RNAi has the potential to generate a new class of safer therapeutics, which are more specific and effective. These therapeutics would take advantage of the body’s own natural processes to silence genes, or more precisely, eliminate specific gene-products or proteins in the cell. Today, there are a number of RNAi product candidates advancing in clinical development – including those from our Pipeline Products and our Partner Products.
In the cell, DNA carries the genetic information required to make each specific protein. Genes are first copied or transcribed into messenger RNA (mRNA), which is translated into protein. Most diseases are caused by either the absence or over-production of a specific protein. For example, a tumor can be caused by the over-production of a protein that stimulates cell growth. If too much of a particular protein is the cause of a disease, the therapeutic approach would be to reduce or silence its activity or amount.
RNAi products can silence or eliminate the production of disease-causing proteins, creating opportunities for therapeutic interventions that are not possible with conventional drugs. To realize the tremendous therapeutic potential of RNAi-based drugs, effective delivery is critical. Successful delivery protects RNAi-based drugs in the bloodstream and facilitates cellular uptake and release into the target cell.
One method to target disease-causing proteins involves developing and using small interfering RNA (siRNA). siRNA are synthetic RNA molecules developed to suppress the production of proteins through the RNAi mechanism. Sequencing of the human genome has provided the information needed to design siRNA therapeutics to target a wide range of disease-causing proteins. Based on the mRNA sequence for the target protein, a siRNA therapeutic can be created relatively quickly compared to the time needed to synthesize and screen conventional small molecule drugs.
siRNA-based therapeutics can bind to a target protein mRNA with great specificity. When siRNA are introduced into the cell cytoplasm, they are rapidly incorporated into an RNA-induced silencing complex (RISC) and guided to the target mRNA. The RISC cuts and destroys the target mRNA, preventing production of this protein. The RISC can remain stable inside the cell for weeks, destroying many more copies of the target mRNA and suppressing this specific protein for long periods of time.
This animation, produced by Nature video, explains how RNAi works.
Over the last decade, we have led the field in RNAi delivery, demonstrating continued innovation of our LNP Delivery Platform to enable the advancement of RNAi therapeutics. In addition to being a leader in RNAi delivery technology, we have a diverse pipeline of products in development to treat serious human diseases, such as cancer, viral infections like Hepatitis B and Ebola, among other therapeutic areas.
Read more about our LNP Delivery Platform here.