RXi Pharmaceuticals (RXII) is a biopharmaceutical company working towards the development and commercialization of proprietary therapies based upon RNA interference (RNAi). RNAi is considered to be a significant advancement in the field. In 2002, the journal Science selected RNAi as the “Breakthrough of the Year”. The discovery team, which included Dr. Craig Mello, one of the founders of RXi, was awarded the 2006 Nobel Prize in Medicine.
It is believed that RNAi, which is a naturally occurring mechanism for the regulation of gene expression, can be used to selectively inhibit the activity of any human gene. RXi hopes to harness the ability to inhibit or “turn off” targeted genes that lead to the development of specific diseases. Founded by leading researchers in the RNAi field, the company hopes to identify leading compounds and move towards pre-clinical and clinical development programs in neurology, metabolic disease, oncology, and a number of other fields.
A major building block was discovered by the neurology studies, which found that some forms of Lou Gehrig’s disease are caused by defects in a gene called SOD1. Early pre-clinical studies have shown promising results using an RNAi compound to selectively inhibit the SOD1 gene. The company is refining the work and, if positive results occur, will enter into formal pre-clinical development. RXi will perform lateral work to deliver RNAi therapies to the central nervous system to explore applicable treatments for neurodegenerative diseases, like Alzheimer’s disease.
In the metabolic disease group, RXi is using in-licensed intellectual property developed by co-founder Dr. Michael Czech on genes that may be important regulators of metabolism. Studies have shown that the inactivation of one of the genes, named RIP140, can cause fat cells to metabolize instead of store fat. Mice used in the studies that did not express the RIP140 stayed lead and non-diabetic, even when given a high-fat diet. RXi is developing RNAi compounds targeting the RIP140 gene for potential application to those who are obese and to those with obesity-related type-2 diabetes.
This vastly new approach of modifying targeted genes is certainly outside the traditional path to curing diseases, but RXi continues to move forward with its research to unlock the key to “turning off” the effects bad genes.
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