Jennifer Doudna

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.

So really exciting, yeah.

Yeah, very good point.

So yeah, we used a version of CRISPR that was created by David Liu at the Broad Institute and published and available.

And so it was possible to create that, again, targeted to the exact mutation that caused baby KJ's disease.

And fortunately, there was also an off-the-shelf way to deliver it because we had access to lipid nanoparticles that were developed for other purposes, including vaccinations.

And the type of disease that KJ suffered from is one that is treatable by editing cells in the liver, which is where the lipid nanoparticle naturally goes.

So we were, you know, there were some, definitely some serendipity here, but it was amazing how all of these pieces were available.

We just had to pull them together to create this therapy.

Right.

Well, the IGI is a joint institute, as you probably know, Eric.