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

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in a way that allows Regulus to attract institutional investors, he says.

Regulus is hoping to keep the momentum going with some more data, which hasn’t yet been published in a peer-reviewed journal, that says that by blocking a different strand of microRNA, known as mir122, it can block the replication of the hepatitis C virus in a laboratory dish, and in chimpanzees, Xanthopoulos says. This is interesting because hepatitis C is one of those diseases in which the animal models tend to be fairly accurate predictors of how a drug will work in people, Xanthopoulos says. Since hepatitis C is undergoing a drug development renaissance of sorts, with more than 40 different drugs in clinical trials, Xanthopoulos is wagering that he can find another partner to help support his company’s R&D. More than one drug company is interested in the data, he says.

MicroRNA, as a reminder to those who may have missed the earlier stories, are tiny strands of RNA that serve like regulatory switches that control full networks of genes. They were first discovered to exist in humans in 2001. Scientists say they have great potential against complex diseases that involve many genes, like cancer, diabetes, heart failure, or autoimmune disease. But there’s a lot of work to do in characterizing what these networks do, not the least of which involves making sure a drug based on microRNAs doesn’t shut down some biological process that turns out to be really important.

One of the big challenges for Regulus is staying disciplined on the programs it has chosen to pursue, and not following every bright idea that comes in over the transom. About 20 to 30 interesting reports about microRNA from academic scientists are getting published every week now, Xanthopoulos says. “An incredible amount of knowledge is being transferred,” he says.

And while some of that knowledge is likely to lead in the right direction, some won’t, and choosing the right balance of what to pursue is part of what makes Regulus a real biotech tightrope act. “We’re not at the cutting-edge, we’re at the bleeding edge of the biological frontier. We are dealing with a big idea, and it’s exciting,” Xanthopoulos says.

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