A Cambridge University spin-out company called Spliceor is engineering a harmless virus to deliver a lethal genetic payload exclusively into liver cancer cells, leaving healthy cells untouched. Current treatments for aggressive liver cancers often fail because the tumours mutate and become resistant to chemotherapy and other drugs. Spliceor’s approach targets molecules found only on cancer cells, using them as a trigger to activate a cell-killing gene inside the tumour. The virus used to deliver this therapy—an adeno-associated virus (AAV)—can infect both dividing and dormant cancer cells without disrupting the host cell’s DNA, and has already shown promise in human clinical trials for eye disease. If this pre-clinical work succeeds, it could lead to a new treatment for liver cancer that is both more effective and less toxic than existing options. The same platform technology could then be adapted to target other cancers with few treatment options. Beyond patient outcomes, the project is expected to help the company grow, create jobs in the UK, and attract further investment to move the therapy toward manufacturing and clinical trials.
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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.
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