Alnylam and Isis’ Offspring, Regulus, Keeps Pushing on Biology’s ‘Bleeding Edge’

Xconomy San Diego — 

Regulus Therapeutics, its CEO admits, is a child of privilege. It was born two years ago with a silver spoon in its mouth—some hot intellectual property to make drugs based on emerging knowledge about microRNA biological networks. It has a couple of parents with wealth and pedigree: Cambridge, MA-based Alnylam Pharmaceuticals (NASDAQ: ALNY), and Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS).

Since we’re watching closely to see how innovation is affected during this economic downturn, I figured it seemed reasonable to check whether ideas on the real frontier of biology can still be sustained at a place like Carlsbad, CA-based Regulus, which we first profiled back in October. CEO Kleanthis Xanthopoulos says he’s always worried about financing—it’s the nature of being a biotech CEO—but enough positive things have happened this year that he says the company has still been able to grow.

That’s no small thing, because companies pursuing microRNA know they are staring at what amounts to a 20-year vision to develop a new class of therapies, as Xanthopoulos pointed out in that first story in October. The key is showing people enticing bits of progress along the way. Regulus did that around Thanksgiving, when it published a scientific paper in Nature that was the first to demonstrate a therapeutic benefit in animals by blocking a strand of microRNA. The company raised $20 million from its corporate parents a couple months later, then scored an undisclosed sum of cash from its big partner, GlaxoSmithKline, back in May when it reached a milestone as part of a collaboration worth as much as $600 million. The company has almost doubled in size, from 22 employees last October, to almost 40 today, Xanthopoulos says.

“Am I concerned about financing? Always. I’m paranoid when it comes to financing,” Xanthopoulos says. “It’s a capital-intensive business, with long development timelines. But we’re really well-capitalized.” He has at least three years of operating cash on hand, and the company recently re-organized … Next Page »

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