A novel delivery system for nucleic acids

Pharmaceutical and biotechnology companies are spending huge sums to develop novel nucleic acid-based therapies for cancer and other hard-to-treat diseases using engineered siRNA/DNA/RNA molecules. A key challenge for these nucleic acid-based therapies is that they need to be successfully delivered intracellularly, ideally to specific cells, with sufficient drug payload to be effective.

To date, companies developing nucleic acid therapies have predominantly used existing commercially available delivery systems such as lipid nanoparticles and viral vectors, but these suffer from several limitations. It is very challenging to load more than one nucleic acid in a controllable fashion and very costly to manufacture at scale. They can also induce the release of systemic inflammatory cytokines meaning the patient will feel unwell and may cause liver toxicity. Targeting lipid nanoparticles or viral vectors to specific cells is also difficult to achieve in practice.

N4 Pharma’s breakthrough silica nanoparticle delivery system Nuvec^® is based on fused silica and has a unique irregular surface structure that simply and effectively traps and protects the siRNA/RNA/DNA as it travels to the cells. The Nuvec^® particles are naturally taken into cells and once inside, the nucleic acid payload is released to deliver a localised response. 

Nuvec^® is safe and well tolerated and can be delivered intravenously, subcutaneously or intramuscularly. Loading with more than one nucleic acid sequence, to produce combination therapies targeting multiple pathways in the cell, is straightforward. Targeting Nuvec^® particles to specific cells has been demonstrated and, perhaps most importantly, Nuvec^® protects the payload from enzymatic degradation and the effects of pH, so that a Nuvec^® therapy can be delivered orally which opens up a range of previously difficult to drug targets in cancer and other indications and potential to avoid costly repeated injections.

With siRNA, Nuvec^® can produce a strong dose response curve showing significant cellular transfection from very low doses, meaning the amount of expensive siRNA needed to be effective can be significantly reduced.

Nuvec^® is available for licensing to third parties to develop their own gene therapies. For more information, please contact us here.