All right. Good afternoon from the 40th Annual J.P. Morgan Healthcare Conference. My name is Cory Kasimov. I'm the senior large cap biotech analyst, and it's my pleasure to introduce Sana Biotechnology and CEO Steve Harr. Please note that following this presentation, we will move right into a Q&A session where you can send in your questions via the conference portal, and I'll do my best to work them into the conversation. With that, Steve, thanks for joining us today. Let me turn things over to you for an update.
Thank you, Cory, and thank you, everybody, for joining us today. We will be making some forward-looking statements today, as you might imagine. Please do take a look at our 10-Q and 10-K. We spent a lot of time on the risk factors, so hope you'll find that useful. Sana was founded with the belief that one of, if not the most important transformation that will occur in medicine over the next several decades is the ability to modulate genes and use cells as medicines. Our goal is to build, you know, one of the leading and sustainable companies of that era. If you take a step back, you know, pretty much every disease and certainly the clinical manifestations of each disease are really caused by damage to or dysfunction to a cell.
Our goal or aspirations are relatively straightforward. One, we would like to be able to fix any cell that's damaged by either modulating the gene or gene expression. Two is we would like to be able to build cells from scratch to replace cells that are too far damaged or actually missing. Three, we wanna do this all in a way that meaningfully expands access for patients. The company started out by trying to understand what are the fundamental challenges to reaching that aspiration, those aspirations, and really go after what I'd say are the most tractable challenges to getting there. A few years into this, I'd say a few things. One, the strategy that we chose to go after is at least as relevant today as it was when we founded the company.
The technologies we brought in are working. That's a ways from saying that they work. We're really, you know, excited about where we are. We've built a pipeline that we think can begin to generate meaningful INDs beginning as early as this year. We have the capability to go forward now, we think, with two to three per year. We're building expertise across really important areas, delivery of genetic material with virus-like particles and other things that we'll talk about, gene modification and gene editing, immunology, using the immune system both as a weapon and importantly, hiding cells from immune rejection, disease and stem cell biology in the manufacturing sciences.
We have the balance sheet to really see this through with $866 million in the bank as of the end of the third quarter, 2021. You often hear companies talk about being a cell therapy company or a gene therapy company, and at the end of the day, what you're trying to do is engineer a cell. We're relatively agnostic as to whether we do that inside the body or out. Importantly, by going after both of these areas, we've been able to build the core capabilities, which are highly overlapping in a competency and scale that we otherwise wouldn't have been able to. The risks, though, in the near term, are actually relatively idiosyncratic.
As we've approached in vivo cells, you know, what people call cell therapy or, sorry, gene therapy, basically what you're trying to do is deliver a payload to a cell that modulates the gene or gene expression. You can more or less do anything you want to a cell in a Petri dish. The real challenge has been delivery, which is what we went after. Our goal is relatively simple, and that's to be able to deliver any payload to any cell in a specific and repeatable way. Every time we do one of those four things, we actually open up a whole host of medicines for patients. The company was founded really on some technology that allowed us to do cell-specific delivery with any payload using things like virus-like particles or VLPs.
As we made progress here, we also have been targeting the ex vivo cell engineering. This is the side of the page where we get to go after the diseases that most of our loved ones will suffer from. It's harder, to be really clear, 'cause to make a medicine, if you distill it down to four things, you have to do all four. We wanna be able to manufacture cells at scale that can graft, function, and persist. The field has been fraught with challenges on each of these. Autologous cells, you know, are complicated to scale, but allogeneic cells or using somebody else's cells have a real problem with immune rejection.
From the outset of the company, we felt that if we were able to overcome this immune rejection and transplant allogeneic cells, and utilize stem cells as medicines, we would have a significant impact on the industry. We really started the company on two core technologies. One was an ability for cell-specific delivery, and the other was the technology that we believe allows us to hide cells from immune rejection. From that, we've been building up our capabilities and pipeline. It's translated into, I think, a pretty robust set of programs moving forward. A couple things to note, these are just some of the assets in our pipeline. First and foremost, you can see that we're pretty balanced between the in vivo and ex vivo cell engineering.
You know, second is we're building meaningful capabilities and pipeline in oncology, and importantly, we're also looking above and beyond that. Third is, you know, hopefully you'll begin to see some of these enter obviously with INDs this year. How did we go about building this? We'll talk about this in more detail going forward. The hypoimmune technology we talked about. Since the beginning of transplant medicine, a real challenge has been that when you transplant, you know, my organ or my cells into you, your immune system will reject them. The way the field has tried to grapple with this today has been heavy immunosuppression, but that's really limited the field.
As people began to understand the potential of stem cells to differentiate into different medicines, they realized that unless you could overcome allogeneic rejection, you wouldn't really get there. When some of the founders of the field went to their immunology colleagues, they got this very simple set of advice, and that is to go figure out the paradox of pregnancy. The paradox of pregnancy is that each of us is half mom and half dad, and the only reason we're on this Zoom call together today is our mothers didn't reject us as foreign. They really set about trying to understand what was different about that maternal fetal border. You have to overcome two parts of the immune system.
One is the adaptive immune system of B and T cells, and many of us figured out a long time ago how to grapple with that. You disrupt class I and class II MHC through gene editing or other mechanisms. Unfortunately, cancers and viruses figure out a long time ago, and we evolved something called the innate immune system, including things like natural killer cells to go after those cells, and they actually get killed very quickly if you don't target the innate immune system. What we found was that overexpression of CD47 is able to turn off the natural killer cells and macrophages of the innate immune system.
This system is something that if we can get to work, we wanna put into iPS cells or pluripotent stem cells that we can then differentiate into all kinds of different cell types and transplant if they're missing. How are we doing? First off, we got the system to work in a Petri dish or in vitro, which is always important, but biological systems are more complex. The first place we went was in the mouse models. We've done this both with mouse cells and with a mouse immune system, as well as human cells with a humanized immune system. What you see on the left-hand side of the page is that our gene-edited hypoimmune cells are able to evade being seen by T cells and antibodies.
That's the top set of graphs. On the bottom, what you see is that when you don't modify the cells, and you put in foreign or allogeneic cells, the T cells and B cells recognize them and reject them. For the right-hand side of the page, what you see is that we're also able to prevent our cells from getting killed by these natural killer cells. Natural killer cells don't recognize on wild type or regular cells. Unfortunately, the B and T cells will. If you just disrupt or knock out class I and class II, you can see that natural killer cells rapidly kill these cells. That's the middle panel on the right-hand side.
If you look at the far right, which I'm circling now, which is the cells that have the gene modifications of our hypoimmune platform, you can see that they have evaded natural killer cells, and over here on the left, you see that they're not seen by antibodies and T cells. It's one thing to do things in mice. It's another thing to do them in non-human primates, which is the closest we'll get to the human primate. Sometimes a picture is worth a thousand words. This is an experiment where we took eight non-human primates, and we injected our cells into the right leg at time zero and follow them over time. At six weeks, in the left leg, we put in the other type of cells.
If the animal got HIP cells or hypoimmune cells first on the right leg, they'd get unmodified cells on the left leg and vice versa. The goal was both to see what happens, you know, de novo and also when you have an ongoing immune response. What you see is for the duration of this study, in the right leg and the top panel, that our cells lived and thrived. Again, a picture being worth it. If you see those, the color is metabolic activity. It did that despite the fact that at week six, in the left leg, we put in unmodified cells, which were rapidly killed. That gave us some confidence that, hey, maybe, you know, our cells will live in the context of an ongoing immune response.
In the bottom panel, what you see is if you inject unmodified cells, they're rapidly killed within a couple of weeks. We can later put our hypoimmune cells in, and they will continue to thrive and not be seen, meaning that even if an animal has a preexisting immune response, our cells will live. What did that look like in terms of B and T cells? What you see. This is what you see here. To make a long story short, basically, B and T cells don't see our hypoimmune cells. They do react to normal allogeneic cell.
Importantly, on the right-hand side, what you see is not only do natural killer cells not see our cells, but we also can have a built-in safety switch, and that is if we make an antibody to CD47, those cells would be killed. That both proves our mechanism as well as provides maybe a bailout if something were to go wrong. This is the first and only time that we're aware of that anybody has shown an ability to transplant allogeneic cells in non-human primates with no immunosuppression. The next step is really just to go and take this into humans. Where are we going? We're gonna start with two cell types: allogeneic T cells and pancreatic islet cells.
Now, the allogeneic T cell, you know, by the way, both of these cell types are cells where the people have already transplanted cells, and that gives us an idea of how to go about this engraftment and persistent challenge. It's also an area where with the allogeneic T cells, we think we have, you know, a lot of the immune system's in our favor. The patient has cancer, they have to get conditioning chemotherapy, which will dampen the immune system for a period. At the end of the day, we only need the cells to live for, I don't know, 3-6 months, something like that, which we'll get into.
Pancreatic islets are kind of the opposite challenge, where patients not only have an intact immune system, but they have an autoimmune response that is already killing the cells, like with type 1 diabetes. That's where those monkey data are so important. Additionally, we need these cells to live for years and years for a patient to have the clinical benefit we wanna see. We have some programs on the right that are a bit further out. Why allogeneic T cells? First and foremost, sometimes people say there are a whole bunch of CAR T cell companies already out there. What do you guys offer? I will start with the fact that, in the U.S. and Europe alone, approximately 100,000 patients a year die of lymphoma, leukemia, multiple myeloma.
I think we all would acknowledge that the CAR T cells have had a tremendous positive impact. In the history of humanity, fewer than 10,000 patients have been treated with these therapies. Of those, many don't respond, and a number of them who do respond relapse. You know, my goal has been to turn lymphoma and leukemia and myeloma into hepatitis C, diseases where the vast majority of patients are cured with a single therapy. There are manufacturing and access challenges, there are safety challenges, and importantly, there are reasons why patients don't respond.
Either the cell type isn't good enough, the cell goes away, probably due to an immune response, and I think we can deal with those with our allogeneic platform, or they lose CD19 in the case of B-cell malignancies we're gonna get into in a minute. With our allogeneic CAR T cell, there are a lot of different approaches out there. When you're gonna make an allogeneic T cell, you're gonna put my cells into you as an example. You have to grapple with a few things. First off, my cells will try to kill you. That's called graft-versus-host disease. The first thing that we will do is knock out the TCR alpha loci, and that's been shown how to deal with.
The second is you have to grapple with host-versus-graft disease or your immune system trying to kill the therapy. Here we think we have a very unique system in our ability to hide cells from the immune system that is quite different from what's available from anybody today. I think one of the things that the field has shown is that cellular persistence or how long cells live in your body is really important to making these cells work. What we'll do is take a donor T-cell, gene engineer it and, you know, create a hypoimmune allogeneic CAR T-cell. We've shown that we can hide these cells from the immune system in non-human primates. We have data that show that we can make CAR T-cells which are hidden from the immune system.
The next question for us is, as we make all these gene changes, does it impact how well our cells function? This is a really important slide for us, and what it shows is that on the left-hand side, the answer is no, that our CAR T cells function really well. The left-hand side is your typical mouse model of leukemia. You can see by the, you know, day 23, they're all red, meaning that they're taken over by the cancer. What you see is a regular CAR T cell in the middle, which again, you know, seems to take care of the cancer at least through these 28 days. What you see is with our HIP-modified cells, comparable efficacy.
We see the same thing in a whole host of assays. We think that with our HIP platform, we can really expand access, we can make a higher quality consistent cell, meaning fewer side effects, and we can evade the immune system, which means that we will allow our cells to last longer. We think that in and of itself can create a tremendously valuable medicine. Beyond that, though, a number of patients lose CD19 or the target of these cells. Over the course of the last year, a group at Stanford has shown that if you utilize a CD22 CAR T-cell, this is an autologous CAR T-cell, and you can really go after these CD19 failures. This is 24 patients. 23 were CAR T failures.
One had a primary cancer that was CD19, it was missing the CD19. What you see is 56% of these patients have a durable complete response, and it's quite tolerable. This morning we announced that we actually licensed the CD22 construct to put into our allogeneic CAR T-cell. That kinda puts together our strategy for you. We hope to file our first IND as early as this year, targeting CD19. I think we can build the best-in-class CD19. We think CD19 and CD22 together give us a tremendous opportunity to increase both the number of complete responses and the durability of those complete responses in a way that we believe can have a tremendously positive effect for patients. The CD22 can also be utilized for CD19 failures.
We also announced overnight we brought in a validated BCMA CAR that we wanna utilize going forward to treat myeloma, which we'll get into. Finally, you'll see us go forward in other areas. We think this is a really exciting program, or platform, I should say, with a number of exciting programs, and you'll see a good bit going forward. It's not where we stop. If we are able to hide cells from the immune system, we think that an area we can have just a tremendous impact is type 1 diabetes. Human insulin has had a huge impact for patients. Despite all of the monitoring and the best insulins, a type 1 diabetic still has about a 15 years shorter expected life than a non-diabetic.
During that time, they have a host of challenges related to low blood sugar, as well as complications of high blood sugar like blindness and kidney failure and stroke. This is a large market. It's around 4 million people just in the U.S. and Europe alone. If we happen to get type 1 diabetes right, we can go after type 2. That being said, there is a lot of evidence that suggests that you can take a beta islet cell, whether it's from a cadaver or now we've even seen evidence from a stem cell, and you can make it into a beta cell, you can transplant it into a patient, heavily immunosuppress that patient, and their glucose will normalize. However, there aren't that many people for whom lifelong immunosuppression is safer than lifelong insulin.
Our goal is to produce a hypoimmune beta cell that can evade the immune system without immunosuppression and normalize blood glucose. To do that, you have to do four things. You have to make really good beta cells from iPS cells, you have to hide them from allogeneic rejection, you have to hide them from autoimmune rejection. Type 1 diabetes is a disease where the immune system kills the beta cell. You have to really turn this into a medicine. Where are we? This is an experiment where we show that we can make, you know, great beta cells. On the left-hand side, what you see is that we have a glucose-dependent secretion of insulin that is very similar to what you have from a normal beta cell.
As far as we're aware from the published literature, this is as good as it gets. What you see on the right-hand side is that you can put this into an animal model of diabetes and it will cure that animal of its diabetes. That's an important first step. The second is you need to be able to hide it from allogeneic rejection. You saw our non-human primate data earlier. We're excited about that. We wanted to show we could also do that in the context of making beta cells. What we did here is we made hypoimmune or normal beta cells and transplanted them again into an animal model of diabetes.
The left-hand side, what you see is if you put an allogeneic unmodified islet cells, they're killed by the immune system and the animal's diabetes goes crazy. What you see on the right-hand side is that our gene-modified cells both live and they function, really normalizing glucose in these animals. That's the allogeneic rejection. Now to do with autoimmune rejection. There is no mouse model of or monkey model where you actually create an immune response to beta cells, so we had to go about this a bit differently. We took the blood from many patients with type 1 diabetes and really tested to see what would happen when they're exposed to our cells. On the left-hand side, what you see are T cells.
If you put T cells from a normal volunteer and expose them to either an unmodified or genetically modified HIP cells or islet cells, you see they don't kill. That's probably you and me. At least I don't have type 1 diabetes. What you see if you take it from a type 1 diabetic, these T cells rapidly kill unmodified islet cells. In the bottom, what you see is that they don't in any way affect our islet cells. That's a real step forward. The next question is, do these people also have antibodies? Do the antibodies recognize and kill these islet cells? What you see on the left-hand side is unmodified islet cells, they will bind to and recognize them.
With our HIP cells, these antibodies again don't see them. We've now shown that we can make great cells, we can evade allogeneic rejection, we can evade autoimmune rejection. Now we need to make a GMP supply chain. It's more complicated and takes more time than you might think. We have to first make a GMP genomically stable cell bank. We've announced license with FUJIFILM or FCDI, as well as made some of our own cell lines. The second is you need to modify the genome, and we announced a license with Beam, you know, in the fourth quarter to go about our gene editing. Now where we are is we are making a GMP gene-edited master cell bank. All of this is complicated, takes time.
You know, we remain right on track like we talked about, and our goal is to file our IND here next year. It won't be this year, but next year. I'm gonna just switch tasks for a minute and go to our in vivo platform. As I mentioned earlier, the real challenge in the field to date has been delivery. Lipid nanoparticles or LNPs are good for getting things into the liver, but you also can't deliver genes. You can only deliver RNA or proteins. AAV allows you to do small DNA sequences, but it's pretty limited to where it goes, and it's complicated to manufacture, and the payload size is quite small. What we wanted to do is create a better-targeted delivery system.
You know, again, when you're faced with a complex biologic problem, one of the things we like to do is see if Mother Nature's already solved it, and if she has, to exploit that system. The left-hand picture is actually just coronavirus. We see it all the time. Those little red spikes are the spike protein that we've learned so much about. That's actually something called the viral fusogen. What it allows this virus to do is only target cells that express the ACE2 receptor. There are a host of different viral fusogens that target specific cell types, and we even use this as mammals for cellular communication and for fundamental processes like the delivery of genetic payload from sperm into an egg.
The sperm does not stop anywhere along the way to deliver its payload, but instead goes specifically to the ovary, again, utilizing a fusogen. The middle is a kind of a schematic of a viral fusogen, and it's a. Think of it as nature's logic gate. There's a G protein or guide, and there's an F protein in fusion. The guide is what gives cell specificity, and the fusion, the F is what drives fusion. What we do is we modify that G protein so that we can target basically any cell type we want. What you see in the right-hand picture is we put those fusogens onto a virus-like particle or VLP, or onto a virus, and that's something called a fusosome. The fusosome allows us to.
for cell specificity, so it binds to that target cell. It then. What you see here is it will fuse the cell membranes, in step two, and we then have delivery of the genetic payload in step three, where it can go into the nucleus and modify the genome. That's basically, in a schematic, how this system works. We've shown that we can get this to work across a host of different cell types, and a host of different genetic materials. Where are we gonna apply it? We're starting with T cells, which I'll show you in a second, and we're gonna go to liver cells and HSCs as well in the not-too-distant future. What's the idea of going after T cells? Our goal is in vivo creation of CAR T cells or inside the body.
The left-hand side is currently what happens with the manufacturing of CAR T cells outside the body. It's a complicated, expensive, and time-consuming process. On the right-hand side, you see our goal, which is in a single doctor visit, to be able to infuse our medicine without any conditioning chemotherapy and create the CAR T cells from that one visit. The way this works is the orange is our fusosome or medicine, and it carries our payload, and it will recognize the T cell and bind to it. You can see that here. It then delivers a payload, we call that minus sign here, a genetic payload, which creates a CAR, which is a chimeric antigen receptor. That's the yellow Y. That CAR will recognize cancer cells.
When it sees cancer cells, it will kill it, the cancer cell, as well as amplify, creating more CAR T cells. You know, does this system work? First of all, we've shown we can do this with targeting CD8 cells. We've shown we can do it targeting CD4 cells. We've shown we can do this targeting CD3 more broadly. We can do this in a specific way, and this is a mouse model showing that it works. This is that same mouse model of leukemia that we showed up above, and on the left-hand side, what you see is what happens if you make typical ex vivo CAR T cells. Again, no different than before. The saline, the leukemia takes over the animal.
With an ex vivo CAR T cell, it works very well over the course of a month to control the tumor. What you see on the right-hand panel is that with a single injection in the tail vein of the mice with our medicine, we will create CAR T cells, and we will eliminate the tumor. We've also shown that we can do this safely and effectively in non-human primates. Our next step is, you know, we're in the process of our manufacturing and GLP tox, and our goal is to file our first IND this year to move forward with these in vivo generation of CAR T cells. Where does that leave us?
We started the company over here on the left-hand side, trying to address the obstacles that we thought were most important and most tractable in really creating engineered cells as medicines. We're moving forward with our first set of human studies, and we think that they will do two things. One, they can create really important medicines, and two, they will validate platforms. If we're able to hide our hypoimmune allo CAR T cells from allogeneic rejection, we think we'll have a technology that we can apply confidently across a host of much more prevalent and larger diseases.
Similarly, if we're able to show with our fusosome platform in CAR T cells that we can deliver a genetic payload safely, efficiently, and effectively to a specific cell in the body, we will be able to apply that not only to make great medicines for cancer patients, but across a whole host of other payloads. We're excited about where we are and really looking forward to the future. With that, Cory, I'll turn it over to you for questions.
Terrific. Thanks, Steve. As we move to Q&A, I'll remind our audience that you can also contribute your questions via the portal, and I'll work them into the conversation. We have about 10 minutes or so for Q&A. I guess, Steve, just to start, I mean, with the first IND you're expecting for CD19 later this year, what are the key gating factors to getting that done at this point, and how much risk is associated with it versus more execution blocking and tackling?
You know, I would say with the hypoimmune platform, right, it is probably more execution blocking and tackling. You know, we need to, you know, do the manufacturing scale-up. It's actually the manufacturing scale-up side. We do the manufacturing transfer and create the drug and get it into human testing. I think with the fusosome, you know, it's an the first time people are trying, anybody we're aware of is trying to deliver this genetic payload in vivo. I think, you know, there's always some risk on two fronts. One, we could run into a safety challenge as we go through the GLP tox. We have dosed animals and, you know, in the past, and we don't have any reason to think we will other than science is unpredictable.
Two, we have to ensure that we get through the manufacturing scale-up and the manufacturing runs. You know, the long-winded way of saying the hypoimmune platform is really pretty straightforward, and I'd say both pre-clinically and in early testing is probably lower risk, and the fusosome, having never been done before, probably entails a bit more risk.
Okay. Makes sense. Then, you know, when you think about potentially developing better cell therapies than either what's out there today or what's in development, how does this ultimately get borne out in the clinic? Like, where do you expect to be better? Is it with the durability? Is it the overall activity? You know, how do you think about this once you are in the clinic with your initial products?
Well, I'll start with allogeneic T cells and move beyond that. Allogeneic T cells, really straightforward. I think data have been really clear to date that cells have to persist for some period of time in order to have durable, complete responses. What we'd expect is if we're able to hide our cells from the immune system, we will have longer cell persistence, and that will lead to higher and more durable complete response rates. I think if you add on to that CD19 and CD22, you know, and you're now targeting CD19 and CD22, we think we can take an entire another step forward. If you then go into
By the way, the other big clinical differentiator is today, if you wanna one of the ways that people deal with your immune system trying to kill my cells, right, is they just suppress the heck out of your immune system, and that comes with its own safety risks, right? What we wanna do is get away from that immunosuppression. When you go beyond oncology, really that immunosuppression becomes intolerable, right? As I said earlier, there aren't that many patients with type 1 diabetes for whom lifelong high-dose immunosuppression is safer and you know, preferable to human insulin. There are a whole the entire field, if you didn't have to immunosuppress them, would like to get rid of the complexity of human insulin as a therapy. There you just dramatically change the art of the possible by not needing to use immunosuppression.
Okay. I guess, you know, piggybacking off that last comment, I'm curious what you make of, you know, it's one patient, but the initial data that came out of Vertex, in terms of starting to prove the concept of what you're doing, albeit with immunosuppression.
First of all, it's great data. I've gotta be thrilled for that patient and it's a real step forward for the field, right? To be able to take a stem cell and make it into, you know, a human islet that has glucose-dependent insulin secretion at a rate that, you know, looks to be a normalizing function for that patient is both a great outcome for Vertex and for that patient. I have to say, it's not trivial to make a great cell at scale. You know, that's something that they've shown they can do. You know, that being said, it
What it does is it validates that this is possible, and if we can add that to our hypoimmune platform and do this in a way that does not require immunosuppression, you go from a disease that can treat, you know, a small number of patients, and I don't know what that number is, I'll let others decide that, to one that is potentially viable for every patient, you know, with type 1 diabetes and potentially viable for many with type 2 diabetes.
Okay.
You are now getting glucose-sensitive insulin secretion, right? That's what we all like.
Right.
You spent some time talking about the work you're doing in terms of the GMP supply chain. I just wanted to ask kind of on manufacturing overall and the progress that Sana is making here, as well as, you know, unique challenges that you may or may not face in this area that might be different from other cell therapies. Or is it really pretty similar to others in the industry?
Yeah. I'll start by saying, you know, we started this company, and we, you know, thought we would over-invest in manufacturing sciences and manufacturing capabilities. You know, it felt that way for a few years. As soon as you start getting a whole host of pre-clinical data and trying to make medicines, you wish you had more, right? It's almost impossible to overinvest right now in cell and gene therapy and process and analytical development. Second is, you know, our strategy around where we would manufacture was really clear. We would use, we've built our own pilot plant. We're building our own plant for late-stage clinical and commercial, and we would use CMOs for phase I.
You know, I think one of the challenges with that is that these supply chains are tight, and CMOs make their money by having more or less 100% capacity utilization. Getting slots, particularly at the time that you want them, is not always easy. So far, you know, that's a risk that we still have going forward. The third thing that I would say is that, you know, if you look at our different programs, they have different levels of novelty, to your last question, how different they are from others. Making a gene-edited allogeneic CAR T cell, there are a number of companies doing that, right? We're doing it a different way, but I don't know if there's anything from a supply chain perspective that is unique and hugely complicated.
Making gene-edited iPS cells that we then turn into, you know, type one, I should say, pancreatic islet cells or heart cells or glial progenitor cells, that's complicated. The number of GMP iPS cell banks in the world that meet our immunologic criteria are not many, I should say. The second thing you have to do is show that they're genomically stable. That is an endeavor that I think the field is still trying to understand how to even do. I think we've made progress. To make a gene-edited genomically stable iPS cell bank, you know, no one's done it yet. There isn't even a manufacturing facility that's easy to do it at. There aren't people that are trained to do it.
Then you have to figure out how you're gonna measure this genomic stability after gene editing. All of those things take time, and they require a lot of resources internally. If we get it right, we think we have a meaningful competitive advantage. That's where some of the novelty is. In terms of manufacturing fusosomes, I mean, it's a technology the others don't have. It's complicated to scale. It's, you know, something where we're still working through getting this to a commercial scale. We think we've gotten it to a scale where we can move it into, you know, human testing, but we have work to do to get it to the scale that we want for the long run.
Okay. A question from the portal here is: Where in the genome locus do you have your knockout in place, your CAR T construct?
We haven't disclosed that.
Okay. I didn't think so. Then I'm just curious, and we're running low on time here, but you, when you think about your initial data, assuming the CD19 program gets to the clinic first, you know, how much does that initial program de-risk the overall platform from a hypoimmune standpoint in kind of what you're trying to do? How much more I mean, you've shown data in mice, you've shown data in non-human primates. When you get that initial data in humans, is that gonna be, like, sort of your aha moment, or is it gonna have to be somewhere outside of CD19 as well?
Yeah. I would say, I believe it will be. I think if you see these cells, and they hide from the human immune system, and you've seen this now in mice and monkeys, you can really get a lot more confidence this technology works. Some of our scientists would say, "Hey, you know, every situation is gonna be different, and we're gonna need to ensure that we look at, you know, beta cells and, you know, different cells as we go." I think if you see these cells live for a long period of time, you have an aha moment. I also think if you see with the fusosome platform that we can deliver the genetic payload specifically to T cells in vivo, right? That is an aha moment for the field of gene delivery.
Regardless of what the cancer efficacy is, I mean, that's our goal is to make a great drug. If it turns out that, you know, you have to add, you know, to provide cytokine support as an example, it will still be a really valuable step forward for the field in terms of delivery. I do think that first set of data, while our goal is to make really important medicines with both of these drugs, at the same time, they provide, you know, really significant insights around both of our platforms.
Okay. We get probably 30 seconds left, so quick answer here from the portal. How long do modified islet cells last in mice? The chart showed 30 days, but have you seen them go longer?
They've lasted as long as we measure them.
Okay. Okay, that's good to know.
Same, by the way, the modified cells in non-human primates.
Okay.
Both mice and non-human primates, they last as long as we can watch them.
Okay. Terrific. Well, listen, this is fascinating stuff. I wish we could sit here and talk about it longer, but we are out of time. Steve, thanks much as always for your time today and best of luck with all the progress.
Thank you, Cory. Great to see you. Enjoy the rest of your week, and thank you everybody for joining us.