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‘Biotech fantasy’? This startup wants to kill cancer by targeting RNA with new generation of pills

| | April 29, 2019

Arrakis Therapeutics is named for the desert planet in the legendary “Dune” series of science-fiction novels. It’s an apt name because the Waltham, Mass., startup is trying to achieve a feat once thought to be biotech fantasy: Develop oral medicines — pills — that can target RNA, the messenger-like molecules that turn genetic instructions into proteins.

On [April 24], the company is announcing an early breakthrough. Arrakis’ screening work has identified “hits” — oral drug-like compounds — that block the RNA machinery needed to make some well-known, cancer-causing proteins long thought to be beyond the reach of drug makers.

Some of the compounds found by Arrakis work against myc, one of the first so-called oncogenes identified in the very early 1980s. Myc is believed to play a role in about 70% of all human tumors, but no one has yet figured out a way to develop a drug against it.

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To be crystal clear, Arrakis hasn’t fully solved the myc puzzle either, but it’s no longer a crazy pipe dream, said Michael Gilman, Arrakis’ CEO.

“I think we’ve achieved escape velocity,” he said, leaning on a rocket ship analogy that befits a biotech with a sci-fi inspired name.

Read full, original post: A biotech startup’s lofty goal: Kill cancer with pills that target RNA instead of proteins

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