Good afternoon, everyone. Thank you for coming here. Welcome to the 43rd Annual JP Morgan Healthcare Conference. My name is Fan Xunzu. I'm an associate within the J.P. Morgan Healthcare Group. Today, it is my pleasure to introduce CRISPR Therapeutics. With us today, we have Dr. Sam Kulkarni from CRISPR. The presentation will be in the format of 20 minutes of presentation, followed by a 20-minute Q&A. With that, I will yield the stage to Dr. Sam.
Thank you very much, and thank you for having us here to describe the journey of our company, CRISPR Therapeutics, and the outlook going forward in what could be one of the most consequential years in the journey of our company. Before I jump in, I will be making forward-looking statements, and I encourage you to go to our website for a full list of risk factors. 1-5-10 is an easy way to encapsulate what we've accomplished over the last decade at CRISPR. We have one approved therapy in CASGEVY, which I'll talk more about, based on the Nobel Prize-winning CRISPR-Cas9 technology. We have five programs in the clinic across different disease areas, including oncology, autoimmune, cardiovascular medicines, and rare diseases, and we have a rich pipeline beyond that in 10 preclinical programs, which are all very promising. Our business is divided into four franchises.
One of the franchises that's core is hemoglobinopathies, or Heme, as we call it. This is where we have our approved drug, CASGEVY, which is partnered with Vertex, and it's a remarkable medicine for patients suffering from sickle cell disease and beta thalassemia. And there's a continuous focus on improving the profile of CASGEVY and expanding it to a larger population with gentle conditioning agents and in vivo gene delivery. We'll talk more about all that. Beyond that, we have the world's CAR-Ts, based on allogeneic modality. What that means is we take healthy donor T cells and have sophisticated edits to make CAR-Ts very potent and are able to address both oncology indications and autoimmune indications with these CAR-Ts.
Beyond that, we have our in vivo approaches, where we take the CRISPR-Cas9, encapsulate the nuclease and the guides in an LNP particle, which is a soapy nanoparticle, and send it into the body to go to the organ of interest. In the first instance, it's the liver, and then modulate genes that are very important in cardiovascular medicine and some rare diseases as well. Finally, we have a franchise that could be transformational in diabetes, where we take cells and engineer them to become artificial pancreas. In other words, we differentiate iPS cells or stem cells into islet cells that function like your pancreas and produce insulin in response to glucose, and that can replace your faulty or defective pancreas. That's a very rich portfolio, and I'll describe each of these franchises and the potential of each as we go along. This is our full pipeline.
Because of the breadth of our pipeline, it's a bit of an eye chart, but as you'll see, we have the one, five, and 10, and all 10 are not shown here on the preclinical side, but we have the five programs that are in seven different clinical trials right now beyond CASGEVY. If I look at the history of our company, we were founded in the end of 2013. 2014 was sort of a seminal year to raise money and evangelize the technology. But really, the last four years have been foundational in the sense that we've had a relentless focus to bring CASGEVY to the market, and all that happened in a very short time frame for a technology as powerful and novel as this. We've diversified into other therapeutic areas in the meantime, using the same competencies that we built for CASGEVY.
Very importantly, we've brought manufacturing in-house and operationalized it to a level where the cost of goods are very low and very seamless. This year promises to be an inflection year because, one, everyone's curious about how CASGEVY is going to do in the market from a commercial standpoint, and we'll have more updates on that. Really, this is the year we have several clinical readouts across all these different franchises that I described, including oncology, autoimmune disease, and diabetes. We also are very active right now on the business development front, both on, as we discussed, things with pharma, but also with other smaller companies that can bring in new technologies to what we're building at CRISPR.
All this positions us to be a sector-leading biotech, not just in the cell and gene therapy space, but across the biotech industry, because we have a path towards profitability and sustainability with CASGEVY and what we expect CASGEVY to do. We have several clinical programs that are going to move into late-stage development and a very rich preclinical pipeline that ensures that we have a sustainable rate of innovation with what we're doing as a company. So starting with, and that brings us to this point around the inflection year in 2025, we have several milestones and readouts that we expect this year. CASGEVY, of course, will have a regular cadence with Vertex, or as we disclose our numbers of patient cells collected and also revenues every quarter, and you see that on the top.
But beyond that, we'll have a very broad update for our CAR-T, both in oncology and autoimmune disease this year. We'll have an update on our CTX131, which is targeted towards both solid tumors and T cell malignancies this year. And it may be surprising to a lot of people, but the first update actually is going to come in the form of our in vivo therapies for cardiovascular diseases in CTX310 and CTX320. And this has moved very quickly through development in the clinic, and we'll have a data update there. And beyond that, we'll have an update on diabetes as well. So it's a catalyst-rich year. We'll have plenty to talk about all throughout the year, I'm pretty sure. So starting with hemoglobinopathies, 2023, December 2023 was a landmark moment in the history of biomedicine with the approval of the first gene-editing medicine in the world.
And the approval by the FDA of CASGEVY signaled acceptance for what I think of as the third wave of modalities in the pharma market. We have small molecules, and now we have biologics that are almost 50% or greater of the pharma innovative pharma market. And now you're going to have this third wave of cell and gene therapies, and CASGEVY is a really important landmark approval in that continuum of the pharma market. It was covered, of course, in a lot of journals and received global attention.
One, because it's the first of its kind gene-editing therapy, but it's also very significant in the sense that 75 years after we all learned about sickle cell disease and understood the molecular basis of a disease, where one letter out of our 3 billion-letter code of our DNA is faulty and that causes such a terrible disease, we're finally able to bring a transformative therapy to patients that had no other options and were living with the disease. And it's not just a rare disease. This is over 60,000 patients if you just consider the U.S., Western Europe, and the Middle East markets. Beyond that, there are more than a million people living with terrible disease around the world with sickle cell disease. And of course, severe thalassemia as well is very prevalent.
But if you look at 2024, which is a foundational year for us, we're now approved in eight jurisdictions around the world. We have more than 50 authorized treatment centers around the world, and we've already started collecting patient cells, and we have more than 50 patients who had cell collections, which is remarkable progress for the first year. What's different about this launch compared to a lot of other pharma launches where you have small molecules and antibodies is as soon as you launch a drug, it's available in a pharmacy or a hospital setting, and you see revenues right away. Here, what we have is this is molecular surgery.
There is sort of a nine-month period where we start a patient on this molecular surgery journey, and then we collect their cells, manufacture, send it back, and then we ultimately treat them, which is not a problem because it is an elective procedure, although these patients will say that every day they live with the disease is terrible and they want to get the treatment as soon as possible. But what that leads to is sort of this different kind of launch curve where we have a more gradual ramp-up, but it builds on itself and ultimately have compounded growth because you have more activated treatment, authorized treatment centers, and each of these treatment centers will do more patients per year. With that, it is more of a device-like launch, and we look forward to providing more updates on how the launch is going.
But what's been very heartening is the global system support for a drug that really meets the needs of these patients that have no other options. In the U.S., you've had recently announced cell and gene therapy access model by CMS, and it had bipartisan support to say that we need to make these medicines available to all the patients suffering from sickle cell disease, and CMS should help all these states. What's been great is even in Europe, where there's a lot of pricing pressure in pharma, there has been support, and there was support from NHS for both thalassemia and sickle cell around the pricing for CASGEVY. And beyond that, an MOU that was signed by our partners, Vertex, in the Gulf region, where we are seeing a large number of patients.
In fact, there may be more patients suffering from severe sickle cell disease and thalassemia in countries like Saudi Arabia, combined Bahrain, UAE, and Qatar than you have in the U.S., perhaps. And that support from the system in the Middle East is very, very important, almost doubles the originally anticipated market that we had. And beyond that, given this patient demand and given this interest and the system support, Vertex and us continue to invest in expanding our manufacturing footprint so that we can meet the needs of all these patients. We're not going to stop there. We're going to continuously innovate, particularly around conditioning agents, where we can use a much more targeted conditioning agent that will shorten the hospital stays for these patients and allow for a much more seamless and more safer approach to changing the long-term hematopoietic cells in their bone marrow.
And so we're making good progress there in animal studies. Both Vertex and CRISPR have two different or parallel efforts ongoing, and we'll put those together and see what's best that can accrue towards these patients that are going to get CASGEVY in the future. And also, as we plan for the next decade, we're looking at in vivo HSCs that can allow us to address patients not just in the developed markets, but all around the world, including Africa. And that's important research that we're doing. So let me move on CAR-Ts. For those who are not familiar CAR-Ts essentially stand for chimeric antigen receptor T cells, but we're taking T cells either from a patient or a healthy donor, engineering them using CRISPR and directing them to attack tumor cells.
We're harnessing the body's own immune system, which is very powerful and has provided curative treatments for many patients suffering from cancer. We have our CTX112 program, which is currently in clinical trials for both oncology and autoimmune disease, and we've expanded the autoimmune disease basket trial given all the data we're seeing CAR-Ts in autoimmune diseases. We have CTX131, which is in trial for both solid tumors and T cell malignancies. And finally, we will bring CAR-T. This is an CAR-T targeting Glypican-3 or GPC3 to patients in the first half of this year. So plenty of rich portfolio there, but CTX112 is probably the best-in-class CAR-T out there, even given all the edits we're making and what we're seeing from a PKPD profile, which is a pharmacokinetic and pharmacodynamic profile. So a little bit of history of allogeneic CAR-Ts.
What we saw in the last seven years was dramatic data from CAR-Ts. We were taking cells from the patient, CAR-Ts with it, sending it back to the patients, and nearly half the patients with terrible lymphoma were cured, and not using the word cured lightly, but five years out, they have no disease, which effectively is a cure. The problem with that is it can only be administered in academic medical settings with hospitals, inpatient, and it's very cumbersome to manufacture. Plus, it costs a lot to manufacture, which leads to a very high price for these drugs, almost $500,000 to patients.
What we can do is take a very young person's healthy T cells and manufacture hundreds of doses or doses for hundreds of patients and make all these edits to make them so potent that they're almost as potent as CAR-Ts while they're easily available off the shelf, and combine that with the fact that we have all this experience with CASGEVY around genomic assessment, manufacturing, FDA approvals, FDA validation gives us a very clear path to be first in class and best in class with an allogeneic CAR-T, so this is a design of our clinical trial. I won't go into all the details, but it's very simple. As patients come into the trial, the median time to dosing is a matter of one or two days for these patients.
We don't have to wait a month like CAR-Ts because the product is available off the shelf. We use standard lymphodepletion, and then you see 30 days later whether you've gotten rid of the cancer or not, and the data we presented at ASH in San Diego in December 2024 were quite remarkable for a couple of reasons. One is there's only 12 patients worth of data, but for very, very sick patients coming into our study who had tried all sorts of other therapies, you were seeing a response rate and a complete response rate in line with CAR-Ts. Nearly half these patients had responses, and they were ongoing responses.
You see the numbers here on the swim lane on the left side and the CR numbers on the right side, the 67% ORR, but what's important is 50% CR rate in these patients that were very, very sick and had very poor prognostic factors. Now, beyond that, what was striking is the expansion data from these cells. Here you see on the left side, and these are new data beyond the data we showed at ASH, where we now have an N of 25. And what we see for dose levels three and four is an expansion of cells nearly; it's close to 100,000 cells per microgram and copies per microgram. And that is autologous-like.
In fact, if you look at the numbers, more than autologous, if you look at the table on the right side, and no other CAR-Ts come close in terms of expansion, and why is that so important? Because I think the expansion of these cells indicates that these cells are seeing CD19-positive tumor cells and are expanding to kill those tumor cells and create an inflammatory environment to get rid of the cancer, and it's a very strong indicator of how potent CAR-T is. Another striking observation from these data, as we've looked at additional data, and I won't present all the additional data because we want to save that for academic conferences, but if you look at this N of 25, we looked at patients who got T cell engagers, what's called TCEs.
These are bispecifics or T cell engagers, and there's a lot of excitement about T cell engagers for this patient population. We looked at patients who had several lines of therapy but had T cell engager therapy prior and either were refractory or relapsed from these T cell engager therapies, and here are six patients that were treated at dose level three and four, and all six of them responded. In fact, at dose level four, all three of those patients had complete responses, including patients who had T cell engagers in the seventh line of treatment, and this just shows that even in this world where everybody's saying, "Who's going to win? CAR-Ts versus TCEs versus CAR-Ts?", and just to remind you, we haven't dosed CAR-T patients, but I suspect that we'll see responses there as well. With TCEs, post-TCEs, we're seeing remarkable activity.
And that bodes really well for CTX112 in cancer, but also in autoimmune diseases for the following reasons. Very clear why CAR-T versus CAR-T will be better. It's much more convenient, etc. But when it comes to autoimmune diseases, there's an underappreciated fact, which is you don't have to take patients off their existing therapies in autoimmune diseases to give them an CAR-T. Whereas CAR-T, you have to take them off their therapy to collect T cells, and that can be problematic. And these are patients. Lupus nephritis patients, for instance, are severe patients. A lot of them are losing kidney function. You can't just take them off therapy. Significantly lower COGS. Order of magnitude or more advantage in COGS and scalability, given the easy manufacturing.
And then versus T cell engagers, the depth of response is going to be deeper versus T cell engagers, which is very, very important in autoimmune diseases, particularly as you go deep into the tissue and get the depth of response at the nerve endings in the deep tissue. And the data I just showed on the post-TCE patients in oncology bode well for what may happen in autoimmune disease. And of course, if you think about us versus other CAR-Ts, you just haven't seen the same level of expansion with any other CAR-Ts. And I think the case studies from even CAR-Ts that are not as potent show that you can have tremendous impact for these autoimmune patients. So all in all, we think CTX112 can be a very, very important drug in both these diseases.
I won't spend too much time on the rest of the pipeline here, but on CTX131, we've talked before quite a bit about CD70 and the potential for this very sophisticated CAR-T construct with the most edits that we know of that are out there in the clinic for both T cell lymphomas and solid tumors, and we'll have more to discuss there as we disclose data at conferences, and then Glypican-3 in an indication, which is hepatocellular carcinoma, where there are really no options. These patients, a lot of them, when they get to metastatic stage, they die within six months, and so there has been encouraging data out of CAR-Ts in hepatocellular carcinoma with this target, Glypican-3, and not only do we have the same target, but we have other potency edits that make CAR-Ts even better than what we've seen so far.
Let me turn our attention to our in vivo approaches. They're targeted towards cardiovascular medicine indications or risk factors. CTX320 is targeted towards Lp(a). Now, most of you will know about LDL and triglycerides, two of the silent killers that we always talk about and think about, but very few people talk about Lp(a). But it turns out nearly 20 million people in the United States alone have elevated Lp(a). And this is a very significant risk factor that can lead to heart disease, MACE events, and even mortality. And it's being recognized now by the pharma industry as a very important target. CTX310 targets ANGPTL3 or angiopoietin-like protein 3. And that's very important to modulate LDL and also triglycerides, two of the other risk factors. And then we have CTX340 targeting angiotensinogen, where I'll show some exciting data in NHPs, and CTX450 for acute hepatic porphyrias.
So very scalable platform that relies on the same LNP vehicle that once you have one target validated, it's very easy to scale into multiple targets and expand the portfolio. So as I said, Lp(a) is emerging as a key target to reduce cardiovascular events. And in the U.S., if you go to the doctor, very few doctors today measure Lp(a). But in Europe now, especially in Germany, Lp(a) is becoming a routine part of your sort of cholesterol measurement or your health measurement. And it turns out that Lp(a) is six times more atherogenic than LDL cholesterol. We talk about bad LDL all the time. Lp(a) is six times worse in terms of being a risk factor for cardiovascular disease. Not only that, it's cumulative.
It's not like you can take a medicine at 60 and all of a sudden all the damage done between your early years goes away. If you don't have an intervention early, it builds up in your arteries and veins. Lastly, with LDL cholesterol, oftentimes when you have a different lifestyle, you have exercise, you can actually modulate that LDL cholesterol pretty easily. There are several agents available, but you can't with Lp(a). There's no other agents available. What we did is, and this is data from NHP experiments in monkeys, where we said, "Here's our CRISPR-Cas9 with a certain guide. Let's give it a single shot into the veins of these monkeys." What we saw was 70%+ editing in the hepatocytes in the liver of these monkeys, which basically means nearly 100% editing in the hepatocytes of these monkey livers.
Now we have data two years out, which shows that you get a sustained reduction of Lp(a), and it's durable for two years, which means it's probably going to be a lifelong reduction of Lp(a) with a single injection. This is a design for our trial. It's not very surprising. We're going to escalate through dose levels, and we are in dose escalation right now with our clinical trials spanning multiple sites and countries, including Australia and New Zealand. We are going to bring it to the US as well. This is where we're already treating patients with very high, even though it's a phase one trial, we're already treating patients with very high Lp(a), which basically means we're going to get a readout in the patient population that ultimately where we're developing this. We'll have several biomarkers, not just the Lp(a) reduction.
I think we'll have data in the first half of this year for 320, as we mentioned earlier. So this last chart, and again, I won't spend too much time on it, which is this is the distribution of Lp(a) levels in the population. What you see is, of course, it's skewed. There's the normal range in green, but then you have quartiles where the Lp(a) levels are very, very high. What you see in the middle chart is your risk factor or your risk of cardiovascular events linearly goes up as you go into the further quartiles of Lp(a) level. At the highest quartile levels, you have a pretty high risk of cardiovascular events. In fact, we were talking to some of the investigators that were telling stories of all these patients.
One of them is a 22-year-old kid who has a 400 nanogram per milliliter Lp(a) level. That person's father, uncles, everybody has had a CABG by the time they're 50. Everyone's had a heart attack in their 40s. They're all fit, exercise, eat well, but nothing they can do about Lp(a). And so for those patients, what are you going to do? You have to intervene early with an Lp(a)-reducing agent. And that's why there's siRNAs in development, but gene editing can be the most powerful of all those approaches because you're going to get a sustained reduction of Lp(a) for life.
And then coming to CTX310 for ANGPTL3, again, this is supported by natural history studies where there are people where their ANGPTL3 is knocked down or have low levels of ANGPTL3, and they all have low levels of LDL and triglycerides without any negative impact to their overall health. So you can safely knock down ANGPTL3, reduce these risk factors, and not have any other adverse events for these patients. And similarly, we had these high levels of editing in NHP studies. We show sustained reduction in NHPs, sustained editing, sustained reduction of triglycerides, and also reduction in LDL levels. And finally, hypertension is one of the biggest killers out there. People take four or five different medications to manage hypertension. There's the high-risk population.
And if you said, "Oh, we're going to do gene editing for hypertension," a few years ago, people would say, "What are you talking about?" But now this is possible. You can go upstream of ACEs and ARBs and treat and actually knock down angiotensinogen using gene editing in the very high-risk population. And you can take patients who are taking four or five medications a day and bring them down to one medication a day that's titratable. And you can also do this not as a single dose. You can titrate the gene editing levels by doing one or two doses or maybe three, and that would get it exactly right for that patient. So this is very, very powerful. And here we show data in mice where we show this reduction in mean arterial pressure as we dose them and edit them to different levels.
On the right side, we show CTX450. This is for a rare disease called a set of diseases called acute hepatic porphyrias, where we go after the ALAS1 target, and by knocking it down, we can actually cure hepatic porphyrias for life based on data from siRNA and approved medicines there. Finally, our very important pillar is diabetes. Today, we live in this world where you have all these GLP-1 drugs and everything else, and we don't know how the world's going to be in the future in diabetes, but what I do know is there are going to be severe patients who are going to need insulin and are going to have, especially Type 1 diabetes patients, who are going to have very high A1Cs, and what we've done is basically created artificial pancreas.
We can take stem cells, differentiate them, and not just cells that are native. We've edited these stem cells, and we differentiate them all the way into islet cells. And that's what we've done. And we have three parallel efforts. On one hand, we've licensed this technology to Vertex, who are scaling it up and bringing it to the clinic across multiple countries and jurisdictions. But we have our own programs where we have ES-based or embryonic stem cell-based efforts with CTX211, which we're progressing inside a device, and CTX213, which is IPS-based and has more advanced edits that we're going to bring to the clinic as well. So finally, why did I say that 2025 could be the most consequential year in the history of our company's journey? One, I think CASGEVY, we believe, is going well.
2024 was a foundational year for CASGEVY, and that's been successfully executed by our partner, Vertex, and what we're going to see here is inflection points as CASGEVY patient demand ramps up, more ATCs come into play, and subsequently that turns into more patients with cell collections and more revenues that we see across quarters that we disclose data for. Beyond that, we have a catalyst-rich year. As I said, we have data for our in vivo program, CTX310 and 320. In the first half of this year, we'll have data, and these data will be data that show the dose escalation and the effect of doing the editing in the liver and the safety profile. We'll have additional updates for CTX112.
I already gave you some snippets of data for CTX112, but that's the additional data on oncology, including durability data and data in autoimmune disease that's probably early but indicative, and additional updates across our pipeline. Lastly, I'll say is we have a strong balance sheet to support all this. Plus the 10 preclinical programs we're working on, we disclose that we have $1.9 billion on our balance sheet, which is an enviable position to be in biotech in an environment where there's very high rates. The high rates actually are good for us because we generate over $100 million in interest income from our balance sheet, which supports our OpEx per year. Our operating expenses for the year are in the $500 million range, but if you look closely, our true burn per year is almost half that because we get a lot of interest income.
As I said, we have milestones that come in from our BD deals. We have other sources of grants. And so ultimately, here we are with a very strong balance sheet. We don't burn a lot of money. We have a very rich pipeline with a lot of updates, and we have a commercial drug that can give us a path towards profitability. So we feel very good. In fact, we're going to take advantage of all those factors to do very opportunistic business development, either to bring in capabilities from big pharma partners or to expand our portfolio with novel assets. So thank you, and I'm happy to take any questions. Go ahead.
Hi. Great presentation. Thank you. A couple of years ago, you were cutting out MHC II as well as MHC I. On your slide right now, you're just doing MHC I.
You got good results in CAR-T. I'm just curious that you find that wasn't all that additive, or are you still cutting out two MHC classes? And then for diabetes, we saw one of your competitors knock in CD47, one patient. I'm curious if you plan to do anything like that as well. So just more about allo and strategies. Thank you.
Yeah, thank you for that question. I think the question alludes to the edits we're making to make CAR-Ts stealth. We're taking allogeneic cells, so we want to make them stealth. And typically, when the host's T cells attack these allogeneic cells, there are different ways to do it. MHC Class I, MHC complex comes into play, and the MHC Class II.
And what we've seen is that the Beta-2M edit for the MHC Class I is very important and critical to make these cells survive longer and not be attacked by the host T cells. The MHC Class II is not as important in CAR-T context. So we're only doing MHC Class I. And we're doing MHC Class I even in the context of diabetes where we're editing these iPS cells. The question refers to data from one of the other companies that is knocking in CD47. What's very interesting about these data in diabetes is that differentiated islet cells are already somewhat hypoimmune. So it doesn't take that much more on the editing to make them very durably hypoimmune. While we don't have long-term data from this other party, I think it's encouraging to see that these cells survive a month.
I think we see the same with our clinical trials and with our cells is that a set of edits that we're making, which include knocking out the MHC Class I, we knock in PD-L1, which is a strategy that tumors use to evade the immune system. And those types of edits actually make it more stealth or hypoimmune. And so more to talk about that in the future.
Do you see a significant safety - sorry. Do you see a significant difference in the safety profile of an CAR-T versus an autologous CAR-T?
Absolutely. It's a very important question. I might have glossed over that point, and it's a really important point. One of the key reasons why CAR-T has not gone into community settings is because you see a lot more CRS and ICANS. These are basically toxicities associated with very rapid expansion of the auto CAR-Ts.
And you see a much lower rate of CRS. And almost in the data we disclosed at ASH, there were no ICANS cases with CAR-Ts. Now, you want to see some CRS because that's a marker of the cells working, but lower than autologous. So here we are with potency almost as good as CAR-Ts with a much better safety profile that can allow us to go into community settings. And I'm glad you asked the question because that's a really important differentiating factor for CTX112 and allogeneic CAR-Ts.
And no GVHD?
Absolutely no GVHD.
Thank you. Excellent presentation. You mentioned that you can make 100 doses from an allogeneic donor. What limits you to go to 1,000, 10,000? Is it T cell exhaustion? And how do you counter that?
Yeah, thank you. I mean, this is an important piece of the work we did.
I talked about the foundational years in the last three or four years. We brought the manufacturing in-house, and we said we really need to knock down the cost of goods to a level that makes it pharma-like. In fact, we brought down the cost of goods for our CAR-T to the $10,000 level, which now allows us to do clinical trials in India and China and, in fact, create a product that can be priced at $25,000 in India and still make a margin on it. How did we do that? We did that by process improvements, but more importantly, by making edits. And these edits, like Regnase-1 and TGF-β, they're like Energizer Bunny edits. These cells keep expanding, but they don't get exhausted, and they keep going. There is no theoretical limit.
We could easily push this to 200 patients' dose per batch or 300. But I think now the fact that we've already brought the cost down to $10,000, I don't know how much marginal benefit there is to bring it down to $5,000, for instance. So we're not taking a chance with how much we're expanding it in manufacturing, and we want to keep the cells more naive. And we measure that, by the way. These cells with the Regnase-1 edit is this bizarre combination where the cells remain more naive and central memory phenotype. At the same time, they're much more cytotoxic and potent. And so it's a very important edit. In fact, we hit upon it, and we developed this product. But Carl June is the founder of this CAR-T era, in a way.
They did a massive screen and came upon the same edit, Regnase-1, as the most powerful edit to make CAR-Ts better. Maybe one last question. If not, thank you very much. Great to see you all.