viernes, 19 de abril de 2019

Drugging the undruggable

The Readout
Damian Garde

Drugging the undruggable

Pills can do amazing things, like lower cholesterol, fight mutant cancers, and treat multiple sclerosis. But over the century or so they’ve been contributing to human health, they’ve pretty much only ever worked on proteins, finding no quarter with the countless other biological drivers of disease.

Arrakis Therapeutics, a Massachusetts startup, is out to change that. And, as STAT’s Adam Feuerstein reports, they’ve just hit a major milestone.

The goal at Arrakis is to craft pills that don’t target proteins but rather the RNA that leads to their production. Yesterday, the company announced that some of its proprietary compounds appear to work against myc, a bedeviling target that plays a role in roughly 70 percent of human tumors and yet has never proven to be druggable.

The company is still years away from testing its discovery in human beings, but the promise was enough to attract $75 million in venture funding and propel Arrakis to what CEO Michael Gilman called “escape velocity.”

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