Accelerating regenerative cell therapies with data-driven cell conversions.

Our understanding of biology and advancements in technology are rapidly changing the way that we both diagnose and treat disease. One area where this change is being experienced is in the area of cell therapy, an area of medicine which involves treating disease by replacing or regenerating patient tissues in order to overcome disease. These tailor-made therapies have the potential to completely change the way that we treat disease since their aim is not is treat the symptoms of a disease but to restore the diseased tissue to its original state. One of the main bottlenecks in cell therapies reaching the clinic is the development of efficient, safe and scalable sources of cells. At present the cells either need to extracted and sorted from the patient or a donor or they are derived via experimental protocols that take years to develop. Cell Mogrify is revolutionising this process through the introduction of its data-driven approach to finding ways to generate any human cell type, allowing us to vastly reduce the development time. Our approach utilises cutting-edge bioinformatic tools that we have developed along with world-class experimental expertise in order to use information extracted from patients cells to predict how to generate that cell outside the body. Typically these cell conversions are performed by artificially over-expressing transcription factors, the biological master regulators of the cell, in order to create cells outside of the body that can later be transplanted to the diseased area of a patient. However in certain circumstances having to perform this reprogramming outside of the body or using transcription factors can be a regulatory or operational roadblock. To this end we have also developed ways to replace these transcription factors with small molecules, allowing us to safely convert between cell types either externally or directly at the disease site. To date Cell Mogrify have utilised the technology to bring one cell therapy to an advanced preclinical stage. This proposal will allow us to demonstrate that this approach is however general for any cell therapy and as such that it is a platform technology for the cell therapy market. In order to do this we will take three further products, at various stages of development, to a preclinic state. In doing so we will both create three new cell therapy products as well as providing a proof of principle for the platform.