UsiRNAs Work Via the RNAi Mechanism of Action but With Minimal Off-Target ActivityBOTHELL, WA, Aug 03, 2009 (MARKETWIRE via COMTEX) -- MDRNA, Inc. (NASDAQ: MRNA) announced today 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 today 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."
About Unlocked Nucleobase Analogs and UsiRNAs
Unlocked Nucleobase Analogs (UNA) are substitutes for nucleotides
that make up conventional siRNAs. UNAs have an open structure in the
place of the ribose portion, making them more flexible than common
nucleotides. UsiRNAs are a duplex siRNAs containing at least one UNA.
UsiRNAs are fully recognized by the cellular RNAi machinery, as
demonstrated by their potent activity. MDRNA has also shown that
inclusion of UNA increases stability to nucleases, substantially
reduces cytokine induction, and reduces passenger and guide
strand-mediated off-target effects. The high potency, and improved
drug-like properties, associated with UsiRNAs provide the potential
to greatly enhance RNAi-based therapeutics.
About the DiLA2 Delivery Platform
The DiLA2 Delivery Platform is MDRNA's proprietary platform for
creating novel liposomal delivery systems based on di-alkylated amino
acids (DiLA2). The DiLA2 Platform enables MDRNA to tailor the charge,
linker length, and acyl chain characteristics to improve delivery of
the liposomes to target tissue of interest. In vivo studies have
demonstrated effective delivery in models of metabolic disease,
cancer, and other targets. DiLA2-based liposomes are well tolerated
for repeat dose, and systemic and local administration. MDRNA is also
utilizing condensing peptides to form peptide-siRNA nanoparticles to
further increase the delivery efficiency of its DiLA2 delivery
systems. In addition, the platform is designed to permit attachment
of peptides and other targeting molecules for delivery to a variety
of tissues, and thus provide for a diverse therapeutic portfolio.
MDRNA Forward-Looking Statements
Statements made in this news release may be forward-looking
statements within the meaning of Federal Securities laws that are
subject to certain risks and uncertainties and involve factors that
may cause actual results to differ materially from those projected or
suggested. Factors that could cause actual results to differ
materially from those in forward-looking statements include, but are
not limited to: (i) the ability of MDRNA to obtain additional
funding; (ii) the ability of MDRNA to attract and/or maintain
manufacturing, research, development and commercialization partners;
(iii) the ability of MDRNA and/or a partner to successfully complete
product research and development, including preclinical and clinical
studies and commercialization; (iv) the ability of MDRNA and/or a
partner to obtain required governmental approvals; and (v) the
ability of MDRNA and/or a partner to develop and commercialize
products that can compete favorably with those of competitors.
Additional factors that could cause actual results to differ
materially from those projected or suggested in any forward-looking
statements are contained in MDRNA's most recent periodic reports on
Form 10-K and Form 10-Q that are filed with the Securities and
Exchange Commission. MDRNA assumes no obligation to update and
supplement forward-looking statements because of subsequent events.
Contact:
Matthew D. Haines
Senior Director, Investor Relations and Corporate Communications
(212) 209-3874
Email Contact
McKinney|Chicago (Media)
Alan Zachary
(312) 944-6784 x 316
(708) 707-6834
Email Contact
SOURCE: MDRNA, Inc.
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