Hope for Ultra-Rare Diseases
Podcast |
Soonish
Publisher |
Hub & Spoke
Media Type |
audio
Podknife tags |
Society & Culture
Technology
Categories Via RSS |
Documentary
Science
Society & Culture
Technology
Publication Date |
Apr 30, 2021
Episode Duration |
00:45:20

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

The Soonish opening theme is by Graham Gordon Ramsay. All additional music by Titlecard Music and Sound.

If you enjoy Soonish, please rate and review the show on Apple Podcasts. Every additional rating makes it easier for other listeners to find the show.

Listener support is the rocket fuel that keeps our little ship going! You can pitch in with a per-episode donation at patreon.com/soonish.

Follow us on Twitter and get the latest updates about the show in our email newsletter, Signals from Soonish.

We’ve spent decades trying to understand human biology, health, and illness at the level of our genes. For people with extremely rare genetic conditions, that work is finally starting to pay off. Thanks to the emerging field of hyper-personalized medicine, and the work of new organizations like the N-Lorem Foundation, we're entering a future where diseases linked to rare mutations don’t always have to be lethal.

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

The Soonish opening theme is by Graham Gordon Ramsay. All additional music by Titlecard Music and Sound.

If you enjoy Soonish, please rate and review the show on Apple Podcasts. Every additional rating makes it easier for other listeners to find the show.

Listener support is the rocket fuel that keeps our little ship going! You can pitch in with a per-episode donation at patreon.com/soonish.

Follow us on Twitter and get the latest updates about the show in our email newsletter, Signals from Soonish.

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