Joe Kernen (1:40)

I don't know if they need chastising or government intervention to tell them how to look.

Becky Quick (1:46)

What I will say is that in an industry like defense where it is so reliant on government spending, maybe they.

Joe Kernen (1:53)

Should have more of a say.

Katie Kramer (1:55)

Plus, a special conversation as we launch CNBC's newest project. CNBC cures 1 in 10Americans affected therapies disparate and slow to reach patients. It is a deep and personal dive into rare disease. Former FDA commissioner Dr. Scott Gottlieb.

Scott Gottlieb (2:12)

Regulation is a roadblock here because it's still hard to do these so called NF1 trials. I think while the FDA has opened up a pathway to this, the FDA hasn't created sort of a reproducible framework.

Katie Kramer (2:23)

It is Thursday, January 8, 2026. Squawk Pod begins right now.

Tom Friedman (2:30)

Stamp Becky by in 3, 2, 1.

Announcer (2:33)

Cuba please.

Becky Quick (2:35)

Good morning everybody. Welcome to Squawk Box right here on cnbc. We are live from the NASDAQ market site in Times Square. I'm Becky Quick along with Joe Kernan and Andrew Ross Sorkin.

Andrew Ross Sorkin (2:45)

President Trump saying that the US could be running Venezuela and pumping oil in the South American country now for years in an interview with the New York Times, the President suggesting the US would maintain oversight over Venezuela for much longer than a year. But he didn't say what might prompt him to send American troops into the country. Now in other Venezuela developments, the President saying on his social media network that Venezuelan would be buying only American made equipment with funds that the country gets from ramping up oil output. Reuters reporting that Chevron is in talks to expand its Venezuela license so it can increase oil exports to its refineries and sell to other buyers. Meanwhile, the Energy Secretary Chris Wright, who was at that Goldman Sachs conference yesterday, telling CNBC that the debts that Venezuela owes ConocoPhillips and Exxon Mobil are not an immediate priority for the administration. So this, you know, to the extent that people thought that money could get shifted from one place to the other, this coming amid a backdrop of the US seizing these two sanctioned oil tankers, including one on the Coast Guard coast that the Coast Guard had been chasing for weeks, that recently flagged itself as Russian. So a whole bunch of headlines embedded in the headlines.

Joe Kernen (3:57)

So we don't want the Trump administration seizing the means of production in the United States and be coming like China and being a state run oil company. How about if we start running the means of production in other countries?

Andrew Ross Sorkin (4:11)

Well, that's what we're doing.

Joe Kernen (4:13)

That's what I mean. But is that I'm like trying to think about how I feel about that. I mean compared to having socialists run it down there where no one gets any of the oil basically because they don't get any of it out of the ground. But it's something, it's like this weird hybrid economic model that we're watching play out. But across the board and all kinds with Nvidia and with all these things, it's like this new way of thinking about things that does involve this is.

Andrew Ross Sorkin (4:40)

This is state sponsored capitalism.

Joe Kernen (4:42)

But now. But it's down in Venezuela.

Andrew Ross Sorkin (4:44)

Yeah, but it's here too.

Joe Kernen (4:45)

Well, it's not really tier 2. I don't agree with that. I think it's a hybrid model. It's nothing like China or I've heard you say, but I don't think it's anything like China.

Becky Quick (4:52)

We had that guest from the CIA who said that Pete Vesa used to be one of the best run companies. It was really when Hugo Chavez came in and the corruption came in that things took a real turn for the worse. When the assets were not flowing, the money was not flowing back to the Venezuelan people, it was getting siphoned off along the way.

Joe Kernen (5:10)

But even if you are a purist about not wanting that in this country, what is this? This is us running the means of production in another country for the good of the Western hemisphere, maybe. And for the good of. I think he wants. But what I said oil down at $50 a barrel and I think he wants whatever flows through to the economy in terms of inflation and.

Andrew Ross Sorkin (5:34)

But you're not in what I imagine has to be boots on the ground.

Joe Kernen (5:38)

I mean, I don't know how you.

Scott Gottlieb (5:39)

Stable.

Joe Kernen (5:40)

We have Tom Friedman. If you can't stabilize, it's important to have a democracy in Venezuela, obviously. And this person that's there right now, I don't know. We have a terrorist. We have a former al Qaeda guy in Syria. Can. Can she change her stripes to be a capitalist or free market person? I don't know. Maybe she's.

Becky Quick (5:59)

Tom's point is if you have us running what is basically the mob there.

Scott Gottlieb (6:05)

Right.

Joe Kernen (6:06)

I don't think he. Friedman didn't, he didn't sound like he thought this was a bad thing that was happening in Venezuela.

Becky Quick (6:12)

He just said democracy is there.

Joe Kernen (6:14)

He would like to see.

Becky Quick (6:15)

He would like to see the democracy is there and that you can't guarantee for any of these big oil companies that they're going to have stability if you are not enforcing democracy.

Andrew Ross Sorkin (6:23)

And if you're not enforcing democracy, you are going to need these boots on the ground. And then what is the cost of that? And this gets to the whole sort.

Joe Kernen (6:29)

Of, oh, that horrible forever war type.

Andrew Ross Sorkin (6:32)

Forever war type thing. That's everything that he actually said he wasn't going to do. Right.

Scott Gottlieb (6:35)

But.

Joe Kernen (6:36)

Well, there's a piece in the Journal Day says that Trump rethinks regime change. It's like this can be something. Is there any way regime change. I don't want to orchestrate it, but if organic regime change were to happen in Iran, is there any way that's a bad thing? No way that's a bad thing. Unless the. I guess unless the new regime was. Unless the new regime was worse. Sometimes the new regime is worse than the old regime.

Becky Quick (6:59)

Hard to imagine in the case it is.

Scott Gottlieb (7:01)

It is.

Joe Kernen (7:02)

Since 1979, we've gotten a pretty good look at exactly what has happened. I feel bad for the people in Iran. President Trump saying on Truth Social yesterday that the 2027 military budget should be $1.5 trillion rather than 1 trillion. That comment is lifting global defense stocks this morning and it followed post earlier in the day pushing for a crackdown on financial moves that the financial. That the defense industry makes like buybacks or dividends. In an earlier post on True Social, the president said companies are buying back too much in stock and paying too much in dividends while not investing enough in plants and equipment. In his words, he said that this situation will no longer be allowed or tolerated. He also slammed executive pay packages as unjustifiable given companies slow pace of deliveries, and said no defense executive should be allowed to make more than $5 million a year until companies build modern factories. In a separate post, the president singled out Raytheon, which is owned by rtx, accusing it of, in his words, the most aggressive spending on their shareholders rather than the needs and demands of the United States military. And shares of defense companies fell sharply during regular trading but gained after hours after the president's comments on budget like any of this really, I have to.

Becky Quick (8:26)

Say Elizabeth Warren does right.

Joe Kernen (8:28)

But I mean, it's a world where we've depleted a lot of weapons. I understand that. And I think we need to make sure that we restock a lot of these because it's a dangerous world. I would think that companies normally would do the right thing through market forces in terms of building out factories, modern factor, doing whatever they had to satisfy the markets. I would think they would do that. I don't know if they need chastising or government intervention to tell them how to spend money.

Becky Quick (9:01)

Look, what I will say is that in an industry like defense, where it is so reliant on government spending, maybe.

Joe Kernen (9:08)

They should have more of a say.

Becky Quick (9:09)

Maybe you should have a little bit more of a say. And I would, I would say there will be people who argue. The same is true of healthcare, where Medicare, pharmaceuticals and Medicaid is paying a huge chunk and basically setting the agenda for what the insurance companies will cover as well. It's complicated, but it's a populist argument. It's one where he is going to find agreement with a lot of Democrats, prominent Democrats that come up with.

Joe Kernen (9:34)

I bet you he doesn't. They might. They'd turn into Republicans if they had to agree with Trump.

Becky Quick (9:39)

No, I would think that they would say. I would think Elizabeth Warren, I'm not even joking. I would think that she would say, yes, we are in favor of making sure that if we're spending this money, and maybe I'm wrong, but if we're spending this money, that it's not just going to go to the executive's pockets.

Joe Kernen (9:56)

He would still call her Pocahontas. Even if she does do that. He would still call her Pocahontas even if they did agree with what another aren't they?

Becky Quick (10:05)

But it's interesting these next stories we're talking about are going to be President Trump agreeing with Gavin Newsom on something.

Joe Kernen (10:11)

Getting back to market for a second. You saw the market today and you saw the market yesterday.

Becky Quick (10:14)

Yeah.

Joe Kernen (10:15)

First thing I did was when was that Wall Street Journal article that said everybody on Wall street says it's going to be a great year? I looked it up.

Becky Quick (10:21)

I think it was Monday.

Joe Kernen (10:22)

No, I think it was Tuesday.

Becky Quick (10:23)

Oh, was it Tuesday.

Joe Kernen (10:24)

A day that will live in infamy. Because it was.

Becky Quick (10:28)

But what they gave back yesterday was just what they added the day before, kind of.

Joe Kernen (10:32)

But now we're down.

Becky Quick (10:33)

We'll see two days in a row.

Joe Kernen (10:35)

I just don't like it. I don't like like that on the front.

Becky Quick (10:38)

Yeah, it makes you nervous. It'll be even better when we get to Davos and we hear what everybody has to say.

Tom Friedman (10:44)

Tease will be next.

Katie Kramer (10:46)

Coming up on Squawk the path forward for Venezuela, we're joined by Pulitzer Prize winning New York Times opinion columnist Tom Friedman.

Tom Friedman (10:55)

The notion that we can decapitate this regime, leave the incredibly corrupt system that Maduro led behind with all the same people and then say to our oil companies, hey, please come in and start pumping oil. I think that's an illusion. Our oil companies need a legal structure in order to operate there.

Katie Kramer (11:14)

Planning a sustainable economic future for a country very much in flux. Right after this break.

Becky Quick (11:24)

What made you confident that you could do something that hadn't been done before? I have no fear of failure.

Katie Kramer (11:30)

Trailblazing women, changing the game.

Becky Quick (11:32)

One of my favorite pieces of advice, think about what your boss's boss needs.

Katie Kramer (11:37)

Leadership can look in many, many different forms. It really does come down to just trusting yourself.

Becky Quick (11:42)

Life is short and you just gotta think big to accomplish big things.

Katie Kramer (11:46)

Julia Boorstin hosts CNBC Changemakers and Power Players. New episodes every Tuesday. Wherever you get your podcasts, This is Squawk Pod.

Andrew Ross Sorkin (12:00)

You're watching Squawk Box on cnbc. I'm Andrew Ross Sorkin along with Joe Kernan and Becky Quick.

Becky Quick (12:04)

Right now we want to get into the situation in Venezuela. New York Times opinion columnist Tom Friedman's latest piece is titled if Trump doesn't bring democracy into Venezuela, he will never get much oil out of it. Tom Friedman himself joins us right now. And Tom, it is great to see you. We've been talking about this situation since it first evolved all week. Your take is that there's no way they can leave the previous administration and setup that is there and think that this is going to work. Why don't you lay out your views on this?

Tom Friedman (12:38)

Well, you know, the situation basically is we removed Maduro, the president of Venezuela, the leader of basically a mafia regime, but we left the mafia regime in place. And now President Trump is basically saying we are taking over Venezuela's oil exports and production. And you'll be meeting with major oil companies, American oil companies, tomorrow at the White House to get them to go in and begin exporting, rebuilding the infrastructure of Venezuela's oil sector and then exporting their oil. And the point I'm trying to make is if, if you're a major American oil company, if you're Exxon or Conoco or Chevron, Chevron's in Venezuela still in a limited way. And Conoco and Exxon were there. Their properties were expropriated. For them to go back, you need a legal structure. They need a new hydrocarbon law. They need assurances of any disputes will be settled by international arbitration. They need security. They need the rule of law and the notion that we can decapitate this regime, leave the incredibly corrupt system that Maduro led behind with all the same people, and then say to our oil companies, hey, please come in and start pumping oil. I think that's an illusion. Our oil companies need a legal structure in order to operate there.

Becky Quick (14:05)

I think that's a very fair point. And I think it'd be difficult to convince them that that structure, first of all, is going to take place really quickly. Second of all, the idea that it would last pass this administration or something beyond that, we're asking them to spend billions and billions, probably $10 billion a year to try and put the infrastructure back up. How, how could you possibly make some of those promises and make sure that they are lasting ones? Because you would need to be there for years in order to get that money back.

Tom Friedman (14:36)

If you talk to the companies, Becky, they'll tell you, I mean, if they started tomorrow, it would probably take them three years to repair everything that's been destroyed in terms of Venezuela's economic infrastructure and then get it going. They could probably do about 500,000 barrels a day with low hanging fruit, you know, in a reasonable amount of time. But to really get it up to the capacity, it would take three years. Three years. Three years. That's the end of Trump's term. They would begin really exporting oil. Right when this administration would change. When you look at the whipsaw nature of our regimes when it comes to our governments, excuse me, when it comes to energy. These oil companies, I think, are going to be very, very wary.

Becky Quick (15:17)

There has been, though, Tom, real investor interest. We've spoken with other investors this week who are raising funds right now. They're not the majors, but some of them used to work for the majors and they think that they can get a couple of billion dollars raised pretty quickly. And I guess that's the question. If we do have a plan to go in and rebuild the infrastructure in Venezuela, try and help the Venezuelan people with this as well, where does the money come from first?

Tom Friedman (15:46)

Yeah, I mean, a couple billion dollars, that's probably about $98 billion less than the estimates of what would be needed. So please tell the people raising those monies, I'm not interested in their prospectus. You know, I really think that the problem we are going to run into here is, is that we decapitated this regime. A terrible leader, but the leader of a terrible regime, we have now basically brought him to trial in New York. But we have decided, the Trump administration decided to leave that terrible regime in place and not move to say to that regime, okay, we've gotten rid of your leader. We need a transition here. You have six months, nine months to organize free and fair elections. That is a political context. I think that would really be attractive to investors and give Venezuela a long term path, you know, to restoration of its democracy.

Joe Kernen (16:42)

That was implied, wasn't it, Tom, that that's the way you would do it, that the military wouldn't back Mikado. They said in the meantime, in lieu of an election, which we want in a year, I thought that was part of what the administration is pushing for at this point because it would have been too. The military is behind for good or bad. The opposition party wouldn't have the support of the military. Then we'd be right back in the same place if there was a. What's it called? Is it a junta or something? Tom, you're well versed in all that stuff.

Tom Friedman (17:14)

You know, Joe, you're raising an important point. I have not heard the president say that at all, that I've not heard him use the word elections at all in terms of Venezuela. And this really gets to the problem. I saw this story unfold in Libya. I wrote about it last week when President Obama and NATO, our NATO allies, decided to decapitate Gaddafi but not put boots on the ground. And what happened basically was nature took its course there and the country basically fell apart into civil war and is still basically in a tribalized situation there. And so I think that's the, that's the danger in these kinds of situations where, when you, when you decapitate one of these regimes, but you don't put in place an alternative structure on the ground, you try to remote control it from afar. I think it's really, really challenging, Tom.

Andrew Ross Sorkin (18:13)

Politically, the idea that we may ultimately have to subsidize these oil companies to even go in there and what kind of game of chicken might go on between some of these oil companies that are even contemplating it and what they may ask for, how do you think that plays back here in the US because at some level, there's this unique paradox, which is if you're, you're trying to lower the price of, of oil, which could be great for consumers to some degree, but by doing that, you could actually put some of these shale producers out of business at the same time.

Tom Friedman (18:45)

Well, the Wall Street Journal today has a lead story that, you know, Trump wants to bring the price of oil down to $50 a barrel. For our majors, that's very problematic. And for the shale drillers, number, I mean, that's, that's really close to break even for them. So Trump's political interest, which I think is to get the price of gasoline to pump as low as possible for the midterms, is going to run up against the economic interests of the oil companies. And at the same time, you know, they have a lot of opportunities around the world. These are also, you know, they've got to report to their shareholders. And so if Trump says, well, we're going to, let's say we're going to give you subsidies to go in, I mean, the amount of subsidies would have to be huge. And let's remember ExxonMobil says it's owed $20 billion by Venezuela for the expropriation of its assets back in 2007. ConocoPhillips says it's owed 12 billion. So before they even go in those debts, there's got to be a process at least in place for eliminating those debts. So my simple point is, in the long run, and we're only less than a week into this, is that ultimately, if you want to exploit, see, Venezuela, it's their oil. Exploit their oil in a way that would be helpful to them and the world market. I think you need to move down the road toward free and fair elections. Let's remember the opposition won the last election by estimates, by 70% of the vote. Joe is right. The people with the guns right now are not those people. And that's why this is going to be Very complicated. Changing a country of 28 million people by remote control and just focusing on oil. I saw that play in Libya. It ended really badly.

Joe Kernen (20:30)

We've talked about the Middle east so many times. I can't believe we don't have time to talk about anything there. I mean, I'm sure your part of you is like I can't believe what's happening in Iran. And you know, things happen gradually and then suddenly I don't know if we're on the cusp of something really unbelievable. But then I think you probably have major concerns with Gaza and what's happening there. But net net, aren't there some really positive things going on? And maybe we can talk about that some other time. And as a longtime watcher, you must have watched some of that stuff saying, wow, this is, this is possibly a really good future setting up for itself. I don't know when, but at some.

Tom Friedman (21:06)

Point, Joe, let's get the positive and the negative because you really got both positive. President Trump organized a cease fire in Gaza, prisoner exchange, hostage release, set the table for a possible peace process. It's now frozen. But that cease fire was a good thing. Same time you have actually now, Joe, Syria and Israel talking to each other about, you know, economic relations and stabilizing their border. Both really important on the, on it's not downside. On the worrying side is you have this uprising in Iran. I think that's wonderful, it's logical. But this is a regime that has proven time and again it will kill as many Iranians as it has to to stay in power. But beyond that, Joe, I would say this, and it applies to Venezuela as well. The opposite of autocracy in the Middle east is not always democracy. It's often disorder that we assume that you get rid of the bad guys, then the good guys come in. What actually can often happen is actually complete disorder. In a country like Iran, nearly 90 million people, that has to be a concern too. It's not rooting for the regime, just the opposite. But everything is about what happens the morning after. The morning after, how does the transition unfold?

Becky Quick (22:25)

Tom, thank you so much. We'll have you back soon. It's good talking to you, to be with you guys.

Katie Kramer (22:31)

Next up on Squawk Pod, a story about rare disease that hits pretty close to home here at squawk box.

Becky Quick (22:38)

My 9 year old daughter Kaylee has a rare genetic condition called SYNGAP1.

Katie Kramer (22:43)

Becky Quick tells her own story and we are joined by former FDA commissioner Dr. Scott Gottlieb who is now advising CNBC Cures, which aims to shine a spotlight on treating rare disease.

Scott Gottlieb (22:55)

What the agency is gonna need to do is to start to think about new drug therapies for these ultra rare diseases, not as single products, but regulating the process that is used to drive the products rather than the product itself.

Katie Kramer (23:08)

The roadblocks for therapies and even cures and bearing the weight of being a rare disease parent.

Becky Quick (23:15)

You were one of the first people I talked to because I just wondered what was possible, what was out there.

Katie Kramer (23:20)

We'll be right back.

Becky Quick (23:27)

What made you confident that you could do something that hadn't been done before? I have no fear of failure.

Katie Kramer (23:33)

Trailblazing women, changing the game.

Becky Quick (23:35)

One of my favorite pieces of advice, think about what your boss's boss needs.

Katie Kramer (23:40)

Leadership can look in many, many different forms. It really does come down to just trusting yourself.

Becky Quick (23:45)

Life is short and you just gotta think big to accomplish big things.

Katie Kramer (23:49)

Julia Boorstin hosts CNBC Changemakers and Power Players New episodes every Tuesday, wherever you get your podcasts. Welcome back to Squawk Pod from cnbc. Here's Becky Quick.

Becky Quick (24:05)

Today we're announcing the launch of the CNBC Cures initiative focusing on advancing awareness and understanding of rare diseases. My nine year old daughter Kaylee has a rare genetic condition called SYNGAP1. It's a brain protein deficiency that results in autism, seizures, developmental delays and there are less than 2,000 other people in the world like her. The patient populations for rare diseases are small, but there are more than 10,000 conditions that you'd call rare. Altogether, it impacts some 30 million Americans. Many of those patients are children and 95% of these diagnoses don't have an FDA approved treatment. Despite those eye popping numbers, millions of people continue to suffer from diseases that often go overlooked by the broader healthcare industry. Small patient populations mean that drug companies and investors may not be willing to invest in the space. And outdated regulatory frameworks make the process of developing life saving treatments too slow for the families that need them to. Joining us right now is one of the first people that I turned to for advice and guidance when we first started to go down the path of being rare disease parents. CNBC contributor Scott Gottlieb. He is the former commissioner of the FDA and he serves on the boards of illumina, Pfizer and UnitedHealth. He's also on the CNBC Cures advisory board. And Dr. Gottlieb, thank you for being here.

Scott Gottlieb (25:23)

Thanks a lot.

Becky Quick (25:24)

Thanks for having me. You were one of the first people I talked to because I just Wondered what was possible, what was out there, what might happen. And you told me about some pretty exciting and amazing things that were taking place in the world of research and things that the FDA was doing to try and help push those developments along. Why don't you talk a little bit about what you first learned at the FDA and how that kind of changed what the FDA was doing at that point.

Scott Gottlieb (25:48)

Yeah. And I remember our conversation at the time, we were starting to think about what we were calling these N of 1 diseases. And this was really galvanized by a single parent, a woman by the name of Julia Vitarello, and a doctor at Boston Children's, Timothy Yu. Her daughter had a rare disease, Batten's disease, but a very specific mutation. And what the doctor was proposing to do was to fashion an SIRNA oligonucleotide. I heard you mention that in the last hour. That would be very specific to the particular mutation that this little girl had. And it could potentially be a treatment. And they wanted a pathway to take this forward. We ultimately created that pathway for them. They were able to treat her daughter. Her daughter did derive some benefit from that treatment. She unfortunately passed away during COVID But it started to change the agency's thinking about how to create a pathway for these so called N of 1 diseases, where you might want to fashion a very specific construct for a very specific genetic mutation. And how could we operationalize this into a policy construct? And this has continued. We made some progress when I was there. You've heard FDA recently talk about trying to advance the frameworks. This was ultimately adopted into forms of policy. I think it was personified in what the President tried to do with. Right. To try to try to get these treatments out to patients more quickly, these gene therapies. And the technology has really advanced since then. There's much better constructs for trying to treat these diseases. And ultimately what the agency is going to need to do is to start to think about new drug therapies for these ultra rare diseases, not as single products, but but regulating the process that is used to derive the products rather than the product itself.

Becky Quick (27:20)

And so if you follow, what's that mean?

Scott Gottlieb (27:22)

Well, if you have a genetic disease that has a sort of common phenotype, and you know that the disease is being driven, for example, by the production of abnormal proteins or a missing enzyme in the brain, which a lot of these diseases are, if you can design a construct to address this, that specific mutation that's going to have the effects that you're intending for. So reduction, for example, in the production of a certain Aberin protein, you should be able to get approval just on the basis of that construct itself, if you can prove that the oligonucleotide or the other construct that you're using is going to have that biological effect. So rather than having to do a clinical trial on every individual patient, because in many cases, you can't find enough patients to enroll in a clinical trial and power it adequately, what you're doing is regulating the process by which you're trying to derive a construct that would have an effect in that biological system.

Becky Quick (28:15)

What is happening on the scientific front that you described, even the things that were happening in 2017, that those are outdated at this point, we're looking at much different ways of pushing forward.

Scott Gottlieb (28:27)

Yeah, that's right. There's been a lot of progress just in those intervening years. I mean, the real what really opened up the field of gene therapy was that development of the AAV vector, a viral vector that could deliver genetic changes to people who were born with genetic diseases and other patients as well. The first time that we used an AAV vector in a patient was really the late 1990s for the attempt to treat cystic fibrosis. And so this is relatively new technology. And there's a lot of shortcomings of the AAV vector, including systemic side effects that you get from it. If you try to deliver it systemically, it often sometimes localizes in the liver and creates certain side effects that can be very dangerous. And we've seen patients die in clinical trials. Right, exactly. And so we've seen new technologies like lipid nanoparticles, where you encase the genetic change that you're trying to deliver in a lipid nanoparticle directly to the target tissue, and you can target it to specific kinds of body tissue. We've seen things like synthetic capsids where you're again encasing the genetic alteration inside a capsid rather than inside a viral vector. Rather than relying on a virus to deliver the gene into new tissue, you're able to more directly target it to that tissue. And so these are relatively new advances. They're in clinical development. They've shown good results in clinical trials. Companies like Beam and Verve are also doing things like base editing, where rather than delivering a whole gene, what you're doing is delivering a particle that will just modify the gene that's already in a person to try to make it a normal gene. And so we're having very good success with this technology.

Joe Kernen (29:54)

It's somatic cells. How do you do it so that it would be. I mean, in other words, you can't get it into the stem cells. Where does it stay in a somatic cell long enough to be expressed and provide the protein?

Scott Gottlieb (30:07)

Yeah, I mean, that's the critical question. So you're not getting into gametes, you're not trying to change is going to be inherited. What you're trying to do is alter tissue that's in a patient's body and.

Joe Kernen (30:18)

It can stay there and do it.

Scott Gottlieb (30:18)

For six months, it could stay there and do it potentially for years and maybe in perpetuity. What we don't know is the durability of a lot of these treatments and whether or not patients are going to need to be redosed with these constructs. And that's also the problem with the AAV vector because many times you can't redose a patient because of neutralizing antibodies.

Joe Kernen (30:34)

It's a one and done if you're sitting dissociated adeno.

Scott Gottlieb (30:37)

Right. So with these other constructs, presumably you can redose patients if the genetic alteration dissipates.

Joe Kernen (30:43)

Wouldn't be nice just to make the protein and introduce it into the. You can't do that.

Scott Gottlieb (30:46)

We're doing that. Well, we are doing that, but it's hard.

Joe Kernen (30:48)

What happens? It gets broken down.

Scott Gottlieb (30:49)

The half it's broken down. You develop neutralized. You can't get it into the brain. You need constant infusions. Patients develop neutralizing antibodies.

Joe Kernen (30:58)

It's better to have it done this way.

Becky Quick (30:59)

Even with the psycho, you'd have to have it done every three to six months. It fades away and it's not an easy injection.

Scott Gottlieb (31:05)

It has to go in intrathecal. Look, we've transformed diseases like Gaucher's disease with these infused enzymes. But if you can deliver a genetic alteration that provides a more durable treatment safely and comparable, if not better efficacy, that could potentially be preferable.

Joe Kernen (31:19)

Well, the messenger RNA vaccines were delivered in a little lipid.

Scott Gottlieb (31:22)

They were delivered in a lipid, not a particle. Is that where the technology really has been perfected? Yeah.

Becky Quick (31:29)

Scott, what's the biggest roadblock? I would say and then the biggest hope you have on the other side of that?

Scott Gottlieb (31:34)

Look, I think the technology is really promising. So I wouldn't say anymore that the technology is the biggest roadblock. I think regulation is a roadblock here because it's still hard to do these so called called N of 1 trials. I think while the FDA has opened up a pathway to this and we're able to do it in this particular case for this particular child. And others since then, the FDA hasn't created sort of a reproducible framework. They put out a paper in November talking about this plausible mechanism pathway that they were going to create that would operationalize this at a larger scale. I think ultimately Mark.

Becky Quick (32:04)

And carry on to talk about that.

Scott Gottlieb (32:05)

Right. I think Congress should intervene here. I think there's room for legislation that would give the FDA very targeted authorities and resources to do this.

Andrew Ross Sorkin (32:13)

Did you.

Joe Kernen (32:14)

You just have a lot of continuing education. I think you're an md, right? Yes. You just read about molecular biology constantly or something.

Scott Gottlieb (32:21)

You're a biochemist.

Joe Kernen (32:22)

Well, I know, but when you talk MDs can't talk the talk like you do.

Andrew Ross Sorkin (32:27)

They just.

Joe Kernen (32:27)

They have. They don't know any of these things you're talking about.

Scott Gottlieb (32:30)

I think. I think a lot do. And I think certainly the people at FDA are very well versed in this because they've had.

Joe Kernen (32:35)

They've kept the literature.

Scott Gottlieb (32:37)

When I was there, we talked about. We put out a statement that we thought there were going to be upwards of 10 cell and gene therapy approvals a year. And at the time that we said that, people thought it was absurd. We're close to that. We've had seven approvals in the last two years. Last year it was down. It was only five approvals. But I think we're getting to a run rate where these things are becoming more mainstream therapies.

Joe Kernen (32:56)

I mean, you are. You're amazing, Scott.

Becky Quick (33:00)

And we are very grateful to have you on the board with CNBC Cures.

Scott Gottlieb (33:03)

Thank you.

Joe Kernen (33:04)

Be good at the fda.

Becky Quick (33:05)

Oh, wait, it's done that.

Katie Kramer (33:10)

Please check out our new podcast special, the Path with Becky Quick about the world of rare diseases. Episode one was published right in the Squawk Pod feed. So wherever you're listening now, the path will be just ahead of this episode. Let us know what you think in the comments on Apple podcasts or on YouTube. We'd love to hear your story. If you are living with a rare disease or caring for someone with one, head to cnbc.com cures and join the community we are building there. Thank you for listening. Thanks for following Squawk Pod with the whole gang. Joe, Becky and Andrew. We'll all meet you right back here tomorrow.

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