MDRNA, Inc., a leading RNAi-based drug discovery and development company, announced that it obtained exclusive rights to develop, make, use and sell UNA (Unlocked Nucleobase Analog)-based diagnostics from RiboTask ApS, a privately held Denmark-based company specializing in the development and synthesis of novel RNA chemistries.

MDRNA expanded its previous agreement with RiboTask for exclusive rights to the development of UsiRNA-based therapeutics to now include the development of UNA-based diagnostics. With the newly acquired exclusive rights to UNA technology combined with the previously acquired exclusive rights to Conformationally Restricted Nucleotide (CRN) technology for both therapeutics and diagnostics, MDRNA has established one of the few intellectual property portfolios supporting a nucleic acid-based personalized medicine platform with the ability to pursue proprietary nucleic acid-based therapeutics and diagnostics. RiboTask retains co-exclusive rights to manufacture, sell and sublicense UNA technology for use in reagents. Financial terms of the expanded agreement were not disclosed.

In addition to UNA based RNAi-therapeutics, UNA-based diagnostics provides MDRNA the opportunity to capitalize upon the rapidly growing fields of personalized medicine and molecular diagnostics. A key component of personalized medicine is the development of companion diagnostics where molecular assays are used to measure levels of proteins, genes or specific mutations to provide a specific targeted therapy to meet an individual patient's needs. Additionally, this information can be used for the early detection of diseases and the relevant stratification of patient populations with the goal of increasing the success rate of therapies, reducing the risks of side effects and decreasing costs.

"The acquisition of the exclusive rights to UNA-based diagnostics adds significant value to MDRNA's IP portfolio and broadens our commercial opportunities in both nucleic acid-based therapeutics and diagnostics," stated J Michael French, president and CEO of MDRNA. "Combined with our acquisition earlier this year of the CRN technology patents for both therapeutics and diagnostics, MDRNA is well-positioned to make a significant contribution to the field of personalized medicine."

UNAs are acyclic nucleomonomers, and when used in the synthesis of a short-interfering RNA (UsiRNA), provide drug-like properties to a siRNA in a novel and proprietary manner; these include protection from enzymatic destruction; reduction, or elimination, of a cytokine response; reduction of so-called off-target effects; and preservation of high efficacy. These characteristics are key to human therapeutic use of an RNAi-based therapeutic.

The combination of UNAs with CRNs, which are novel nucleoside analogs in which the flexible ribose sugar is locked into a rigid conformation by a small chemical linker, provides the opportunity to tailor on a position-by-position basis the specificity of a nucleic-acid based diagnostics.

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 in which non-nucleotide acyclic monomers (UNAs) have been included, 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-alkylated 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 41 pending patent applications, 134 pending foreign patent applications and 6 PCT applications.

MDRNA is a biotechnology company focused on the development and commercialization of therapeutic products based on RNA interference (RNAi).