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Therapeutic Thursday: A Fringe RNA Drug Is Now First Of Its Kind In The Clinic

An empty forest road climbing toward misty light, one vehicle partway up.
Photo: Nick Chung / Unsplash, who found the road so we didn't have to.

Today is a quiet kind of vindication story, the sort the industry doesn't hand out often. A founder spent 15 years insisting that the junk stretches of the genome could treat disease, walked away from a tenure-track career that was his for the taking, and has now put the first drug of its kind into patients.

The science is worth your time. But the part to carry into your day is smaller and more useful: how a roughly 60-person biotech turned a fringe idea into a platform a pharma giant pays to borrow, without betting the company on a single molecule. The proof is still a couple of years out. The template is already here.

The Prospector Who Wouldn't Quit

For a decade and a half, Samir Ounzain had a pitch much of his field didn't want to hear: the vast stretch of human DNA written off as junk was doing real work, and one day you would treat disease by tuning it. He made that case as a career researcher at Lausanne University Hospital, where tenure-track offers were on the table and the safe path was clear. He took the other one, co-founding HAYA Therapeutics in 2019, to chase the biology into the clinic. "The rubber is hitting the road, so to speak," he told In Vivo, which this week profiled him as a 2026 Rising Leader.