Co-founder, Mammoth Biosciences
Jennifer Doudna Reflects on Long Path to Making CRISPR Gene Edited Therapies More Accessible
In recognition of GenomeWeb's 25th anniversary, we are conducting a series of interviews with leaders in the field to reflect on the last 25 years of genomics, as well as what the future may hold.
The full series is available here: https://www.genomeweb.com/topic/genom...
In this interview, we speak to CRISPR pioneer Jennifer Doudna, a professor in the chemistry department and the molecular and cell biology department at the University of California, Berkeley. Doudna in 2012 developed the CRISPR gene-editing technique with Emmanuelle Charpentier, earning them the Nobel Prize in chemistry in...
About Jennifer Doudna
Jennifer Doudna, co-founder of Scribe Therapeutics and a Nobel laureate, appeared on Bloomberg Technology on July 24, 2026, following Scribe Therapeutics' IPO, which raised $128.7 million. Doudna discussed the company's lead therapy, which aims to lower LDL cholesterol with a single treatment using "epi editing," a method she described as making changes in DNA that are not permanent but alter protein production. She stated that this approach could allow the therapy to be used "safely and effectively for common disease." Doudna also commented on the role of artificial intelligence in science, saying that while AI is "an incredible tool" that can accelerate work, it "doesn't replace scientists" and that she does not see AI coming up with "brand new idea[s]." In a June 24, 2026, interview on "The Circuit" with Emily Chang, Doudna reflected on the pace of CRISPR's commercialization, stating that the promise of the technology was not overstated but that "we're just early." She expressed a desire for a future where patients with rare diseases can be quickly diagnosed and receive a genetic therapy through a "smooth pipeline." Doudna also addressed the impact of funding cuts to scientific research, calling them a risk to the United States' economic success in science and technology.
Transcript (17 segments)
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Jessica Kim Cohen0:00
In 2012, Jennifer Doudna developed the CRISPR gene editing technique with Emmanuelle Charpentier, earning them the Nobel Prize in Chemistry in 2020. GenomeWeb reporter Jessica Kim Cohen spoke to Doudna about the impact CRISPR has had in rare disease and her recent work to improve human and planetary health.
There have been some major CRISPR advancements in patient care in recent years. We've had the FDA approval of the first CRISPR-based treatment for sickle cell disease. The first infant was treated with a personalized CRISPR base editing therapy earlier this year. I'm curious, is this what you envisioned when you discovered how CRISPR-Cas9 could be used to edit genomes? And what do you think 2025 could look like for CRISPR in patient care?
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Jennifer Doudna0:50
It's just extraordinary what's happening right now in the world of CRISPR, especially in terms of therapies. It's an amazing time. Could I have predicted any of this? No. Did I hope that we would see a day like this? Yes, absolutely. It's very hard often to predict the timing of technology impacts. You can see capabilities with technologies that are there, but for a variety of other reasons, it can often take a long time for those to actually come to bear in the real world. I think what's one of the really exceptional things about CRISPR is that, partly because so many people have gotten involved in the field, we have now wide-ranging participation from not only scientists like me that are doing very fundamental research, but also clinicians, regulators, stakeholders like patient advocacy groups, foundations. It's really been exciting to see all of those different players coming together to make sure that we move the field forward as quickly as possible.
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Jessica Kim Cohen1:56
And looking back over the last 25 years, in your view, what has been the most important genomics advancement in medicine?
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Jennifer Doudna2:05
This really goes back to actually the first whole genome sequence for a bacterium published in 1995. I was a young professor at the time, and that really did kick off an era in which we've been able to take advantage of those kinds of data. But of course, sequencing data don't mean much if you don't know what they encode. We have really made a lot of progress in the last 25 years in dissecting the coding of various kinds of molecules in the genome, not just proteins but also RNA molecules, some near and dear to my own heart, and the kinds of interactions between those components that make it possible to do things like edit genomes with CRISPR. So I think it's been extraordinary to see these things coming together: sequencing information, being able to understand the content of the genome, and now with technologies like CRISPR, being able to rewrite that content. It's an extraordinary combination.
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Jessica Kim Cohen3:13
I know you founded the Innovative Genomics Institute as a collaboration between UC Berkeley, UC San Francisco, and UC Davis with a goal of using genome engineering to support human health and well-being. Of the projects that the institute is working on, what are you most excited about right now?
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Jennifer Doudna3:31
Well, I should say we're working on healthcare at large, so human and planetary health. I'm very excited about what we're doing with microbiome editing. There's a large cohort of scientists now that we've hired who are funded in part by the TED Audacious program, and they are teaming up to use CRISPR in microbes in ways that are allowing them to manipulate individual genes or pathways in individual species, but in the context of a whole microbiome. Whether it's in the human gut, the cow rumen, or the soil, we're starting to get insights into the behaviors of these organisms in their native contexts, as well as to be able to manipulate what they're doing there. I'll just give you one quick example: in the cow rumen, we know that microbes are responsible for producing methane, one of the most powerful greenhouse gases emitted by human activities, namely agriculture. Imagine a day when we can edit the microbes that do that to redirect their metabolic pathways to producing more milk and meat in cows and no methane.
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Jessica Kim Cohen4:48
And researchers at the Innovative Genomics Institute were involved in the first personalized CRISPR therapy that was given to baby KJ earlier this year. How far off are we from that becoming a common way for treating patients with these types of extremely rare genetic diseases, and what needs to happen in the industry to get more of these personalized therapies to patients?
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Jennifer Doudna5:11
We were delighted to support the clinical team at the Children's Hospital of Philadelphia for that extraordinary case with baby KJ and being able to create a CRISPR therapy for him in real time. But what do we do to take that N-of-1 example and expand it to so many others that could benefit from that kind of therapy? There's really two big barriers that I see. One is access: how do we do this in more places so that more people can actually interact with clinical teams that have the capabilities that we saw so critically applied for baby KJ? And the other is cost: how do we reduce the manufacturing costs of these kinds of molecules and even the time it takes to make them and test them? I think that's going to be critical. It's a combination of figuring out how to make the molecules more inexpensively, but also working with regulators to make sure that we have a safe but streamlined process for testing.
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Jessica Kim Cohen6:17
There's been much reported on the patent dispute over the use of CRISPR-Cas9. What are the implications of that dispute for the biotech field, and has that dispute changed how you think about scientific discovery?
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Jennifer Doudna6:33
What I've seen as somebody working away in the lab and really working on the kinds of applications that we've been talking about that I think are going to be so impactful is that, lawyers aside, we're not stopping or slowing down the research that we're doing, especially in a nonprofit setting. It really does not impact us much at all. So that's a good thing. I think it's not slowing or stalling the science. I think this is an important thing to keep in mind: why do we patent things? The idea there is really to protect ideas and discoveries and technologies so that they can be deployed commercially in a process that might take many years and still be able to recover value for investors. So that's definitely important in a company setting. But when we're talking about academic labs and nonprofits, as we've been discussing here, we can just forge ahead. It's an important thing to keep in mind. It can be an important advantage in terms of nonprofit activities.
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Jessica Kim Cohen7:40
What do you think that precision medicine will look like in the next 25 years?
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Jennifer Doudna7:45
Boy, it's always hard to predict even a year or two ahead, so 25 is hard to say. But I guess what I hope happens here—I'll tell you what I hope happens. I really would like to see CRISPR become standard of care in certain diseases. Right now I'm really excited about the momentum that we have. I think with baby KJ in particular, as well as the FDA-approved therapy of Casgevy for sickle cell patients, there's been a lot of momentum created because a lot of people now see what's possible. They're not imagining it anymore.
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Jessica Kim Cohen8:24
I know that some people have worried that gene editing could further stigmatize certain genetic illnesses and disabilities, or create a future where only those with the means to access pricey treatments can garner their benefits. How can the healthcare and the biotech industries contend with those concerns?
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Jennifer Doudna8:45
It's very important to understand what it's going to take to make sure that a very exciting technology like CRISPR is ultimately made available to everyone that can benefit from it. That's a tall order for sure. One of the really important organizational principles of the Innovative Genomics Institute when we started it 10 years ago was exactly that. It was saying, 'Look, we're researchers at a public university. We want to make sure that our work ultimately benefits everyone that can be helped by it.' It'll keep me busy for a while, for sure, but it's amazing to see how inspiring that vision is to so many people.
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Jessica Kim Cohen9:29
As we're thinking about this work, of course genetics experts will need to be involved as gene editing continues to move into patient care. What other experts or types of organizations also need to be engaged?
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Jennifer Doudna9:43
Well, I think it's been great to have all of the interactions that we've had over the years at the IGI with bioethicists in particular, people who are thinking deeply about the future of the human race and the future of our societies globally. The challenges that we face are really very large, and how does genome editing intersect with some of those challenges? We want to make sure that we're using it to make the world a better place. We need to be working with people who are not scientists themselves, but are informed about science and can help the scientists like me understand all of those other aspects that come into play when you think about applications of very powerful and very exciting but potentially risky technology. And by the way, we're seeing this playing out with AI right now as well. There are lots of groups that are forging ahead with technology development for AI, but I think that ultimately they need to be working with people that are outside the field but can provide perspectives that are so essential, so critical for ensuring that powerful technologies are used responsibly.