An Adeno-Associated virus (AAV) gene therapy for tumour cell specific treatment of liver cancer

Spliceor is an early-stage Cambridge University spin-out company from the Department of Medicine developing novel gene therapies for aggressive cancers for which current treatments are ineffective, often due to mutational evasion developing towards standard therapies. Spliceor targets molecules found exclusively in cancer cells using them to generate a lethal gene product within the cancer cell itself without harming surrounding non-cancerous cells. The Spliceor team has identified a specific class of molecules that provide ideal targets for this selective approach. By exploiting this overall approach, Spliceor aims to generate a platform technology to many cancers especially those with unmet medical needs. This new application is focused initially on developing a new treatment for liver cancer using adeno-associated viruses (AAVs), small non-pathogenic viruses infectious for human cells. Several features make AAVs attractive as viral vectors for gene therapy, as they can infect both dividing and quiescent cancer cells. They can also persist without integrating into and disrupting the DNA of the host cell. Recent human clinical trials using AAVs for gene therapy in the eye have been encouraging. The new project is a development from preliminary data we have already obtained using other vector systems but will now focus on generating pre-clinical study data using AAVs to deliver a liver cancer therapy. The work will help to progress the therapy towards manufacturing, safety testing and, ultimately, 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 improved healthcare outcomes and much needed economic growth in the United Kingdom (UK). While health outcomes in the UK have improved substantially since the National Health Service was established in 1948, it now lags well behind many other European countries in key health outcomes in areas such as cancer survival rates. This project, if funded, is focused on reversing that trend for liver and many other cancers aiming to improve both survival and quality of life for patients diagnosed with cancer.