Latest / Making Medicine / The Relay Continues: Building Biotechs That Save Lives | How America Makes Medicine Ep. 3

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The Relay Continues: Building Biotechs That Save Lives | How America Makes Medicine Ep. 3 · Making Medicine

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Speaker 1: At Making Medicine, we're reporting on the headlines, not making
0:03
Speaker 1: medical recommendations. For personal health questions, always ask your doctor.
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Speaker 1: Nothing in this episode constitutes investment, financial, or legal advice.
0:13
Speaker 1: Consult your own advisors before making decisions.
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Speaker 2: I think we should be learning from China and implementing
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Speaker 2: a lot of things that China has. I honestly don't
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Speaker 2: think you can be compatitive in the gene therapy and
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Speaker 2: cell therapy areas these days if you don't utilize the
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Speaker 2: Chinese system to advance the development of your drugs period
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Speaker 2: and for patients, at the end of the day, patients
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Speaker 2: want cheaper drugs, and you know, as a breast cancer survivor,
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Speaker 2: I don't care where the drug comes from. I don't
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Speaker 2: care if it comes from China, at Africa, the US.
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Speaker 2: I want a drug that's going to treat my disease.
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Speaker 1: Welcome back to the Making Medicine podcast or Science Policy
0:58
Speaker 1: and Innovation Meet. I'm Your Hope's John Stamford. On today's show,
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Speaker 1: we're digging into what happens after a scientific breakthrough leaves
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Speaker 1: the lab, when an early stage company has to turn
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Speaker 1: a discovery and is something that can actually reach a patient.
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Speaker 1: This is the third episode of our seven part series
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Speaker 1: How America Makes Medicine, celebrating two hundred and fifty years
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Speaker 1: of American pharmaceutical innovation. Our guest has gone down that
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Speaker 1: entrepreneurial path more times than almost anyone in the industry.
1:27
Speaker 1: Sheila McHale co founded ask Bio in two thousand and
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Speaker 1: one and built it into one of the most consequential
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Speaker 1: gene therapy platforms in the country. She went on to
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Speaker 1: found Bamboo Therapeutics, later acquired by Pfizer, and Chathaman Therapeutics,
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Speaker 1: later acquired by Baxter, before Ask Bio itself was acquired
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Speaker 1: by Behar in twenty twenty for four billion dollars. Today,
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Speaker 1: she's co founder and executive chair at M thirty four.
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Speaker 1: Chila is going to walk us through what it actually
1:54
Speaker 1: takes to build a company around a scientific discovery, the funding,
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Speaker 1: the risk, and the decisions that determine whether a promising
2:00
Speaker 1: idea ever moves forward. It's a great conversation, so let's
2:04
Speaker 1: get into the show. As with the rest of our
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Speaker 1: How America Makes Medicine series. Before the guests on, we
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Speaker 1: like to do a little history lesson, so let's talk
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Speaker 1: about how the modern biotech company came to exist at
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Speaker 1: all because for most of American history, the handoff that
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Speaker 1: we described in the teaser for the episode from a
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Speaker 1: university lab to a company didn't really happen at all.
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Speaker 1: For decades, discoveries made in university labs mostly stayed there.
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Speaker 1: Even when federal funding poured into research after World War Two,
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Speaker 1: there was no clear mechanism for turning a slightly government
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Speaker 1: backed funded discovery into a commercial product. The patents typically
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Speaker 1: sat with the federal government or the university, and companies
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Speaker 1: had little to no incentive to license and develop them.
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Speaker 1: Two things change that, As we've mentioned before on the
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Speaker 1: science front. In nineteen seventy three, Stanley Cohen and Herbert
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Speaker 1: Boyer developed recombinant DNA technology, splicing genes from one organism
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Speaker 1: into another. This, for the first time, made it possible
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Speaker 1: to manufacture biological molecules like insulin at scale. Boyer went
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Speaker 1: on to co found Genentech in nineteen seventy six with
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Speaker 1: venture capitalist Robert Swanson, widely considered the first true biotech
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Speaker 1: company built specifically to commercialize university born science, but it
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Speaker 1: was actually a couple years later that something truly transformative happened,
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Speaker 1: and it didn't happen in the lab. It happened in
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Speaker 1: Washington because Congress had taken note that university invention, sometimes
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Speaker 1: on the taxpayer dollar, really was going nowhere. So they
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Speaker 1: passed the bi Dole Act, which let universities and small
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Speaker 1: businesses keep ownership of inventions developed with federal funding as
3:54
Speaker 1: long as they worked to commercialize them. That single law,
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Speaker 1: and that single moment and time is why tech transfer
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Speaker 1: officers at Marquee Universities exist. It's why licensing deals happen,
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Speaker 1: and why a discovery in a university lab like the
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Speaker 1: ones we're going to hear from from Sheila, can become
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Speaker 1: the seed of a company instead of sitting in a
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Speaker 1: filing cabinet. The bi Dole Act is truly the hinge
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Speaker 1: point between our first episode in the series, talking with universities,
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Speaker 1: and today's episode where we talked with that early stage ecosystem.
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Speaker 1: It's simple enough to say that without the by Dole Act,
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Speaker 1: we simply would not have this area of the ecosystem.
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Speaker 1: We would have large manufacturers but very little innovation. The
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Speaker 1: Bidole Act is also one of the biggest American policy
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Speaker 1: exports that countries around the world in Europe and in
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Speaker 1: Asia particularly are trying to emulate it's been that successful.
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Speaker 1: So on today's show, we're picking up with that next
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Speaker 1: leg how a licensed discovery becomes an early stage company
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Speaker 1: that can actually carry it forward on to Shila. Sheila,
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Speaker 1: welcome to the show.
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Speaker 2: Oh, I'm so glad to be here today.
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Speaker 1: Well, we are in the midst of our How America
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Speaker 1: Makes Medicine series and we thought you'd be the perfect
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Speaker 1: voice for We had a great conversation about what universities do,
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Speaker 1: but at some point the entrepreneurial journey needs to begin
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Speaker 1: and that early stage ecosystem. We'd be fortunate if you
5:19
Speaker 1: had navigated that once and could tell us how it works.
5:23
Speaker 1: But you've navigated a heck of a lot more than
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Speaker 1: one time to multiple levels of success. So looking forward
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Speaker 1: to unpacking that with you today. And maybe though for
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Speaker 1: folks who don't know your story, how did you find
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Speaker 1: yourself early two thousands ready to take this plunge with
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Speaker 1: you and see what was your path to being someone
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Speaker 1: who was going to commercialize university innovation?
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Speaker 2: Yeah, it wasn't intentional. So I was teaching a course
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Speaker 2: at the University of North Carolina as a guest lecture.
5:54
Speaker 2: I was actually called in the last second to step
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Speaker 2: in for somebody who got sick, and the topic was
6:00
Speaker 2: how to spin up companies from the universities. It just
6:03
Speaker 2: so happened that doctor Richard Jude Simalski, who is known
6:08
Speaker 2: for his work in and no associated viruses, was in
6:13
Speaker 2: the audience, and he came up to me afterwards, and,
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Speaker 2: you know, said hey, could you help me? Could you
6:18
Speaker 2: represent me as a lawyer. I had had a small
6:21
Speaker 2: firm called Life Sciences Law located in Chapel Hill, and
6:26
Speaker 2: he said he was having a lot of difficulty getting
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Speaker 2: the license from the university, and I said, sure, no problem,
6:33
Speaker 2: you know, so I helped him for about a year
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Speaker 2: and we tried to attract a CEO to come in
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Speaker 2: and run the company. That was a requirement for spinning
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Speaker 2: out the organization. And because gene Therapy had gone through
6:48
Speaker 2: some difficulty there was a death of a little boy
6:51
Speaker 2: named Jesse Gelsinger. Nobody believed that gene Therapy was viable,
6:56
Speaker 2: so venture capitalist wouldn't put any money into it, and
7:00
Speaker 2: so there came to a point where the company couldn't
7:03
Speaker 2: go any further because nobody was willing to step up
7:07
Speaker 2: and be the CEO without you know, any assurances of
7:10
Speaker 2: a paycheck anytime, you know, on the horizon. And it
7:14
Speaker 2: really came to a pivotal point when the Muscular Dystrophe
7:18
Speaker 2: Association approached to and said that they were willing to
7:21
Speaker 2: give him eight million dollars for the advancement of a
7:25
Speaker 2: therapy that he had been working on for descends. But
7:28
Speaker 2: they said, you need a viable CEO. And so at
7:31
Speaker 2: that point in time, it was almost because I was
7:34
Speaker 2: the personal last resort, nobody else would do it. I
7:39
Speaker 2: stepped in. I saw the promise and especially you know,
7:43
Speaker 2: the potential to help little boys who had a devastating
7:47
Speaker 2: diagnosis with no treatment options. I really felt like it
7:50
Speaker 2: was a moral imperative. So I worked with you for
7:54
Speaker 2: over a decade, running my law firm and also running
7:59
Speaker 2: as bio without any paycheck. And you know, it was
8:03
Speaker 2: funny because Jude sort of sold me on it. He's
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Speaker 2: very persuasive, and he said, when this works, it wasn't if.
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Speaker 2: When this works, it will change the world. And I
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Speaker 2: really do think that Jude's technology has changed the world.
8:17
Speaker 2: And I was very privileged to be a small part
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Speaker 2: of that journey.
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Speaker 1: Well, I think larger than a small part. I can
8:25
Speaker 1: almost see the movie. You know, you're here lecturing and
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Speaker 1: this incredible doctor sticks around afterwards to ask you questions.
8:34
Speaker 1: That's almost too surreal to be true, and yet here
8:38
Speaker 1: it is in two and a half decades later, asked,
8:42
Speaker 1: bio has been just this key part of your resume.
8:46
Speaker 1: But so to have Bamboo and Chatham, like you said,
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Speaker 1: you're a serial entrepreneur. Talk a little bit.
8:52
Speaker 2: Now thirty four.
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Speaker 1: Yes, now we're going to have to get to what
8:56
Speaker 1: you're actually doing today. So talk us through those two
8:59
Speaker 1: and then maybe finish on M thirty four and what
9:02
Speaker 1: you're doing today. Because you've you've already had three time success.
9:08
Speaker 1: Some people might be saying, why is she still at this?
9:11
Speaker 1: What's M thirty four gonna? How can she even contribute more?
9:14
Speaker 1: I'm curious. There was a lot of questions at once,
9:16
Speaker 1: so being.
9:17
Speaker 2: Oh, I can go ahead and talk about that. I
9:21
Speaker 2: think the bottom line is you have to see work
9:23
Speaker 2: as a way of expressing what you're passionate about. And
9:28
Speaker 2: you know, I really feel sorry for people who see
9:31
Speaker 2: work as drudgery and they can't wait until they get
9:33
Speaker 2: to retirement. I really see it as an incredible privilege
9:37
Speaker 2: to learn about science, to interact with patients, to work
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Speaker 2: with employees and colleagues who are world class, and all
9:45
Speaker 2: of us, I think fundamentally just want to alleviate human
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Speaker 2: suffering and really, you know, in some small way of
9:52
Speaker 2: advanced health. And so you know, when you're around a
9:55
Speaker 2: bunch of thoroughbreds and you're racing really hard, it's fun.
9:59
Speaker 2: It's it's not drudgery. It's exciting and interesting. And you know,
10:05
Speaker 2: I still remember a few years ago when I was
10:08
Speaker 2: raising money. The venture capitalist at that point asked me,
10:11
Speaker 2: so what do you do for fun? And I was like,
10:13
Speaker 2: I really don't have any fun outside of work, right,
10:16
Speaker 2: But I said, you know, because when you are in
10:19
Speaker 2: the middle of these things, and the time, as bio
10:22
Speaker 2: was growing really fast and I had hundreds of employees,
10:26
Speaker 2: you know, there's not a lot of time. It is
10:28
Speaker 2: twenty four seven, and even my dreams were all about
10:32
Speaker 2: the issues that I was dealing with, and you know,
10:35
Speaker 2: I was trying to solve them subconsciously. But it wasn't
10:40
Speaker 2: a drudgery. It was really you know, just an immersion
10:43
Speaker 2: is something that I totally and fully enjoyed. I think
10:47
Speaker 2: I'm a little bit more balanced these days than I
10:51
Speaker 2: used to be, But in the middle of it I
10:53
Speaker 2: was quite quite immersed. So what are the different companies?
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Speaker 2: I spun out Bamboo and Chatham because we couldn't get
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Speaker 2: funding for gene therapy. It was very difficult in the
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Speaker 2: early two thousand, again because of the death I referred
11:10
Speaker 2: to earlier at the Jesse Gelsinger DA. There was just
11:13
Speaker 2: no institutional venture capital money available for the advancement of
11:19
Speaker 2: our science. And so essentially my responsibility was to build
11:23
Speaker 2: a company, advance the science, get clinical data without access
11:27
Speaker 2: traditional funding sources. I mean, if I had been a
11:32
Speaker 2: little bit poor, I think experienced, I would have walked
11:37
Speaker 2: away knowing how difficult or really how impossible that task was.
11:42
Speaker 2: But there's something about being naive. I think sometimes you
11:45
Speaker 2: do better than you should because you don't know better,
11:47
Speaker 2: and you do a lot of things untraditional, like oh
11:50
Speaker 2: we can manage this, we just will try X, Y,
11:52
Speaker 2: and Z, and so I funded the company and built
11:56
Speaker 2: a company using non traditional funding sources, and one of
12:01
Speaker 2: those approaches was to spin out different companies from the platform.
12:06
Speaker 2: So asked Bio retained the platform lots of patents at
12:10
Speaker 2: the end I think we had two hundred and eighty patents,
12:13
Speaker 2: and then we spun out different companies focusing on particular uses,
12:19
Speaker 2: like certain indications of that intellectual property. So Chatham was
12:24
Speaker 2: focused on hemophilia bleeding disorders, and we were able to
12:29
Speaker 2: fund that through a five million dollar SBIR. We were
12:33
Speaker 2: able to generate human clinical data on the basis of
12:37
Speaker 2: that SBIR, and with that then we got a collaboration
12:41
Speaker 2: with Baxter, and eventually Baxter bought it and it became
12:44
Speaker 2: part of Taketa. So that was Chatham, and then we
12:48
Speaker 2: got a little bit of money. Each time we did that,
12:50
Speaker 2: we made a payout to our equity holders, but we
12:54
Speaker 2: also retained a portion for Asked Bio. And then we
12:58
Speaker 2: reinvested in a Mamboo and Bamboo advanced the Descends muscular
13:03
Speaker 2: didystrophe program. Again, we had gotten some money from the
13:06
Speaker 2: MDA to demonstrate our first clinical trial safety and so
13:12
Speaker 2: we took that and then went forward and did additional
13:15
Speaker 2: clinical trials. Unfortunately, I had a very difficult time with
13:20
Speaker 2: Wall Street, where some ruthless vcs we're trying to squeeze
13:27
Speaker 2: Jude and me out of the company. I mean, wall
13:29
Speaker 2: Street is tough and so I sort of surprised them
13:32
Speaker 2: rather than them squeezing us out. I sold the company
13:35
Speaker 2: to Peiser, and Peiser advanced that program into Phase three
13:39
Speaker 2: clinical trials. It allegedly did not meet its endpoint. We
13:43
Speaker 2: think Pfiser did not design the clinical trial appropriately by
13:48
Speaker 2: including some people we said should not be included in
13:50
Speaker 2: the trial, but say Lavi. And then finally the funding
13:55
Speaker 2: environment changed and I was able to raise money in
13:58
Speaker 2: as Bio. So then we focused on the hold co
14:01
Speaker 2: and I raised two hundred and thirty five million and
14:03
Speaker 2: then exited fourteen months later in a four billion dollar transaction.
14:08
Speaker 2: Now Jude and I have some money in our pocket,
14:10
Speaker 2: so now we basically finance our own companies. We started
14:15
Speaker 2: one company called Gerrata. We saw what was happening with
14:19
Speaker 2: the COVID vaccine and distribution throughout the world. We found
14:24
Speaker 2: some technology that thermal stabilizes vaccines and gene therapies, and
14:29
Speaker 2: so we invested in that, and that technology has been
14:34
Speaker 2: used in therapies now that are being given to gene
14:38
Speaker 2: therapy patients, but also vaccines. We can effectively thermal stabilize
14:44
Speaker 2: vaccines at very high temperatures and very low temperatures without
14:47
Speaker 2: any need for refrigeration, and then finally we started M
14:53
Speaker 2: thirty four. M thirty four is really science fiction. Again.
14:59
Speaker 2: Jude and I are funding our own companies so we
15:01
Speaker 2: can take really big bets. We believe that we can
15:05
Speaker 2: stop and maybe even reverse neuroggenitive diseases like dementia and
15:10
Speaker 2: Alzheimer's by delivering via an ad NO associated virus AAV,
15:16
Speaker 2: a reagent that would essentially change trends differentiate certain cell
15:21
Speaker 2: types in the brain to create more neurons. So when
15:25
Speaker 2: you're born, you only have so many neurons, and as
15:28
Speaker 2: you age, you lose those neurons, you don't get new ones,
15:32
Speaker 2: and that's over time how we get neuroggeneritive disease. So
15:35
Speaker 2: we believe that we can essentially change cell types and
15:39
Speaker 2: create new change them into new neurons that would prevent
15:45
Speaker 2: dementia and other diseases like that, And so that's what
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Speaker 2: we're working on the big bet.
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Speaker 1: It's incredible. There are so many things to pick at there.
15:55
Speaker 1: M thirty four really does sound like science fiction and
15:58
Speaker 1: I can't wait for it to just be signed. And
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Speaker 1: I mean, it sounds like you're there, but even your
16:03
Speaker 1: work on the logistics of medicine delivery and getting pat
16:06
Speaker 1: What we did learn about refrigeration is great when you're
16:10
Speaker 1: in a fully developed first World country in an urban area.
16:14
Speaker 1: I mean, there's still there are plenty of places in
16:15
Speaker 1: the US where refrigeration in rural America was a challenge,
16:19
Speaker 1: let alone in developing parts of the world. So I
16:23
Speaker 1: think we'll have to have you back on to hear
16:25
Speaker 1: more about both of those companies. But something you also
16:28
Speaker 1: said about the early days stood out to me only
16:31
Speaker 1: because we just got out of this SBIR turning the
16:34
Speaker 1: lights back on fight that incubate was a part of.
16:38
Speaker 1: Is it fair to say that without SBIR you couldn't
16:42
Speaker 1: have moved forward at the time, or at least it
16:44
Speaker 1: would have looked very different. And if you could speak
16:47
Speaker 1: to policymakers about SBIR and STTR, is there anything you'd
16:51
Speaker 1: say to them?
16:52
Speaker 2: Yes, sbirs STTRs are very essential for early stage technology
16:56
Speaker 2: because investors won't invest in that, you know, stage of
17:02
Speaker 2: development for technology. They want to see at least in
17:05
Speaker 2: vivo animal data, and even more so these days they
17:10
Speaker 2: want human clinical data. Right, So how do you get
17:14
Speaker 2: between how do you advance from the time when you're
17:17
Speaker 2: in the lab and you are working in petrie dishes
17:21
Speaker 2: and you have some in vitro data to getting it
17:24
Speaker 2: into maybe you get it into mice, right, and that's
17:28
Speaker 2: one step that's great. I don't put a lot of
17:30
Speaker 2: credence into mice data. We've cured cancer and mice, you
17:34
Speaker 2: know many times over. You really want to see large
17:38
Speaker 2: animal data like non human primates, and that typically can't
17:42
Speaker 2: be done at an institution like UNC or Duke. You
17:46
Speaker 2: have to outsource that to a cro and that costs
17:50
Speaker 2: Google's amounts of money. And in our space, right, you
17:53
Speaker 2: have to manufacture the product that you're going to inject
17:56
Speaker 2: in the non human primates and the monkeys, and that
17:59
Speaker 2: also costs lots of money, and then getting into human
18:02
Speaker 2: clinical trials it costs even more. So you have a problem.
18:05
Speaker 2: You have this value of death between when you prove
18:08
Speaker 2: something in a lab of context and when you get
18:11
Speaker 2: it out into you know, larger animal models, and who
18:15
Speaker 2: finances that. Historically people rely on SBI rs STTRs to
18:20
Speaker 2: get through that value of death. The suspension of SBI
18:26
Speaker 2: r STTR funding has detrimentally impacted our competitiveness and I'm
18:33
Speaker 2: not sure we're going to be able to rebound so quickly.
18:37
Speaker 2: Once you turn off the spickett, people let go writing employees.
18:41
Speaker 2: Technology reverted back at the universities. A lot of scientists
18:46
Speaker 2: left the area because the US, because you know, everybody
18:51
Speaker 2: wants to see their technology eventually, especially in life sciences area,
18:55
Speaker 2: translated into humans and become a product, right and so well,
19:00
Speaker 2: it was really stupid, I don't know how else to
19:02
Speaker 2: say it, and extremely detrimental to the competitiveness of this country.
19:09
Speaker 2: And it's not so easy now that it's back on.
19:11
Speaker 2: It's not so easy. You can't just flip a light
19:15
Speaker 2: switch and expect the life sciences industry to get back
19:19
Speaker 2: into year and to be back at full throttle. It
19:23
Speaker 2: has a real drag on our industry and our prospects.
19:29
Speaker 1: I think stupid is the right word. I mean, I
19:31
Speaker 1: don't know and if we have any number of data
19:34
Speaker 1: sets from red states and blue states and green states
19:38
Speaker 1: and whatever states that turning this thing off was a mistake,
19:42
Speaker 1: and it seems like I'm curious what you think of this.
19:46
Speaker 1: We're in the midst of this debate about China, and
19:49
Speaker 1: we've covered on different episodes that the US government is
19:53
Speaker 1: considering taking some pretty protectionist steps. I would say limit
19:58
Speaker 1: our ability to engage with the innovation that's coming out
20:01
Speaker 1: of China. And I guess my perspective is I don't
20:05
Speaker 1: know that I see China so much as the problem
20:08
Speaker 1: as I see shooting ourselves in the foot at every
20:11
Speaker 1: point of the value chain, price control, Yeah, shutting sbir.
20:15
Speaker 1: Do you is that the problem because one of the
20:18
Speaker 1: things we get told is well, the early stage ecosystem
20:21
Speaker 1: can't survive because China's too competitive. How do you see it?
20:26
Speaker 2: Yeah? No, I think we should be learning from China
20:29
Speaker 2: and implementing a lot of things that China has, So
20:34
Speaker 2: I started transitioning my early stage development work to China
20:39
Speaker 2: several years ago. I honestly don't think you can be
20:43
Speaker 2: competitive in the gene therapy and cell therapy areas these
20:47
Speaker 2: days if you don't utilize the Chinese system to advance
20:53
Speaker 2: the development of your drugs period. I mean, we have
20:57
Speaker 2: not only have we hurt ourselves by too much regulation
21:01
Speaker 2: in this country too much on certainty, all the changes
21:04
Speaker 2: at the FDA were extremely problematic, but in addition, the
21:11
Speaker 2: Chinese have facilitated the advancement of drugs. I think of
21:17
Speaker 2: our colleagues there while we were cutting the sbirs, STTRs
21:23
Speaker 2: and other funding for early stage life sciences companies. In China,
21:28
Speaker 2: my colleagues, if they wanted to start a company, they
21:31
Speaker 2: were given a significant amount of money. One of my
21:34
Speaker 2: colleagues got fifty million dollars from the Chinese government. They
21:37
Speaker 2: were given a you know, an R and D facility.
21:41
Speaker 2: Very nice. Here in the US, I have to go
21:44
Speaker 2: to Alexandria or you know, Longfellow. They're fine, they provide
21:50
Speaker 2: really incredible facilities, but it's very expensive, extremely expensive. So
21:54
Speaker 2: my operating costs are significantly higher. The cost of labor
21:59
Speaker 2: in China is lower and then in the US, and
22:04
Speaker 2: the productivity is higher. They work, you know, basically nine
22:11
Speaker 2: to nine, six days a week. You can't compete. You
22:15
Speaker 2: just can't compete. Also, because the regulatory path I can
22:19
Speaker 2: get drugs into human clinical trials is called a Chinese
22:23
Speaker 2: investigator initiated trial to figure out if my drug works
22:27
Speaker 2: are not within fourteen months. In the US that would
22:31
Speaker 2: take four to five maybe even six years, and in
22:36
Speaker 2: China I can do it four a quarter of the cost.
22:39
Speaker 2: Now once I have that human clinical data. You know,
22:42
Speaker 2: of course, the Trump administration, unlike the Brits are saying
22:47
Speaker 2: they won't take that data when you bring it back
22:49
Speaker 2: into the US. That's fine, but at least now I
22:52
Speaker 2: know if a drug works or not. Right, So I
22:55
Speaker 2: think today, if you don't utilize, at least in my
22:58
Speaker 2: space sellingen therapy, if you don't don't utilize the Chinese structure,
23:03
Speaker 2: which has you know, reduced cost, has an expedided pathway
23:09
Speaker 2: and less regulation. And also it's easier to recruit people
23:13
Speaker 2: in China into clinical trials just because two reasons. One
23:18
Speaker 2: just the population they have so many people, but also infrastructure.
23:22
Speaker 2: They have high speed trains where they can you know,
23:24
Speaker 2: basically transport people into the major medical center Shanghai and
23:29
Speaker 2: Beijing very quickly and easily. Like we're losing, uh. I
23:34
Speaker 2: think we're losing the battle on many fronts. We just
23:37
Speaker 2: haven't made the necessary investment in infrastructure. And I think,
23:46
Speaker 2: you know, our regulatory hurdles are just two uncertain and
23:50
Speaker 2: too difficult relative to to the Chinese. And you know,
23:55
Speaker 2: when you take on top of that all the cutbacks
23:57
Speaker 2: and funding, it's like most of the the companies I
24:01
Speaker 2: know in this space are struggling just to survive. And
24:03
Speaker 2: I'm thriving because I have rewritten the playbook, right. I
24:08
Speaker 2: basically have looked across the world and said, where are
24:13
Speaker 2: where the barriers lower, and where can I achieve what
24:17
Speaker 2: I need to achieve with a reduced budget, right, with
24:20
Speaker 2: smaller source of cash. So I am totally against putting
24:26
Speaker 2: up more protectionism in China at this point. I think
24:28
Speaker 2: we should utilize China. There are certain competitive advantages there
24:32
Speaker 2: and drug developers should take advantage of those. And for patients,
24:38
Speaker 2: at the end of the day, patients want cheaper drugs.
24:42
Speaker 2: So you know, if you can develop it faster and cheaper,
24:44
Speaker 2: that means that the cost of the drugs should be lower.
24:47
Speaker 2: And you know, as a breast cancer survivor, I don't
24:49
Speaker 2: care where the drug comes from. I don't care if
24:51
Speaker 2: it comes from China, Africa, the US. I want a
24:55
Speaker 2: drug that's going to treat my disease.
24:58
Speaker 1: I would that last point I think is one of
25:02
Speaker 1: the strongest. I mean, how do we look American patients
25:05
Speaker 1: in the I don't think Americans are xenophobic. When the
25:09
Speaker 1: FDA has approved of medicine, I think they say, I
25:12
Speaker 1: don't care where the innovation came from if it saves
25:15
Speaker 1: my child, or myself or a loved one, and so
25:18
Speaker 1: I think that's really profound. But I also think something
25:23
Speaker 1: else we sort of hear is you know you mentioned
25:27
Speaker 1: the IITs I guess the way I hear it. Would
25:32
Speaker 1: you agree if the cost for some of the things
25:36
Speaker 1: you pursued wasn't at the IT level but was a
25:39
Speaker 1: much higher one, we just simply would be putting fewer
25:43
Speaker 1: shots on goal. And I've had other investors come on saying,
25:47
Speaker 1: with but four the lower cost point, these three assets
25:51
Speaker 1: would have never moved forward. There never would have been
25:53
Speaker 1: the capital to explore that space.
25:56
Speaker 2: Yeah, no, I totally agree. I totally agree. But look,
25:59
Speaker 2: I mean, you know, I'm all about American ingenuity, American
26:04
Speaker 2: you know can do spirit Protectionism is not the answer.
26:09
Speaker 2: It is looking at us and you know, basically modernizing
26:15
Speaker 2: how we do drug development now with you know AI,
26:20
Speaker 2: it's just amazing what you can do through AI right
26:24
Speaker 2: to really reduce the uncertainty before you advance a drug
26:29
Speaker 2: into clinical trials. Recently, you know, the FDA said that
26:33
Speaker 2: they're not as interested in the animal testing and large
26:38
Speaker 2: animal testing, and that was because there was a shortage
26:41
Speaker 2: for a while of non human primates we just couldn't
26:43
Speaker 2: get access to them. And you know, again China was
26:46
Speaker 2: restricting the supply, and so the FDA had to sort
26:50
Speaker 2: of evolve and say, hey, we're not as interested in
26:54
Speaker 2: animal testing. Let's figure out different approaches. One approach that
26:58
Speaker 2: my company is using M thirty four is and it
27:03
Speaker 2: was unprecedented. I don't know why, and it took me
27:07
Speaker 2: four years to get this approach actually effectuated. We now
27:13
Speaker 2: are testing our delivery systems are AAV for biotropism to
27:18
Speaker 2: figure out. You know, we basically re engineer each of
27:23
Speaker 2: our AAV's, our viruses to deliver to particular targets. So
27:28
Speaker 2: the easiest way to think about this is our AAVs
27:33
Speaker 2: are like biological fed X trucks. There are a virus
27:37
Speaker 2: that we can put a zip code and say, deliver
27:40
Speaker 2: this payload, which is usually a gene to a particular tissue.
27:45
Speaker 2: We can say delivered to deliver delivered to the heart.
27:48
Speaker 2: So if you have a genetic disease, what that means
27:51
Speaker 2: is you have a gene that doesn't work properly. And
27:53
Speaker 2: so with this biological fed X truck that we now
27:57
Speaker 2: put in a zip code saying go to the heart
27:59
Speaker 2: or go to liver. We're delivering a therapeutic gene to
28:02
Speaker 2: do the work that the defective gene could not do.
28:05
Speaker 2: And that's how we treat genetic diseases. Pretty simple in theory.
28:11
Speaker 2: But what we are now doing is we're testing these
28:15
Speaker 2: biological FedEx trucks and making sure that we have the
28:19
Speaker 2: right ZIP code. We're testing them in decedents, human decedents,
28:24
Speaker 2: people who have passed their brain dead, and we inject
28:29
Speaker 2: them into people who are deceased, essentially decedents, to see
28:35
Speaker 2: if they actually have the tropism that we think that
28:37
Speaker 2: they have. Now, why is that important? Why is that transformative?
28:41
Speaker 2: It's transformative because in the past, we used to test
28:44
Speaker 2: them first, our biological FedEx trucks in mice, and then
28:50
Speaker 2: we would go after we prove that hey, we're trying
28:53
Speaker 2: to target the heart, oh, yes, it works in mice,
28:55
Speaker 2: then we would go into non human primates monkeys, and
28:59
Speaker 2: sometime times we would get consistent results, but often we didn't.
29:04
Speaker 2: If we got consistent results, then we went into humans,
29:07
Speaker 2: but then we often found surprises. We got surprised, and
29:10
Speaker 2: you would say, well, of course, right, it's pretty obvious
29:13
Speaker 2: a human is different than than a monkey, and a
29:16
Speaker 2: monkey is different than a you know, a mouse, and
29:19
Speaker 2: so to get rid of that interspecies variability, which costs years,
29:25
Speaker 2: you know, five six, seven years to do that and
29:29
Speaker 2: costs you know, tens of millions of dollars, we just said,
29:32
Speaker 2: let's just go into human decedents. Let's go into the
29:35
Speaker 2: human model, right, and test our regions in humans. So
29:38
Speaker 2: I think to this date we're probably the only group
29:41
Speaker 2: that is doing this, but it really reduces the variability,
29:46
Speaker 2: reduces the drug development costs. And for people who have
29:51
Speaker 2: you know, deceased individuals, they're used to, you know, people
29:55
Speaker 2: are used to giving organs for transplant. This is just
29:59
Speaker 2: an their opportunity where people can make some sense out
30:03
Speaker 2: of death and add value, right, help the next development
30:08
Speaker 2: of drugs, next generation of drugs. And so that's how
30:13
Speaker 2: we're addressing some of these issues.
30:15
Speaker 1: I had no idea this is where the conversation was
30:18
Speaker 1: going to go. What It's also very interesting that you
30:22
Speaker 1: had to pilot that that we had that no one
30:26
Speaker 1: had really explored that before. I am just genuinely curious,
30:30
Speaker 1: is there a time window when you can still conduct
30:34
Speaker 1: these from passing to that or can you really work
30:39
Speaker 1: with the deced and population a long time after death.
30:42
Speaker 2: No, so we want to be respectful of the families.
30:45
Speaker 2: We typically after death we have about three days that
30:50
Speaker 2: we can work with the deceit and then of course
30:53
Speaker 2: we want, you know, to make sure that the family
30:56
Speaker 2: can have closure. But that's more than enough time for
30:59
Speaker 2: us to get the scientific answers that we need. And again,
31:03
Speaker 2: you know, it's it's transformative. It advances science much more
31:07
Speaker 2: quickly without as many errors that you get going from
31:11
Speaker 2: you know, animal models that are not really relevant. Uh,
31:15
Speaker 2: and it reduces costs. And so I think, again, American ingenuity,
31:21
Speaker 2: we need to rethink and challenge the paradigms. I guess,
31:26
Speaker 2: you know, because of the fact that I was not
31:28
Speaker 2: always able to access substantial amounts of capital, I've always
31:33
Speaker 2: had to rethink how things are done. And you know,
31:36
Speaker 2: when everybody would say there's a certain template to getting
31:39
Speaker 2: a company finance scaled and you know, exited, I had
31:43
Speaker 2: to do it differently. And so I think it's the
31:45
Speaker 2: same mindset when we look at drug development. Why is
31:49
Speaker 2: it that everybody says it has to be done this
31:52
Speaker 2: way or you advance from mouse to non human primate
31:56
Speaker 2: to human. Is that really necessary, or there are other
32:00
Speaker 2: approaches that can reduce the costs and also, you know,
32:05
Speaker 2: expedite drug development. It's the same thing M thirty four
32:09
Speaker 2: or Another thing that we have done is again, you know,
32:13
Speaker 2: a lot of these gene therapy drugs are very expensive.
32:16
Speaker 2: We want to make sure that they're accessible not just
32:19
Speaker 2: to people in the US who have good insurance coverage,
32:23
Speaker 2: but around the world. And so one of our missions,
32:26
Speaker 2: which we successfully met, was could re reduce the cost
32:30
Speaker 2: of manufacturing significantly. So manufacturing has typically been one of
32:36
Speaker 2: the highest costs for selling gene therapies, and we took
32:40
Speaker 2: it upon ourselves to say that we were and people
32:43
Speaker 2: thought we were crazy and still don't believe that we've
32:45
Speaker 2: done it, and you're skeptical, but we have. We said
32:48
Speaker 2: we're going to reduce the cost of manufacturing to about
32:51
Speaker 2: ten thousand dollars per patient, and we went in and
32:55
Speaker 2: redesigned celgensma. So Gensma is one of the most successful
33:00
Speaker 2: drugs right now in the market. As bio was instrumental
33:04
Speaker 2: in making that drug work. We added a key piece
33:07
Speaker 2: of technology that essentially reduced the toxicity and increase the
33:12
Speaker 2: potency that allowed it to be viable and it's treated
33:16
Speaker 2: lots of people with spinal muscular atrophy SMA who would
33:21
Speaker 2: have otherwise died right They would have not lived to
33:25
Speaker 2: be five years of age. They would have had a
33:27
Speaker 2: horrible death beforehand. It's a disease where people are cognitively aware,
33:31
Speaker 2: but they can't use their muscles, and so we said,
33:36
Speaker 2: can we redesign that drug to reduce a cost but
33:41
Speaker 2: also make it more potent, and we were able to
33:45
Speaker 2: do that. So right now our understanding is Novartis has
33:49
Speaker 2: a very difficult time manufacturing enough drug to supply to
33:54
Speaker 2: all the SMA patients. We redesigned it such that with
33:58
Speaker 2: one two hundredly bioreactor, we could supply enough drug for
34:02
Speaker 2: all the patients in the world, and we reduce the costs,
34:07
Speaker 2: like I said, to about ten thousand dollars per patient.
34:10
Speaker 2: We are now in the process through our foundation, Columbus
34:15
Speaker 2: Children's Foundation, which focuses on the development of gene therapies
34:19
Speaker 2: for ultrarare disease, where there's fewer than one hundred kids
34:23
Speaker 2: in the world. We're now supplying the drug that we created,
34:27
Speaker 2: the competitive drug to cell gensma, free of charge to
34:31
Speaker 2: patients in Southeast Asia, and we can do that because
34:35
Speaker 2: we were able again to redesign it in a way
34:38
Speaker 2: where it's just a pretty cheap drug. So I think
34:44
Speaker 2: again this goes back to what our industry needs to do.
34:47
Speaker 2: We need to re examine things and figure out more
34:52
Speaker 2: efficient strategies for drug development. But also, once you successfully
34:56
Speaker 2: have a drug, how do we make that drug more
34:59
Speaker 2: for right for patients across the world and even in
35:04
Speaker 2: the US, even though we have this incredible insurance policy
35:07
Speaker 2: where people are willing to pay you know, insurance companies
35:10
Speaker 2: to three million dollars for these drugs, it's just not sustainable.
35:14
Speaker 2: So while you know, big Pharma can say, well, we
35:18
Speaker 2: want to get as much money as we can, I
35:22
Speaker 2: just don't think it's like eventually our industry and I
35:25
Speaker 2: think there's a lot of pushback already. Eventually the US
35:28
Speaker 2: system can't manage these high price drugs, and you know,
35:34
Speaker 2: patients aren't going to be able to access them. And
35:36
Speaker 2: so it's just sort of silly for all of us
35:38
Speaker 2: who went into drug development not to get you know,
35:41
Speaker 2: the highest price possible for a drug, but to really
35:44
Speaker 2: make sure that patients get access to transformative therapies. We
35:49
Speaker 2: have to take it upon ourselves to figure out how
35:51
Speaker 2: do we make those drugs more cost effective.
35:55
Speaker 1: Well, it's interesting that you have not just innovated in
35:58
Speaker 1: the lab and moving a drug forward, but even the
36:03
Speaker 1: underlying logistics of the manufacturing to meet sort of the
36:08
Speaker 1: needs not just of the US population but also the
36:11
Speaker 1: global population. I also think one of the points we're
36:15
Speaker 1: trying to make to listeners and policymakers is even if
36:20
Speaker 1: the even if you use a CRO or CDMO in
36:25
Speaker 1: China to move it forward, I think you're the perfect
36:27
Speaker 1: story of you've had this incredible success, and from a
36:32
Speaker 1: monetary and resources standpoint, that success, generally speaking, is staying
36:37
Speaker 1: here in the US, and you're choosing to reinvest it
36:40
Speaker 1: through the Foundation, through M thirty four, through others to
36:43
Speaker 1: move things forward. And so I mean part of my
36:46
Speaker 1: view about like, oh, it's wal off China is well,
36:49
Speaker 1: a lot of the upside that comes out of that
36:51
Speaker 1: or any other country ends up staying here in the
36:54
Speaker 1: form of people like you who then choose to redeploy
36:56
Speaker 1: it at some other challenge. And when thirty four is successful,
37:02
Speaker 1: you're going to just get to continue to grow that
37:04
Speaker 1: impact in the US.
37:05
Speaker 2: Yeah, And that's absolutely true. But also all the Chinese
37:09
Speaker 2: companies that I know, they want to partner with Americans, right.
37:13
Speaker 2: They want to get their drugs into the US market.
37:16
Speaker 2: This is where the big middle classes, this is where
37:19
Speaker 2: you know, they can get a return for their investors
37:22
Speaker 2: on the drugs. They don't want an isolationist policy. They're
37:27
Speaker 2: not out to beat us. They're out to advanced science
37:31
Speaker 2: to collaborate with us. That's that's my sense, right. I've
37:35
Speaker 2: never had the feeling that China. Maybe the government has
37:40
Speaker 2: a different perspective, but the people that are in the
37:43
Speaker 2: CROs and the scientists there and the hospitals, they're all
37:48
Speaker 2: sort of like us. They just have the mission of
37:50
Speaker 2: creating drugs and getting them out to patients. And in China,
37:55
Speaker 2: the reality is they don't have an insurance system, right
37:59
Speaker 2: that can for wide distribution of the drugs to their population.
38:04
Speaker 2: And so everybody who is doing good drug development China's
38:08
Speaker 2: trying to figure out how do we partner with US
38:12
Speaker 2: companies right to get transformative, innovative drugs, you know, to
38:16
Speaker 2: the market. Their mission is just like us. They have investors,
38:21
Speaker 2: and those investors are looking for a return on their investment,
38:25
Speaker 2: and that often means getting the drugs to the US.
38:28
Speaker 1: Yeah, well, and I think that'll be the market plays
38:32
Speaker 1: an incredible role. I want to switch gears a little
38:35
Speaker 1: bit to take advantage of the time we have you on.
38:39
Speaker 1: You know, part of this broader series is talking about
38:42
Speaker 1: the relay race of drug development. I think you did
38:44
Speaker 1: a great job of giving some tangible examples of when
38:48
Speaker 1: a company when it's ready to leave UNC it was okay,
38:54
Speaker 1: maybe we can get from the petri dish to mice,
38:58
Speaker 1: but manufacturing and and going into primates where that's when
39:03
Speaker 1: let's build a company. Let's use sbr When do you
39:07
Speaker 1: know you're unique in the sense that you've got your
39:11
Speaker 1: own personal data set. When did you know that you
39:14
Speaker 1: were at the other end of the spectrum that it
39:16
Speaker 1: was time to hand something off to a Pfizer, to
39:20
Speaker 1: a Baxter to others. What are some of the telltale
39:24
Speaker 1: signs that that early stage entrepreneur and company is ready
39:30
Speaker 1: to now hand it off.
39:31
Speaker 2: What do you look for so bamboo that occurred when
39:35
Speaker 2: our drug looked very successful and we had to scale
39:39
Speaker 2: up manufacturing. Building a large manufacturing facility is very difficult
39:47
Speaker 2: because most venture capitalists do not want to invest in
39:50
Speaker 2: brick and MOTAR. Unfortunately, at that time there were no
39:54
Speaker 2: CMOS contract manufacturing organizations where I could outsource that manufacturing too.
40:02
Speaker 2: They just were not as familiar with gene therapy at
40:05
Speaker 2: that time, and you know, didn't have a process, didn't
40:09
Speaker 2: understand what needed to be done, and so we were
40:12
Speaker 2: going to have to build it. At that point, I
40:15
Speaker 2: knew I was going to have to partner with a
40:18
Speaker 2: big pharma. And honestly, while we understood how to manufacture
40:24
Speaker 2: at small scale my team it was about thirty six
40:27
Speaker 2: at that point in time, we did not know how
40:29
Speaker 2: to manufacture for the needs of a later stage clinical
40:32
Speaker 2: trial or for the market for commercialization, as are higher
40:36
Speaker 2: manufacturing requirements called good cGMP current good Manufacturing Practices. We
40:43
Speaker 2: did not have that understanding. It's a lot of SOPs,
40:47
Speaker 2: a lot of regulatory requirements, and so, you know, because
40:53
Speaker 2: we didn't have one hundred million, hundred two hundred million
40:56
Speaker 2: dollars required to build a manufacturing and plus we we
40:59
Speaker 2: didn't have expertise, we decided it was time to exit
41:02
Speaker 2: to a big pharma and we sold to Pfizer. That
41:04
Speaker 2: was the right decision. And then with regards to asked bio,
41:09
Speaker 2: when I exited to Bear. It was because we had
41:13
Speaker 2: two big indications, Parkinson's and late stage heart failure, where
41:19
Speaker 2: you have hundreds, possibly thousands right of people that are
41:23
Speaker 2: required to be in a clinical trial, global clinical trials.
41:28
Speaker 2: I could not build that the staff necessary to support
41:32
Speaker 2: those clinical trials. They're just complicated too. These were you know,
41:36
Speaker 2: we moved away from monogenetic diseases where one gene was
41:41
Speaker 2: not working, to pathway diseases where multiple different genes were
41:45
Speaker 2: contributing to the disease. Date, we needed sophistication of a
41:50
Speaker 2: big pharma's clinical trials organization as well as you know,
41:55
Speaker 2: the biostatistics and understanding, right deep understanding of those diseases.
42:01
Speaker 2: And so that's why we sold to Bear. Yeah, there
42:05
Speaker 2: comes a point I think in every company's lifespan where
42:11
Speaker 2: you're just not going to get access to the capital
42:14
Speaker 2: that you need. And I would say in areas like
42:17
Speaker 2: the Southeast and you know US and other areas like
42:21
Speaker 2: that where you don't have as extensive of a venture
42:24
Speaker 2: capital private equity group, those days come earlier than if
42:29
Speaker 2: you were in you know, Boston or in Silicon Valley.
42:34
Speaker 2: You just can't you can't access the cash you need.
42:37
Speaker 2: That's number one to continue to scale and build. But
42:40
Speaker 2: two right there becomes a competency issue, infrastructure issue where
42:46
Speaker 2: leveraging off the infrastructure of big pharma, especially for large
42:52
Speaker 2: you know, clinical trials, it's just a lot easier and
42:55
Speaker 2: it ensures it gives a higher likelihood that your drug
42:58
Speaker 2: is going to be successful.
43:00
Speaker 1: I mean, again, you're so great about pointing to sort
43:02
Speaker 1: of the tangible moments in time. Manufacturing and clinical trials
43:07
Speaker 1: at scale were two tell tale signs that it needed
43:11
Speaker 1: to be handed off. I guess again, you come with
43:16
Speaker 1: a perspective of having done this so many times. Is
43:20
Speaker 1: this the right way? This relay race? You do this?
43:24
Speaker 1: You do this? I sometimes hear from members of Congress,
43:28
Speaker 1: why can't our small biotechs go on to do it themselves.
43:32
Speaker 1: I don't like this relay race. I don't like this handoff.
43:36
Speaker 1: I find it to be very efficient, but I'm curious
43:39
Speaker 1: you've been through it is the way we do these things.
43:42
Speaker 1: The innovation comes out, we build up these small to
43:45
Speaker 1: mid sized biotechs, and then we hand it off and
43:48
Speaker 1: the manufacturers who really do navigate manufacturing and global clinical
43:53
Speaker 1: trials and leverage their capital bases is this the right recipe,
43:59
Speaker 1: or there some tweaks you'd make.
44:00
Speaker 2: Yeah, So I have mixed feelings about this. So each
44:03
Speaker 2: time I've handed off my companies, I've learned something right
44:09
Speaker 2: about how my programs do and my teams do in
44:13
Speaker 2: big corporate structures. The first time, when we exited to Baxter,
44:18
Speaker 2: we you know, the company was fully integrated and disappeared.
44:22
Speaker 2: You know, Chatham no longer exists. The second time, that
44:25
Speaker 2: didn't work out so well. Our program got lost because
44:31
Speaker 2: Baxter became Basalt Up and then it was acquired by
44:34
Speaker 2: Shire and eventually it became Taketa, and our program got
44:36
Speaker 2: lost and it wasn't a good experience. So the next time,
44:40
Speaker 2: I said, Okay, I'm going to do better. I'm going
44:42
Speaker 2: to keep Bamboo as a separate company with a separate
44:46
Speaker 2: management group. And that worked pretty well for a while,
44:51
Speaker 2: but eventually Peiser said okay, enough of this nonsense, and
44:54
Speaker 2: they integrated us into Feiser, and then Feiser decided didn't
44:59
Speaker 2: want to be gene therapy anymore, and the programs got
45:03
Speaker 2: stuck and lost. Right, So I said, okay, third time
45:07
Speaker 2: is a charm. Third time I'm going to do it right.
45:09
Speaker 2: And so when we negotiated the sales ass biode Bear.
45:13
Speaker 2: Not only did we have a separate management team, but
45:18
Speaker 2: money is everything, right, I negotiated a separate P and L.
45:21
Speaker 2: So we had predetermined budgets and allotments of money to
45:25
Speaker 2: make sure that I could actually govern the company independently.
45:29
Speaker 2: You know, if you have to. If you have if
45:31
Speaker 2: you're quote unquote in charge, but you have to go
45:34
Speaker 2: back to the parent company for all your funding, then
45:37
Speaker 2: they really are the ones in control. Right is everything? Yeah?
45:41
Speaker 2: So I figured that piece out and I had a
45:44
Speaker 2: separate board and that worked pretty well until I had
45:49
Speaker 2: to step down. I was diagnosed with breast cancer and
45:53
Speaker 2: had to step down after three years of operating it,
45:56
Speaker 2: and leadership is everything. Since I've left, the company does
45:59
Speaker 2: not have it's the same level of autonomy anymore. But
46:03
Speaker 2: I think that's the right answer, right. I like that
46:06
Speaker 2: where you have the resources cash, clinical trial, infrastructure and
46:12
Speaker 2: regulatory of a big PHARMAUP, but you keep your independence.
46:16
Speaker 1: Uh.
46:17
Speaker 2: And you know asked bio had its own independence, had
46:20
Speaker 2: its own P and L, had its own management team,
46:23
Speaker 2: had its own board of director, was able to create
46:25
Speaker 2: its own strategy. That is sort of the winning I
46:29
Speaker 2: think formula, but like anything, right, you need leadership that's
46:33
Speaker 2: really committed to that dog it. Every time Bear used
46:36
Speaker 2: to step over the line, I was like a rabid dog, right.
46:42
Speaker 1: That's not what you don't want you on my oil
46:44
Speaker 1: sheet when I've disappointed you.
46:47
Speaker 2: Yeah, yeah, exactly. But look, I would love to take
46:52
Speaker 2: my companies all the way and stay with them. But
46:56
Speaker 2: I also think you have and I tell my management
46:59
Speaker 2: teams this all the time. As individuals, as leaders, as
47:04
Speaker 2: founders of companies, we also have a relevant life cycle,
47:08
Speaker 2: if that makes sense. When I sit down with my
47:12
Speaker 2: founding you know, team, when we start a company, I
47:16
Speaker 2: always give the speech about you probably won't be here
47:19
Speaker 2: right three to five years from now. And it's not
47:21
Speaker 2: because you didn't do everything right and you weren't an
47:25
Speaker 2: incredible asset. It's just when the company gets to the
47:28
Speaker 2: next stage of development, there are different skill sets that
47:31
Speaker 2: are required. And so even myself, you know, I'm really
47:37
Speaker 2: good at the early stages. I'm good at scale up.
47:40
Speaker 2: When it comes to commercialization, I've never done that. There
47:44
Speaker 2: would be better people right to commercialize, to lead a
47:49
Speaker 2: company that is actually selling drugs, and I've never taken
47:53
Speaker 2: a company public I've always chosen not to do so.
47:57
Speaker 2: I just don't want to run a public company. I
47:59
Speaker 2: don't want to be subject to quarterly uh you know results.
48:04
Speaker 2: I want to stay focused on the science and do
48:06
Speaker 2: the right things for the drug and for the science
48:08
Speaker 2: long term. So I've always chosen to keep my company's
48:12
Speaker 2: public until I can't anymore and then exit to you know,
48:16
Speaker 2: big pharma. And that's another reason why I've always chosen
48:21
Speaker 2: to exit. But you know, at some point, right my
48:26
Speaker 2: skill set Peter principle, I would have advanced beyond my
48:31
Speaker 2: capabilities or my experience level. And uh, it's hard, I
48:35
Speaker 2: think to put in a management team to fill founders' shoes.
48:41
Speaker 2: The whole culture, right, the people they all follow founders,
48:48
Speaker 2: And when you put in a new management team, there's
48:50
Speaker 2: a lot of hmm, it's a lot of difficulty in structures.
48:55
Speaker 2: It's just it's different. So I think I think there's
48:59
Speaker 2: just a natural life cycle for most companies that spin
49:02
Speaker 2: out of universities. I think it's very difficult to take
49:05
Speaker 2: something all the way through, especially these days. I mean
49:10
Speaker 2: the biogens of the world, the genet techs. I think
49:14
Speaker 2: there was an ear for that, but I don't think
49:16
Speaker 2: that era is today. I don't think it's so easy
49:19
Speaker 2: today to do that.
49:21
Speaker 1: But I love and I know our listeners will love
49:24
Speaker 1: your takeaways. As you went, not all exits and acquisitions
49:29
Speaker 1: and intakes by a large company are the same. And
49:33
Speaker 1: as you even went, you progressed to know what to
49:36
Speaker 1: ask for. And I know the founders who listen to
49:38
Speaker 1: the show will say, Okay, I need to follow Sheila's
49:41
Speaker 1: path on the Bayer side. She figured it out then
49:45
Speaker 1: to say, hey, I want my own P and L.
49:46
Speaker 1: Because you're right, the money is everything. It's almost like
49:50
Speaker 1: instead on all of our graphics explaining drug development, we've
49:53
Speaker 1: got Arrows University, Aero small Biotech, Aero large Pharma, much
49:59
Speaker 1: more Van diagrams. It's how much of your stage can
50:01
Speaker 1: you keep while also getting all the benefits that you
50:06
Speaker 1: need out of the next stage, and then eventually you
50:09
Speaker 1: exit stage left closing. You have been incredibly generous with
50:13
Speaker 1: your time. Your resume is too deep to kind of
50:16
Speaker 1: get through every nuance. So we have to have you back.
50:19
Speaker 1: What is the next two hundred and fifty years? And
50:21
Speaker 1: that's a tricky question. So what's the next fifty years?
50:24
Speaker 1: Although with your neurodegenitive work. Maybe you and I'll still
50:27
Speaker 1: be here in two hundred and fifty years, But what
50:31
Speaker 1: what do you see next? What keeps you excited as
50:35
Speaker 1: we sort of look back on where we've gotten to.
50:37
Speaker 2: Yeah, AI is going to be so transformative. It's going
50:40
Speaker 2: to let us do things faster and cheaper with fewer mistakes,
50:45
Speaker 2: and so I just think it's going to be an
50:47
Speaker 2: exciting era. I think, you know, M thirty four and
50:51
Speaker 2: others are resolving some of the issues related to making
50:56
Speaker 2: you know, gene therapy more prevalent and more unif versuly used,
51:01
Speaker 2: you know, using cancer vaccines to prevent cancer, to prevent reoccurrences,
51:10
Speaker 2: immune therapies, right to use the immune system to get
51:15
Speaker 2: rid of malignancies. It's just it's an incredibly bright future.
51:20
Speaker 2: And I hope right that we can come together as
51:23
Speaker 2: a global scientific community and resolve these issues together as
51:28
Speaker 2: collaborators rather than you know, I don't know, spending a
51:33
Speaker 2: lot of time fighting against each other. We should learn
51:37
Speaker 2: best practices on a global system. What are the Chinese
51:40
Speaker 2: doing right? What are the Brits doing right that we
51:43
Speaker 2: can and what is you know we have now some
51:46
Speaker 2: states like Montana right, who are going to go on
51:49
Speaker 2: their own and make themselves unique drug development hubs. What
51:52
Speaker 2: are the best practices that we can do to reduce
51:56
Speaker 2: the barriers right to efficient drug development, Get the regulators
52:02
Speaker 2: in line where their value add not confusing, and you know,
52:09
Speaker 2: giving mixed signals where there's just a lot of uncertainty
52:13
Speaker 2: in our space, freeing up money again, there's a lot
52:18
Speaker 2: of studies that show that government investment in life sciences
52:21
Speaker 2: gives a big payoff in terms of job creation and
52:25
Speaker 2: economic development. Let's make those investments. Those are good investments,
52:31
Speaker 2: you know, Reform insurance, reform hospital care. I think that
52:36
Speaker 2: if we're looking at inefficiencies, I'm not sure, you know,
52:41
Speaker 2: these PBMs and whatnot. I'm not sure what role they play.
52:44
Speaker 2: They're not doing the innovation, but they take fifty cents
52:47
Speaker 2: of every dollar. Why is that. If we're looking where
52:51
Speaker 2: to cut money, we should be looking there. And hospitals
52:55
Speaker 2: right should start incorporating AI more universally, because I know
52:59
Speaker 2: a lot of doctors are protecting their jobs, but it's
53:02
Speaker 2: better for patients and it's better for the system. We
53:04
Speaker 2: can't keep going on with the high cost of care
53:07
Speaker 2: that we have. There's lots of opportunity, and you know, again,
53:11
Speaker 2: I believe in American ingenuity. We just have to rise
53:15
Speaker 2: up to the task and get it done. And our
53:18
Speaker 2: politicians we need younger people who are more tech savvy.
53:21
Speaker 2: And we should not be we should not be a
53:24
Speaker 2: country run by seventy and eighty year olds. You know,
53:27
Speaker 2: they were using typewriters right when they were in college
53:30
Speaker 2: and whatnot.
53:31
Speaker 1: Sheila, thank you so much. That's a great place to
53:34
Speaker 1: leave it. Obviously, we'll have to have you back on
53:36
Speaker 1: the show, but thank you for being here.
53:38
Speaker 2: Thank you so much for the opportunity.
53:40
Speaker 1: That wraps up this episode of the Making Medicine podcast,
53:43
Speaker 1: our third episode in our How America Makes Medicine series.
53:47
Speaker 1: A huge thank you to Sheila for joining us today
53:49
Speaker 1: on today's episode. Be sure to subscribe on YouTube and
53:53
Speaker 1: follow us on other channels so you don't miss this
53:55
Speaker 1: or any other episodes. Thanks for listening, and as always,
53:59
Speaker 1: keep in a
54:00
Speaker 2: Ecouting thingting thing thing.