New pre-clinical data on RXi Pharmaceuticals' proprietary rxRNA compounds presented

The data were presented in four posters that are available on RXi’s website, www.rxipharma.com/scientific_publications_presentations.html.

• Novel, Chemically Modified RNAi Compounds with Improved Potency, Stability and Specificity – Describes the development of novel, chemically modified, 25-mer blunt RNAi compounds (rxRNAori™) with enhanced stability, reduced off-target effects and reduced immune stimulatory activity. This novel configuration enables rapid identification of potent RNAi compounds for potential therapeutic development due to improvements in the algorithm used to identify extremely potent candidate molecules.
• Novel “Self-Delivering” RNAi Compounds with Enhanced Cellular Uptake and Distribution Properties – Outlines the development of “self-delivering” RNA (sd-rxRNA™) compounds that efficiently enter cells without the need for an additional delivery vehicle. sd-rxRNAs are reduced in size compared to conventional siRNAs and are chemically modified to improve cellular uptake without interfering with the ability to modulate gene expression.
• Robust Intradermal Efficacy with Novel Chemically Modified Self-Delivering RNAi Compounds – Demonstrates efficient intracellular uptake of fluorescently-tagged sd-rxRNA into dermal cells and significant reduction of targeted mRNA using intradermal injection as a model of local delivery in vivo. The data presented establish the efficacy of locally-administered sd-rxRNAs in animal models and support the use of these novel compounds for clinical applications where direct or local administration is possible, thereby potentially enabling broad opportunities for therapeutic development using local administration routes.
• Highly Potent RNAi Compounds Derived from a Single, Short Oligonucleotide Sequence - Illustrates the development of a 27 nucleotide RNA construct with silencing potency equal to duplex RNAs. RXi’s single-oligo RNAi triggers (rxRNAsolo™) act as homo-dimers and have shown high, seed-specific potency. In addition, the poster shows that a hetero-dimer (rxRNAduo™) can simultaneously silence two different genes.