RXi Pharmaceuticals is a discovery-stage biopharmaceutical company engaged in the discovery, development and commercialization of proprietary therapeutics based on RNA interference (RNAi) for the treatment of human diseases.  RNAi is a naturally occurring mechanism for the regulation of gene expression that has the potential to be harnessed to selectively inhibit the activity of any human gene. It is believed that this inhibition may potentially treat human diseases by “turning-off” genes that lead to disease.

RXi’s founders include world-renown researchers in the RNAi field, including Dr. Craig Mello, recipient of the 2006 Nobel Prize for Medicine for his co-discovery of RNAi, and Blais University Chair of Molecular Medicine at the University of Massachusetts Medical School.   The management team has extensive experience in the field of RNA interference and in moving RNA/DNA based drugs into the clinic.

 

The rxRNA™ compounds used by RXi are distinct from the traditional small interfering RNA (siRNA) compound used by other companies developing RNAi therapeutics.  Based on our internal research, rxRNA compounds are highly active and potent and have increased nuclease stability, show reduced toxicity levels and are readily manufactured.

 

Many genes associated with disease have been identified but have been difficult to target with conventional medicinal chemistry.  RXi believes that RNAi technology may play an important role in targeting these genes and potentially treating diseases.  With our pioneering work developing the RXi technology platform, the company believes that it will discover many drug candidates than can be developed as therapeutics.  To move as many of these therapeutic candidates forward into clinical trials as possible, RXi’s is seeking to work with partners in the discovery and development process.

 

Distributing RNAi compounds to the right tissues of the body is essential for RNAi therapeutics to be effective.  RXi is using various technologies, including glucan encapsulated RNAi particles (GeRPs) and nanotransporters to deliver its rxRNA™ compounds to the appropriate tissues and cells.

 

RXi intends to use its expertise in the field and its proprietary RNAi technology platform to identify lead compounds and advance towards pre-clinical and clinical development programs in two main therapeutic areas: inflammatory and metabolic diseases. 

 

Inflammatory disease:

Inflammation is a key component in the initiation and progression of many diseases including rheumatoid arthritis, Crohn’s disease, atherosclerosis, inflammatory bowel disease, diabetes and others.  As such, inflammatory disease represents an area of great potential opportunity for RXi.  The company is using a novel system that would allow for oral administration of our rxRNA™ compounds and delivery to certain types of inflammatory cells called macrophages.  Oral administration provides a much less invasive and more convenient alternative than injection, the current standard of care for patients with these diseases, and so should result in improved compliance and potentially greater market acceptance.  By specifically delivering the drug to macrophages, we aim to target the cells and genes involved in the pathology of inflammation.

Metabolic disease: 

Within metabolic disease, RXi has two primary areas of focus. First, the company is targeting a gene thought to be responsible for elevated cholesterol. Second, RXi has in-licensed intellectual property developed by RXi co-founder Dr. Michael Czech, Professor and Chair of Molecular Medicine and Professor of Biochemistry and Molecular Pharmacology at the University of Massachusetts Medical School (UMMS) covering genes that appear to be important regulators of metabolism. Studies conducted in Dr. Czech’s laboratory at UMMS and by others at Imperial College of London have demonstrated that inactivation of one of these genes, called RIP140, can cause fat cells to metabolize rather than store fat.  Mice in these studies that did not express RIP140 remained lean and non-diabetic even when maintained on a high-fat diet.  RXi is currently designing rxRNA™ compounds targeting RIP140 as a potential treatment for obesity and obesity-related type 2 diabetes.