The potential uses of gene silencing

Home / Patients / Gene Silencing Explained / The potential uses of gene silencing

What are the potential applications for gene ‘silencing’ therapy?

Silence Therapeutics is working to make the best use of RNAi technology for new medicines for a wide variety of diseases caused by genetic factors. We are currently focusing on three key areas of unmet need: cardiovascular (heart and blood vessel) disease, hematological disorders (a type of blood condition) and complement-mediated disorders (the complement system is part of the immune system).

Silence Therapeutics has already developed a delivery system targeting the liver and is aiming to explore delivery platforms for other areas of the body including the lung, central nervous system (brain and spinal cord), skin, kidneys, muscles, heart and gastrointestinal tract (digestive system, which includes the stomach and intestines).

Have gene silencing therapies been approved yet?

Gene silencing therapies have already been improved for the treatment of high cholesterol and some rare diseases, including hereditary transthyretin amyloidosis (hATTR) and acute hepatic porphyria (AHP). Early stage research using gene silencing therapies is being carried out in other rare diseases, including Huntington’s disease and porphyria. The concept is also being explored in other common diseases, such as cancer and dementia, which have a genetic link.

However, existing siRNA programs have only scratched the surface of the liver target space. Although all of the different cells in our bodies contain the same DNA, they express different sets of genes that determine the set of proteins and functional RNA they produce. This in turn gives cells their unique properties. Currently, it is estimated that only 1% of the genes expressed in the liver have been targeted by known siRNAs. This creates an immense opportunity to identify new drugs that target the remaining 13,000+ liver-expressed genes.

< Our approach to gene silencing at Silence Therapeutics