Eric Topol
๐ค SpeakerAppearances Over Time
Podcast Appearances
So that, I think, is a great substrate for starting a new center.
And so maybe back to you, Priscilla, as to what your vision was when you're working with Jennifer and IGI to go through with this.
Yeah, well, it's pretty remarkable because here you have incurable, ultra rare diseases.
If you can help these babies, you know, just think of what this could do in a much broader context.
I mean, there are a lot of common diseases have their roots with some of these very rare ones.
So how do you see going forward, Jennifer, as to
where you see Berkeley, Gladstone, UCSF.
I'm envious of you all up there in Northern California, I have to say.
We'll pull this off.
How will you get the first similar case to KJ Muldoon going forward?
Do you think it's possible to go any faster right now than the six months that it took for KJ?
Yeah.
Yeah, and I also would note, as you both know well, this administration is really keen on genome editing, and they've had a joint announcement regarding their support.
And in my discussions with the FDA commissioner, this is something I'm very excited about.
So this timing of the new center
for pediatric CRISPR cures is aligned with the current administration, which is good to see.
It's not always the case.
Now, going back, Priscilla, to your point that not just for the liver, because delivery has been an issue, of course, and if we're going to try to get after a lot of these really rare diseases, it's going to go beyond the liver.
So this is also an exciting new dimension of the center, as you said, to go after the bone marrow for
hematopoietic cells, perhaps other organs as well?