Synpromics, a leading synthetic biology company, will use its unique approach to develop synthetic promoters with upregulated, liver cell specific, activity whilst also decreasing the promoter molecular size to optimize the packaging efficiency of Adeno-Associated Virus (AAV).
AAV is a small virus which infects human cells but does not cause disease, which turns it into a perfect vector for gene therapy uses. AAV-based vectors have been found to affect long-term therapeutic protein expression in humans, which can result in long-lasting treatment for patients.
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.