MicroRNAs contain only 22 letters of genetic code. They regulate production of individual proteins by binding to control points in genetic code readouts called messenger RNAs, thereby raising or lowering the levels of individual proteins that control cell growth or death. Too much or too little can push cancer cell growth, kill heart cells, inflame joint cells, or kill brain cells. Unlike conventional drugs, which only target one or two genes or pathways, microRNA blockers simultaneously modulate multiple disease gene pathways.
MicroRNA imbalances drive many forms of cancer. So far, there are hundreds of well-validated microRNAs active in cancer, heart disease, arthritis, and brain disease, giving us enormous opportunities for therapeutic use.
There have been many attempts since 2000 to block individual microRNAs in order to inhibit cancer. There are no FDA approved microRNA blockers. However, there are several FDA approved messenger RNA-targeted blockers using the same fundamental design, a string of letters of genetic code that turn off a single disease gene.
Our technology promises greater potency, and greater gene specificity, because our design platform yields shorter microRNA blockers than standard approaches.
The design platform uses a next generation RNA analog with drug-like properties that efficiently destroys target cancer-related microRNAs at a low dose, with negligible toxicity for normal cells, negligible immune reactions, and negligible gene disruption.
In addition, our drug delivery method concentrates microRNA blockers in diseased cells in any part of the body, minimally affecting normal cells. Conventional delivery methods for RNA analogs use large toxic synthetic polymers that inevitably concentrate in the liver and kidney.