MDRNA Reports Positive In Vivo Data on its Proprietary UsiRNA Technology
News Aug 07, 2009
MDRNA, Inc. has announced positive data on its proprietary UsiRNA technology, demonstrating that its UsiRNAs are highly potent against metabolic disease and cancer targets in rodent models. Further, the data establish that the knockdown of these targets is achieved via an RNAi-mediated process.
Of particular importance is that the data demonstrate that strategic placement of UNA (unlocked nucleobase analogs) results in the ability to manipulate, either increasing or decreasing, strand-specific activity thus minimizing off-target activity and providing the ability to impart improved drug-like characteristics to the UsiRNAs.
The data are being presented by Michael V. Templin, Ph.D., Vice President, Discovery Research and Pharmaceutical Development of MDRNA, at the IBC Drug Discovery & Development Week, Oligonucleotide Therapeutics: From Concept to Implementation, in Boston, Massachusetts.
"UNA-modified siRNAs are novel and proprietary constructs that have high potency in target mRNA reduction. In addition, we have recently determined that strategic placement of UNA in the siRNA reduces micro-RNA-like off-target activity. This observation is distinct from, and in addition to, our previous work in which we have demonstrated that UNA residues have a dramatic effect on reduction of cytokine induction by siRNA," stated Barry Polisky, Ph.D., Chief Scientific Officer of MDRNA.
"We are encouraged by these significant results that demonstrate the versatility of UNA and UsiRNAs for improving the safety and specificity of RNAi, and reinforce our confidence in our UsiRNA platform."
"Repeated positive results from multiple in vivo studies in rodents using our UsiRNAs and DiLA2 delivery platform has led us to the initiation of preclinical safety and efficacy studies in non-human primates," stated J. Michael French, President and Chief Executive Officer. "We are one of a handful of companies to have initiated non-human primate studies with a liposomal-based delivery system and we look forward to reporting positive data this quarter."