therapy interventions, research and development process, clinical trials, regulation, bioprocessing, reimbursement, translation cycle, law and ethics.
Research, development and full commercialisation (including post market surveillance). Impact of stage of development (including animal studies, clinical trials), timelines, resources and outcomes. Development cycle and product life. Patents and other IPR approaches.
Impact on bioprocessing unit operations on production of cells for therapy from the perspective of the economics, speed to market, regulation, current Good Manufacturing Practice (cGMP) including current Good Clinical Practice (cGCP). Students will work in small groups (<5), to create reports and presentations focused on clinical trials of advanced therapies for final assessment.