Five years ago, a small startup led by biotech veteran Michael Gilman announced progress against a cancer gene that had bedeviled drug developers for 40 years.
Known as Myc, it was one of the first so-called oncogenes ever discovered. It’s mutated or dysregulated in perhaps 70% of all cancers. The challenge is that the protein it creates is “intrinsically disordered” — which is chemist-speak for “spaghetti-esque”, lacking a clear pocket where chemists can aim a deactivating molecule.
There are many proteins like this, known cellular miscreants that persist because scientists just can’t find a way to take them down. Gilman’s startup, Arrakis, had a radical solution. It would skip the protein. Instead, the startup — which derives its name from the frontier planet in Dune, source of life-extending “spice” — claimed it could do what chemists also thought near-impossible: Create molecules that intercept mRNA, preventing the protein from even being produced.
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