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At Arrakis, Gilman Teams With Biogen Vets to Target RNA With Pills

Xconomy Boston — 

A year after selling his latest biotech startup—the second since 2012—Michael Gilman is back. He emerged today as chairman and CEO of a two-year-old startup named Arrakis Therapeutics, which aims to use chemical drugs to go after an unlikely target: RNA, the molecules that turn our genetic blueprints into proteins.

Named after the dangerous desert planet in the science fiction epic Dune, Arrakis has also closed a $38.5 million Series A round from three venture firms (Advent Life Sciences, Canaan Partners, and Osage University Partners), two large drugmakers (Celgene and Pfizer), and former Genzyme CEO Henri Termeer. Canaan led the funding.

In Dune, Arrakis is inhabited by deadly giant sand worms and the only place to mine the “spice,” a psychoactive drug that everyone in the universe craves. “There’s essentially a [central nervous system] drug there,” Gilman (pictured) says.

Arrakis the biotech, formed in 2015, is also wading into foreboding territory, pursuing drug development for neurological disorders and other diseases. To do so, it will try to do intentionally using new methods what a few others, Gilman says, have done accidentally: find small molecules—traditional chemical-based drugs—that block the function of RNAs that are causing disease.

A number of chemical drugs either approved or in development that target RNA—like Merck’s ribocil—weren’t known to do so when they were first discovered. Arrakis is taking common tools that drug companies use to search through vast libraries of proteins and adapting them to RNA, Gilman says. Arrakis has come up with new algorithms to help find what could be “druggable” sites on RNA. Once it finds those sites, Arrakis can verify if its small molecules are hitting them just right.

The idea is to get after drug targets inside cells that to date have been out of the reach of small molecules and biologics. Small molecules can slip into cells but often don’t match up with the desired targets, like mismatched keys and locks. Biologics, particularly monoclonal antibodies, are popular because they can be designed to bind to almost any target, but only on the surface of a cell.

Those limitations are significant. They’re the reason that the entire universe of small molecules and biologics can currently only get to … Next Page »

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