RXi's RNAi eye cancer drug gets government grant infusion

RXi Pharmaceuticals will use a new government grant to pursue preclinical work on an RNAi-related treatment for a nasty form of eye cancer. The news underscores the fact that research in the field is advancing, even as progress toward development of new RNAi treatments continues to be frustrating and sluggish.

The Westborough, MA, company said it won a new $300,000 grant to work with researchers at the University of Massachusetts and Memorial Sloan-Kettering Cancer Center on the discovery and preclinical development of sd-rxRNAs to treat retinoblastoma. The Small Business Innovation Research grant comes through the National Institute of Health's National Cancer Institute.

Their idea behind the technology builds on previous RXi and UMass research suggesting that self-delivering sd-rxRNAs will penetrate the retina and regulate gene expression inside. In theory, they hope that same process will enable gene expression regulation within intraocular retinoblastoma cells. Retinoblastoma is a retina cancer (back of the eye). While it is rare, it is the most common eye cancer to occur in children, according to cancer.org.

RNAi drugs are designed to turn off bad genes that cause disease, but researchers and companies have struggled to develop one that can survive through clinical trials and reach the marketplace.

RXi launched based on the research work of Craig Mello, a Nobel Prize winner and member of the company's scientific advisory board. While the company has one drug--RXI-109--in a Phase I clinical trial as a treatment for scar prevention, it hasn't been immune to RNAi setbacks. About a year ago, the company split in two in the face of slow RNAi research progress, launching Galena Biopharma ($GALE) to focus on experimental cancer vaccines and keeping the RNAi technology under the RXi name.

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