Jason Mast

But the advancements had come to the point where you could make these really fine-tuned changes in DNA. And that... both creates some opportunities and some challenges. And the opportunity is there you can like treat as, you know, happened with KJ ultimately.

You can treat an individual patient's mutation, but you can only treat maybe that patient or this small handful of patients with this one very precise mutation. In cases like KJ, you're going to need to actually make that treatment really quickly.

You can treat an individual patient's mutation, but you can only treat maybe that patient or this small handful of patients with this one very precise mutation. In cases like KJ, you're going to need to actually make that treatment really quickly.

You can treat an individual patient's mutation, but you can only treat maybe that patient or this small handful of patients with this one very precise mutation. In cases like KJ, you're going to need to actually make that treatment really quickly.

And so they were like, can we do this in time ourselves? Can we go from an infant and make a treatment in time to help them?

And so they were like, can we do this in time ourselves? Can we go from an infant and make a treatment in time to help them?

And so they were like, can we do this in time ourselves? Can we go from an infant and make a treatment in time to help them?

Yes. And they had been running what they called sort of time trials where they like would pick a variant that's in the literature or that Becca would encounter. And they would see how fast can we do this? Not with any intention of we're going to put this in a patient, but we can see how fast can we do this?

Yes. And they had been running what they called sort of time trials where they like would pick a variant that's in the literature or that Becca would encounter. And they would see how fast can we do this? Not with any intention of we're going to put this in a patient, but we can see how fast can we do this?

Yes. And they had been running what they called sort of time trials where they like would pick a variant that's in the literature or that Becca would encounter. And they would see how fast can we do this? Not with any intention of we're going to put this in a patient, but we can see how fast can we do this?

And initially it takes over a year, and that was a couple years ago, but they get faster and faster until they can do it in what seems like a number of months. And what that starts is basically a six-month sprint to can we build this therapy in time? And he brings in researchers and companies.

And initially it takes over a year, and that was a couple years ago, but they get faster and faster until they can do it in what seems like a number of months. And what that starts is basically a six-month sprint to can we build this therapy in time? And he brings in researchers and companies.

And initially it takes over a year, and that was a couple years ago, but they get faster and faster until they can do it in what seems like a number of months. And what that starts is basically a six-month sprint to can we build this therapy in time? And he brings in researchers and companies.

in California, in Boston, in Vancouver, in Iowa, all sprinting, working overtime to see can we build this treatment before KJ either needs a liver transplant or has an ammonia attack that's going to really cause some long-term damage.

in California, in Boston, in Vancouver, in Iowa, all sprinting, working overtime to see can we build this treatment before KJ either needs a liver transplant or has an ammonia attack that's going to really cause some long-term damage.

in California, in Boston, in Vancouver, in Iowa, all sprinting, working overtime to see can we build this treatment before KJ either needs a liver transplant or has an ammonia attack that's going to really cause some long-term damage.

They managed to get it tested in mice and monkeys. They managed to, you know, do some testing on it to make sure that it doesn't accidentally edit the wrong portion of the genome. They get companies who basically are willing to, like, manufacture this at relatively low cost or for free. It's not clear exactly... But they were able to sort of cut a deal with these manufacturers.

They managed to get it tested in mice and monkeys. They managed to, you know, do some testing on it to make sure that it doesn't accidentally edit the wrong portion of the genome. They get companies who basically are willing to, like, manufacture this at relatively low cost or for free. It's not clear exactly... But they were able to sort of cut a deal with these manufacturers.

They managed to get it tested in mice and monkeys. They managed to, you know, do some testing on it to make sure that it doesn't accidentally edit the wrong portion of the genome. They get companies who basically are willing to, like, manufacture this at relatively low cost or for free. It's not clear exactly... But they were able to sort of cut a deal with these manufacturers.

And at six months, baby KJ is treated with the first dose. This is in February. It's not amazingly effective, the first one, because it's a very small dose. They want to be very careful. But then they go for a second dose a few weeks later. And then the third dose, not yet clear how the third dose has gone.