Silence Therapeutics is a leading RNA technology company.
We have developed proprietary modifications to improve the robustness of RNA sequences, together with advanced liposomal chemistries to enhance the delivery of therapeutics. Our technology can selectively silence or replace the expression of any gene in the genome, modulating expression up as well as down in a variety of organs and cell types, in vivo.
This allows the development of therapeutics for diseases with high unmet clinical need. Silence’s technology is currently in the clinic in a Phase 2a pancreatic cancer trial.
RNA (ribonucleic acid) is one of the two types of nucleic acids found in all cells – the other being DNA (deoxyribonucleic acid). RNA is the messenger that takes a copy of the genetic information stored in DNA in a cell’s nucleus and translates it into instructions for the manufacture of proteins in that cell.
RNA interference (RNAi) is a method of regulating or ‘silencing’ gene expression. It is a naturally occurring phenomenon which can be used to selectively supress the genes which cause some diseases. Genes are the blueprints for proteins, the building blocks of life, and this technology can selectively silence potentially any gene in the genome, preventing the overexpression of that gene and reducing the production of disease-causing proteins.
The ability to specifically target these proteins could open the door to types of therapeutics that current treatments such as small molecules and antibodies cannot address.
Our patented RNAi platform is known as AtuRNAi®. It is based on specific, proprietary short interfering RNA (siRNA) modules, stabilisation technology and exclusive tailored features.
siRNA requires delivery systems in order to enable it to enter into target cells within the body. Silence has its own delivery systems focused on a variety of organs:
- AtuPLEX® targets the endothelial cells of the vascular system
- DACC targets the endothelial cells of the lung
- HepaPLEX targets the hepatocyte cells of the liver and the liver’s vascular system
We use our technology in our own pre-clinical research and clinical trial programmes, resulting in a promising pipeline. We also license our technology to other drug development and pharmaceutical companies.
Rather than decreasing gene function, mRNA delivery is an exciting new method of introducing messenger RNA activity into the cell and stimulating it to produce a target protein, in conditions where this is of therapeutic interest, including genetic deficiencies. This way improved gene expression is achieved without having to make permanent changes to the genome, thus avoiding safety concerns.
We have already demonstrated our ability to deliver functional mRNA in vivo and are actively exploring new opportunities in this space.