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In order to understand the workings of the pre-clinical and clinical developmental stages of cell and gene therapies, it is necessary to understand the process by which all therapeutics are characterised during their developmental procedure. This module aims to impart this knowledge into students by covering the key concepts of pharmacokinetics and pharmacodynamics and demonstrating how information around these topics is gathered in order to instruct on the efficacy, toxicity and dosing of a new therapeutic agent. The pharmacokinetics teaching will address the mechanisms governing absorption, distribution, metabolism and excretion (ADME) of small molecule drugs and contrast how ADME applies to novel cell and gene therapies.

The pharmacodynamics teaching will illustrate how efficacy and toxicity is characterised pre-clinically using in vitro and in vivo disease models and introduce students to the type of data produced at this stage of development. Finally, the module will cover the clinical stage of development by explaining the clinical trial design process and the routes, approaches and strategies required for clinical trial creation and management (with a link to the regulatory demands made during the development of a novel cell or gene therapy).

On completion of this module, students will be able to:

1. Demonstrate the difference between pharmacokinetic and pharmacodynamic principles and illustrate how these principles link to efficacy, toxicity and dosing
2. Evaluate the key purposes/goals and associated design aspects to both pre-clinical and clinical studies
3. Evaluate the importance of disease modelling in therapeutic development and demonstrate how disease models are created.
4. Analyse pre-clinical and clinical efficacy and toxicity data
5. Infer how information from pre-clinical and clinical studies will impact on commercialisation prospects.