familial adenomatous polyposis

FAP is an inherited, colorectal cancer syndrome characterized by the growth of colorectal polyps. Though the polyps are initially benign, they become malignant in nearly all cases in the absence of colectomy, according to Marina.

The company's vice president of corporate development said that Marina is "committed" to signing two partnerships before year end, but indicated that it may miss this goal if the terms of the deals aren't right.

"Clinical site approval at MGH marks the last regulatory requirement prior to moving our first drug candidate into human trials," Michael French, president and CEO of Marina, said in a statement.

CEQ508 is based on Cequent Pharmaceuticals' so-called transkingdom RNAi technology, and is designed to inhibit the oncogene beta-catenin, which is expected to prevent new polyp formation associated with the disease.

The company said the toxicology work, which is being conducted in non-human primates, will lay the groundwork for a phase II study to begin in 2011.

The drug is designed to treat advanced familial adenomatous polyposis by inhibiting the oncogene beta-catenin, which is expected to prevent the formation of polyps characterizing the disease and possibly slow the progression to malignancy of existing ones.

The company also remains on track with its more advanced familial adenomatous polyposis drug and plans to file within the next two weeks an investigational new drug application to begin testing the therapy in humans.

The data, which the company said represent the first proof of activity of an oral RNAi drug, set the stage for an investigational new drug application filing later this year.

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An analysis of UK Biobank data finds hemochromatosis to be more prevalent than thought, according to the BBC.

An analysis finds that female biomedical researchers receive fewer prizes than male ones, and when they do win prizes, they are less prestigious.

In Nature this week: improved genomic analysis using a graph genome reference, tumor mutational burden could predict clinical response to immune checkpoint inhibitors, and more.

Federal researchers tell the Los Angeles Times that the shutdown is causing missed research opportunities as they try to keep their experiments going.