Eric Topol
๐ค SpeakerAppearances Over Time
Podcast Appearances
You know, we recently spoke, I know, on Ground Truths about things as diverse as editing the gut microbiome for an asthma and potentially someday Alzheimer's.
But here you were very much involved, IGI, with the KJ, baby KJ microbiome.
Muldoon.
Maybe you could take us through this because this is such an extraordinary advance in the whole CRISPR cure story.
It's so amazing.
I don't think people necessarily graph this.
This timeline that we'll post with this,
It's just mind-boggling how you could, as you said, Jennifer, in about six months, to go from the birth and sequencing through cell-specific cultures with the genome mutations, through multiple experimental models with non-human primates even,
looking at off-target effects, you know, through the multiple FDA reviews, and then dosing, you know, cumulatively three dosing to save this baby's life.
It's really just amazing.
Now, that is a template.
And before we go to this new center, I just want to also mention not just a timeline of compression, which is unimaginable,
and the partnership that you've had at IGI with, I guess, Donaher to help manufacture, which is just another part of the story.
But also the fact that you're not just even with CRISPR 1.0 as being used in the approvals previously for sickle cell and beta thalassemia, but now we're talking about base editing in vivo, in the body, using mRNA delivery.
So maybe you could comment on that, Jennifer.
Yeah, no, it is amazing.
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.