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Cequent to Present First Proof of Activity of an Oral RNAi Drug in Non-Human Primates at February Keystone Conference
Date:1/6/2009

lly administered bacteria expressing short hairpin RNA (shRNA) against beta-catenin significantly reduced messenger RNA (mRNA) levels of beta-catenin in the mucosa of the small and large intestine, an effect that was reversible after termination of dosing. Samples obtained from the gastrointestinal mucosa indicate that oral tkRNAi treatment was effective in up to 84 percent of animals, and target gene levels were suppressed between 58 to 64 percent compared to control animals. Pharmacokinetic studies verified the presence of the engineered shRNA in the intestinal mucosa, proving successful delivery of the active hairpin RNA component by the bacterial system. Cequent developed the current tkRNAi candidate on the basis of an E. coli delivery platform obtained from the laboratory of Patrice Courvalin at the Institut Pasteur. Cequent is planning to start a Phase I clinical trial for the indication of FAP in 2009.

About Cequent Pharmaceuticals, Inc. (www.cequentpharma.com)

An early-stage biopharmaceutical company, Cequent is pioneering the development of novel therapeutics to prevent and treat a wide range of human disorders - from inflammatory disease to cancer - based on the company's proprietary technology, TransKingdom RNA interference (tkRNAi). Cequent's first products, now in pre-clinical development, are drug candidates targeting colon-cancer prevention and inflammatory bowel disease. The company designed its powerful tkRNAi technology to deactivate specific disease-causing genes safely and effectively, using non-pathogenic bacteria as an engine to produce and deliver RNAi directly into cells. It is based on ground-breaking scientific research originating at the Institut Pasteur (Paris, France) and at the Beth Israel Deaconess Medical Center/Harvard Medical School. A privately held company based in Cambridge, Massachusetts
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