Wilson disease

A patient and advocate.

Um, I had a transplant due to Wilson's disease in 2022.

Oh, wow.

So, um, um, Teresa Davison is with, uh, Mid-South Liver Alliance, and she invites me every year to come because I have the rare autoimmune disease of Wilson's.

I never liked a sweet potato until after I had a transplant.

Oh my goodness.

So, um, it's really, um, I should say rare, um, like one in 500,000 or something like that have Wilson's disease.

And I may be off on that number, but I think that's right, around that.

His achievements include the 2025 Breakthrough Prize in Life Sciences and the 2026 Franklin Institute Bower Award Prize for Achievement in Science.

And the day before we recorded this interview, Prime Medicine, a company he co-founded, announced that it had dosed the first patient in its trial of an in vivo prime editing treatment for Wilson disease, delivering the editing machinery directly into the body.

David also co-leads the Center for Therapeutic Genetics, working to bring individualized genetic medicines to patients with diseases too rare to attract conventional drug development.

For families like mine, the question is deeply personal.

So...

The first in vivo prime editing in a patient was just announced yesterday.

And that was Prime Medicine's dosing of an in vivo prime editor delivered with an LMP to the liver to correct a mutation in ATP7B that causes Wilson disease.

Wilson disease is also a severe disease that is characterized by having excessive copper levels in the blood.

Well, it came about, um, another one of those unfortunate, unplanned events in my life.

In '97, I was diagnosed with Wilson's disease, and I'd seen several specialists along my journey and ended up at the Mayo Clinic in Rochester, Minnesota, some of the greatest minds in the world.

Um, it was a very, very rare disease, so there wasn't a lot of people who could really pinpoint, you know, how it was gonna impact me and what the outcome was going to be.

And it's a terminal illness, and at the time in '97, there wasn't a lot of case study and research.