Intellectual property cornerstone now established for use of engineered bacteria as therapeutics that produce and deliver RNAi mediators against various disease-related genes, including cancer oncogenes
CAMBRIDGE, Mass., June 22 /PRNewswire/ -- Cequent Pharmaceuticals, Inc., a pioneer in the development of novel products to deliver RNAi-based treatments to prevent and treat human disease, announced that it has secured key intellectual property rights for its TransKingdom RNAi (tkRNAi) technology. The European Patent Office has delivered a notice of Intent to Grant a patent entitled "Compositions for Bacterial Mediated Gene Silencing and Methods Using Same," and the Korean Intellectual Property Office delivered a Notice of Decision for Patent of the same title. Allowed genus claims cover attenuated bacteria (such as modified E.coli) as a therapeutic engine for the delivery of short interfering RNA (siRNA) molecules against disease-related genes, including cancer genes such as beta-catenin, whose up-regulation is implicated in the formation and proliferation of colorectal and other cancers.
The patents encompassed by these two notices are family members of the seminal patent application filed by the Beth Israel Deaconess Medical Center (BIDMC) relating to its researchers' invention of the tkRNAi technology. Cequent has an exclusive license to the tkRNAi technology from BIDMC, and the company has subsequently filed many new U.S. and international patent applications to protect its improvements to this broadly applicable platform for the generation of new drug candidates. Cequent was founded in 2006 to advance the then bench-stage discovery into a versatile therapeutic platform and promising therapeutic products suitable for administration to humans. In December 2009, the U.S. Food and Drug Administration (FDA) approved Cequent's Investigation New Drug (IND) application allowing initiation of human studies with the company's most-advanced tkRNAi therapeutic candidate, CEQ508, an oral therapy targeting the cancer gene beta-catenin. A clinical trial of CEQ508 is slated to commence this summer in FAP (Familial Adenomatous Polyposis) patients.
"Important jurisdictions have now confirmed the novelty and utility of our innovative technology for the delivery of mediators of RNA interference," commented Cequent CEO Peter Parker. "The ability to achieve delivery of siRNAs to target cells in order to block disease-related genes has been hobbling the promise of RNAi-based therapies envisioned since Mello and colleagues made the Nobel Prize-winning discovery of RNAi in the late 1990s," he added. "Today's announcement highlights a major regulatory advancement of our tkRNAi platform, a technology specifically designed to overcome this difficult problem of delivery."
Chiang Li, MD, FACP, whose laboratory at BIDMC, Harvard Medical School, first invented and reduced the tkRNAi technology to practice, said, "By combining the potential of RNAi with the versatility of microbes, our tkRNAi bacteria have been able to induce gene silencing on a wide variety of targets in the gastrointestinal tract after oral dosing, including the silencing of previously 'undruggable' intracellular targets. The tkRNAi platform may serve as a powerful drug-discovery engine, holding significant promise for the treatment or prevention of a range of important diseases – from cancer to Inflammatory Bowel Disease (IBD) to metabolic disorders." Dr. Li co-founded Cequent and is currently a board director and the Chairman of the Scientific Advisory Board of the company.
About Familial Adenomatous Polyposis (FAP) and CEQ508
FAP is a rare inherited gastrointestinal disease that causes hundreds to thousands of precancerous polyps to form in the colon of an affected individual. Today, without prophylactic removal of the colon, people with FAP almost inevitably develop cancer, and there is no generally accepted pharmacological treatment available. CEQ508 is an oral therapeutic designed to down-regulate beta-catenin, which is implicated in the formation of colonic polyps and in the progression of polyps to colorectal and other gastrointestinal cancers. An intracellular protein, beta-catenin has to date been an undruggable target using conventional therapeutic modalities, such as small molecules. However, in previous trials with non-human primates, Cequent's tkRNAi therapeutic candidates have demonstrated potent silencing of beta-catenin, and CEQ508 exhibited an encouraging safety profile when administered as a daily oral therapeutic in animal trials.
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 entering clinical development, are orally administered drug candidates targeting colon-cancer prevention and inflammatory bowel disease. The company designed its powerful tkRNAi technology as a therapeutic 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, Cequent was established in 2006.
SOURCE Cequent Pharmaceuticals, Inc.