Engineering and preliminary safety assessment of an RNA trans-splicing therapeutic vector for liver cancer.

Spliceor Ltd is an early-stage University of Cambridge spin-out company developing novel gene therapies for certain types of aggressive cancers for which current treatments are ineffective. Spliceor targets molecules found exclusively in cancer cells using them to generate a lethal gene product within the cancer cell. The Spliceor team has identified a specific class of molecules that provide ideal targets for this approach. Using these, Spliceor aims to generate a platform approach to many types of cancers, beginning with liver and pancreatic cancer, to generate curative and well tolerated gene therapies for cancer patients. The project will consolidate the evidence we have already obtained and give us important pre-clinical study data to guide therapeutic dosing range for subsequence pivotal pre-clinical efficacy studies to help to progress the therapy towards manufacturing, safety testing and clinical trials. It will also help to refine our business plan and serve as a stepping stone to secure future pivotal investment for the company. This funding will allow the company to grow in scale and create new job opportunities, contributing to improving healthcare outcomes and much needed economic growth.