Priscilla Chan

Two, they can make research so much better due to their insights as patients and patient advocates.

And I think they close the distance between basic science and impact on patients.

And so we've been working on that since 2019 and has been a passion of ours.

I think the thing that's incredibly exciting, you mentioned that at CZI, our mission is to cure, prevent, and manage all disease.

And when we talked about this 10 years ago, it felt like this far-off idea that

but every day it seems closer and closer.

And I think the, the part that's super exciting about this is the direct connection between the basic science that's happening in CRISPR and the molecular and sort of down to the nucleotide understanding of these mutations and the ability to correct them.

And it's, it's,

I think many of us have our imaginations have included this possibility, but it's very exciting that it has happened with Baby KJ and CHOP.

And we need to be able to do the work to understand how we can do more.

treat more patients this way, how to understand the obstacles, unblock them, streamline the process, bring down the cost so that we better understand this pathway for treatment, as well as to sort of increasingly democratize access to this type of platform.

And so our hope is to be able to do that, you know, go from take the work and inspiration that IGI and the team at CHOP have done and continue to push forward and to look at more cases, look at more organ systems.

We're going to be looking in addition to the liver at the bone marrow and the immune system.

and to be able to really work through more of the steps so that we can bring this to more families and patients.

I mean, with the expertise and feasibility, the immune system is going to be the next target.

Jennifer Puck has been a pioneer in this work.

She's the one who designed the newborn screen that will be the tool that picks up these patients as they are born.

And I think the thing that's tremendous is the immune system, first of all, is active in many, many diseases, not just these cases of children born with partial or absence of immune systems.

And the course right now that these babies are left with is complete isolation and then a very long and arduous course of a bone marrow transplant with high morbidity and mortality.

And even if after the transplant, you have complications like graft versus host and immunosuppression.