We spend a lot of time talking with our guests about building biotech companies. That’s true of this episode too, but today we’re taking a step further with John Leonard, M.D., longtime CEO at Intellia Therapeutics. In addition to how he’s built late-clinical stage Intellia, Dr. Leonard shares the foundational elements of a biotech community, particularly around rapidly-advancing technologies such as CRISPR, and what biotech ecosystems offer emerging biotechs in terms of resources, intellectual property, and support. Turns out that despite his proximity, Boston isn’t the prerequisite it used to be. This is an instructive conversation with an industry vet who’s happy to share the wealth of his knowledge.
You've listened along for years -- now you can watch along, too! Go to bioprocessonline.com/solution/the-business-of-biotech-podcast, where you can put faces to voices as you watch hundreds of interviews with the world's best biotech builders. While you're there, subscribe to the #BusinessofBiotech newsletter at bioprocessonline.com/bob for more real, honest, transparent interactions with the leaders of emerging biotech. It's a once-per-month dose of insight and intel that you'll actually look forward to receiving! Check it out at bioprocessonline.com/bob!
We spend a lot of time talking with our guests about building biotech companies. That’s true of this episode too, but today we’re taking a step further with John Leonard, M.D., longtime CEO at Intellia Therapeutics. In addition to how he’s built late-clinical stage Intellia, Dr. Leonard shares the foundational elements of a biotech community, particularly around rapidly-advancing technologies such as CRISPR, and what biotech ecosystems offer emerging biotechs in terms of resources, intellectual property, and support. Turns out that despite his proximity, Boston isn’t the prerequisite it used to be. This is an instructive conversation with an industry vet who’s happy to share the wealth of his knowledge.
You've listened along for years -- now you can watch along, too! Go to bioprocessonline.com/solution/the-business-of-biotech-podcast, where you can put faces to voices as you watch hundreds of interviews with the world's best biotech builders. While you're there, subscribe to the #BusinessofBiotech newsletter at bioprocessonline.com/bob for more real, honest, transparent interactions with the leaders of emerging biotech. It's a once-per-month dose of insight and intel that you'll actually look forward to receiving! Check it out at bioprocessonline.com/bob!
The business of biotech is produced by LifeScienceConnect and its community of learning, solving and sourcing resources for biopharma decision makers. If you're working on biologics process development and manufacturing challenges, you need to swing by bioprocessonlinecom. If you're trying to stay ahead of the Cell or Gene Therapy curve, visit cellenginecom. When it's time to map out your clinical course, let clinicalleadercom help, and if optimizing outsourcing decisions is what you're after, check out outsourcepharmacom. We're LifeScienceConnect and we're here to help.
Matt Pillar:Ask anyone who's been my guest on the business of biotech and they'll tell you that it takes more than a novel molecule to build a biotech A lot more IntelliAtherapeutics. President and CEO, dr John Leonard certainly knows it At his core. He's a molecule guy that is postdoc and molecular virology and spent the formative years of his career in R&D. But he's also a business builder and he's learned that it takes a community of diverse resources an ecosystem if you will to build a successful biotech. I'm Matt Piller. This is the Business of Biotech, and on today's show we're sitting down with Dr Leonard for a discussion on the building blocks of biotech, building blocks that reach well beyond the four walls of the business. Dr Leonard, welcome to the show.
John Leonard, M.D.:Great to be here.
Matt Pillar:I'm thrilled to have you here. When this idea, the conversation idea, was thrown my way, I thought man, I've been excited for this one for a while because I think it's a great, great topic and it's super timely. Right now we're at sort of an inflection point in the way that we do business. I mean, you know well, I should say a few years into an inflection point in the way we do business. And I'm curious. I've got lots of questions for you about how that inflection point has impacted specifically the biotech business and we'll get into that. But I want to start with a little bit of you right, to kind of get your perspective, get some context as to where you come from and what you bring to this conversation. And that starts with you know, when I look at your CV I see R&D, r&d, r&d, svp Pharmaceutical R&D at Abbott, svp Global Pharmaceutical R&D at Abbott, svp Pharmaceutical R&D at Abbott. And then you've got this MD and molecular, molecular virology back in the 80s, not to date you there's a little bit of-.
John Leonard, M.D.:You just did, but that's fine.
Matt Pillar:I did too Blatantly. There's a little bit of a gap there I'm curious about, like when you came out of academia, you know pretty prestigious academic experience. Then there's a bit of a gap before you joined industry, at least date-wise. So I'm just curious, like what you were doing then? What sort of field that? Yeah, it's.
John Leonard, M.D.:There's really no gaps. Everything led directly into the next thing. I went to medical school in the early 80s. I graduated 1983, so there's a date for you.
Matt Pillar:You've officially made an official date.
John Leonard, M.D.:Very, very official and but it's an important date because at that time the AIDS epidemic was just beginning. In fact, when I was in medical school, some of those very first case reports came out. So I was in medical school on the East Coast, at Johns Hopkins in Baltimore, which really was not the epicenter of AIDS, and in fact when I was doing my basic medical training or just you- know, maybe a few hundred case reports in the literature, it wasn't called AIDS.
John Leonard, M.D.:Even at that time, I mean, it was a condition that had been noted. When I graduated from medical school, I went to the West Coast, I did my medical internship and residency at Stanford and at that time we rotated through as interns at San Francisco General Hospital. So in the few years between, you know, just beginning my ward work and actually, you know, being an intern, the AIDS epidemic had exploded, and at San Francisco General Hospital they actually had an AIDS ward. So when I got there as an intern, you know the disease now had a name, the numbers were skyrocketing.
John Leonard, M.D.:In a place like San Francisco, which was certainly one of the epicenters, it was inescapable. And it's really that experience, I think, that in many, many ways influenced my ultimate career and career choices. Because while there, you know, you see a new condition appear that you know, just a few years ago nobody knew about. You see, a condition for which we don't know what the cause is. And if we don't know what the cause is, we have very few means actually treated, certainly not the disease itself. And so it was one of these things where I'm starting out my medical training and immediately you're at the end of pharmacopoeia, so to speak.
John Leonard, M.D.:So it's like where do these things come from? How do they happen? Where does medicine come from? Which, really, you know I'd say, started my, you know, you know initial thinking that maybe I would do something other than, you know, traditional medicine. And you know, maybe the gap you're referring to is after my internship in residency. I wanted to get more molecular because I just, I just seen that if you're gonna understand, understand the answer to some of these questions, or at least how to address them, I need to have something other than just my medical training. So I went to the NIH and that was 86-89, and worked in a laboratory that happened to be one of the two AIDS labs at that time at the NIH and you know, you abandond the clinical thinking because that's just not part of what that work is.
John Leonard, M.D.:And you're surrounded as a physician by PhDs, immersed in a world that was quite different from you know the medical training that I'd have and that I would say that was sort of the dividing point for me as to whether I would go and be sort of a standard academic clinician or, you know, be in a position that can, you know, continue a line of research. That, ultimately, is what I did.
Matt Pillar:Yeah, that's fascinating. So was it? I mean, was at that point in time sort of that the AIDS influence that moved you? Like prior to that, I should say prior to that, were you intending to be sort of the traditional academic clinician?
John Leonard, M.D.:You know I was always interested in clinical medicine. I enjoyed learning about it. I learned a lot from my patients, you know, when we were in a position where you could actually make a difference, that I was rewarding in its own way and I liked the process of, you know, trying to figure out diagnostically what's going on and then intervening. But I think it was so influenced by, you know, that experience with AIDS, along with, I guess, well, stanford, it's a, you know, at that time it was a tertiary referral center for oncology and you know lots happened in these last 40 years.
John Leonard, M.D.:So at that time it was in many respects similar to, you know, hiv infection, which is we had some ideas, but the you know, the tails and the granularity of our understanding was just really beginning to be what it is. So I became just really interested in going beyond recognizing the disease and trying to intervene and peering more deeply I guess is a way of saying it into what the disease process was and finding other ways to actually, you know, intervene at the source. And you know, as I was at the NIH and I got more deeply into, you know, the strictly research approach, I realized that it was going to be very hard for me to go and do clinical work and research at the level that I was hoping to do it in what was available to me at that time. So I decided to embark on a career in industry, which I realized while I was at the NIH is where most the drugs came from, and I wanted to be a part of that process.
Matt Pillar:Yeah, yeah, and what you know. So your experience in R&D speaks for itself. I mean, you had a long, storied career with some big companies in R&D Excuse me, and you know, I could spend the next 45 minutes talking with you about that. In fact, I'm going to go on public blast. I'm going to put you on blast publicly right now. We have a we're doing an R&D a bioprocess online live event in January on R&D. I'm going to publicly invite you right now to be a panelist on that event.
Matt Pillar:I'll follow up with your PR team on that.
John Leonard, M.D.:I'll have my people talk to your people.
Matt Pillar:That sounds good, but we're going to skip. We're going for the. For the intent of this, this conversation, we're going to fast forward, all right. So you know you did a bunch of R&D work at Abbott and AbbVie and we're going to fast forward all the way to 2014 to start talking about your, your leadership and building Ventelia. You joined the board there in 2014 and and took on a role that was familiar to you again in R&D EVP of R&D before assuming that had post there just just a few years later. So I guess, before we get off the R&D train completely, I'm curious about how your experience, your long experience in that realm, influenced your role in the C-suite as the leader, ed, and tell you.
John Leonard, M.D.:Yeah, I had been at Abbott for 22 years and I was in the R&D organization and I led it for a third of that time. When I stepped out of AbbV, I did not plan and you know, recapitulate my career, having some operational role, and had been asked if I'd be willing to, you know, provide some advice and thinking with respect to where you know CRISPR technology might go, you know, in terms of putting a company together. So I agreed to think about that. You know, from a strategic point, it's the difference between the cool molecular biology that had been reported in actually making a product and having it be relevant for patients. I was not certainly a CRISPR scientist, but I did have a lot of experience thinking about what matters for patients and how to get technology there. That's why I tried to help, you know, thinking down the road as to where this work could be applied and where it could take us and ultimately, while you're right, I was on the board. They had this advisory role. I ultimately came into the company to actually help with some of that.
Matt Pillar:Yeah. So that's a good place to start talking about sort of these building these big foundational building blocks of building a biotech and beyond right, building a biotech hub, building an industry, if you will, an ecosystem around a technology. I mean, technology is one of those foundational elements. So I want to, I guess, get your sense of how fundamental CRISPR in this case is in terms of, like, being one of the foundational elements necessary to building momentum, not just within a company, but within an ecosystem or a region or a you know, quote unquote biotech hub, however you might define that Like. Would you say that something new to grab onto? You know, 30 years ago, monoclonal antibodies were kind of a corollary.
John Leonard, M.D.:You know, it's interesting the way you asked the question.
Matt Pillar:In a modeled way.
John Leonard, M.D.:it's an interesting no, I wasn't thinking about that, but I had occasion to give a talk recently in Chicago you know Abbott's there and I have many ties to the area about building, you know, a biotech hub and what it takes. And so, as I thought about what to, you know, tell that group, I went and I looked at the history of the Boston Cambridge Biotech hub and it was really interesting. You know, it's what I saw. At least what I took away from the history that I was able to glean was at least in the case of Boston.
John Leonard, M.D.:I think it's also true in the Bay Area, where there was a new technology it's recombinant DNA and you know the pharma industry at that time was largely staffed by chemists, much more so than molecular biologists, and some of the true innovators were academicians who had proficiency with this new approach and believed that you could go and use it to think about, you know, making medicine.
John Leonard, M.D.:In the early days it was very much about cloning genes. You know insulin and clotting factors and things that would grow hormone, for example, things that were missing from certain disease states. So I think there's certainly precedents where a technology, a major advance, can serve as the basis for a burst of innovation and depending who does that and where that takes place, you can imagine as was, I think, the case in both Bay Area and Boston that serving as the kind of the night is for something new happening and then all of the kind of attendance related sort of technologies spring up around it. I think now with CRISPR, you know, I see it certainly having the same sort of impact, but I don't see it, so far at least, driving new biotech hubs, I see it, you know sort of nesting, if you will, in the established hubs and drawing from these other technologies that have grown up around you know, microbiology and genomics and delivery modalities, gene-based medicine, et cetera, and enabling CRISPR in a way that would not have been possible many, many years ago.
Matt Pillar:So do you think that's a product of the portability of information and, I guess, the portability of people now compared to perhaps the back when Boston and the Bay Area were?
John Leonard, M.D.:I think a virgin. Well, I know, if I understand your question, I'd say, if imagine that CRISPR had appeared 40 years ago, 50 years ago, it would have been a fascinating technology that my guesses would have, you know, spent a lot of time in the laboratory and a lot less time, as it's doing now, headed toward the clinic and patients, etc. Because there were a whole set of other things that needed to happen, that the need to exist. When CRISPR came along to be able to be, you know, applied technology for making a medicine, and when I think about that, it's okay, you gotta have a basic understanding.
John Leonard, M.D.:Genomics, I mean the human genome worked out the ability to Get large-scale genomic information very, very fast, efficiently. When you think about these studies that we do to determine where CRISPR goes, how it behaves, specificity of it, if we were sequencing DNA today the way that we did 40 years ago, it would be prohibitively expensive and incredibly slow delivery technologies that have only evolved in the last. You know 10, 15 years or so needed to be there for CRISPR to be relevant. So you know it's different from, you know, recombinant DNA, when it was the idea you could splice a piece of DNA into another piece of DNA in a very specific way, hear the ability to, you know, go into the genome, change a particular nucleotide in some part of the body.
John Leonard, M.D.:It just requires all of these other things to exist that have evolved and come to be Over the, you know, the course of these last 30, 40 years. So it's like a lot of things. You know there's certain things that are ahead of their time and there are certain things that you know Come in the being when you know the time is right and take off and are successful, and I think that's certainly been the case here. You know I guess it's a long-winded way of saying that you know CRISPR, you know, to your question about biotech hubs, benefits from the existence of the already present hubs and the technology that they've built up. Yeah, it can be applied.
Matt Pillar:Yeah, and that the technology that they've built up, and I'm also assuming the, the research resource. So, to talk a little bit about that, like you know, if I'm in Boise, take CRISPR out of the equation. I'm a Boise. I discover a novel molecule and I'm like, okay, cool, I got something I want to build right. Like, I want to build something around this. I need some, I need some intellect, I need some people, I need some reach research capacity, right, like and it's not handy right. So how? How important is that? Is that Boston like physical and IP Infrastructure?
John Leonard, M.D.:it's, you know it's. It's a really interesting question because there are a lot of cities right now that see the success that Certainly has played out here in Boston or in the Bay Area to a certain extent, and Research Triangle in San Diego and places like that.
Matt Pillar:Well, I'm gonna, I'm gonna, I'm gonna throw my, my home state in there real quick, john. So Philadelphia wants to stake its claim right now as a, as a Selangino and ATMP Hub, and they're doing a great job, you know. But but, similar, similarly, you know, similarly, philly has resources like Penn and a great, a great healthcare network, a great you know chop they get a great hospital system down there. So, like, similarly, you know, they're, you know, they're, they're, they're aided by, by a pretty good Extended network or infrastructure.
John Leonard, M.D.:Yeah, that was an unintended omission. I mean absolutely. That's certainly the case. You know, I would say this you can do it almost anywhere, but it gets progressively harder as you move away from places where the skill sets are resonant. And call it abundant quantities, right, I Actually I think the pen is Example you gave is a really excellent one where they are leading many respects the whole CAR T revolution.
John Leonard, M.D.:It came from, you know, carl June and and a group there that came up with an idea that a plated out and Spread from there as it was picked up in other areas. So it's like well, what are the critical elements? You need an insight, you know you need People who can come and work on that. You can be virtual up to an extent, but depending on how you know large the undertaking is and how far you want to go, I think Virtual organizations have many, many limitations.
John Leonard, M.D.:You need to have a source of funding and you know money is Certainly portable, although it tends to reside preferentially in certain areas where you know Venture groups etc set up and have access to you know individuals and institutions etc. But it's portable. I think really one of the key things is an entrepreneur, you know somebody who's Driven to make something happen, who's got a notion of what's possible in that? Ideally For our exceptions, but ideally the experience To go and understand what it takes to make something happen. I mean you can have a lot of inspired people who want to go change the world, whatever.
John Leonard, M.D.:Yeah but if you don't have, you know, some experience, or can't, if you can't partner partner with somebody who does you can make a lot of mistakes, waste a lot of money, waste a lot of time and lose the enthusiasm or the support of the people that you know, the support of the people with the money that you need to make this stuff work. So you know, I think Biotech is In the early days, you know very much local phenomena, but it is Far more portable as the number of people who have, you know, played in the area and gained experience Grows. And so you know, for the that entrepreneur in Boise and for all I know, there are companies I've been looked into.
Matt Pillar:It was the. It was the first like remote sounding city that popped into my head. So, and a fine attention.
John Leonard, M.D.:You know, and I'm sure somebody wants to do it and you know it's. I wouldn't say it's impossible, I just say it's harder than in other places.
Matt Pillar:Yeah, even in places where Places like Boston and Philadelphia in the Bay Area that are flush with with with intellect and research capacity. Some of these newer technologies, like I mean you know you talk about the early days were common at DNA in Boston. You know CRISPR. Now there are multiple, you know, atmps where they're being developed right now that Perhaps require or could benefit from skill sets that aren't even fully developed at MIT. So so let's talk about that a little bit.
Matt Pillar:I want to, I want to kind of get your sense like as you built in, tell you, you know, as you, as you, as you've Doug deeper into some technology that's new, some stuff that's not been seen before. How do you develop that talent? How do you make, how do you ensure that you've got people who you mentioned? You said earlier, you've got to have people who can work on that specific researcher, that specific, but in some cases I want to say that there's people who it might be more important to have, people who want to Like maybe aren't trained to, but want to, Would you say that's true.
John Leonard, M.D.:Yeah, it's a really interesting question. I mean, by its nature, innovation means doing something new, and if you're doing true innovation, you're going to be doing a lot of new stuff, in almost all cases, building on things we know, drawing from experiences we've had, et cetera, but trying to channel that in a way that accomplishes something that hasn't been done before. And it's not like some quantum theorist looking at the world for the first time and coming up with just a novel way of seeing things. That feels like a departure from where we've been.
Matt Pillar:Oh, so you're opening the curtain up on this Wizard of Oz-like perception that so many people have.
John Leonard, M.D.:Yeah, that's why you say that, because I do think when I talk to people that I know, who are not technical people but friends and colleagues from other things, when I talk about what we do, it very quickly becomes a black box for them or, in the most extraordinary sense, probably magic.
Matt Pillar:With high expectations, no less. That's the thing of it. Right, the black box is the black box, but there's such high expectation, yeah.
John Leonard, M.D.:I mean I'll stick with the magic thing for a minute, because I don't mean the sound, I don't know what proper word is Like, devoid of reality or anything like that.
John Leonard, M.D.:But when you're talking to a person who doesn't understand some of the basic scientific aspects of what we do, when people live in the world of science understand, one thing leads to the next and something's derivative of the other and the related, et cetera. But if you've never thought deeply about that and you're having a conversation with somebody's working on your house, for example you know who may not have thought about this previously and you describe that you're going to go into a person's body, into cells of their name, the organ of your choice, find a particular gene and change some atoms in that gene so that gene behaves differently, they are either swept up in this is extraordinary, this is telling me more or it just it does not compute, it doesn't have the technical wherewithal to really understand what that means, and it can be very frightening, it can be very alarming, or it just depends on where one starts with all this stuff.
Matt Pillar:Yeah.
John Leonard, M.D.:But yeah.
Matt Pillar:So we went on a rabbit hole there, chased the new shiny moment. But what prompted that question was sort of my challenge that when you're entering into a new research area, curiosity, just as much as curiosity of the scientific mind, just as much as knowledge, right like a skill set in that particular technology, might drive progress. And you were being able to explain that it's not necessarily as you said, it's not magic.
John Leonard, M.D.:Right Exactly. And that's back to the notion of experience. I mean it's knowing how to pose questions and what questions are important and how to set up an experiment that is interpretable, that builds on the knowledge you have. And it really comes down to some of the new tools that we have the ability to pose different questions and do it in a way that is meaningful from the standpoint of a product that ultimately what helps to make. So that's where I say the experience matters. I mean it's one thing to get your PhD and say CRISPR wouldn't it be cool we could do X, y and Z Right.
John Leonard, M.D.:But if you intend to actually make that X, y and Z thing, that product, there's a process that one goes through and you need to know how to build the basis of understanding what the experimental tools and processes are to actually get to what you want to achieve. And it's experienced scientists, who may be coming from other areas that are somewhere related, will understand how to do that and they will get you there faster. They will do it in a way that has fewer zigs and zags and, ideally, they'll do it in a way that is robust, and I'll give you an example. So here at the company as we've grown we've brought in folks who have extensive experience, leading groups, leading scientists, who have these individuals have done things, different systems, but they understand how to carry out that research process. So you can take the enthusiast, the new scientist and have that individual be very, very productive. And it's certainly played out in our work in the clinic thus far.
Matt Pillar:Yeah, is there competition for those folks?
John Leonard, M.D.:Absolutely. I mean it's you know things have cooled off a little bit in the last year, year and a half, I think it's going in the market out there, but for I'd say, three years or so there was intense competition for people who have delivery technology, who have, you know, basic insights into, you know, bioinformatics, which is core to what we do here, people with experience with nucleotide chemistry, etc. He needs those people because there's so many different related efforts that draw on those expertises that you know are playing out in companies around the country.
Matt Pillar:Yeah, how do you, how do you get them and keep them?
John Leonard, M.D.:Well, one hopes that the mission, the culture etc. Is really inspiring and one wherea scientist, when he or she encounters that, feels like they can live a life of science in our organization. So it's not just a job, it's not just a paycheck. You have to be competitive with that, of course. But we try really hard to create an environment where people feel very connected to each other. They understand how the science comes together, irrespective of the particular piece that they may do. That may be, you know, 10 steps away from you know somebody else, and then we try very hard to connect them to the patient in the end. So they understand what this all adds up to and the difference that it can make.
Matt Pillar:You mentioned, you mentioned capital, you mentioned, you know, finance, money being a foundation element, and you also mentioned that things have cooled off recently, as we're all aware.
Matt Pillar:So, from your purview, right like if you're looking at the ecosystem, the people, the research, the intellect, the you- know, the region, the resources that it takes to create a biotech community, finance being one of those resources and any one of these resources is not working right. As a biotech builder, you ask yourself why and how do we fix that Right? I mean, I guess, fundamentally, like you think, oh, why isn't that working right? Why don't I have that resource? Why don't I have the you know whether it's intellect, people, money, whatever so yeah, so yeah, basic question like what's wrong with the market right now and how do we fix it? It's a basic but big question. But I mean, it's just in your perspective. Yeah, it's just one more real, quick piece of context.
Matt Pillar:You know I have conversations. We drop 52 episodes a year. I have conversations week in and week out with biotech execs. In the past three years since we launched this podcast. Like, I've seen the growth of the technology and the tools just in the past three years, incremental, like it's incredible, right, the growth. No, I don't think there's a time in here. I'm not going to say that because I'm I don't have the history, but I would venture, I would challenge, I would. I think I would say it's arguable that there's been, you know, no time in history prior to this where the technology and the science have come together to the point where we have tools, like we do today, and yet, you know, despite that advance and that promise, people are having really hard time getting money and they're putting programs on shelves and backburner things that have great promise for patients, because they can't fund them. What's going on?
John Leonard, M.D.:Well, first of all, I think most people would agree with you about the tools and insights comments that you made. I mean, it is unique. You know and this is unprecedented the things that we can do, and there's a lot of different people trying to do a lot of different things, which is exciting. You know, if one setting up a company, you're trying to get to a future that any investor will look for as coming with a payoff of some sort, a return on that investment and it's true, whether you're in a big company, you know, that has revenues and you're making decisions about how to, you know, set up the various programs that you can work on there. Or if you're starting out with a new company and you have a new technology and you're going to try to solve a problem, if one lives in the pre-commercial space, like here at Antelio, meaning that there's no products at this company that produce revenues, then you know, in some respects it's a simple economic exercise. You say, okay, well, what is that future? What might it be? What might it be worth? And as the economic circumstances change in the world interest rates going up, more economic uncertainty related to macroeconomic events that no particular individual can control, you know people make a judgment that the risk is going up as well. And so you know, if you think about what is a share price, you know, on the stock market is the discounted value of future earnings, and if the future earnings are discounted at a higher rate because interest rates are up, that means it's worthless today.
John Leonard, M.D.:And you can see that as you look across the industry in the pre-commercial space, in fact, especially those companies that are pre-pre-commercial, if you will, they're just starting out They've all paid the price of having their future discounted very, very aggressively. And that means that, you know, in the eye of the economist, the technologies are just not worth as much. You know the return is not going to be there. And in fact, you know you actually have some people saying well, I'll put my money in the money market and make my 5% the year, as opposed to taking a chance. I will lose if I do everything on some novel thing that we'll see if it works out or not. And that's what we're confronting right now.
John Leonard, M.D.:And plus, I think it's fair to say that you know the pendulum's swinging a little bit where. You know, a couple of years ago, when money was virtually free, you know interest rates were almost at zero. There was an abundance of companies that you know they could make that math work because it was discounted almost at zero, and so all of those companies at the same time are looking for ongoing, continuing investments. You know, in this environment that is progressively more demanding and so you know there's only so many investor dollars to go around. So I think our look at much more stringently than you know a few years ago and you're seeing the consequences of that and it's not inherently bad, but it does make it harder and one needs to be just better at what you do.
Matt Pillar:Yeah, I was going to ask you. I was going to ask you for your advice for new biotechs, of new biotechs who are maybe, like you said, pre-preclinical, like just starting out now, one piece of advice might be like find something else to do for a couple of years until things straighten out.
John Leonard, M.D.:Or don't start something that requires many, many years of progressive insights. I think you know companies that probably will do well. Starting now are ones that you know are shortened the path to a drug. I think investors will always be interested in something that you know will give them a return in just a few years, and you can see that with some of the companies that have been successful.
Matt Pillar:Yeah, yeah. So, adam, tell me, like what have I mean? Can you share any, I guess, experiences in terms of your leadership of the company through this kind of blip elite? I don't know if you'd call it a blip on the radar, but through this tough time we'll just yeah, it's beyond a blip. I think, yeah. I mean, is there, you know, is there anything creative that you've learned or had to do, or any lessons that you can kind of share with us around how to manage a company through this?
John Leonard, M.D.:Well, we never take our funding for granted and we try to be really thoughtful about what are the best places to deploy the capital. You know it's going to be a balance between things that you know maybe we're one or two steps away from an insight or those things that are just required more effort. So we try to balance that. You know. We try to take an approach with our budgets that you know that is tight, you know, and make sure that we're getting the most out of our people, that we want them to understand. That you know the cliche money doesn't grow on trees. That you know these are valuable resources that you know make it possible to do our work, and so we don't hide that stuff. I mean, we talk about it, you know, and I think that helps inspire better planning and thinking.
John Leonard, M.D.:And you know, but I'm always out there thinking about how to continue to keep this company fine, because we never want to be in a situation where the runway is so short that there's uncertainty about, you know, will we get to the next step where there's true value creation and where I was, you know, thinking about. You know, think about it as you know, stepping stones, going across a brook to get to the other side. We always want to make sure that we have sufficient runway to. You know, step across a few of those stones to make progress, so that investors understand where we're going and feel confident that we will get to keep the promises that we make.
Matt Pillar:Yeah, I want to get back to the community aspect right, like the Biotech Hub sort of community, great, greater, you know, I guess it takes a village sort of mentality around Boston and the Bay Area and Philly and other places like that. I'm always fascinated in this industry. Since I've been covering it, I've covered a lot of industries in my career and in most of them, you know, I covered high tech, I covered retail, like years and years ago, and competition was competition Like it was. There were very thick walls between you and your competitors In this space. The competition would seemingly be fueled, you know, fueled and just. It seems like it would be almost violent competition for the rewards of success right in this business. And there is like it is.
Matt Pillar:It can be cutthroat, but at the same time I've never been in an industry before where that community of like, sharing and progress in advance pre-clinical, pre-commercial obviously has been so collaborative, open. You know it's, I don't. I, you know it's really hard for me to get my mind around it. I'm that simple person who's like you know, we're either we're either friends or enemies. You know what I mean. Like it's fascinating to me. So, like in a community, in a Biotech Hub community. You know how do you? I guess I was looking for your perception on how you, how you, how you put up walls, how you guard yourself, how you allow collaborative exchange that fuels advance.
John Leonard, M.D.:I'll give you something that, an example of something I thought was extraordinary. I saw it, as we said early on. I worked at Abbott Laboratories for a long time and in the morning you would go through the gates with guards and security things and it was a square mile in the headquarters.
Matt Pillar:Yeah, I've been there. Yeah, I've been there, I can picture it.
John Leonard, M.D.:Yeah, I'm in a special place and we did a lot of great things, but the notion of having some person from a competing company there, it's just like you do everything you could to exclude that from getting into the company Early on. I had an experience here where I was in a room talking about some things, that Intellia and we got up to lower the shade because Nextdoor was a building with other scientists from another company who could just watch the board just for doing the work, and so I mean it's almost as if they were in the same room and that proximity was just mind blowing for me. You know, I'd say what I found. I'd say it this way. Well, it's certainly true that there are certain treatments where it's maybe winner-take-all If you cure something and you set the standard and it's very hard for that standard to be overcome and you're the first one there. That would be an example like that.
John Leonard, M.D.:There's not many of those, but it's not a zero-sum game. You know, in most cases where there's learning that takes place in the broader community that you know is applicable in so many different ways with so many other uses, that there's this kind of you know growing that takes place as people get insights that leads to formation of new companies, or collaborations or technologies that need to be shared, because nobody can do it all, particularly in smaller companies, and that's what I see. I mean I've been really impressed by the incessant networking that takes place where people who have similar positions or roles in companies. I mean they don't talk about their intellectual property, they don't talk about their key experiment, but they talk about what is to do their job, things about shared problems in companies, how to make them better, etc. That makes the community stronger, as opposed to being on an island trying to figure every single thing out for yourself, not drawing on the experience or wisdom of others, which I would say characterizes me for the first 20 years of my experience in this industry.
Matt Pillar:Yeah, yeah, I don't know. I just I get this, like you know, similar to the. You know the non-insiders' perception of the way that the work that happens inside the black box and the dramatic expectations. One can easily conjure up this vision of Boston where, you know, dr John Leonard is nervous on Friday nights because he knows that his scientists are out and about on the town talking with other scientists, right, I mean, you know you're hanging out in certain areas Like it's, you know it's the biotech up. You just get the sense like, how do you main? At what point do you say, okay, lock it down. You don't have to lock it down, keep it safe. Yeah.
John Leonard, M.D.:On a Friday night, typically I'm really tired, so you're not worried about anything. But I guess I would draw a distinction between secrets, trade secrets, intellectual property, which are zealously guarded and protected, versus ways of thinking, ways of working. Those are different things but are related. You know, we all want to have the patent that says you know this antibody or this construct, whatever it is, has unique attributes and is. You know there's value, that's protected, etc. There's not sharing of that.
John Leonard, M.D.:But how to get there, the ways to do work, is something that I think is is. It may be not freely flowing, but it is brisen in the community because we're all trying to solve similar problems with different ultimate end uses, and so I mean there's conferences every week about some of these things and it may not be you know the most, you know that morning's news, but it might be for people coming into the space, useful information for them to bring to their work, etc. So that's what I'm talking about, just the resident expertise in the community, versus the particular bit of information that relates to you know an individual product or you know a very, very unique and valuable insight.
Matt Pillar:Yeah, dr Lahren, we're getting short on time here and I want to respect your time, but I do want to give you an opportunity to talk a little bit about what is going on you know we talk about. I liked your conversation around cash preservation and the stepping stone analogy is that's a fantastic one, so I'll play off of that. Like what stepping stone are you on it and tell you right now and what's the next stone you need to skip over to?
John Leonard, M.D.:Yeah, I mean, we have a long-term strategy that we're trying to play out in a very deliberate fashion and we there's really three parts to it. One is the CRISPR space is ever expanding and there's new insights and derivative forms etc. We want to have what we call toolbox that allows one to edit DNA in whatever is the relevant format for a particular disease state. So we continue to do that, etc. But it's really the application of that that has the stepping stone approach, where you know we've tried to take existing technology, apply it in a use case that would give us the confidence that things were behaving the way that we're supposed to behave, and then expanding out from that.
John Leonard, M.D.:So the example would be knocking out a gene or first program, which is TTR amyloidosis, knocking out a gene in the liver. Then the question becomes well, can you build up a gene in the liver and then can you do that outside the liver? How do you extend that to other tissues that are more difficult to reach? And we're doing that work now, where it's this, I would say, a creation of insights, where one thing builds on the other, and for those examples where you can take a cell outside the body, what we've tried to do is take a similar step-by-step approach. Where can we make cells that can come from another person and go into a different individual, and do that in a way that enables all kinds of different approaches, whether for treatment of cancer or autoimmune diseases or, ultimately, regenerative medicine? So one thing enables the next, but we want to make sure we get the foundation right.
Matt Pillar:Excuse me. So from like a clinical perspective, can you give us an update there, like where are you on that path?
John Leonard, M.D.:Right. So in that first format knocking out genes in the liver we've got a program for TTR, amyloidosis gives, cardiomyopathy and polyneuropathy. We're now in phase three and that is a step before the drug becomes available to patients. And we have another condition, hereditary angioedema an unusual disease but one that's ideal for a CRISPR approach knocking out a gene in the liver which will likely be in phase three studies next year as well. So things have progressed and we're just beginning to treat patients where we build up a gene that's deficient in the liver for alpha-1 antitrypsin deficiency, a pulmonary if it's a genetic form of emphysema. And that's that stepwise format and in the next many months I suppose we'll be in a position to take those same technologies outside the liver into whether it's the brain or muscle or the eye, for example. All that work is underway.
Matt Pillar:Yeah, very exciting, super exciting, just real quick. One follow-up question on the clinical pipeline I often have conversations with companies that maybe have a late-stage candidate and a real early-stage candidate and they're like, oh, we kind of got something filling to do right, like what's coming behind these two later-stage candidates? I didn't tell you.
John Leonard, M.D.:Yeah, it's again alpha-1 antitrypsin which is in phase one. We have, in the later stages of discovery, ophthalmological diseases which you know is a very delicate genetics that go with that and we're poised to begin some of our allogeneic exfivo work which we'll go into the clinic and the immunoconcology setting. So we don't have anything right in phase two after these later-stage programs. But those programs are coming quickly.
Matt Pillar:Yeah, Very nice. Well, I appreciate the time. Dr Leonard, this has been very insightful. I know we meandered a bit, there's no doubt, which is that's typical of my line of questioning. You know we covered some ground, but valuable ground. I really appreciate your insight and your transparency around what it's taking at Intelia and your thoughts on what it takes to build a biotech community. So thank you for coming on. It was my honor to have you and we look forward to paying attention to where Intelia goes from here.
John Leonard, M.D.:Thank you.
Matt Pillar:It's my pleasure. So that was Intelia's Dr John Leonard. I'm Matt Piller. This is the business of biotech. We're produced by Life Science Connects Bioprocess Online, which offers a trove of peer-generated content for biotech leaders at bioprocessonlinecom. If you like listening into conversations with biotech leaders like Dr Leonard, subscribe to the business of biotech podcast, sign up for our newsletter at bioprocessonlinecom backslashbob and, as always, be sure to leave us a review. Let us know how we're doing, and thanks for listening.