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Reports Its Proprietary
MDRNA, Inc. (NASDAQ: MRNA),
a biotechnology company focused on the development and commercialization of
therapeutic products based on RNA interference (RNAi), announced today 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 are
being presented today by Michael V. Templin, Ph.D., Vice President, Discovery
Research and Pharmaceutical Development of MDRNA, at the TIDES Oligonucleotide
and Peptide(R) 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."
UsiRNAs are duplex siRNAs that are modified with non-nucleotide acyclic monomers,
termed unlocked nucleobase analogs (UNA), in which the bond between two adjacent
carbon atoms of ribose is removed. UsiRNAs are fully recognized by the RNAi
machinery and provide for potent RNAi activity. Placement of UNA within UsiRNA
minimizes the potential for off-target effects by the guide strand as well as
undesired activity of the passenger strand. Further, the change in sugar structure
renders this unlocked nucleobase analog conformationally flexible. The flexibility
of the monomer escapes the body's surveillance mechanisms associated with cytokine
induction, as well as providing protection from nuclease degradation.
DiLA2 is MDRNA's proprietary platform technology for creating novel liposomal
delivery systems from amino acids. The platform enables MDRNA to tailor the
charge, linker and acyl chains of amino acids in order to optimize the liposome
for delivery to the target tissue of interest. In addition, the platform is
designed to permit attachment of various peptides and other targeting molecules
to improve a variety of delivery characteristics. In addition, MDRNA is utilizing
peptides for nanoparticle formulations to increase cellular uptake and endosomal
release.