Sana Biotechnology, Inc. (SANA) Earnings Call Transcript & Summary

Your biotech analyst here at Citi. And today, it's my pleasure to be hosting Sana Biotechnology for a fireside chat at Citi's Global Healthcare Conference. I'm joined today by CEO, Steve Harr. Steve, thank you so much for being here today.

Thank you for having me, Samantha.

Well, so why don't we just start off with a little bit of an overview on Sana. I know you recently streamlined our pipeline to focus on SC451, which is your T1D islet cell product. And also SG293, which is your in vivo CAR T product. Just talk a little bit about that decision and what Sana looks like going forward?

Sure. First of all, thanks, everybody, for joining us, both in the room and online. And I think you guys know we'll be making forward-looking statements. So feel free to check out our risk factors. And we spend a lot of time out of and so they're using worth reading. A couple of thoughts here. One, I'll start with type 1 diabetes. So type 1 diabetes is actually really well understood disease, right? It's a -- from just the highest level, the patient's immune system has attacked and knocked out the pancreatic beta cell. And the pancreatic beta cell is the only cell in the body that makes insulin. So prior to the advent of insulin therapy about 100 years ago, it was a death sentence and patients would rapidly start to death over the course of a few months and a pretty gruesome death. And even like 40 or 50 years ago, when you talk to people, the only way that people were taking insulin, but the only way to check their sugars and things like that was by their urine, right? So it's been a very gradual improvement of patients' quality of life and outcome. But if you look today, there are over 9 million people, almost 10 million people who have type 1 diabetes. It's growing at a rate where it's supposed to be about $15 million within 15 years. The patient -- even if they manage it with the best possible care today, they have on average about a 10-year shorter expected lifespan. During that time, they make about 140 executive decisions every single day about what they're going to eat, how much insulin to take, what carbohydrates they have? Are they feeling a little sick. For a young woman, it might be what time of the month is it, all those types of things impact how people think and what they take. And at the same time, they have to worry that they could die from too low blood sugar and that most likely they're going to face a long-term future, where they have risk of blindness, amputation, heart attack, stroke and death. And so it's a disease. It's a giant unmet need. And it's been known for about the last 20 years that if you can replace pancreatic islets, patients can come off insulin. They actually do quite well. And there is a group up in Canada led by James Shapiro, who started transplanting cadaveric islets into people with type 1 diabetes. The problem is it's not a very scalable source. They get them from cadavers, right? It's a very variable source based upon kind of things that happened around the time of death for the donor. And patients have to be on lifelong immunosuppression, really no different than having an organ transplant. That leads to all kinds of side effects. There really aren't that many people for whom lifelong immunosuppression is better than lifelong insulin. So it's done. People get transplants around the world, and there are hundreds of them done every year, but it's never really scaled into a real solution. Over the last several years, several groups have shown that you can take stem cells, pluripotent stem cells and grow them into pancreatic beta cells. And when transplanted, they will also very predictably actually improve patient outcomes and get them off of insulin. So it's more scalable it's certainly more replicable, but you still have this problem of immunosuppression. And over the last several -- over the last year, what we've shown is we can make a few gene modifications, I'm happy to get into and that with those gene edits, these cells are invisible to the immune system. They both overcome allogeneic, meaning kind of transplant rejection as well as the autoimmune destruction that typically occurs in type 1 diabetes. So now all the component parts are there for a onetime curative therapy, which is our goal, a single treatment. In our case, it's intramuscular injection that allows these patients to live the rest of their lives with no more insulin, no immunosuppression, no monitoring and normal blood sugars. And our goal is an IND and to begin a clinical study next year. We think that it will be very quick to read out whether or not first we've overcome immune rejection with our stem cell-derived therapy and then also to see hopefully euglycemia or normal blood glucose. Then you're really looking at a challenge of scaling the manufacturing -- and I think as you've heard from -- we'll get into this, I think, later from the FDA today, as recently as today, but also we've heard from regulators around the world that should be a relatively straightforward, at least clinical path to the market. That's that one. We're really quite optimistic. We own 100% worldwide rights to it. We think it can be one of the more important medicines that has been created. But there also were a lot of challenges to us getting there. The in vivo CAR T cell is a bit different. It's obviously -- so here what we do is -- we take a virus-like particle and engineer it to deliver a payload directly to a specific cell type and the in vivo CAR T case is to a T cell. And the goal is to be able to give a patient a single treatment with no lymphodepleting chemotherapy like what people have with autologous CAR T cells. And be able to go after a host of different blood cancers, whether that's lymphoma, leukemia or ultimately myeloma as well as autoimmune disorders like lupus, scleroderma and others. At the beginning of this program, we made 2 critical bets, and they're really important to understanding how good our platform is. One is that cell specificity matters, meaning you want to deliver the payload just to your target cell. And we believe that to be true because one, just manufacturability, right? If most of your cells are in T cells. So if it's going to deliver most of your -- even if a small percentage of liver cells are transduced, it will be the vast majority of the drug product. The second is immunogenicity, right? If you get in into antigen presenting cells, you're going to have problems with immune responses against CAR T -- against the CAR. And the third is just general safety. That's part 1 bet that cell specificity matters. The second is that you need to integrate into the T cell to get the right level of killing. You're probably making a couple of hundred million CAR T cells, but you're trying to kill a couple of hundred billion target cells, which means you have to log rhythm -- multi-logarithmically grow or expand your T cells. And so our thought is that just simply put an mRNA and it will get diluted. So if it turns out we're wrong on those 2 bets, we've made things complicated, right? If it turns out we're right. I think we have a best-in-class medicine. I'm quite convinced if we're right and if you were a nonhuman primate, you would want our medicine. It really -- it looks quite promising. And we need to get that into humans. We've given our guidance for 2027. There are paths we get this done next year. The path we get it done, actually get data next year, we'll have to see kind of how that all plays out. But that's also exciting. It's a little bit different. It's a more competitive space, right? There are -- and -- but I think it is a relatively well understood and straightforward space for us to develop into. So those are the 2 things that we're really focused on in the company right now.

It's a great overview, Steve. It gives me a lot to work with here. So why don't we just start with type 1 diabetes. And you mentioned that the IND and potentially a Phase I, both cleared IND cleared potentially and Phase I start as early as '26. And I know you have multiple interactions with FDA over the last several months. And as you noted, we had a keynote speaker this morning, Dr. Marty Makary, who called out specifically type 1 diabetes islet cell transplants without immunosuppression as a key focus for the administration. So I guess just given your guidance and FDA's interest, can you just share a bit about where you currently stand in preparing the IND and if you're able to share any context from what FDA feedback has been on that process?

Yes. So first off, our goal is to both to bring this drug forward in the United States and also to bring it forward in a few other geographies. So we're engaged with dialogues with regulators around the world. I think the feedback has been generally very consistent. And the 2 things that we need to do, which I'll come back to where the important risks are the 2 things we need to do to move this medicine into human testing are 1 complete or nonclinical toxicology package and 2 is complete GMP manufacturing. So this is a -- so what the medicine is we took a -- you take a donor cell, a single donor a long time ago, reprogram that cell back into a pluripotent stem cell. That's your starting material. We then gene modify it once. We knock out 2 genes and we knock 2 in. And that cell is rigorously tested and that, that cell is a starting material, hopefully forever for our drug product. It took us a long time to make that cell and not see genomic mutations pop up from time to time. And so we really -- it took us several years of really hard work, and I would argue maybe a little bit of luck to see this really play out as it has. But we now have a master cell bank that retains pluripotency and does not mutate as you go through what is likely trillions of divisions over time, right? And so that's been hard for us. So now our main risk -- the main risk of this drug is safety, right? I mean it will probably work. We've proven every component part of it. And there are really 2 safety risks that we worry about. One is just in the very short-term severe hypoglycemia, cells die, release insulin, It's about 24 hours. It's been seen in other trial, super easy -- it shouldn't be complicated to manage, right, just monitor the patient if they have it just get a little glucose. The second is that over the long-term, off-target sales becoming -- or target cells with mutations becoming tumors, right? And so that's what we really have to work hard on preventing as we go through our preclinical testing as we make modifications in our manufacturing process to ensure that we have the safety that is necessary for a population, while this is a very, very high unmet need, and it is a very unsatiated patient population, they will live for decades, but for us, right? And so we have a real responsibility to put -- to make this medicine as safe as we can. And so that's where we spend a lot of our time right now is that safety part of it. Generally, regulators. So to your point, I mean, I think we still have to ensure we have global consensus around what our clinical protocol is going to look like. I think we know we have alignment with at least several geographies and we can then move into human testing, I think, in a really straightforward way. You can look at one of our -- there's a company out there that's done a Phase I/II/III study with immunosuppression. It's going after a smaller patient population, right, people have a very severe form of the disease. Their Phase I/II/III program in aggregate was around 50 patients, about 13 in Phase I and about 37 in Phase II/III. I think that's a reasonable guide. I could make arguments why we should be smaller. We don't have immunosuppression. I can make arguments why we should be more patients. We're going to go after a much broader patient population, pretty much anybody with type 1 diabetes. And we'll have to see kind of how our safety profile emerges and if it ends up probably being pretty close to that 50 number.

For the nonclinical piece on safety, how are you preparing that in terms -- in the way that gives FDA confidence on the potential cancer aspect of the safety package?

Yes. I want to get into too much of it, but there are I mean, first off, just a rigorous testing of the genome of the cell that we started with. And there are some things you have to really worry about at the outset off-target edits easy to look for, right? Genomic recombinations as you go through division. You have chromosomes that are a little bit unstable because there are stem cells and also chromosomes that could be a little bit unstable because they've been gene-edited. That's complicated to look for. We have to do that. The third is not seeing mutations at predisposed patients or should predispose those cells to become tumors. And that's been -- that's taken us a long time to deal with, but the testing is relatively straightforward. And so that's part of the safety profile, and then you have to do things to ensure your product purity is high, meaning most of the tumors that you would worry about, let's just -- you're going -- going from a stem cell to endoderm and then you go to like primitive gut and ultimately, you go into making a pancreatic islet, which is a beta cell and it's called support structure. Any off-target cell along the way, particularly something like stomach or gut could continue to divide, right? And you see that in the academic literature all over the place. And you really don't want a bunch of stomach over 20 years dividing in your arm, right? It going to become much more than a new sense if it keeps going. And so really working hard to ensure we have the right product purity so that, that doesn't happen.

Right. Okay. That makes sense. But the takeaway here is that you've had alignment that you can take that lead GMP master cell line forward for this ID. Is that confirmed at this point?

Yes.

That's great.

We're good to go.

Okay. Love to hear it.

And that was like just -- I think the reason that for those who aren't as close to us, you may not have heard people talk a lot about a master cell bank and making any product in the past very frequently. It's like the cell that you start with. We really struggled with this, and it took us a few years to really make this happen. And if you talk to large companies around the -- in our industry would tell you that these just don't exist. These like gene modified pluripotent master cell banks and GMP made it GMP. And ours is also O negative, meaning that it can go into any type of a donor. And so putting that all together has taken us many years and several more years than I thought it would, which is why we had to be so transparent upon it because we struggle with it. And it's now in the rearview mirror.

Great. I'm glad that we've passed that. So looking forward to hopefully hearing when you have that IND cleared. And from that standpoint, how quickly do you think you could initiate a Phase I trial and get it up and running?

Pretty quickly. It's not overnight, right? That's why it wasn't true. When we changed our guidance at the end of the third quarter to say our goal was both to get the IND done to start the trial next year. That was intentional to give you a little sense that we're increasingly confident of the timeline and it's not towards the very end of the year to be able to pull that off.

Right. Right. Of course. And for the manufacturing for Phase 1, I think you've said in the past that you have capacity to be able to support a Phase I trial, correct?

Scale. I mean capacity is now probably scale is hard. It's only like 13 patients. We can do this for Phase I, but it's a Phase I process. I don't want to fool anybody. We have real work to do to turn this into a process that we're comfortable with a scale for launch, right? And so you have to have your launch process finish before you can start a registration study. You're not going to be able to make any substantial changes. And so that really, I think, is a long pole in the tent to us probably starting a registration study, although we've begun to really, I think, figure out what we need to do and make some progress around what that would look like. And again, if you just say there are 10 million-ish people on this, if you just kind of just even 2 million in the United States, 100,000 people a year it would take you 20 years to treat the people in the United States, assuming no new patients, right? It would take you 100 and some years to treat the global population. So we have a lot of work to do to get to even a scale that is important for patients. I don't think we'll start at 100,000 people. I'll be very clear. I think that's something that's really kind of aspirational. But our goal is to not be at something where we're smell like a CAR T cell, right?

I mean, so is it possible that you would be able to complete your Phase I and then have a gap clinically as you solve the scaling problem. Like I guess I'm trying to get a feel for like the timeline of when you might have enough to support the Phase III and also have that process locked in sufficient for commercial.

I think the clinical path is super straightforward, but to know exactly how long it will take probably requires us to have global alignment around any staggers that might be in that study as well as any dose escalation. But my assumption is a long pole in the tent is starting a registration study is actually manufacturing. It won't be terrible from your perspective because you'll get some longer-term follow-up, right? And -- but you'll know the first patients pretty quickly, if this is working. I mean there are people around the world who would have approve this regulator is on a very small number of patients that happens to work because the competitive products you have -- you're giving with a known toxic drug, right? You're giving it with substantial immunosuppression or patients are stuck with insulin that's really problematic for them. But we have some work to do on the manufacturing side to ensure we can consistently deliver at a scale that's important something for patients.

You're not the only one that's developing an iPSC cell line for various indications. There's Parkinson's, there's other type 1 diabetes programs. I mean is there a community or learnings that as a field can help aid you in the manufacturing process? Or is it truly like Sana specific based on this...

There are learnings -- now just order of magnitude, the number of dopaminergic neurons that someone might transplant is probably less than 10 million, right? If you look at the order of -- the number of islets cells that someone might transplant, it's circa 1 billion, right? And there are way more patients with type 1 diabetes and Parkinson's disease. And so you just have a -- it's a bigger scale problem. But there are elements of this that are very, very universal. As an example, you want to grow your pluripotent stem cells at the beginning before you start differentiating that would be used for any product, right? Whether it's -- and that would be true probably for -- there are a number of people using embryonic stem cells and induce pluripotent stem cells. And that should be very similar. You're trying to do to maintain pluripotency and genomic stability, right? And that learning is something that will be important across the field.

Is there anything that partnering with the FDA can help accelerate and obviously, the clinical piece, but does FDA have any sort of insight into the manufacturing scalability piece as well or?

I think if the FDA had insight, they wouldn't tell us. I mean my experience with the FDA is they're extraordinarily good at maintaining company's trade secrets. They're just a really well-run organization.

Right. No, I didn't mean it from that perspective. Okay. All right. Got it. So then...

There's something the industry could come together and work on, right? That's different. But industry consortiums or things like that or working with the CDMOs, but it won't come from regulators.

Okay. So then, I guess, what are your latest thoughts on partnering type 1 diabetes? Is that on the table at all in the near-term future? Is that something you would need to finance the company?

Well, We can finance the company, right? I mean we can raise equity capital is expensive for us, right? So as I think about a partnership, they can do 2 things, right? They -- we know we're selling off a portion of our future cash flows, right? If that happens to work. So in exchange, you'd like to have 2 things happen. One, we'd really like to increase and improve the company's financial resiliency. I mean I just look at something like this. It's like a stem -- a gene-modified stem cell-derived therapy. The probability we face a little hiccup along the way is pretty high, right? And financial resiliency gives you the ability in the rearview mirror, you look at that, and that was a little speed bump. No financial resiliency and it can have an impact on returns for your shareholders and stakeholders, even your ability to move it forward in the right way. So -- but the second is you'd like to think it improves the probability of success or in some other way, increases the size of the pie because otherwise, you're just fighting over crumbs, right? And -- so if we're just dividing up the pie, it's not useful. We want to really see an improvement and probably success. So as we talk to potential partners, I mean, one, we have a high bar for doing this, doing something just given owning 100% worldwide rights for this as we start to unlock data over the not-too-distant future, can be very valuable for our stakeholders. And there aren't companies that have inherently substantial capabilities in bringing forward scaled stem cell-derived therapies, right? Just hasn't yet -- there hasn't been one approved in the planet yet. So we're looking for someone who can really help us with that, improve our probability of success, right? And that's going to have to be a good economic deal too. We're not going to do this for some royalty in the future or something that would be just a death of the company. The -- and the 2 biggest challenges outside of capital because capital drives time, right? We'll be scaling this manufacturing to something that could meet the demands. I kind of think of it, though, is there are 2 elements of scale. There's number of doses per manufacturing run and then there's a number of manufacturing runs. And a number of doses per manufacturing run is a science problem. Number of manufacturing runs is a capital problem, right? And so finding a partner who helps us with the science problem is way harder than finding a partner who can help us with the capital problem. And so we've really been pushing anybody who wants to just engage in partnerships, say, how do you help us -- how can you help us solve this science problem, which is making more cells every manufacturing run. The second is it's not going to be straightforward, and I don't like to give people thinking about things that are not, but it's not going to be straightforward commercializing a curative therapy, right? I mean it's a onetime payment and this is a disease that affects millions of people. Hepatitis C was a challenge for the system to digest and it was much smaller. And it was a relatively short course of therapy. Society is winning in a big way from all the work that Gilead and others did to really solve the hepatitis C epidemic. But that was a challenge for a few years for society to digest, and this is a much, much, much -- this is millions and millions of people, right? So some -- we know we're going to need a partner to help us at some point, it's going to be different solutions in different parts of the world. It's not going to be -- and even in the United States, there's likely a private market and a government-based solution that we're going to have to grapple through with and work our way through. So those are the things we ask to a partner. I'm sure that someone will come up with something to help us over time. But we're in no rush because we can take this forward ourselves, at least for the foreseeable future because the risk we're grappling with are things where I think our team is really well situated to solve.

Right. So you can definitely get through Phase I proof of concept in humans on your own?

No problem.

Yes. Okay.

I think we can solve the scale. I think we'll solve the scale problem on our own. It won't be a big company does it for us. The capital -- long-term, we might be better off with a partner to solve some of those other things. But the science part of it, I actually think we're making we're kind of making progress what we need to do.

Got it. Okay.

Yes. Can you do you mind us using your microphone since we're on a webcast. If not, I'll just repeat it.

Yes. I was wondering here if part of scaling up for you guys is also about data management, right? I mean the very few patients, but the data type that you're looking at is very heavy, right? I mean it's molecular data, it's very, very dense. As you scale up the company and deliver your drug your therapies to kind of a greater population. Do you see like a growing challenge around data management for you guys? Or is that not really part of the picture?

I don't think so, but I'll give you a caveat. This -- if you're going from a -- every manufacturing run is going to be its own -- it's going to be a little bit different, right? And if we want to treat -- let's say it's around 1 billion cells. That means every 1,000 people is 1 trillion cells. It means every -- and so we are like 30 trillion cells, right? We have all kinds of mutations all over our body and a system that's set up to control them. We're going to need to figure out how do we tease out when bad things are happening, what led to them and how do we make that less likely and/or how do we identify it early and stop that run. I said something, sorry. So that part of it -- I think the manufacturing part of it will be very significantly related to data management. The -- some of the other clinical developments should be really straightforward here, right? I mean our goal is patients come in, they have to take insulin many, many times a day, all kinds of form, they're of it, right? It's like it's 0,1, right? So hopefully, we're going to have an analog type outcome on this.

Bit of naive question. How do you actually inject the cells into the pancreas? Or do you have some other mechanism -- and the number of cells what happens to these sales long-term. I'm sure you've done some animal work despite what our commissioner said that you've done some sizing pigs or some other animals to show how well do. I don't think we go to pancreas.

I don' think we'll go in the pancreas. It's going to be super -- we're going to do something much easier, but a little different. So the current standard just take a step back. So there have been thousands of panc islet transplants done. And what is done is a large bore needle is put into the inferior vena cava and they are injected up -- sorry, the portal vein and they're injected up into the liver. And that's what they -- but we don't want to do that for a host of reasons. It's space limited. There are a lot of toxic. So what we've actually been doing in -- we did in our first in-human studies put it in the muscle where you have a lot of capacity. We know that every year, there are approximately 14,000 thyroidectomies done in the United States. And each of those, the parathyroid is dissected out ground up and put into the forearm of the patients. So you can put -- you can put endocrine tissue and it functions very well in the muscle. It's done some in Europe where they now are putting islet transplants into the muscle. And so we're doing muscle. It's not quite as simple as you just inject it. If you go through the New England Journal of Medicine paper, it was done on a 18 different tracks very slowly was how they did it so that you don't end up creating a big bolus of cells that can't get oxygen essentially and sugar to survive. And so we'll just put in muscle. And the goal is, again, they've been gene modified, so the immune system doesn't recognize them. And some of them almost certainly will die in the course of that transplant. And so part of what we need to continue to do is work to ensure we solve that Amazon last mile problem, which is getting the cells into -- from our hands into the muscle of the patient.

And how do you get the cells to stay in the muscle?

Do you say it again?

How do you get the cells to stay.

They just grab, they stay. If they -- and in fact, what happens if they leave, is they'll end up in the bloodstream and they'll be killed. It's one of the reasons not to put them you have some -- we all have something called the immediate blood mediated immune response. And it will -- we're not supposed to have somatic cells in our bloodstream, right, because it's probably a tumor or something. So they get killed almost instantaneously. And so the goal is they need to engraft and stay, and they do. Like that's part of the -- one of the things that we do as part of the pre-IND work as you do biodistribution work to make sure you can't find cells in other parts of the body. And it's been done now in humans and other places without a problem. And we've done it in nonhuman primates and in animals, other animals.

And the level of insulin produced by a certain number of cells at that constant is that constant level as you get a steady state?

You don't want a constant level of insulin.

I'm asking you.

You want a glucose-sensitive insulin secretion, right? And so just like our natural pancreas. One of the beautiful parts of this drug is there is no such thing as far as I'm aware, as an overdose, right? You and I have a lot of islets and so you get this glucose sensitive insulin secretion. That's part of the release criteria of the product is ensuring that you do have a glucose sensitive insulin secretion. And did you have a follow-up?

About the regulatory approval process across regulators, MHRA, European regulators, Asia-based versus FDA. In your experience in early conversation, is it the same path when you submit for...

So my experience in cell and gene therapy broadly is it's similar but different. And different countries may have different clinical requirements and certainly, different countries have different manufacturing requirements. Within the context of this, what we've started out from the outset is to try to get alignment in different areas, really, hopefully, using the FDA as the template for others to grapple with. And then go forward with that. But the goal is to do a universal application and a universal process. And then this disease is really very much focused in the United States, Europe, the Middle East. It is in other parts of the world that's in Asia, but it's not nearly -- if you look at -- there's a very much of a geographic and maybe a hereditary -- or definitely in a hereditary component of this is somehow mix to create this higher risk. And so Nordic region, the United States, Middle East countries, those are some of the higher prevalences, Canada prevalence of disease.

Steve, is there anything that didn't get brought up yet about type 1 diabetes that you think it's really important for everyone to know.

I mean I just always think -- I think the thing that can be lost in all of the science and all of the -- this is a really unsatiated patient population. They're very engaged, right, and pushing a medicine forward. And it's a very large opportunity. Like there are very few opportunities in my career that I've been around for a long time that I've seen where it's this many people with this type of impact that you can have. And what we need to do is to make sure we do this both urgently and safely. I think that will be our biggest challenge is making sure we are again, despite the fact that there's very high unmet need and patients really want something different. They will live for a long time without this therapy. And so we need to make sure we're not doing anything that puts that at risk and only makes it better.

Got it. Okay. And so I wanted to spend our last several minutes just on the in vivo CAR T platform. You gave us a little teaser. Can you just tell us how that works and why it's differentiated from the other approaches that are in the field.

So there are basically 2 approaches in the field. There's people who are trying to take lipid nanoparticles, similar to what was in like vaccines and things like that to deliver some type of a payload to cells. Those tend to go to the liver, but they've worked really hard to have less of it go to the liver and all some of it go to T cells and things like that. Those companies tend to put mRNA in it, which doesn't integrate into your DNA, It sits there, which has some safety advantages, at least theoretical safety advantages, but these cells tend to go through many, many, many divisions. I'll come back in a second. So there's a group of companies that do something called virus-like particles or VLPs. And really, what you're doing there is taking some virus structure and modifying it so that it doesn't replicate anymore so that hopefully we will target a certain cell type and that it can deliver the payload that you want since what we've done. So what we do is we take a -- we're the only company who does this take a paramyxovirus and modify it. And the beautiful part of it a paramyxovirus is it's a logic-gated system to enter a cell, right? So we're able to get both cell-specific delivery, and we never go into the endosome and instead, we go directly in the cytoplasma in the cell we target. And the reason that's super important is any other delivery mechanism pretty much requires endocytic to uptake into an endocyte before it goes into the cell. So they end up anything that sticks to, whether -- if they have a CAR on the cell surface, which every VLP does it may end up in a tumor cell, right? If you have a -- if you end up on an antigen-presenting cell, you're going to end up in that cell, and that's going to present for immunogenicity that's going to kind of create immunogenicity problems. And so we get very cell-specific delivery in a way that others don't. And we just go into T cells. We've shown this across multiple nonhuman primate studies. That's the main difference of what we're doing. We've done this. We can do this in HSCs. We'll publish a paper very shortly that shows cell-specific delivering of gene editing agents, either kind of a CRISPR/Cas9 or base editing to modify hematopoietic stem cells. I think that's really exciting, but we need to focus first on getting it to work in the first place, which is we're targeting which is in vivo CAR T cells.

And that's a good lead-in because there's been a lot of interest from pharma strategics in the in vivo CAR T, but there's also potentially a lot of interest in HSC editing as well from an in vivo standpoint given busulfan conditioning that's required today for sickle cell disease, gene editing therapies. How do you think about potentially partnering this program across all of the cell types that you can target?

I think it would be an excellent one for the company to partner. It's something where -- we have said I think the company's capital -- we're not limitless in our capital. We're pretty capital constrained. I think our shareholder base is very aligned on the importance of type 1 diabetes. And so to the extent that we are paying to develop anything in this in vivo delivery capability, it's a little bit with our shareholders gradually being drug along. Because they really are big believers in type 1 diabetes. It doesn't mean we shouldn't do it. But I do think a partner can really help us accelerate what we're able to do. And within -- even within the fusogen platform, you have so many different places, so we can do in vivo CAR T, CD19, in vivo CAR T cancer and autoimmune. You can go into BCMA, we can go into -- and we have constructs for all of that. We can go into novel targets, we can go into solid tumors. We can go into things like CD22 who then can go into HSCs, right? So all of those -- you can partner this away without selling the farm in type 1 diabetes is a single asset, right? And so that becomes more complicated for us to partner and retain long-term value in other assets.

Have you had any interactions with pharma on any of these programs, if you're able to share?

Sure. I would say the big difference I tell people this for a while is that within the context, it's changing a little bit. But in the context of the stem cell drive therapy, the interest is relatively narrow. That's not for every company in the world, but it's pretty deep where we're having dialogues more. Within the context of the in vivo CAR T, I think there's a lot of just kicking tires. You're seeing more companies get very -- we have such, I think, good nonhuman primate data that there's more interest in that. But we are -- there's still -- there are kind of 2 camps that the strategics fall into. They want the simplicity of the LNP mRNA that happens to work it's just a lot easier to manufacture, right? We will have made this system really complicated. If it doesn't work, then you're going to need something like what we're doing. And I do think that what we have is very differentiated nonclinical data, and we need to get human data to see if it compares with others that are starting to create in the clinic with patients, and we haven't done that yet.

And you mentioned in the beginning that 2027 is your current guidance for IND for this program, but maybe you could pull that forward. Could you just talk through some of the levers that could allow you to do that?

I just need to go to a different geography, right? So there are just some complexities in manufacturing, we have to grapple with that we don't have to deal with in some other places that allow us to move faster. Finally, you get data before we can get a U.S. IND done.

Okay. Was there a question? Okay. So I guess, what would make you choose to move to the other geographies?

We need to make sure it's something we can do, right? We just need to make sure it's something we can do. I mean we're pretty -- for a company that's relative -- again, going back, I don't want to overplay, but we're relatively capital constrained, right? We're a pretty small company. And so speed to human data is something that can be very important for us being able to properly invest in these assets, understand them.

Got it. Okay. Well, then in our last minute, Steve, maybe you could just recap some of the things that we can look forward to over the next 12 months? And anything you really want to emphasize for investors to understand about Sana?

Well, let's start with, I think we're entering a period where it's -- I feel like [indiscernible] is finally coming. We've been working on this type 1 diabetes program and trying to move into humans for a while. And we've gone through a number of stages that we've meaningfully derisked, right? We've actually done these gene edits in cadaveric islets and seeing that we can transplant cells into people or into a person and that they survive and function for the long-term. And that's a massively derisking event both for the type 1 diabetes, but also for the platform more broadly. This should work, right? I mean again, I think that every part of the type 1 diabetes platform that we put together has been tested in a human and been shown to work. And so now we put all the component parts together and make it happen. And we're at the cusp of having that happen. We will be hopefully in the clinic next year. That means data -- and data will come very quickly once that happens. It's longer than I hoped it would be from when we start out, it's taking more capital than I thought it might. It also looks to be way more transformative and much more likely to happen than I thought it was. I mean that's a good part about these things. The in vivo -- so that is the company's major focus going forward will be type 1 diabetes. I don't see that changing. The in vivo CAR T offer a wonderful opportunity for us to continue to diversify a little bit or just begin to diversify and/or to bring in a bit of capital for the company. It's a super promising platform. Again, if you were a nonhuman primate, I'm very convinced this is the medicine of all of that have been brought forward you'd want to take. And I know none of you are. And so we need to see if that translating to people. And I'm optimistic it will, but we have to see that happen. And that all can happen -- it's all going to happen finally relatively short-term for this industry. So it's an exciting time for us. That's how I'll end it.

Yes, absolutely. Very exciting next couple of years for Sana. Well, thank you, Steve. This has been wonderful. I really enjoyed it, and thank you so much for being here.

Thanks.