Regulus, the MicroRNA Child of Alnylam and Isis, Offers Litmus Test for Biotech Hope in 2011

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raising traditional venture capital, Xanthopoulos says.

Why all the fuss? The money and brainpower is being directed in an effort to turn Regulus into the leading developer of microRNA drugs. Biologists didn’t even know these tiny microRNA switches existed in humans until 2001, but a big push in research has shown these molecules have potential to regulate full networks of genes, not just one gene or protein at a time, like most conventional drugs. The microRNA strategy is thought to have promise in treating complex diseases like diabetes, cancer, or autoimmunity, where multiple genes can get thrown out of whack. This is all very early stuff—back in the summer of 2009, Xanthopoulos called it “bleeding edge” in an interview with Xconomy.

Most of the research at Regulus to date has been about developing specific microRNA silencing molecules against certain targets, and seeing what kind of effect they have in mice. Several times during our chat, Xanthopoulos talked about “therapeutic effects” that have been seen in mouse models of microRNA drugs in the pipeline, as opposed to merely showing a drug can hit its intended biological target. Work is now advancing into primates, with an eye toward mounting an initial clinical trial in another 12 to 18 months, he says. Xanthopoulos didn’t say which program is furthest along in development, although Regulus lists four disease categories on its website—cancer, autoimmunity, hepatitis C, and cardiovascular disease.

Any time you plow ahead in a new field of biology, with all its complexity, an almost infinite number of things could go wrong. There are hard methodical steps that must be taken to win over academic peers, regulatory agencies, partners, and, yes, public investors. But enough of those methodical steps have happened in the past couple years that Regulus is in position to dream very big, and maybe even go public before it has a drug showing promise in clinical trials.

“This will be about us going into humans, targeting a specific microRNA we know is dysregulated, injecting patients with a specific oligonucleotide, and seeing whether this injection correlates with a return to normal,” Xanthopoulos says. “That’s the challenge we put in front of 55 brilliant people in the next three years. It’s an aggressive, but not an unrealistic goal.”

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