Jennifer Doudna

Thank you, Eric.

Great to be here.

Yes, we have.

We've known each other for a while.

And of course, I've admired the progress at the CZI on fundamental science.

I was an advisor very early on.

And I think actually that's how we got to know each other.

Right, Priscilla?

Yes, Eric, it's a very exciting story, and we're very, very proud of the teamwork that went into making it possible to cure baby KJ of his very rare disease.

And in brief, the story began back in August of last year when he was born with a metabolic disorder that prevented him from digesting protein.

It's called a urea cycle disorder and rare, but extremely severe and to the point where he was in the ICU and facing a very, very difficult prognosis.

And so fortunately, his clinical team at Children's Hospital of Philadelphia reached out to Fyodor Urnov, who is the director of translational medicine at the IGI here in the Bay Area.

They teamed up and realized that they could quickly diagnose that child because we had an IRB approved here at the IGI that allowed us to collect patient samples and do diagnosis.

So that was done.

We created an off-the-shelf CRISPR therapy that would be targeted to the exact mutation that caused that young boy's disease.

And then we worked with the FDA in Washington to make sure that we could very safely proceed with testing of that therapy initially in the lab and then ultimately in two different animal models.

And then we opened a clinical trial that allowed that boy to be enrolled with, of course, his parents' approval and for him to be dosed.

And the result was spectacular.

And in fact, he was released from the hospital recently.

as a happy, healthy child, gaining lots of weight and looking very chunky.