Good morning, everybody. Thanks for joining us. I'm Terrence Flynn, Morgan Stanley's U.S. biopharma analyst. Very pleased this morning to be hosting CRISPR Therapeutics. Today, from the company, we have Samarth Kulkarni, who's the company CEO and Chairman. Just before we get started, for important disclosures, please see the Morgan Stanley Research Disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative. Sam, thanks so much for joining us today. Really appreciate you taking the time to be here with us. I thought first to start, we'd start on strategy. Obviously, the company has CASGEVY approved with Vertex Pharmaceuticals, and you have three additional pipeline areas in cardiovascular, autoimmune, and oncology, which I know we'll talk about.
Maybe you could just talk about your priorities, what you're focused on for the remainder of this year heading into next year, and then we can dig into some of these topics.
Yeah. Thank you, Terrence, for having us. It's always great to be back at this conference. We're sitting in a very interesting place as a company. We're about more than 10 years into the journey of the company to take this powerful editing platform called CRISPR and turn it into transformative medicines. Ten years out, we have one approved drug in CASGEVY, which I'll talk more about. We're very encouraged by what we're seeing in how it can change the lives of patients with sickle cell disease and also what it means for us as a company. We're then sitting on a number of pipeline drugs where we're going to have readouts in the next 12 months. As you said, we have different verticals. We have our cardiovascular vertical, where we're going after important targets like ANGPTL3 and Lp(a), the gene editing in the liver.
This is in vivo gene editing. We also have an siRNA targeted towards factor XI. That kind of was a nice bolt-on to that cardiovascular franchise. That's sort of one pillar that we're quite focused on, and we're building capabilities. The second pillar is in autoimmune and oncology with the same asset, CTX112, which is CD19 allogeneic CAR T. I know there have been lots of ups and downs in the allogeneic field, but I think we truly have the best-in-class allogeneic CAR T that acts just like autologous CAR T. In autoimmune disease, I think this could have transformative potential, given what we're seeing with the data with autologous CAR T. If you can recapitulate that or replicate that with allogeneic CAR T, I think it's that much more scalable across a number of indications.
Obviously, there's a lot in the news about in vivo CAR T, and I'll talk about how that plays out. Again, in oncology, the opportunity remains. Even after having all these auto CAR Ts and biospecifics, there are still a number of patients with DLBCL that don't get access to any of the advanced medicines. We continue to pursue our oncology efforts with CTX112. Finally, as we look beyond that, we have other drugs that we're developing in our diabetes field. There's a publication showing you can actually transplant islet cells into a patient, and they can be hypoimmune for several months. I think we have something that could be very competitive in the diabetes space as well.
All in all, I think we're feeling very good about the pipeline, lots of readouts to come over the next 12 months, and from a balance sheet standpoint, really well positioned to prosecute all these assets.
Yeah. Okay. Great. That's a good segue to my next question. Obviously, a lot of different programs you walk through right now. You guys do have one of the stronger balance sheets in the SmeCap biotech space. Are there select opportunities to partner here? Obviously, you took that approach with Vertex, but as you noted, the company was maybe in a different place when you did that deal. How are you thinking about the opportunities set on partnering business development here on the forward for a lot of these different assets?
Yeah. I mean, we definitely have seen a lot more pharma interest in the last year than the two preceding years. I mean, I think in the 2018-2019 time frame, we saw a lot of pharma interest, and it kind of ebbed and flowed a little bit. What we've realized, we've come to the realization that if our cardiovascular assets work, when I say work, they're like best-in-class. They have really good data, and we have autoimmune potential. We may not be able to take all of these forward ourselves, not from a dollar standpoint, but from a resource and bandwidth standpoint. We have seen a lot of partnership interest, particularly in the cardiovascular area lately. You saw a deal this morning, for another important target, IL-6, in cardiovascular. That's, by the way, how we ended up with factor XI.
We were pursuing ANGPTL3 and Lp(a), and we were talking to a lot of the key opinion leaders about what are the targets that's really interesting. Factor XI kept coming up. Actually, by the way, the other one that came up a lot was IL-6, in ASCVD. Now, Lp(a) is a, you know, could be a huge blockbuster. The number of people with high Lp(a), and it's recognized that it's a, it's, you know, very high iatrogenic risk. There is a lot of interest there. There's emerging interest in ANGPTL3, especially with the recent deal that we saw with siRNA and ANGPTL3 as well. This could be a target that could be better than PCSK9, potentially even for LDL reduction. We'll see how the data all turns out when we put it all side by side, but not just triglycerides, but also LDL reduction. Autoimmune, there continues to be interest.
There is a little bit of churn in the space as pharma companies try to figure out what's better. Is the best solution for a patient an allogeneic CAR T with the condition that we have right now? Is it, or is it in vivo CAR T, but there's no conditioning, but you have to do repeat dosing? I think my belief is allogeneic CAR T is better. In fact, especially as we get to gentler conditioning, you see much better elimination of these germinal centers in the lymph nodes and the secondary, if you take secondary lymph node biopsies, etc. I think there's interest there as well. Long story, long-winded way of saying that we haven't made any decisions on what we would partner right now, but there continues to be a lot of discussion with pharma.
I think over the next six months, as the cards unfold on our data, I think we're better positioned to decide what we want to partner and what we don't want to partner.
Okay. Great. Do you see most of the inflection around your own data? Are there external data points too? I mean, Lp(a), for example. I know Novartis, everyone's kind of waiting for their study here. What other external data points do you think any of these companies are kind of looking to as they think about opportunities?
Yeah. I mean, I think that is, you know, the Horizon readout from Novartis is an extremely important readout for the space in general. I think that is one of the reasons we're positioning behind that. I think what we want to do is we want to position our gene editing assets ahead of some of these or right around some of these big readouts. The Horizon trial for Lp(a) will be the first trial where, in spite of, you know, we know that with all the population studies that low Lp(a) is better. Does a pharmacological intervention or a pharmacological reduction of Lp(a), does that lead to better outcomes or not? That's going to be the first readout. What are all the subsets we're going to see? Does it matter if they have high Lp(a) and high LDL versus low LDL?
I think I'm very interested in some of the subset analysis. I think it'll most likely read out positive, and the subset analysis would be very interesting. We're positioned ahead of that, and I think that can be a very valuable asset for us. As I said, there are trials around factor XI as well with Novartis and the recent acquisition by Novartis from Anthos. I think that trial readout is next year as well. Those will position us as we begin our phase two study here.
Okay. Great. Just one last one on the strategy front. You and I were talking about the policy environment right now. I'd just be curious to get your take on kind of FDA on genetic medicines broadly, where you think the pendulum stands in terms of your kind of FDA interactions. Are they leaning in on genetic medicines? Are they being more cautious? Is there anything about the framework that they're approaching these therapies through?
Yeah. My take is the leadership of the FDA, Doctor McAree in particular, are leaning in on genetic medicines. How much that's percolated into all the teams on the ground level is, you know, it takes some time. I think, for instance, you know, and I'm very close to Peter Much before as well, and we worked on quite a bit around CASGEVY. The previous regime, I would say the FDA would lean in more on clinical data, but had very high requirements around CMC. I think that's one of the reasons the entire cell and gene therapy space was a little bit at a competitive disadvantage, which is it costs a lot to develop these assets. Now that we have an siRNA asset, it's almost 3X cost to get to human POC with gene editing versus siRNA, even if it's in vivo.
That's just not a good competitive situation as you have all these competing modalities. I think this regime is very sensitive to that. I think there's active work ongoing to both change guidelines and regulations to make it such that we can do clinical trials in the U.S. easily, have some of the CMC requirements be phased where you don't have to do it all in the early going. That's a very good development. How the current regime is positioned relative to data and how they look at approvals, etc., it's an unknown. We don't know yet. There haven't been good case studies where they've had to make a decision one way or another. There will be a few coming up, actually, in cell and gene therapy. All in all, I'm pretty encouraged. There's actually a task force looking at everything quite closely on the regulation side.
The next few months will tell us where we may land.
Yeah. I know the Commissioner had done these listening tours. Did you get any feedback from any of those or hear anything from the biotech listening tours?
The listening tours were 90% listening and very little feedback. Off the record, there has been feedback. I think just around the complexity of what's been built on the regulations. I think with cell and gene therapy, we have these guidelines and regulations all overlapping, right? It's unclear what the primary thing to go by is, the guideline or the regulations. The second is, oftentimes with these CBER drugs too, the therapeutic area teams have come in at a certain point of the drug to help. Who's primary? Is CBER still primary, or is it the regional hematology, for instance, that actually makes the decision? I think some of those things are getting sorted out. I would say they're getting decluttered to a certain extent.
Okay. Great. Maybe we'll move over to CASGEVY, and you can kind of give us an update on the launch. I mean, I think you and Vertex posted sales in the second quarter, about $30 million. You reached your target of activating 75 sites, and I think about 250 patients have been referred into those sites. Maybe just talk us through some of the dynamics that brought you there, but also on the forward, how are you thinking about the outlook? I'd say the back half is pretty well understood. As we think about 2026, maybe you could just give us some of the puts and takes that we need to consider as we think about the run rate.
Yeah. I mean, for those who are not familiar with CASGEVY, CASGEVY is an autologous cell therapy product where we're taking the patient's cells. It takes a two or three-day collection cycle to take the cells and return it to our manufacturing facility, manufacture it, send it back. The patient has to undergo hemoablation. They basically get the cells, and then, you know, hopefully, it's a cure for life. I think the cell cycle or the way the commercialization model works is much more akin to medical devices than it is a traditional pharma launch. You're selling a mini procedure in a way, and the same thing that happened as in medical devices. Let's say you're getting a hip implant or something else. You kind of get, you know, you see the doctor, you get measured for it. Things are ordered. You schedule surgery, and then you get the surgery.
It used to be a lot longer cycle. It gets shorter and shorter over time as the centers get experience. I'm very encouraged by the fact that we have over 75 ATCs or authorized treatment centers globally, 10 countries or 10 jurisdictions where CASGEVY is approved, very strong payer reimbursement. If you look at the trajectory of patient initiations or referrals, that's already starting to inflect. The question is, how long before the cell collections start inflecting and how long before the revenues start inflecting? There's like a three-part funnel with CASGEVY revenues. One is how many patients are coming in. It's very clear to me that the demand equation is not the problem. There's a lot of demand from patients. The word of mouth and everything else that's happening in that population is pretty strong. There's like WeChat groups and WhatsApp groups and all that kind of stuff.
The question is, it's taking a little time in cell collections. The reason for that is a lot of patients are opting to do exchange transfusions where they get transfusions for a couple of cycles before they go in and schedule a collection. Some patients are choosing to do fertility preservation ahead of doing the cell collection. Then you get to the cell collections. After the cell collection, the manufacturing itself is relatively fast. Some patients may require more than one cell collection. Even if after it's manufactured, there's definitely patient preference of when they want to get dosed because they have to be in the hospital for three weeks or so. A lot of patients in the U.S. don't want to get dosed in the summer. Patients ex-U.S. may want to get dosed in the summer because there's nothing else they can do during that time.
There are definitely some interesting dynamics in terms of how this all goes. If you look at the secular growth in patient initiations, you're going to see compounded growth for a long time to come. That gives me a lot of confidence.
When you look at the referrals into the sites, is it pretty equally spread out, or do you have kind of like a concentration among the big sites that were involved in the trials, or is it pretty evenly spread across these?
There are definitely different sites with different productivity. I think if you look at the, yes, Skarta sales at the end of the day, the sites got to a point where they were doing 8 to 10 a month, right, once they got fully used to doing an autologous product. I think you might see the same increase in productivity over time with the sites. Not only do you have more sites, you're going to have more productive sites. That's just going to lead to greater and greater throughput of cell collections, and that's the key to dosing patients. There's really not much falloff once your patients get their cells collected to actual revenues, although it takes a little bit of time. I think those are all very, you know, those are leading indicators, and they're very positive looking ahead into the next couple of years.
Where is most of the falloff? Is it from the referral to the cell collection, or is it from cell collection to infusion? Like, meaning which step would a person decide like, "Okay, I'm going down this path," but then they, for whatever reason, they make a decision not to? Does it happen upstream, or does it happen more downstream if you look at it on a % basis?
Yeah. Once you get cells collected, I think very little falloff. I think from referral to collections, also, there's been no falloff because we have a pretty strict criteria for what we call initiation or referral. By the time they've come into, sort of gone through the referral and everything else, they generally have done prior authorization and are ready to get scheduled for collections. I would say that funnel is pretty tight in terms of the correlation between referrals and ultimately revenues.
Okay, just two more here before we go to the pipeline. The CASGEVY JV profitability, just remind us kind of how you guys have framed that, where you are in that ramp, and then what that means for your balance sheet, I guess.
Yeah. I mean, I think our goal as a company is to be breakeven if not profitable by 2028. That's something that we've said in the past too. I think we feel like, you know, that's a real possibility. In terms of near-term profitability or in CASGEVY, we're not commenting on that. Vertex may do some guidance early next year. In general, I think with the franchise, one of the things we'll also realize is that the costs do go down in some areas beyond a certain point, especially as we finish up clinical trials. We have clinical trials ongoing right now for a pediatric population. There's long-term follow-up, etc. Some of those costs will start coming down as well. The true cost of commercialization or what classical SG&A is not as high as people think it is. COGS obviously are higher than a typical pharma launch.
All in all, I think we're happy with how the profile is developing.
Okay. Do you anticipate an improvement in cost of goods over time as you get more throughput, or is COGS more of like steady state at this point?
Absolutely. I think Vertex is now leading the charge to continue to improve both cost of goods just from a cost perspective, but also just making it more seamless, faster manufacturing, faster throughput in a way. Lots of efforts ongoing. We continue to invest. We're all very bullish.
You mentioned in vivo approaches broadly in the beginning in terms of some of your pipeline opportunities. We do occasionally get questions on in vivo editing approaches for sickle cell beta fail. Maybe update us on your thoughts on how far away those are, if there's any prospects that those could ultimately reach the market.
Yeah. Eventually, I mean, I think the way we see the lifecycle on, where obviously Vertex Pharmaceuticals and us are very committed to the space, so we're all in. We're doing all approaches. The next step might be gentle conditioning. I think there are a lot of agents out there that have shown tremendous progress on gentle conditioning. These are ADCs where you can actually selectively ablate the long-term hematopoietic stem cells and not ablate the neutrophils, etc. You don't need a much longer hospital stay, possibly a three or four-day hospital stay to get these cells transplanted. None of the side effects of potential glucophan, etc. That might happen in the next four years. I think in vivo HSC editing could be eight years by the time it's on the market.
While I'm very encouraged by the fact that we can actually edit in vivo now in mice and even in NHPs, one of the things that's really important is you actually want to have a very selective profile. You want to edit only the hematopoietic stem cells and not other cells because, for a disease like sickle cell disease, the bar will be high in terms of off-tissue editing. I think that work, we're spending a lot of time thinking about that, and there are ways to do it. It'll just take some time to move to clinical trials.
Yeah. Okay. Great. Maybe we'll pivot to the pipeline now. You know, we'll go through cardiovascular first. We were talking a little bit about Lp(a) at the beginning. Maybe I guess the one update from your program is your data now is coming first half of 2026. Maybe just talk to us about, you know, what drove that. As you look at that Novartis data that's coming out, what are you going to be most focused on there beyond just obviously the kind of primary endpoint? Are there any other nuances as you think about, "Okay, should we pull the trigger to, you know, advance our program further because of X, Y, Z," which we saw from the data?
Yeah. I mean, I think it made sense for us to wait for the Horizon data. I think the key things we're, you know, first of all, is Lp(a), you know, pharmacological reduction of Lp(a), that results in better outcomes, right? One of the things that we're looking for from the data, that data set, would be how does the baseline level of Lp(a) correlate with improvement in outcomes? I don't know what the powering is going to be as you look at, you know, people with, let's say, greater than 200 nanomolar per liter Lp(a) versus greater than that or something like that, right? There are different cutoffs that we want to look at, and that's going to be important. The second is how do other markers correlate?
Like, for instance, you know, if we have high LDL and high Lp(a), is that far worse than high Lp(a) alone, for instance, and low LDL or normal LDL? I think those two are really important markers I'm looking for. Other than that, I think there'll be a lot to learn in terms of level of reduction needed. Everybody talks about reduction in terms of percentage terms, right? Even Lilly presented their data, and they basically were saying that, you know, they're getting 90+% reduction. It all depends on baseline. I think what is the target you want to get to? With the LDL world, it's very straightforward. Just get it down as much as possible. In the Lp(a) world, that paradigm has not been set. The recent ESC conference, there was a lot of discussion about what is the target, you know, on Lp(a) reduction.
There was always the school of thought that Europeans had a different view versus the U.S. American cardiologists. I think they arrived somewhere in between, as to what the, you know, ideal target is for Lp(a) reduction. Those are all things we'll be watching for ahead of, you know, presenting our own data on phase one.
Yeah. What about, this is obviously a secondary prevention trial. I know the companies have talked about high-level, you know, primary prevention studies as well. How do you think about translatability from here to primary? Because when we talk to KOLs, they'll tell you that, you know, primary, it's the bigger commercial opportunity, and that's really where you need to get to. How much read-through is there in your view from this study to primary?
Tremendous read-through. I mean, ultimately, you know, as humans, I think we are going to be in a different world where we're going to see risk factors and change of risk factors early on in life. We had this case of a 22-year-old kid who came, whose Lp(a) levels were like 600 nanomolar per liter. That kid's father, uncles all had a heart attack in their 40s. You know this kid's going to have an MI event or some MACE event early in life. It's genetic. You know that the levels are high. Why would you not reduce the levels of Lp(a)? I think that's one example. By the way, the administration is super interested in this. I actually did a five-site change effect with Dr. Oz, and he was highly interested in Lp(a).
He's like, as you think about national priority vouchers and things like that, these are national priorities in their mind, targets like Lp(a), things that are a huge burden to society in terms of not just cost of the healthcare system, but productivity, etc. If you take something like Alzheimer's, if you're born with two copies of APOE4, if I knew I had two copies of APOE4, I'd be doing everything I can to say, how do I prevent this risk of Alzheimer's? You know you're going to get it. It doesn't matter when. I think we're going to get to a world where people are starting to do these longevity genetic tests now. You can do these 15 tests and tell you what your longevity score is, for instance. Lp(a), Alzheimer's, etc., is on there. You're going to want to change these early on.
The world is going to move towards primary prevention. I don't know what the regulatory framework required would be to get to that point. We just want to make sure. That's why I say the world's going to change. We want to be, this is going to be a secular growth space in gene editing. We want to be around to see all those major inflections. I think Lp(a) will be one of the first test cases for primary prevention.
Is the National Priority Review Voucher your speculation, or do you think Dr. Oz is, there's already companies that are talking to him about that for Lp(a)?
I don't know. Lp(a) is one example, but I know there are a lot of companies talking to the regulators to see what the National Priority Review Voucher really means because there are elements of national competitiveness in there, what the target means for the American population. I think it's not 100% clear to define what they meant. I think these types of targets do make sense from what we're hearing, which is reducing overall cost of healthcare, right, is a national priority. American competitiveness, getting ahead on certain medicines ahead of other countries, and obviously, all the unmet need for patients, etc. Not to say that Lp(a), we haven't heard anything directly on Lp(a), but those kinds of things seem to fit with what we've heard.
Okay. Great. Maybe before we get to ANGPTL3, I wanted to go to your factor XI, because I think this is a newer program for you. Maybe just from a strategic side, first talk to us about a decision to go into RNAi or siRNA here, with this, given your platform in kind of CRISPR and how this aligns with that platform.
Yeah, absolutely. As I said, the origins of looking at factor XI was we were quite enthusiastic about Lp(a) and ANGPTL3. We were having a lot of discussions with different key opinion leaders. We always have, you know, when we have dinners with them, we'd ask them, "What else are you interested in? What else is interesting in the target space?" Factor XI came up a lot. People said, "Look, you know, there hasn't been much innovation in the clotting, anti-clotting space for a long time." It's similar to what happened to LDL. You had this dead period of like a decade where not much innovation happened after these big, you know, CETP trials, etc. failed. Everybody got out of it. There was this, now there's a boom in terms of, you know, lipid management happening right now from an innovation standpoint.
The same thing had happened in the anti-clotting space. The other one that came up was hypertension. There's all of a sudden new renewed excitement in the hypertension space, after sort of a decade where there wasn't much happening. We looked at factor XI and said, you know, this is not something you want to gene edit, right? You don't want to permanently reduce factor XI. The holy grail of, you know, preventing clots and everything else is you want to prevent clotting, but you want to not have bleeds because those, you know, you could be on, you know, factor Xa after surgery, and then you, like, you know, hit something in the shower, you have a nosebleed or something like that, and you could literally end up in, you know, you could die or you could end up in the hospital because it's uncontrolled bleeding, right?
These bleeding events happen. Some of these are external bleeding events. Others are internal bleeding events that could cause other complications. Factor XI is very interesting. You get APTT levels above 2 in a sustained manner with this siRNA. We found this siRNA. This is a former Arrowhead CSO who founded this company with sort of the next-generation backbone modifications in siRNA that lead to durable six-month effect. A lot of the Arrowhead drugs right now are on a three-month dosing schedule. I'm sure they're working on longer term, but, you know, the siRNAs we're moving towards potentially even once a year. It's that durable with all the modifications that are being made. What we have, we got with this deal from Sirius is a phase II ready asset. The phase Is, two phase Is have been completed.
The phase II is starting now in a TKA study, a total knee arthroscopy, where you're comparing with the low molecular weight heparin to see if you can prevent clotting post-surgery with one injection that's done pre-surgery, and then you don't have to worry about it for six months. What are the bleeding rates associated with that, right? This study is a key sort of leveler. It'll tell you where you stand versus Milvecs, GNS, IndexGen, or other siRNA or ASOs that are in the space, or antibodies, which we have from both Novartis and Regeneron now. All in all, I expect excitement to only keep going up with this target.
Yep. How do you think about that long half-life? Because on one hand, it's good from an adherence perspective, but on the other hand, if there is some issue with bleeding, how do you think about that as the trade-off if you do need to, like for example, like my father's on, you know, warfarin, for example, had a surgery he had to go on, he had to come off of the warfarin. How do you think about that trade-off?
That's the beauty of siRNA. You can just supplement with factor or other ways too. That just reverses it, right? With antibodies, it's actually very hard to reverse it. If you have, with the Anthos and the Regeneron antibodies, you know, this month, once a month dosing. If you have a complication, I don't know what their strategy is going to be, but it's actually hard to reverse it because even if you add factor, it still binds to it. With siRNA, you don't have that complication. It is actually a beautiful setup, long-acting but has reversibility. I think once in six months is probably durable enough. I don't think you need to get to a once a year in this setting. In fact, you probably don't want to because you want to see your physician every six months.
Yep. What, and then I guess the other question that we get a lot more so for, you know, the oral factor XI is, you know, read-through from the various programs to each other. As you think about, I think Bayer has got into seeing some data for their program, S-Induction, later this year in secondary stroke prevention. Again, similar to the Novartis question I asked you earlier on Lp(a), like what are you looking for in that data set to, you know, maybe give you confidence in your program or inform, you know, trial design? TKR is always, like you said, kind of proof of concept, very quick readout, easy, but you typically do it versus, you know, Lovenox or something. For these bigger, you know, AFib, ACS, SSP, I think there's still question about like what people want to see from these bigger studies. What are you focused on?
I think two factors of S-Induction, which is one is there's a lot of controversy about, you know, they had their own proprietary assay for S-Induction. You know, how much target engagement were they getting, right? I think when you're going after, with these small molecules, you're going after the activated factor XI, so not factor XI itself. There's a notion that by the time the small molecule acts, you will already have an activated factor XI that started a cascade through factor Xa. With the siRNA, the advantage of siRNA is you're actually preventing factor XI from even being formed, so you don't have factor XIa. You may get a very different target engagement with factor XI siRNA versus the small molecules. I think the current thinking in the field is that Milvection may be a stronger binder than S-Induction. Milvection readout is going to be very important.
That said, I think one of the things that these big pharmas were trying to do is directly go up against the factor Xa inhibitors, which are amazing drugs, right? I mean, our goal is not necessarily to beat them and show that factor XI is better than factor Xa drugs in all these indications. There's a significant population of people that are ineligible for DOACs. They just can't take DOACs for various reasons. In all those patient populations, factor XI is a beautiful solution. I think there's different reasons for why they're ineligible, but that's one setting. The second setting is, I think in cancer-associated VTEs and sort of venous thromboembolism indications, definitely this is the only solution that actually makes sense, versus DOACs. There are patients that have multiple complications like warfarin, like you mentioned with your father.
There are actually people who are, who genetically can't take warfarin, for instance. It's a very complicated space. Patient by patient, depending on what other drugs they're on, this could be a very low-burden drug they can add on to everything else they're taking.
Great. Maybe just, I think we got one minute left. Just on any last catalyst you want to highlight quickly of the other programs that we should pay attention to before year end?
Yeah. I mean, I think CTX112 and allogeneic CAR T. This is the only allogeneic CAR T that has shown expansion levels to show up to 50,000 copies per microgram. That's sort of the traditional currency which you measure cell expansion. Autologous CAR Ts, the ESCARDA and VAND and others, they get to that 50,000 level copies per microgram. Some slightly less, some slightly more. All the other allogeneic cell therapies, whether it's NK cells or T cells, have not, have gone to about 5,000 copies per microgram. CTX112, with the edits that we've made, is the only one that gets to autologous-like expansion without the same side effects of CRS or ICANS. It's actually a really great asset. We're developing in autoimmune diseases. All the data from George Schett indicate that you could get 99% ablation of B cells, but it's the last mile that counts.
You want the last mile ablation in the germinal centers that are formed by these B cells. If you don't do that, the disease is going to come back. I think that we have confidence that our asset could get to that 100% reduction, which would be very powerful in autoimmune disease.
Great. Thank you so much, Sam. Really a pleasure and best of luck.
All right. Thank you very much.
Thank you.