Advanced Cell and Gene Therapies

This module provides a brief introduction to human genetics and the clinical presentation and manifestations of inherited diseases. The module will also illustrate how traditional and current advanced strategies and techniques have been used to identify genes responsible for both common multifactorial and rare inherited diseases. The module will also develop skills for communicating biomedical research to a lay audience.

This module will introduce 'omics' technologies, such as genomics, transcriptomics, proteomics and metabolomics, and how they are applied in disease areas such as cancer, rare inherited diseases and infectious diseases. Students will experience the experimental and bioinformatic pipelines used to evaluate pathogenicity of variants and learn about the use of different methodologies, such as microarrays and RNA sequencing in estimating levels of mRNA, micro RNAs and long non-coding RNAs. Students will also learn about the advantages and limitations of each method, to assess which is the best method for a particular experimental question.

This module will offer a comprehensive overview of the principles of advanced therapies (cell and gene therapies), including introduction to viral and non-viral vectors, vector design, gene editing, gene replacement, gene silencing, vector delivery, stem cell and pluripotency, tissue engineering, ex-vivo gene therapeutics and CAR T cell technology. Gene and cell therapeutics for monogenic rare diseases and other common diseases (e.g. cancer, heart, immune system) will be discussed. Students will be delivered tutorials on cell and gene therapy study design. They will also have a chance to learn about career opportunities in advanced therapeutics.

This module will offer an overview of advanced therapy approaches other than cell and gene therapy. These include small molecule drug development, intermediate molecules (protacs, peptide), antibody therapeutics and their variants, electroceuticals and scaffolding for regenerative medicine. The module will focus on the different stages of preclinical drug discovery and highlight any specific issues relating to toxicology and clinical trials for each approach. The module will also cover IP and commercialisation in and explore the growing fields of novel therapeutic modalities such as protacs and bicyclic peptides, applied to therapeutic areas, from neurology to cancer, and how this complements cell and gene therapy.

This module will offer a comprehensive overview of the translational pathway of advanced therapies (cell and gene therapies), including the regulatory requirement overseeing the clinical development of advanced therapies, Good Laboratory Procedures (GLP) pre-clinical regulatory studies, Good Manufacturing Procedures (GMP) for plasmid, vector and cell manufacturing, quality control and assurance. This module will also give an overview on procedures to secure clinical trial approval (CTA) and Market Authorisation for advanced therapy products. Students will have a chance to learn about career opportunities in regulatory and GMP manufacturing through interaction with industry.

Students will select a research project title from a list of projects relevant to advanced therapies or translational neuroscience (as appropriate). They will research the scientific literature to identify a gap in current knowledge and with assistance from an academic supervisor, formulate a hypothesis and series of specific aims that could be used to address the research problem.

The aim of the module is to provide the opportunity to learn and apply research methodologies to test a specific scientific hypothesis. A list of projects will be made available and students will be asked to select their top choices. Having been assigned a project of their choice, the students will carry out a 20 week research project, culminating in an oral presentation and dissertation concisely written in the style of a scientific paper describing their research. Students will be expected to join in with the Departmental seminars, journal clubs and supervisor meetings, to learn and experience the role of a scientific researcher.