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MDRNA Demonstrates Significantly Reduced Off-Target Activity for Proprietary RNAi-Based Compounds
Results Show Greater Target Specificity for Novel UsiRNA Constructs Based on Proprietary Unlocked Nucleobase Analog Technology
| Source: MDRNA, Inc.
BOTHELL, WA--(Marketwire - January 19, 2010) - MDRNA, Inc. (NASDAQ : MRNA ), a leading
RNAi-based drug discovery and development company, today announced the
presentation of data highlighting the ability of the UsiRNA construct to
significantly minimize off-target effects while maintaining potent RNA
Interference (RNAi) activity. Narendra Vaish, Ph.D., MBA, Associate
Director, Discovery Research and Pharmaceutical Development, presented the
data at the Keystone Symposia on "RNA Silencing: Mechanism, Biology and
Application" from January 14 to 19, 2010, Keystone Resort, Keystone,
Colorado.
UsiRNAs are MDRNA's proprietary constructs in which Unlocked Nucleobase
Analogs (UNA) strategically replace ribonucleobases. Placement of UNA in
the passenger strand specifically blocks the activity of this strand to
function in RNAi, thus decreasing off-target activity. Placement of UNA
within the guide strand confers greater specificity upon RNAi by inhibiting
micro-RNA-like effects. Using microarray analysis to simultaneously
measure the expression levels of more than 30,000 genes, a UNA in the
passenger strand reduced the off-target effects by greater than two-fold as
compared to a standard siRNA. More importantly, a UsiRNA, with both
passenger and guide strand UNA placements reduced the off-targets effects
by more than 10-fold compared to a standard siRNA.
MDRNA also reported delivery of UsiRNA against Survivin and PLK-1, by
MDRNA's proprietary DiLA2-based liposome formulations via systemic (liver
cancer) or local (bladder cancer) administration, and demonstrated potent
and persistent target knock-down and inhibition of tumor growth.
Significant knockdown in the expression level of a liver gene has also been
described in non-human primates following intravenous (systemic)
administration of a UsiRNA using DiLA2 liposomes.
"These new data highlight the ability of our UsiRNA construct to inhibit
passenger strand activity and achieve greater guide strand specificity,"
said Dr. Barry Polisky, Chief Scientific Officer of MDRNA. "These
advantages are reflected in the microarray data which indicate UsiRNAs
mitigate off-target effects that can occur in RNAi. The inhibition of
non-cancer, and cancer targets in two divergent models via different routes
of administration, demonstrates the strength of pairing the UsiRNA and DiLA2
delivery platforms for the development of RNAi-based therapeutics."
About MDRNA, Inc.
MDRNA is a biotechnology company focused on the development and
commercialization of therapeutic products based on RNA interference (RNAi).
Our goal is to improve human health through the development of RNAi-based
compounds and drug delivery technologies that together provide superior
therapeutic options for patients. Over the past decade, we have developed
substantial capabilities in molecular biology, cellular biology, lipid
chemistry, peptide chemistry, pharmacology and bioinformatics, which we are
applying to a wide range of RNAi technologies and delivery approaches.
These capabilities plus the in-licensing of key RNAi-related intellectual
property have rapidly enabled us to become a leading RNAi-based
therapeutics company with a pre-clinical pipeline in oncology. Through our
capabilities, expertise and know-how, we are incorporating multiple RNAi
technologies as well as peptide- and lipid-based delivery approaches into a
single integrated drug discovery platform that will be the engine for our
clinical pipeline as well as a versatile platform for establishing broad
therapeutic partnerships with biotechnology and pharmaceutical companies.
We are also investing in new technologies that we expect to lead to safer
and more effective RNAi-based therapeutics while aggressively building upon
our broad and extensive intellectual property estate. By combining broad
expertise in siRNA science with proven delivery platforms and a strong IP
position, MDRNA is well positioned as a leading RNAi-based drug discovery
and development company. Additional information about MDRNA, Inc. is
available at http://www.mdrnainc.com.
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.