RNAi has the potential to generate a new class of safer therapeutics, which are more specific and effective. These therapeutics take advantage of the body’s own natural processes to silence genes, or more precisely, eliminate specific gene-products or proteins in the cell. The development of RNAi drugs allows for a completely novel approach to treating disease, which is why RNAi is considered one of the most promising and rapidly advancing frontiers in drug discovery. Today, there are a number of RNAi product candidates advancing in clinical development – including those from our Oncology, Anti-viral and Metabolic Programs and our Partnered Programs.
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 RNAi therapeutic approach would be to reduce or silence its activity or amount.
In order for RNAi drugs to be effective, they must be delivered and released to the appropriate target cells. Tekmira has developed a proprietary delivery platform called Lipid Nanoparticle or LNP, which has become the gold-standard in RNAi development, further establishing Tekmira’s role as a leader in this new area of innovative medicine.
One method to target disease-causing proteins involves using synthetic RNAi trigger molecules. These molecules are developed as drugs that specifically suppress the production of disease-associated proteins through the RNAi mechanism. RNAi trigger molecules are designed using the gene sequence coding for the target protein. RNAi drugs offer the potential for safer and more effective treatments because of their specificity. Additionally, based on the mRNA sequence for the target protein, RNAi trigger molecules can be created relatively quickly compared to the time needed to synthesize and screen conventional small molecule drugs.
By encapsulating the RNAi trigger molecules in lipid particles, Tekmira’s proprietary LNP technology enables efficient delivery and uptake into the target cells, preventing the production of the disease-associated protein.
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. Tekmira’s expertise in this area has resulted in the development of an exciting pipeline of RNAi therapeutics across a range of indications for serious conditions with limited treatment options.
Read more about our LNP Delivery Platform here.