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Posted: April 19, 2010
MDRNA, Inc. Announces Patent Allowance Covering siRNA and Delivery Systems
(Nanowerk News) MDRNA, Inc., a leading RNAi-based drug discovery and development company, today announced that the Intellectual Property Office of New Zealand (IPONZ) has issued a Notice of Acceptance for patent application 553828, titled "Methods of Treating an Inflammatory Disease by Double-Stranded Ribonucleic Acid." Allowed claims cover small interfering RNAs (siRNA) directed against the tumor necrosis factor (TNF) gene as well as several of the Company's key nucleic acid condensing and delivery peptide motifs in combination with a siRNA directed against TNF. This patent application was filed in 2005 as part of MDRNA's global intellectual property strategy which comprises a broad comprehensive patent portfolio protecting the Company's proprietary siRNA constructs, nucleic acid chemistry, nucleic acid delivery platforms, and its extensive library of siRNAs directed against therapeutic targets.
"The TNF family of cytokines includes some of the most validated targets in drug development and the ability to inhibit a specific member such as TNF-alpha remains a key therapeutic approach for many diseases," stated Barry Polisky, Chief Scientific Officer of MDRNA. "Highly potent siRNAs that inhibit TNF may have direct application in the treatment of inflammatory bowel disease, rheumatoid arthritis, psoriasis and other inflammatory conditions. Efficient delivery to specific cell types involved in these diseases is critical and the peptides protected under this patent provide a basis upon which we can modify our DiLA2 technology to specifically target TNF producing cells."
TNF-alpha (TNF-a) is the most studied member of the TNF family of cytokines. First described as a factor that causes necrosis (death) of tumor cells, subsequent research revealed TNF-a plays a key role within the immune system. TNF-a is produced by cells in blood and most tissues, and virtually all cells respond to TNF-a. It is the role that TNF-a plays in mediating chronic inflammatory diseases, such as rheumatoid arthritis, psoriasis, and Crohn's disease, that has been the focus of drug development efforts. Besides attaining blockbuster status in the treatment of rheumatoid arthritis, TNF inhibitors (e.g., Enbrel(R) and Remicade(R)) have revealed a potential role in multiple diseases. The ability to deliver a TNF-a siRNA to specific cells involved in inflammatory diseases and cancer provides a unique opportunity for an RNAi-based therapeutic.
"One of our main objectives in building an industry-leading RNAi company is to develop a patent portfolio that is both comprehensive and distinct from other RNAi companies," stated Mark Bales, Ph.D., Intellectual Property Counsel at MDRNA. "We believe this allowance demonstrates that MDRNA has a leading position in the field of RNAi therapeutics and that it is an excellent addition to our growing patent estate. We expect to receive additional similar allowances globally that will expand the breadth of our patent estate and further distinguish MDRNA as a leader in the RNAi field."
About MDRNA's Technology
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 17 issued or allowed patents, and has 36 pending patent applications, 125 pending foreign patent applications and 7 PCT applications.
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.