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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

CAMBRIDGE, Mass., Jan. 6 /PRNewswire/ -- Cequent Pharmaceuticals, a pioneer in the development of novel products to deliver RNAi-based treatments to prevent and treat human disease, announced that the company has recently completed a successful toxicology study of its candidate CEQ501, an orally administered tkRNAi therapeutic targeting the primary oncogene (beta-catenin, CTNNB1) in FAP (familial adenomatous polyposis). FAP is an inherited gastrointestinal disease that causes hundreds of polyps to form in the colon. Today, without prophylactic removal of the colon, people with FAP almost inevitably develop colon cancer, and there is no medical treatment available. Cequent will present its findings, the first-ever proof of activity of an oral RNAi drug in non-human primates, in a poster at the Keystone Symposia Conference, Therapeutic Modulation of RNA Using Oligonucleotides. The conference will be held February 8 - 13 in Lake Louise, Alberta Canada.

"Delivery of RNAi into the targeted cells and tissues remains one of the biggest challenges in the development of RNAi-based therapeutics - a new class of drugs designed to work by effectively deactivating the specific gene or genes implicated in the progression of a disease," said Cequent President and CEO, Peter Parker. "We have shown previously in cell-culture assays and in a mouse model for human colon cancer that our tkRNAi technology effectively suppresses beta-catenin, a key oncogene in FAP. These new results are very encouraging, and represent another significant milestone in our work to turn the promise of RNAi into safe and effective therapies to treat devastating diseases, like FAP, and improve the lives of those affected."

The study showed that tkRNAi targeting beta-catenin was well tolerated at high dosing levels, with no product-related adverse events, no increase in serum cytokines, and no gross or histopathologic abnormalities noted. Ora
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SOURCE Cequent Pharmaceuticals
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