In this interview, Rachel Haurwitz, President and CEO of Caribou Biosciences, explains CRISPR‑Cas9 gene‑editing as a precise tool that cuts DNA at programmed locations using a guide RNA, likening it to a Microsoft Word for the genome. She outlines the vast medical potential, citing 6,000‑7,000 single‑gene disorders that could be treated and the promise of gene‑edited cell therapies such as off‑the‑shelf CAR‑T for cancers. Haurwitz predicts that within a few years CRISPR‑based treatments will move from clinical trials to commercial reality, though broader applications will take longer. Addressing ethical concerns, she emphasizes Caribou’s strong opposition to germline editing, embedding prohibitions in licensing agreements to prevent partners from using their technology for embryo editing. Currently, Caribou devotes resources to two pre‑clinical programs: “bugs as drugs,” which edits the microbiome, and allogeneic gene‑edited cell therapies for cancer. She expresses excitement about the progress and future prospects.
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Hi, I'm here with Rachel Horowitz, President and CEO of Caribou Biosciences. Thank you for joining me today. If I could just start with, with the technology that you, that you're involved in. If I'm saying it right, it's the CRISPR-Cas9 technology. Technology. Could you explain, as you've had to do many times, I'm sure, but could you explain to me what that is and how it works?
Sure. So CRISPR-Cas9 is a technology for gene editing, which is basically the ability to go inside of cells and precisely change DNA sequences. For a lay audience, I like to think of it as sort of a Microsoft Word for the genome. But of course, those of us who live in the life sciences know it's not that easy. And really fundamentally, it's about being able to cut the DNA at the exact site that we want to change it. And so the Cas9 system lets us do that in a very carefully programmed way. Cas9 is actually the name of a protein, and it's really the central player in this field. And it's able to cut the DNA, but it needs a piece of RNA to drag it to the right place in the genome. And so the scientist can design an RNA that takes it to a very specific place and make that cut. And then at that point, we're relying on the cell to actually repair that cut. Cells will die if they don't repair that break. And it's how they repair it that lets us ultimately edit the genome at that location.
And so what at the moment are the other medical, you know, what is the medical potential for that specific technology?
The medical potential for this is vast, and it will have a huge impact on the entire field of gene and cell therapies. There are, depending on who you ask, something like 6,000 or 7,000 different genetic disorders that are caused by a single gene mutation, many of which potentially could be treated, possibly even cured using a gene editing technology. Likewise, in the field of immuno-oncology, in the cancer space, there's huge, huge potential for using gene editing technologies to make gene-edited cell therapies like off-the-shelf CAR-T therapies and other programs to treat a number of cancers that are either untreatable or poorly treated today.
Yeah. And looking out at the time frame roughly, I mean, do you see— what do you see as that for when this is becoming used on a on a regular basis, if you like?
This field is moving fast, probably faster than anything we've ever seen before. Already there are multiple clinical trials ongoing testing CRISPR in a couple of different settings, and we'll see a wave of additional CRISPR and other gene editing technologies being tested in the clinic in the next few years. And so I think this is, in a handful of years, going to be a commercial reality. It'll be the tip of the iceberg, right, to ultimately get to many different kinds of cancer and many in genetic diseases will take a long development time.
A question I feel obliged to ask, I guess, is about the legal and ethical challenges. We've heard news from China and things like that about CRISPR, and maybe that's something that confuses the layperson on this as well. What's your reaction, though, to those sort of issues in that area of the debate?
Right. The ethical question is really important to us at Caribou and something that we've spent a lot of time thinking about and talking about. And it really comes down to the fact that you can modify different kinds of cells that has different potential for those changes. So you fundamentally can either edit a somatic cell or a germline cell. Somatic cell, of course, is most of the cells in our bodies. If that change is made in me, that change will die with me. It will not move on to future generations. However, you could, in theory, use this technology to edit the germline. So early embryos, sperm or egg. And that, of course, then has the potential to go on through many generations. We at Caribou feel very strongly that embryo editing is inappropriate. We do not do it ourselves. And in fact, we even bake it into license agreements where appropriate to prevent our partners from using our technologies to do germline editing as well.
And so where are you now then at the Caribou story? What's the future hold? Are things changing? Pretty rapidly there.
Yeah. So at Caribou, we're devoting most of our resources to developing gene-edited cell therapies in two primary areas. One is what we call bugs as drugs. So actually manipulating the microbiome using gene editing technologies. And the other is off-the-shelf gene-edited cell therapies for cancer. So allogeneic CAR-T programs and other programs like that.
Okay. So it sounds like an exciting point in your career and in the career of the company?
Absolutely. We're still in the preclinical stage, but very excited about the progress that we've made so far.
Well, thanks, Rachel, for joining us, and that's been most useful and illuminating. Thanks a lot. Okay. Thank you.
