RXi Pharmaceuticals is a discovery-stage biopharmaceutical company pursuing the 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-leading researchers in the RNAi field, such as 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.  RXi intends to use its expertise in RNAi and the RNAi technology platform it has licensed from leading researchers to identify lead compounds and advance towards pre-clinical and clinical development programs in four therapeutic areas: neurology, metabolic disease oncology and additional diseases as indicated.

Neurology:

Initially, RXi is pursuing research in ALS (Amyotrophic Lateral Sclerosis, commonly known as Lou Gehrig's Disease).  Some forms of ALS are caused by defects in a gene called SOD1.  Early pre-clinical studies conducted by our advisors, Dr. Tariq Rana and Dr. Zuoshang Xu at the University of Massachusetts Medical School, showed promising results in animals using an RNAi compound to selectively inhibit the SOD1 gene.  RXi is refining and extending this work and, if successful, will move into formal pre-clinical development.  RXi also intends to leverage its experience related to the delivery of RNAi therapeutics in the central nervous system to explore development of RNAi-based treatments for neurodegenerative diseases other than ALS, including Alzheimer's Disease.

Metabolic disease: 

RXi co-founder and Scientific Advisory Board member Dr. Michael Czech is a leading metabolic disease researcher. RXi has in-licensed intellectual property developed by Dr. Czech on 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 RNAi compounds targeting RIP140 as a potential treatment for obesity and obesity-related type 2 diabetes.  RXi also continues to evaluate genes in Dr. Czech’s database for candidate targets.

Oncology:

RXi is initiating a program to develop RNAi compounds for use in oncology.  This strategy is led by key RXi scientific advisors, Dr. Greg Hannon and Dr. Nicholas Dean, both of whom are leading researchers in targeting oncogene pathways.  Additionally, RXi’s management team has expertise in developing programs targeting genes involved in cancer.  Dr. Pamela Pavco, Vice President for Pharmaceutical Development, previously managed the pre-clinical programs targeting genes involved with cancer while at Sirna Therapeutics, Inc. (recently acquired by Merck & Co.).

Additional Indications:

There are many well-studied genes associated with numerous diseases that have been identified but have been difficult to target with normal medicinal chemistry. RXi believes that RNAi technology may play an important role in targeting these genes and potentially treating these diseases.

With the pioneering work being done in developing the RXi technology platform, RXi believes that it will discover many more drug candidates than can be advanced into clinical trials.  RXi will seek to identify and work with partners in the discovery and development process to build the RXi development pipeline.

Download company overview fact sheet (pdf)