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.
One of our core technologies is RNA interference. 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 reducing or ‘silencing’ gene expression. It is a naturally occurring phenomenon which can be used to selectively turn down 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.
Take a look at our ‘Introduction to RNA interference’ video here.
Another core focus is messenger RNA (mRNA) therapeutics.
Our messenger RNA technology allows us to restore missing gene expression or artificially encourage gene expression where it may be clinically useful. Currently at early stage testing in rodents, this technology opens up a wide range of potential treatments for genetic deficiencies, protein deficiencies, infectious diseases and even cellular reprogramming.