MDRNA’s RNAi-Based Compounds Demonstrate High Potency Against Multiple Targets in Preclinical Studies
News May 25, 2009
MDRNA, Inc. has announced positive in vivo efficacy data showing that its proprietary UsiRNA constructs are highly potent and highly specific against ApoB and Factor VII message.
The data was presented by Michael V. Templin, Ph.D., Vice President, Discovery Research and Pharmaceutical Development of MDRNA, at the TIDES Oligonucleotide and Peptide® Technology and Product Development Conference in Las Vegas, Nevada.
"Data from recent in vivo studies using our UsiRNAs targeting ApoB message confirm that RNA interference is the mechanism of action by which knockdown occurs, giving us high confidence that our UsiRNAs work by a sequence-specific mechanism," stated Barry Polisky, Ph.D., Chief Scientific Officer of MDRNA.
"Data from the Factor VII studies indicate that a siRNA in our lead formulation achieved greater than 90% knockdown at 1 mg/kg with duration of effect of up to 28 days. This level of inhibition and duration of effect meets or exceeds published data for Factor VII."
Dr. Polisky added, "Data from rodent studies with our DiLA2 delivery system also continue to show encouraging results, with potency, specificity and duration of effect data now achieved for four independent liver targets, including ApoB, PCSK9, DGAT2 and Factor VII. The absence of histological changes in the liver with doses up to 9 mg/kg in rodents confirms that our DiLA2 liposomes are well tolerated."