Using the power of RNA silencing to impact disease.
Mitotherapeutix was created to take advantage of advancements in RNA chemistry and its use in developing siRNA based drugs that can target otherwise difficult to address disease targets. Our initial gene target is a novel protein that controls metabolic activity/efficiency in the mitochondria. The ability to control mitochondrial activity is significant because > 90% of the energy of the cell comes from the mitochondria. This gene target is a negative regulator. Thus, in the presence of the regulator, metabolism is slowed down and the removal of the regulator safely increases metabolism. We have explored the function of this metabolic regulator in different tissues and disease states and have found that turning off the regulator can have a positive effect in disease treatment. Targeting this gene product creates the opportunity of producing a drug that can treat a wide array of diseases.