(Announcer For a conversation on the nex frontier in medical innovation.

Please welcome founder of the Innovative Genomics Institute and Nobel laureat in chemistry, Jennifer Doudna, with The Atlantics Jeffrey Goldberg.

(Instrumental music and applause (Jeffrey Goldberg) Thank you all.

Thanks very much.

It's a real pleasure and honor to have Jennifer Doudna with us today.

Winner of the Nobel Prize in Chemistry.

[applause] Thank you.

They've heard of the Nobel Prize, it's amazing.

The leader of the Innovative Genomics Institute, at Berkeley, professor at Berkeley, obviously one of the great innovators and inventors of our age.

I want to talk about, obviously, the signal, achievement of your career so far, Crispr, the invention of CRISPR technology.

I obviously can explain CRISPR, in a both erudite yet accessible way, but I thought I would let you do it.

(Jennifer Doudna I want to hear your explanation.

(Jeffrey) No, no.

I'll, I'll fill in the cracks after you go.

But maybe you should just explain what CRISPR is to our audience for a minute, and then we can go on from there.

(Jennifer) Well, Jeff, it's great to be here.

So CRISPR, what is CRISPR?

it's a technology that came out of curiosity driven science.

Never thought it would be where it is today, and I'm sure we'll get into that.

But it started as a projec to understand how bacteria fight viral infection.

So in nature, it's a bacterial immune system.

And by studying the chemistry of that system, how it works we figured out that it could be harnessed as a powerful tool to manipulate DNA precisely, making targeted changes t the letters of the genetic code, the code of life to understand the function of genes, and importantly, to actually alter the function of genes.

So it's become a tool that's now used globally in most biological research.

(Jeffrey) Right.

I want you to talk a little bit, you know, we're obviously in conversation on the stage this morning, has suggested this.

We live in a in a strange and discomforting and and sometimes unhappy, sometimes very unhappy age.

Possibly the best news, underreported, of the last few months has been news out of Philadelphia, out of Children's Hospital in Philadelphia.

There's a baby goes by there identifying the baby as K.J.

And I want you to talk for a minute about what CRISPR has been able to do for this baby and what it portends for the future.

(Jennifer) Well this is an extraordinary story.

The story began last August when this boy was born with a rare metabolic disease that made it impossible for him to digest protein.

So you can imagine that that creates a very severe, situation.

He was in the intensive care unit.

Bad prognosis.

Fortunately, his clinicians at the Children's Hospital of Philadelphia recognize that he had a genetic disease.

They were able to quickly diagnose that disease and create a CRISPR therapy.

And this was in partnership with a number of folks, including scientists at the Innovative Genomics Institute, to figure out how they could make a chang to this baby's DNA in his liver that would correct the disease causing mutation.

And the extraordinary outcome of that nine months later is that this boy was treated with the CRISPR therapy that was designed for him and manufactured in real time with, approvals from the Food and Drug Administration to proceed.

And now he's home with his family, and he appears normal.

So it's extraordinary.

(Jeffrey It's actually one of the most.

[applause] I know scientists don't traffic in terms like miracles, but it's a miracle of modern science.

(Jennifer) It feels that way.

(Jeffrey) The kid, no.

The kid is going to be able to live a normal life because something that you did so that's pretty amazing.

Talk about, talk about the wider application.

I know that you're focuse a great deal on rare diseases.

Maybe orphan diseases, however you phrase it.

Talk about the use of CRISPR in those and in the cost associated with it and sort of the balancing act that has to go on there.

(Jennifer) Well I think one of the very exciting things that happened in the in the field of gene therapy and CRISPR was at the end of 2023 when the Food and Drug Administration and the equivalent organization in the UK approved the first CRISPR therapeutic for a disease, sickle cell disease.

And again, it's an extraordinar story of teamwork of companies and academic researchers working over several years to develop a CRISPR therapy that would be curative or at least, palliative for peopl that suffer from this disease.

And so we now have a we're now in a situation where dozens of people have received this therapy.

I've met Victoria Gray, who is the first US patient to receive the CRISPR therapy.

She has an extraordinary stor of her life really transforming with, this this therapy, which is, by the way, a one and done treatment.

It's really a quite, a quite a change in the way we think about therapeutics, to think about something that could literall be delivered once into a patient and go to the source and make a change that is corrective for, for a disease like this.

(Jeffrey) One hack, one line of code, and then.

(Jennifer) That's the idea.

(Jeffrey) You're done.

(Jennifer) But, you know, you brought up the issue of cost.

And this is this is a real challenge right now.

Is that these therapies are quite expensive.

They cost a couple million dollars per patient.

So it's clearly a price point that won't be accessible to most people around the globe that could benefit.

And that's really where, you know, I feel motivation every day to change that situation.

I want to see us get to a stage where we can provide CRISPR therapies to people that can benefit from it.

We have to do that by reducing the cost, and we're going to do that through a combination of continued scientific and technology advances, but also working with manufacturers, working with companies, working with investors to make sure that we can, together, create a scenario, really a pipeline for these types of therapies that makes them much more affordable and beneficial for everybody.

(Jeffrey) Stay on the subject of K.J., this child, it's a heritable disease that the child, was suffering from.

But the fix is not going to transfer down the generation.

Is that correct?

(Jennifer) That's correct.

(Jeffrey) So, there are a lot of controversies about trying to fix diseases, fix flaws that extend past the individual patient to the next generation, generation after.

Talk a little bit about that, talk about the dilemmas, because you yourself have spoken about the danger or the worry about permanent fixes or fixing heritable diseases all the way down the line and try to just give us a little insight into what the what the benefits are and what the challenges o troubles are with all of that.

(Jennifer) Well, I' certainly I'm not against that.

But, I think I think the thing to keep in mind is that today, the therapies that we're talking about are therapies that, as you said, treat the individual.

They don't make germline changes.

They don't create heritable changes in DNA.

And that's because that's clearly, you know, very powerfu use of a technology like CRISPR that has to be carefully thought through, carefully regulated.

And I think that today there is not enough knowledge about how CRISPR really works in embryos or eggs or sperm to make it safe enough to proceed for probably any indication in the future.

Will that be possible?

I think it is.

And so this is why I've advocated for open, transparent discussions around how to procee with heritable germline editing.

We have to be able t you know, think that through and have a framework in place for the day that the the technology is ready.

(Jeffrey Are you worried now about people or countries, institutions doing, germline editing in ways that you don't approve of or you think is too dangerous or too many unknowns?

(Jennifer) Well, I think it's again, I just I think we have to be very thoughtful as we proceed with that type of use.

I think there could be cases.

And you brought this up where if you have a mutation that is inherited in a family and it causes very severe disease, and you have a technology tha can safely revert that mutation, you might argue that it would be unethical not to use it for that purpose.

(Jeffrey) Right.

Right.

By the way, as your as you're talking about, the conscientiousness with which you and your colleagues are bringing the concerns that you and your colleagues are bringing to this conversation, I'm thinking about the equivalent conversation in the AI world, which is not quit as conscientious at the moment.

I would say, about not knowing what a technology is going to do.

And being careful abou introducing it into the world.

What could bad actors do with CRISPR?

(Jennifer Well, we talked about heritable editing, making changes in humans that could enhance certain features, create further inequities across our society.

I think that would be very damaging.

(Jeffrey) That sounds like eugenics.

(Jennifer) It does.

Right?

Yeah.

And the other application that has received a lot of attention and discussion scientifically is the use of CRISPR in populations, not humans, but in, say, insects, where it might be possible to use CRISPR to create a, spread a trait very quickly through a population even create sterile, organisms.

Now, if you're applying it in mosquitoes, there could be public health benefits to that.

But there's also risk if something like that were to get out of control.

So there's been a lot of discussion.

(Jeffrey) What would be the risk of of making sterile, an unpopular insect?

How would it jump to something that.

(Jennifer) Oh, you're a bat and you eat mosquitoes... (Jeffrey) Right.

(Jennifer) It could be problematic.

(Jeffrey) It could be problematic.

So I think I thin we just have to be thoughtful.

When you have applications in the environment, there's.

You know, I'm from Hawaii, where there were a lot of, things that happened in the environment that turned ou to have unintended consequences.

So I think we just have to be careful.

(Jeffrey) Right.

You mentioned Hawaii.

Let's talk about that for a second, because I'm always fascinated by the motivation of someone who went into science for sciences sake for because you're curious talk about your youth on the Big Island, and how you came to the place you are today.

(Jennifer) Right.

Well, you know, my family moved to Hawaii in the early 70s, an it was an incredible experience.

We were in Michigan before that.

So it was a quite a culture shock.

I was about seven years ol at that time, and I found myself just surrounde by an incredible environment of of, you know, plants and animals that had adapted to this unique island environment.

And I was curious.

I thought, it's fascinatin that we have blind cave spiders.

I mean, how did these evolve?

I wasn't thinking anything about DNA in those days.

But as I got sort of... (Jeffrey) When did you realize that you were preoccupied with blind cave spiders?

Like what?

What?

At what age did you, do You remember saying, you know what?

That's, I mean, it's not a normal pursuit of an eight year old, but when did when did you realize, hey, there's something I can find out something?

(Jennifer) We did a lot of you know, it's very different, maybe from a lot of kids growing up now.

But, you know, I think in those days we did a lot of just running around outside and exploring every afternoon with our friends.

And so, you know, there was just all kinds of opportunities to observe these, these crazy creatures.

And I ended up, you know, taking a, chemistry class in high school with a teacher, Miss Wong, who was very, you know, very excited about the scientific method.

And she really taught kids that science isn't about memorizing a bunch of facts in a book.

It's about discovering things.

It's about asking a question “Gee, I wonder how that works”.

And then designing an experiment to figure it out.

And I just found that really exciting, (Jeffrey) Right.

Well, congratulations to Ms.

Wong for finding..., (Jennifer) Indeed, indeed.

(Jeffrey) for winnin a Nobel Prize by transference.

Talk a little bit about, yo mentioned the scientific method.

We were talking, yesterday, and I, I mentioned that, one of the most astonishing facts of American history is that George Washington, who ordered the inoculation of the Continental Army against smallpox, had a more, I don't know how you would phrase it, scientifically progressive understanding of vaccination than some people i our government 250 years later.

And as absurd as this is to ask, are you surprised that we've entered this period where provable therapies, proven through time and study, are being doubted in the way that they are?

(Jennifer) Well, Jeff, you know what I, what I would say is, you know, you could argue that vaccination has been so successful it's almost been too successful.

And, at least in this countr and in some parts of the world, we've been able to effectively wipe out certain diseases such that there's been a collective forgetting that, you know measles and mumps and rubella.

These used to be diseases that would, you know, kill people and fairl large numbers, mostly children.

And so I think, you know, we have to get back to, you know, just an understanding that vaccination is something that, you know there's a huge amount of data, as you said, it goes back a long time about how effective this approach is for protecting u from disease and protecting us at the population level from disease.

(Jeffrey) Why is this happening now?

I mean, frame it the way howeve you think about it, but, things that seem solid and generally accepted no longer seem solid and generally accepted.

We see this manifes in the wild cutting of budgets.

And let's just talk about the National Institutes of Health for, for starters, wild cutting and not logical cutting either.

Give us your understanding of what's going on and what the dangers are right now.

(Jennifer) Well, I'll just point out that currently Congress has, said they're going to support funding of the NIH and the the National Science Foundation at the current levels, which is which is good.

So hopefully that that continues.

But, you know, look, I mean I think we have to be thinking about the future of our country and where discoveries come from.

Government plays an incredibly important role in supporting innovative work and discovery research.

This is work that just, frankly doesn't get done in companies.

It can't because, you know, companies don't have the timelines.

They can't take the risk.

A lot of fundamental researc doesn't go anywhere or doesn't doesn't lead to, something that is curative of disease.

But every no and then you get a breakthrough and you don't know where thos breakthroughs are coming from.

That's what we want to support.

And by the way, government funding is essential for supporting the next generation of scientists, something that we're really working hard to do at Innovative Genomics Institute right now to make sure that scientists who are just starting their careers have opportunities to make breakthroughs that are going to drive the next generation of health car and other kinds of innovations.

(Jeffrey Talk about the role government funding, government support played in your career developmen that brought you to this place.

(Jennifer) Well, you know the story that comes to mind is, I remember when I was, you know, I was finishing college and I thought, you know, I really want to d more science.

How do I do that?

Well, maybe I'll go to graduate school.

I didn't really know what that was about.

I told my parents, you know, that I wanted to go to graduate school.

And, you know, my parents didn't have much money, and they were puttin two other kids through college.

And they said, well, you know we're really sorry, but we can't we just don't have the money to support you to do that.

You'll have to, you know you'll have to figure out a way to maybe work your way through that.

And I I found out that actually, the National Institutes of Health had a training grant program, which they still have, by the way, that supports graduate students in their, research and to get a PhD.

And so I called my father back and I said, you know, dad, I just found out that I' going to be paid $8,600 a year, which sounded like a lot at the time, to, to get an education, to get my PhD.

And he was stunned.

There was silence on the phone, and he was shocked.

And he was, of course, delighted.

But he said, you know, this is this is just extraordinary.

And he was astounded to find out that this was the case.

And so, you know, thi has really been true for decades now that the government has supported young students who want to get an education.

Why do we why do we do this?

Well, we use taxpayer money to do this because we understand that these are the student that are going to get trained, and they're going to go out there and make the next discoveries are going to drive business innovation.

They're going to drive academic innovation that keeps America number one in science.

(Jeffrey) I want to obviously, we're in this moment right now of confusion about public health.

We have a acting CDC director who's neither a physician nor a scientist, probably the first time in history.

There's all kinds of strange things going on.

What would you say if you were sitting with Donald Trump right now, which you're not.

What would you say to him about the importance of maintaining the integrity and primacy of the American federally supported health care research complex?

(Jennifer) Well, I point out that, you know, for every dollar of National Institutes of Health funding that's invested, we get $2.50 back.

That's a pretty good that's pretty good deal.

And that's been consistent over a long period of time.

And I want to keep America number one.

(Jeffrey) Come back and in our last few minutes, come back to the CRISPR.

We'r at the beginning of the story.

Take us five years ahead, ten years ahead.

And obviously no one's going to hold you to it, but.

But where do you think you're heading in this?

And what do you thin you're going to be able to solve in the next five, ten, 20 years with this technology that you've developed?

(Jennifer) Well, Jeff I'm incredibly excited right now because I think we're seeing an extraordinary moment in which CRISPR is just beginning to have real worl impact across a bunch of sectors where there's, you know, huge opportunities for growth.

And this will happen both through nonprofits and academic work.

But frankly, through a lot of, company and investor work as well.

So we talked about baby K.J.

We talked about the sickle cell disease therapeutic.

I think there's an opportunity right now to expand the way we're treating rare disease.

Frankly, probably this won't be done in pharmaceutical companies, at least not right now because the market size is you know, is small.

Although if you add up all the people affected by rare disease, it's actually not a small number.

So what I'd like to see u do over the next 5 to 10 years, I hope it doesn't tak ten, is to get to a point where we have a very clear, smooth pipeline for getting from diagnosing a rare disease to creating the therapy that's going to be, the appropriate treatment for that person and then getting it to them and doing it in a cost effective way.

So that's something we're focused on right now with a new center that we've created in San Francisco to do this, and we hope to roll it out at a number of other medica centers over the next few years.

And then we didn't speak about applications in other areas... (Jeffrey) Agriculture I wanted you to address very excited about the applications in agriculture, because I think that what I'm seeing, and this is not research that I'm doing myself, but I see it going on with my colleagues and in companies, is that it' increasingly possible to make 5 or 10 genetic changes in plants at a time with CRISPR that create the kinds of traits that make these plants more productive, drought resistant, less, les requiring of nutrient additions.

So they're beneficial to farmers.

They're beneficial people.

And by the way, CRISPR is a precise tool, so it doesn't make random changes along the way.

It does things precisely.

So it creates a much more precise way to do plant breeding in the future.

(Jeffrey) Right.

Right.

Give us what do we have we seen anything yet in the, in, in the market that has benefited from CRISPR on the agriculture side or is it still theoretical?

It's not theoretical.

So there's a there's a CRISPR tomato out there that has claimed nutritional value.

There are, there are drought resistant versions of rice that have been created that are being deployed already in South America and are being tested in other countries.

I think these are some of the types of applications, and I know of a number of other things that are being developed and companies that are also very exciting that involve increasing plant productivity, but without increasing nutrient requirement.

(Jeffrey) I was just picturing a you're talking about the tomato, I mean, the obvious joke is that it's a tomato that will walk right into your basket, without even picking up, but which comes to the science fiction, you know, which comes to the science fiction and the and and the possibly dystopic applications.

How in this period of widespread mistrust of science, do you convince people tha changing the genetic structure of a food you're going to eat is actually healthy.

You have an FDA that you still trust?

I think?

(Jennifer) We have to work with the FDA.

(Jeffrey) We have to work with the FDA.

Do you trust the FDA the same way that you used to trust the FDA in the past?

(Jennifer) Well, you know, in selling gene therapy, which is the only area where I really have had interactions with the FDA, mostly through my colleagues.

We've seen real support for for CRISPR and for for, selling gene therapies more broadly.

So, let's hope that continues.

(Jeffrey) I guess what I'm asking you is to articulate in just a few seconds, if you can, the argument, the best argumen someone from science can make to the American peopl that you can trust scientists, not all scientists, obviously, but that you can we're entering this dangerous phase of widespread almost axiomatic distrust.

What's the argument about the way science is done in America that actually would convince people that the science that is being done is actually scientifically proven?

(Jennifer) I mean, scientists work with data, right?

I mean, we love data.

We love facts.

We love information.

We also recognize that, you know, not all data is of the same quality.

So you have to be a little bit, you know, you know, you have to be discerning about it.

But I just would invite people to, you know, look into the dat behind statements that are made.

I think it's dangerous to look at a TikTok video or, you know, take something off, the internet that is unvetted as fact.

You really need to dig in a little bit and figure out where it came from.

(Jeffrey) I look forward to your appearance on The Joe Rogan Show.

That's all I can say.

I want to thank you very much for coming here.

I know it's fascinating to our audience.

Thank you so much for doing this.

I appreciate it.

[applause] [instrumental music]