Dicerna’s compounds to interact with an enzyme called “Dicer,” hence the company name. This is important because Dicer is involved at an earlier step in the RNA interference process than where other drugs come in, Jenson says—intervening earlier gives the firm’s drugs increased potency and duration of effect. The extra few nucleotides also give the drug extra precision in targeting diseased cells, he says.
The extra length also gives Dicerna the ability to attach other compounds like peptides or antibody fragments, and to make other modifications that could enable Dicerna to avoid the problems other drug developers have encountered in trying to deliver RNAi drugs, Jenson says. If given directly, most small RNAi drugs get flushed out via the kidneys in a few minutes—before they can do any good. Companies like Alnylam and others are working on a host of delivery techniques to solve the problem.
The critical patents for Dicerna’s techniques come from John Rossi of the City of Hope Beckman Research Institute in Duarte, CA, and Mark Behlke, chief scientific officer of Coralville, IA-based Integrated DNA Technologies.
Like other RNAi companies, Dicerna envisions using this approach against many of the biggest diseases in the developed world—cancer, cardiovascular disease, inflammation, neurological conditions, metabolic diseases like diabetes, and infections like hepatitis C.
The company will spend this year laying the groundwork for its first clinical trial of a cancer drug, with a goal of getting there “sometime in 2010,” Jenson says. As with any RNAi company, if Dicerna rushes into the clinic and fall short, it could greatly harm the field. “We want to be very careful and thorough,” Jenson says.
