Synpromics will use its unique approach to develop synthetic promoters with up- regulated, liver cell specific, activity whilst also decreasing the promoter molecular size to optimize the packaging efficiency of AAV.
The work is also supported by Scottish Enterprise through a grant awarded from its SMART: SCOTLAND scheme, which provides financial support to Small to Medium Enterprises across Scotland.
Dr David Venables, CEO of Synpromics, commented, “Synpromics are very pleased to undertake this collaboration with uniQure, and have a further opportunity to demonstrate the utility of our synthetic promoter technology to gene therapy applications. We have now advanced several projects in different areas and this collaboration highlights that our technology is being increasingly validated.”
About Gene Therapy And Adeno-Associated Virus (AAV)
- Gene therapy is the introduction of genetic material into a patient’s cells to replace missing genes, compensate for abnormal genes or make a beneficial protein. Genes are typically delivered into the cell using a carrier known as a vector.
- Viruses are often used as vectors because they can effectively deliver a new gene by infecting a cell. Adeno-Associated Virus (AAV) is a small virus which infects human cells but does not cause disease.
- The ability of AAV to infect both dividing and non-dividing cells, together with its inability to replicate on its own and that it is unlikely to spread within the body, make it suitable for gene therapy applications.
- AAV-based vectors have been found to affect long-term therapeutic protein expression in humans which can result in long-lasting treatment for patients.