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New RNAi Player in Town: Cequent Pharma


The RNAi therapeutics field got a little bigger late last year with the establishment of Cequent Pharmaceuticals, a Cambridge, Mass.-based firm developing RNAi drugs incorporating a novel bacteria-based delivery technology.

Called transkingdom RNAi, Cequent’s technology involves engineering non-pathogenic bacteria to express shRNAs. It was developed by company founder and director Chiang Li at Beth Israel Deaconess Medical Center.

Having closed a $6 million financing round in November, Cequent is now aiming to prepare the technology for clinical testing this year and file its first investigational new drug application in familial adenomatous polyposis by the second quarter of 2008, according to Cequent President and CEO Peter Parker.

FAP is an inherited colorectal cancer syndrome characterized by the growth of polyps on the colon. According to the Stanford Comprehensive Cancer Center, the condition, which affects about one in 8,000 individuals, leads to colorectal cancer in nearly all cases in the absence of colectomy.

Though FAP is a relatively small indication, affecting an estimated 40,000 people in the US, Parker says it is a good starting point for Cequent to demonstrate the efficacy of its technology.

Patients “would have to be treated for their whole life,” he adds. While Cequent doesn’t anticipate its treatment, an oral drug targeting beta-catenin, would be “terribly expensive,” there is a “very big market when you start thinking about long-term care.”

Parker also notes that production of the bacteria used in the transkingdom RNAi technology is straightforward enough to allow the company to file a series of INDs very rapidly -- as many as one a year.

As such, Cequent expects to file an IND on a second indication in the first half of 2009, he says.

“What we’re furthest along in next is IBD,” he says. “[But] we honestly don’t know what we’re going to bring to the clinic next.”

- Doug Macron

Short Reads

Research centers at Vanderbilt University and the University of Michigan have joined Sigma-Aldrich’s RNAi Partnership Program, part of the RNAi Consortium. Terms of the membership call for the Vanderbilt-Ingram Cancer and the Comprehensive Cancer Center at the University of Michigan to receive Sigma-Aldrich genomics products.

Antisense drug firm iCo Therapeutics said that the US Food and Drug Administration has accepted the company’s investigational new drug application for its diabetic macular edema drug iCo-007. The drug, which was licensed from Isis Pharmaceuticals late last year, targets c-Raf kinase.

Lentigen signed a deal under which Wave Biotech will provide process and equipment expertise for Lenitgen’s lentiviral vector manufacturing platform.

RXi Pharmaceuticals signed a three-year agreement with the University of Massachusetts Medical School that will allow the company to license all unrestricted therapeutic RNAi technology the school develops during that period, according to RXi parent company CytRx. Financial details were not disclosed.

The Gulf Coast Consortium for Chemical Genomics, a group of six Texas research institutions studying genomics and disease, will use Invitrogen’s siRNA library and screening tools to study cancer, diabetes, and metabolic disorders.


US patent application 20070025969. RNAi expression constructs. Inventors: Petrus Roelvink, David Suhy, Alexander Kolykhalov, and Linda Couto. Filed: February 3, 2006.

This invention “provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues, or organs of interest in vitro and in vivo so as to treat diseases or disorders,” according to the abstract.

US patent application 20070004040. RNAi agents for maintenance of stem cells. Inventors: Sarah Brashears and Elisabeth Evertsz. Filed: January 4, 2006.

This patent covers “compositions and methods suitable for delivering RNAi agents against genetic targets in stem cells so as to direct cell growth and differentiation. [It] provides stable, effective siRNA and ddRNAi reagents and methods for use thereof to control the differentiation and proliferation of stem cells by altering the level of expression of one or more transcriptionally active genetic regions that are directly or indirectly associated with the differentiation and proliferation of stem cells.”

Data Point


Stratagene licensed rights to more than 150 microRNA sequences from Max Planck Innovation, the commercial arm of the Max Planck Society. Under the agreement, Stratagene has co-exclusive rights to use the sequences to make and sell molecular diagnostic kits based on its FullVelocity platform.


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