Rxi secures SBIR grants to develop novel RNAi therapeutics for ALS

RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biotechnology company focused on discovering, developing and commercializing innovative therapies addressing major unmet medical needs using RNA-targeted and immunotherapy technologies, today announced that it was awarded two Small Business Innovation Research (SBIR) grants from the National Institutes of Health (NIH).

ALS Research Support. The National Institute of Neurological Disorders and Stroke (NINDS/NIH) will provide funding for an ongoing collaboration between RXi Pharmaceuticals and Robert Brown, MD, DPhil, Chair of the Department of Neurology at the University of Massachusetts Medical School in Worcester that is focused on the preclinical development of novel RNAi therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. The project aims to generate an optimized self-delivering RNAi compound (sd-rxRNA™) for ALS that can be moved into clinical development as well as establish an optimized sd-rxRNA platform for spinal cord delivery. RXi was awarded $304,559 for Phase I of the project and may apply for additional Phase II funding to continue its research in this area.

RNAi Chemical Optimization Project. A grant from the National Institute of General Medical Sciences (NIGMS) will provide funding for a project seeking to improve the delivery of RNAi therapeutics through medicinal chemistry. More specifically, RXi will explore further chemical optimization of its sd-rxRNA compounds to improve in vivo potency and expand bio-distribution for clinical applications. RXi was awarded $273,824 for Phase I of the project and may apply for additional Phase II funding to continue its research in this area.

"We are pleased to have received support for our innovative research programs that could have a significant impact on our progress lead to powerful new approaches to therapy," said Anastasia Khvorova, Ph.D. Chief Scientific Officer of RXi Pharmaceuticals. "The funding of these grants reflects the strength of our scientific team, demonstrated progress, and our determination to successfully meet the delivery challenge of RNAi therapeutics with our unique sd-rxRNA technology."

The projects described are supported by Award Number R43GM096548 from the National Institute of General Medical Sciences and Award Number R43NS074671 from the National Institute of Neurological Disorders And Stroke. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institute Of General Medical Sciences, the National Institute Of Neurological Disorders and Stroke or the National Institutes of Health.