21EBTA Engineering Biology for Cell and Gene Therapy Applications

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Cell therapy and gene therapies (CGTs) are interrelated areas of biomedical research and treatment that aim to treat, prevent, and potentially cure diseases. Cell therapy aims to treat diseases by restoring or altering certain sets of cells or by using cells to carry a therapy through the body. Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into a patient's cells. Both cell and gene therapies overlap in that they necessitate the transfer of new genetic material to cells to produce what could be thought of as "living medicines". The most commonly used cell therapy at present is Chimeric Antigen Receptor (CAR) T-cell therapy for treating blood cancers. This involves genetic modification of patient's own T-cells to express a CAR specific for a tumour antigen, following by ex vivo cell expansion and re-infusion back to the patient enabling to the engineered T Cell to identify cancer cells and destroy them. The global cell and gene therapy manufacturing market size was valued at USD 13.1 billion in 2020 and is expected to expand at a compound annual growth rate (CAGR) of 20.3% from 2021 ($17B) to 2028 ($57.4B). Despite their promise, these therapies are limited providing little control over their dosage, timing, or localization and are often prohibitively expensive. These shortcomings can be overcome by using Engineering Biology to create the next generation of cell and gene therapies. We will use our unique automated facilities to develop new engineering biology tools, and solutions for the bottlenecks in the production CGTs and enable new, inexpensive and safe therapies for future clinical applications. The research will be split into four Engineering Biology Work Packages:1. Genetic Devices for Control in CGTs, 2. Delivery Mechanisms, 3. Standardisation of Cell Lines and 4. Responsible Research and Innovation.