Gene therapy for human cancers caused by fusion genes

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Research

Cancer is the second leading cause of death worldwide. Available treatments are invasive and have severe side effects. In many cases, such as for some types of leukaemia, colon and prostate cancer, the origin of the tumour lies in so-called ‘fusion genes’. Formed by two previously separated genes, these hybrid genes can sometimes acquire high oncogenic potential. The recent development of the CRISPR/Cas9 gene-editing technology, which allows DNA to be manipulated with precision, offers unparalleled potential to develop new treatments in this area.

Aim

To develop a gene-editing treatment technology to fight all types of cancer caused by oncogenic fusion genes.

Problem to Solve

In 2017, cancer was the cause of death for almost 1.3 million people in the EU. Cancerous cells evolve rapidly and are heterogeneous and difficult to target. Around 20% of all cancers are driven by fusion genes, and those tumours are commonly treated by using a combination of chemotherapy, radiotherapy, surgery and, more recently, targeted and immune therapies. However, all these treatments have mixed outcomes and can result in severe side effects. Moreover, the financial costs of cancer are high for both the patient and for society as a whole.

Innovation

FuGe Therapeutics technology is based on an innovative CRISPR/Cas9 gene editing approach to target cancers caused by fusion genes. The gene-editing approach has shown antitumor properties in sarcoma and leukaemia in both in vitro and mouse models.