Hope for Ultra-Rare Diseases

Soonish

In this episode of Soonish you'll meet Stanley Crooke, the former CEO of Ionis Pharmaceuticals and the head of a new nonprofit called N-Lorem, which is working to make mutation-correcting "antisense oligonucleotide" drugs available free for life to people with uncommon genetic diseases.

These are conditions so rare they often don't have a name. But while the diseases themselves are unusual, the problem isn't: as many as 350 million people worldwide are thought to carry mutations that give rise to unique "N of 1" health problems.

The debut of hyper-personalized antisense medicines is a topic I covered in a March 2020 episode of the podcast Deep Tech for MIT Technology Review. Back then, N-Lorem was just getting started. So I was excited to connect with Crooke one year later and go into more depth how antisense drugs work, why they're well-suited for treating some genetic diseases, and how Crooke realized he could give some patients personalized versions of these drugs for free—and for life.

"It was literally impossible until just now," Crooke says. Listen to find out what changed—and what it could mean for the future of drug discovery and the way we regulate and pay for advanced therapies.

For more, head to soonishpodcast.org, where we've got the full transcript and additional resources.

Notes

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