siRNA Directed Against JAM-1 Gene Also Has Potent Drug Delivery PotentialBOTHELL, WA, Sep 29, 2009 (MARKETWIRE via COMTEX) -- MDRNA, Inc. (NASDAQ: MRNA), a leading RNAi-based drug discovery and
development company, today announced that the U.S. Patent and
Trademark Office (USPTO) has issued a Notice of Allowance for patent
application U.S. 11/624,630 covering an siRNA directed against a
junctional adhesion molecule-1 (JAM-1) gene.
The siRNA of the allowed claim has a broad array of potential
applications, most notably as a therapeutic for the treatment of
certain cancers, thrombosis, atherosclerosis, strokes, and
hypertension; and enhances the delivery of drugs across the skin and
the blood-brain barrier. JAM-1 is a tight junction protein that plays
a key role in maintaining epithelial and endothelial barrier
functions, leukocyte transmigration and platelet adhesion and also
functions as a pro-inflammatory molecule.
"This allowance validates our patent strategy for our gene target
portfolio, and reinforces our belief that we will obtain patent
protection for our extensive library of siRNAs directed against more
than 140 therapeutic gene targets," said J. Michael French, President
and CEO of MDRNA. "As our scientific team develops more novel RNAi
technologies, we expect to expand and strengthen our IP estate."
Small interfering RNA (siRNA), sometimes known as short interfering
RNA, is a class of double-stranded RNA molecules that play a variety
of roles in biology. siRNA is involved in the RNA interference
pathway, where it interferes with the expression of a specific gene.
A Notice of Allowance confirms the substantive examination of a
patent application and will result in a final issuance of a U.S.
patent after an administrative process is completed.
MDRNA has a broad intellectual property estate that encompasses four
key RNAi technology platforms: siRNA constructs, chemistry, nucleic
acid delivery, and gene targets. The MDRNA-owned siRNA constructs and
chemistry include its proprietary UsiRNA construct, which is a duplex
siRNA chemically modified with non-nucleotide acyclic monomers (UNAs),
and is distinct from the standard siRNA construct used by others in
the industry. UsiRNAs are fully recognized by the RNAi machinery and
provide for potent RNAi activity while specific placement of UNAs in
a duplex siRNA minimizes potential off-target effects by the guide
strand and reduces undesired passenger strand activity. Furthermore,
UsiRNAs escape the surveillance mechanisms associated with cytokine
induction, and provide protection from nuclease degradation.
The MDRNA delivery platforms include DiLA2 and nanoparticle forming
peptides. DiLA2 is an MDRNA proprietary delivery platform of novel
synthetic di-alklylated amino acid compounds used to make liposomal
delivery formulations. The DiLA2 platform enables MDRNA to tailor the
charge, linker and acyl chains of amino acids in order to configure
liposomes for delivery to target tissues 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. The MDRNA peptide nanoparticle platform includes
exclusively in-licensed and developed IP surrounding the use of
peptides for nanoparticle formulations that increase cellular uptake
and endosomal release of siRNAs. MDRNA is currently biopanning its
patented phage display library to identify additional peptides for
targeted delivery, cellular uptake and endosomal release of siRNA.
MDRNA owns or controls 4 issued patents and has more than 50 pending
patent applications, 103 pending foreign patent applications and 17
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
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.
Mark Bales, Ph.D.
Matthew D. Haines
Senior Director, Investor Relations and Corporate Communications
(312) 944-6784 x 316 or
SOURCE: MDRNA, Inc.