Ark said that the discovery, which was made by its scientists in Finland, represents a major advance on existing RNA silencing technology and has the potential for use in the treatment of a variety of human diseases involving defective genes, either where the genes do not work or are overactive.
According to Ark, its new small hairpin RNA technology is unique in that it appears to be actively taken to the nucleus of the cell where it acts on the promoter of the target gene via an epigenetic mechanism. After processing in the nucleus, it can both down-regulate (silence) or up-regulate (increase) gene expression within the cell (endogenous gene expression).
Ark added that its new technology can also perform multiple gene up- or down-regulation with the same vector construct. Importantly, it can be delivered using Ark’s existing and established viral vector technologies according to the length of time an effect is needed.
Nigel Parker, CEO of Ark, said: “This is extremely exciting technology which in many respects complements our previous discovery of targeted gene insertion two years ago and which we believe has the potential for broad utilization in the treatment of disease.
“We now have a full toolkit which provides the flexibility to both selectively insert beneficial genes or correct defective genes, enabling us to develop a wide range of DNA-based medical treatments, for example to switch off cell proliferation or migration in the treatment of cancer and vascular disease.”