Jun 12, 2026 · 18 min · 12 segments
We love to hear from our listeners. Send us a message. In Episode 3 of our in vivo-focused special series of Cell & Gene: The Podcast, Ascidian…
Mike EhlersGuestErin HarrisHostWe're going to get to specifically Ascidian's RNA exon editing approach a little bit later in our discussion.
Before we do, I want to talk a little bit more broadly about in vivo therapies and get your perspective on it.
Yeah, well, in terms of what do you think the field still needs to prove here, I can think of three things.
We need to consistently hit the right cells, achieve meaningful and lasting biology, and do it with safety profiles that scale beyond rare disease into broader populations.
And I think what the field still needs to produce is proof of reliability that these therapies behave as expected across patients and across disease, not just in idealized settings.
I do believe that kind of the end state of disease biology and therapeutic intervention will ultimately be forms of genetic medicine.
This will obviously happen at different paces and different indications, but I think it will expand over time as we solve some of these problems.
We're going to get to specifically Ascidian's RNA exon editing approach a little bit later in our discussion.
Before we do, I want to talk a little bit more broadly about in vivo therapies and get your perspective on it.
Yeah, well, in terms of what do you think the field still needs to prove here, I can think of three things.
We need to consistently hit the right cells, achieve meaningful and lasting biology, and do it with safety profiles that scale beyond rare disease into broader populations.
And I think what the field still needs to produce is proof of reliability that these therapies behave as expected across patients and across disease, not just in idealized settings.
I do believe that kind of the end state of disease biology and therapeutic intervention will ultimately be forms of genetic medicine.
This will obviously happen at different paces and different indications, but I think it will expand over time as we solve some of these problems.
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