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Cequent Delays Lead Candidate Program


Cequent Pharmaceuticals will not meet its previously announced goal of filing an investigational new drug application this year for its lead RNAi drug candidate, a treatment for familial adenomatous polyposis, a company official disclosed at the RNAi World Congress in Boston.

The IND now is expected to be filed sometime in 2009, says Johannes Fruehauf, Cequent's vice president of research.

He also unveiled two recent additions to the company's pipeline — a treatment for human papillomavirus and a modified version of its core delivery technology that is expected to open the door to systemic administration — and gave an update on its inflammatory bowel disease program, to which Novartis holds an option.

The delay in the FAP program is primarily the result of a request by the US Food and Drug Administration for additional mouse studies evaluating drug dosing, which is something "that we didn't have in our plans," says Fruehauf. "We wanted to have this IND filed in 2008, and now we're not going to make it."

Once these studies are completed, however, Cequent expects development of the candidate, called CEQ501, to proceed apace, and Fruehauf provided meeting attendees with an overview of the company's planned phase I trial for the drug.

Cequent was founded in 2006 to develop a novel bacterial-based delivery technology created by Fruehauf and colleagues at Beth Israel Deaconess Medical Center. Called transkingdom RNAi, the approach involves using attenuated Escherichia coli to transcribe therapeutic shRNAs.

According to the company, the bacteria express the protein invasion on their surface, which allows them to enter a host cell. They also express listeriolysin, which permits the shRNA payload to escape after bacterial entry.

In 2006, Fruehauf and colleagues published data showing that oral administration of E. coli expressing shRNAs against the oncogene beta-catenin resulted in decreased catenin expression in the intestinal epithelium of a mouse model.

Doug Macron

RNAi Notes

Regulus Therapeutics, a joint venture between Alnylam Pharmaceuticals and Isis pharmaceuticals, has an exclusive license to Stanford University's worldwide patent applications on methods and compositions for antagonizing microRNA-181a to regulate immune responses. miR-181a modifies how immune cells respond to stimuli and the company thinks it could lead to new ways of treating inflammatory diseases.
Sigma-Aldrich will be working with the RNAi Consortium, a public/private alliance of RNAi researchers, to functionally validate 160,000 cloned, lentiviral-based shRNA vector constructs.

Agilent Technologies expanded its nucleic acid active-pharmaceutical ingredient-manufacturing facility in Boulder and acquired Dowpharma's nucleic acid medicines business.


Novartis bought 213,888 unregistered shares of Alnylam Pharmaceuticals stock for $5.4 million. Novartis' stake in Alnylam is now approximately 13.4 percent.

Funded Grants

$52,898 /FY2007
Components of C. elegans P-granules and regulation of their function
Grantee: Ekaterina Voronina, Johns Hopkins University
Began: Jul. 1, 2007; Ends: Jun. 30, 2010

Voronina will be studying the regulators of the germ granules in C. elegans. Using RNAi, she will functionally screen germline-expressed genes and visually screen where fluorescently tagged P granules localize. She then plans to isolate the P granule complexes to identify its protein components.


Broad Spectrum RNAi Therapeutics for Flaviviral Encephalitis
Grantee: Manjunath Swamy, Immune Disease Institute
Began: Jul. 16, 2007; Ends: Jun. 30, 2012

Swamy will be using this grant to develop a delivery system for an RNAi-based therapeutic for flavivirus infections, such as the West Nile and Japanese encephalitis viruses. They will be focusing on a peptide from the rabies virus glycoprotein that, when combined with a positively charged polymeric arginine peptide, has been a promising therapeutic after injection into mouse brains.

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