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

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Jan 20 2010

RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that new pre-clinical data using proprietary rxRNA™ compounds was presented at the Keystone Symposia’s RNA Silencing: Mechanism, Biology and Application held January 14-19 in Keystone, Colorado.

“RXi has progressed substantially in advancing its technology and building a robust and differentiated RNAi therapeutic platform. Our cutting-edge innovation has led to the discovery and development of novel RNAi compounds with improved drug-like properties that may enable the development of therapeutics for a wide range of indications. By having such a broad platform, we believe that we are well positioned to focus on the most promising therapeutic areas and to select the right candidates to take forward into development,” commented Anastasia Khvorova, Ph.D., RXi’s Chief Scientific Officer.

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.