All right, welcome everyone to the 42nd annual J.P. Morgan Healthcare Conference. My name is Anupam Rama. I'm one of the senior biotech analysts here at J.P. Morgan. I'm joined by my squad, Lyra Hall, Priyanka Grover, and Malcolm Kuno. Our next presenting company is Solid Biosciences, and presenting on behalf of the company, we have CEO Bo Cumbo. Bo?
Yeah, thank you very much, Anupam, and thank you, J.P. Morgan, for the invitation. I really do appreciate it. I'm gonna be making a number of forward-looking statements today. Please take time to look the forward-looking statements on the deck. We also filed on the 8-K. Obviously, results may vary, so please take a moment to read this. So today, we're gonna be talking about Solid and really the transformative changes that we've made over the last year. It really leading platform, partners, pipeline management, and a very strong balance sheet. Our management team, we just recently added our new Chief Medical Officer, Dr. Gabriel Brooks, to the team. He really rounds out the entire team now. He's a cardiologist by training, most recently came from Pfizer Gene Therapy programs, and joined the team in October.
So now we have a full team of very experienced professionals in the biotech world. Our pipeline was put together very thoughtfully and strategically, where each and every program builds upon each other. High large populations, high unmet need, and significant mortality that we're gonna try to correct. We have collaborators around the world now, so it's no longer Solid on its own. It's UCLA, Nationwide Children's Hospital, University of Missouri, Duke, Wisconsin, Iowa. All of these universities and thought leaders are supporting us and helping us with our, with our program. So we really do have depth and breadth outside of Solid to transform lives.
We have platform capabilities that we believe are unmatched, not only from a cardiac and skeletal capsid library, but also our manufacturing capabilities, and we'll talk a little bit about that, focusing on not only yields, but also the full-to-empty ratios that really do matter when you're thinking about delivering gene therapies. We'll spend a lot of time today on our first IND that's now open for 003, our next generation Duchenne muscular dystrophy drug. This is our pipeline that we'll talk about. I'll obviously spend most of the time today talking about Duchenne at the top, but then I'm gonna skip down and focus in on CPVT, the RYR2 and CASQ2 mediated CPVT, which we will have an IND roughly 12-15 months from today. We're very excited about that program.
Then I'll spend time on BAG3, dilated cardiomyopathy, and I'll also talk about our platform, technologies, capsids, and manufacturing. So we'll dive into our lead program. Everyone knows what Duchenne is. It's top of mind of a lot of different companies, and rightfully so, because it's a high unmet need, and where we need to make changes, dramatic changes to these kids' lives. We have what we believe is truly a next generation program, and it really starts with three aspects of the program: the transgene, the capsid, and the manufacturing process. From the transgene, we have a very unique transgene, one that has domains that other companies do not have. It starts with the repeat domains of R16 and R17.
When you have R16 and R17, you're able to recruit for a couple different proteins, alpha-syntrophin and others, and then that creates a binding spot for nNOS. In preclinical models, when you have nNOS, you have vasodilation, ultimately increased blood flow, and that creates healthier, heavier muscles. Now, we think that this is gonna play a role in the, in the longevity of the children. When we look at mouse models, when we look at fast twitch muscle recovery in the EDL, you see a re, it come right back to normal, and this, we believe, is due to the vasodilation, ultimately to the blood flow. We also have removed a couple of the hinges to create a very flexible, microdystrophin in nature.
So as the muscles are contracting, the microdystrophin that we've created has enough flexibility to move with the muscle. Therefore, you try not to have breakdown of muscle, and you do see some ring fibers in preclinical models when you have different hinges, and so we've removed those hinges to make sure that we have a very flexible construct. And it really, what's the most important is not just the transgene, but it's can we get that transgene into the nucleus, into the muscle? And it really starts with the capsid. And we have a very unique capsid, never been used before, first in human studies, but preclinically, we have a lot of data that shows that this capsid is significantly different than the parental capsid, AAV9, and I'll talk a little bit about that.
It all starts with delivery. How fast and how robust can you get this transgene in? At day 4, at day 4, we're getting 50%-75% microdystrophin expression into the heart, into the diaphragm, and the quadriceps. It's really unheard of. You don't see transduction and expression within a week, but this capsid has increased binding capacity. We've actually put this capsid in the face of IVIG at 0.5, 1, and 2 grams per kg. Looking at can we bind and transduce and express in the face of antibodies? And the answer is yes, and we have significant expression even in the face of IVIG. Now, one of the things we wanted to understand is, now that we have expression at day 4, what does expression look like at peak, at day 29 or day 30?
We are going to take biopsies in our children at day 90. So does that expression hold to day 90? And the answer is yes. As we are looking at different muscle groups, there's 6 on the screen today, you can see once again, day 4 expression, which is unheard of, a very robust peaks at day 29, and it holds all the way to day 90. That gives us a lot of confidence as we're taking our biopsies at day 90 in our children, that we're going to get robust expression. Now, one thing that we didn't really notice in the original study is a dose curve, because at 3E13, 1E14, and 3E14, we're all getting to 100% expression. So where is this dose curve? So we went down.
We went all the way down to 2e12. This capsid is acting different than other capsids. You can see very robust expression in all the muscles. However, we found the dose curve. You also see that nNOS expression, which we believe is ultimately very important, especially when blood flow, and you're thinking of endurance in these children. Blood in nNOS is 100% at 3e13. Keep in mind, we are dosing our children at 1e14. Even though we're getting significant amount of expression at 3e13, we're going higher. One, we have the safety window to do so, and I'll talk about that in a second. But two, we really want to put our best foot forward and really try to help these children and deliver as much microdystrophin expression as possible.
Now, how does it compare to Solid's original program? We're using AAV9. Well, in biodistribution, expression, or disease progression, looking at CK, it's significantly different than Solid's original program using AAV9. Of note, in the non-human primate study, when we did our GLP tox, in the diaphragm, we were getting five to ten times greater biodistribution in the diaphragm, which is a very hard muscle to drug, five to ten times greater in the diaphragm. Think about all the children that have pulmonary function decline. We don't know yet if that's gonna make a difference, but our hypothesis is the increased biodistribution will increase expression in the diaphragm. We already know it's getting to the heart very well, and this will help a lot of the non-ambulatory children. Now, we looked at grip strength.
I already mentioned we looked at fast twitch muscle, and the EDL can recover. We also looked at grip strength, treadmill exhaustion, and mass spec. Now, take a look at the doses. This is 3e13. Once again, at the high dose, we're dosing in our human subjects 1e14, and I showed you before, we're already at 100% before. Now, we're looking at mass spec percent normal dystrophin. We're getting to 100% at very low doses. And we already know that we can change grip strength, change treadmill exhaustion, change recovery of the EDL. So we're very excited about that. It looked the same in the non-human primates. When you look at biodistribution, the skeletal muscle, cardiac muscle, and it was also liver detargeting, very similar to what we saw in the mouse.
Now, I'll be very clear on GLP tox, because Solid of old had issues when they were dosing their children. This, we did not receive one single question from the FDA in our IND on safety, not one. It was well tolerated in both groups throughout the study. There were no more mortality events. There were no unscheduled takedowns. We didn't have to intervene at all in the study. There were no pathology findings, no organ weight changes. The liver enzymes were comparable to vehicle at the target clinical dose, and we dosed up to 3 × 10^14. So we feel very comfortable in the study. Now, what is our primary objective? Well, let me start by saying that we are actually trying to get IRB approvals right now.
We should have that approval by the end of this month, maybe early in February. We will be dosing patients mid-quarter to the end of quarter. That'll start our first patient dosing. We have to wait 30 days in between patients. Our primary objective is obviously safety. Our second is gonna be looking at efficacy and focus on microdystrophin expression and some other clinical endpoints. Our first trial, our first cohort, is going to be age 4, 4-5 years of age, low weight, and we're gonna be dosing, as I mentioned, minimum of 3, most likely 4 patients in this cohort over the course of the next couple of months. Now, we have here cohort two.
However, if cohort one looks really well, you know, microdystrophin expression as well as safety, we will speak with the regulators and think about amending to go into a double-blind, placebo-controlled trial as fast as possible. So as I mentioned before, primary endpoint, looking at safety and then looking at expression. Timing. Well, if everything goes well, we'll be dosing patients over the next couple of months. We should finish dosing right around summer. So over the summer months, we should be able to announce that safety, and that we're through our cohort one.
If that timing holds up, that means late Q3, early Q4, we will have all the microdystrophin expression for everyone to see, as well as patient videos, baseline, and day 90, and some clinical endpoints, albeit, you know, 90 days after you start the dose. Now, we are going. As I mentioned before, we're gonna give all that readout in Duchenne, late Q3, early Q4, if our timing holds up. At that point, we're 5 to 6 months from our next IND, which is another fatal young disease. We talked about Duchenne. Everyone's focused on Duchenne because it's such a horrific disease in children. If you don't know this disease, you should get to know it. Catecholaminergic polymorphic ventricular tachycardia. You get diagnosed age 7 to 12, sometimes as low as 5. Mortality is 40% in 10 years.
Your quality of life is severely impacted, and you could have a spontaneous arrhythmia at any time and die. There is no drug on the market to treat the underlying cause of this disease, and we believe that we have a solution. What we're trying to do is soak up the excess calcium that you see in the sarcoplasmic reticulum, that you can see here, that causes these arrhythmias. Once you overexpress calsequestrin, the normal rhythm should come back to these children. When we look at animal models, this is for RYR2. By the way, 20,000 patients in the United States have this specific mutation here. RYR2 in three different doses at 12 weeks, all arrhythmias are eliminated.
For CASQ2, which is much smaller population, roughly 1-2,000 patients in the United States, once again, at all the doses, at all the time points, all the arrhythmias have been eliminated. We're very, very excited about this program, and we will be in the clinic roughly about 15 months from today, and about 5-6 months after we read out our Duchenne program. So a lot of inflection points for investors to get behind. Now, what we're gonna do, this will be a global trial. We'll start in the United States. We'll have multiple sites. We already have orphan drug designation. We will be seeking rare pediatric disease designation. We're very hopeful to get it. We don't have it yet, but this is a childhood disease, and we believe that we can get it.
We're also gonna plan to apply for the Innovative Licensing and Access Pathway, so we can start talking to payers about reimbursement for this deadly disease. As I mentioned, it's 20-22,000 patients in the United States. It's larger population ex-US. Now, as we're going into this IND for CPVT in Q1, we'll be dosing patients in the first half of the year. Two to three quarters later, another IND for another fatal dilated cardiomyopathy called BAG3. Codes for this BCL2-associated athanogene 3 protein. And once you have these reduction in proteins, you end up with cardiomyopathy, you ultimately end up with heart failure. We're taking Rh74 with this very specific promoter, and we're applying to this. We're working with Dr. Eric Adler out of UC San Diego. We're very happy to have his lab supporting us on this.
This is a big, you know, big population in the United States, greater than 20,000 patients. Now, our drug specifically goes right where you need it. You need it to go to the heart, you don't want it to go other muscles. And you can see that we're not going to the gastroc, we're not going to the liver, we're going straight to the heart. We use a very specific cardiac promoter with Rh74. We get to 80% of the cardiomyocytes, and it's positive for BAG3, so we're very excited, and we actually don't believe that we need 80%, so we can pull this dose hopefully back down to lower levels. When you look at the mouse model on the left, we wait until we see a phenotype, and that's when we dose.
And on the right-hand side, we can eliminate, you know, cardiac dysfunction right back to wild-type vehicle. So it rescues cardiac function at week 24 at a very low dose, 2E13. Now, our platform technologies, I believe we have some of the best, not only manufacturing, but capsids. This is our next capsid. It's called SLB-134. Kevin likes to call it Snow Leopard. It's because it sneaks up on you. It goes right to the heart, but it bypasses the liver, and it's a thousandfold liver detargeting compared to what the parental capsid was. So this is a mouse. We want to see if it's gonna be the same in the monkey. We're going in non-human primates this quarter.
If the non-human primate holds up to what we see in the mouse, this can change the way we think about gene therapy and all the events that you could have by overwhelming the liver. It's a thousandfold liver detargeting. However, it still is cardiac tropic, as you can see, the parental capsid. Now, our manufacturing platform, we believe, is one of the best, not only from yields, which we've increased yields about 20 times. We've also moved away from triple plasmids to dual plasmids. And you can see when we moved to dual plasmid, you can see increase in yields. It obviously lowers COGS, and we're significantly proud of our achievements there. But we're also proud of what we're seeing in our full-to-empty ratio.
Compared to other CMOs and other companies that we know about, we have the highest full to empty ratio, with the exception of one company that I know of in the space. Now, when we talk fulls, we talk true fulls. We're not talking partials. Partials, we lump into empties. Some companies lump partials in with fulls. If we did that, we would be saying 97%-98%. However, if you want to talk from a purist point of view, true fulls, batch to batch, somewhere between 77%-81% true fulls. It really does matter. When you think about how you're dosing children, you're dosing in vg to kg, and so you're gonna have to increase your viral capacity for that child, and also increase your COGS, to get to that true dose.
So when you start at a very high full-to-empty ratio like we have, 1, hopefully, it should help on safety, 2, on COGS. So a lot of achievements that we have, a lot of milestones that we're gonna have this year. 1, we're gonna be dosing our first patient, hopefully this quarter. We're going to read out safety over the summer. That will put us in timeframe of late this year to also read out our, our microdystrophin expression, late Q3, early Q4. We're also gonna be filing CTAs. If we have great data, why not go global, and why not go into a double-blind, placebo-controlled trial? There's gonna be a lot of patients, not only in the U.S., but especially ex-U.S., that are gonna need a drug. So we're gonna file our CTAs.
We're gonna wait and see what we have, and then we'll quickly move into another trial. At that time, we're only five or six months away from CPVT IND. I told you before, highly fatal, very young, and life-altering, and we believe that we have a way that we can solve this. Couple quarters after that, BAG3 IND. Our capsids are gonna go through multiple routes of non-human primates as well as pigs, and our platform technology from manufacturing is gonna continue to advance. So with that, I'll say thank you very much. I appreciate your time, and once again, thank you, Anupam.
Thanks, folks. So I'll just remind folks, like I have all week, that there are three ways to ask a question, right? There's raise your hand, the old school method, I'll call on you. If you have access to the question portal, you can submit it, and it'll show up on this iPad, and I'm happy to ask on your behalf. I guess there's an intermediate strategy where you can email me, and I'll do the same thing. So. But I will start, if that's cool?
Oh, it's cool.
So, on SGT-003, you talked about, you know, enrolling that first set of patients. Have you identified these patients? Have you identified, you know, mapped them to a site?
Well, yes and no. I mean, we need to get IRB approvals first. Obviously, so our sites, Nationwide Children's Hospital and UCLA, Dr. Perry Shieh, Kevin Flanigan, very, you know, well-known experts in the field. We have a number of families that are trying to get into the clinical trial. They've been calling. They're even changing their children's steroid regimen because you have to be on a 3-month stable steroid regimen prior to getting into study. So they're already calling and asking if they can get in a trial. Of course, as I mentioned before, it's only 4 patients, so there's not a lot of room. We really can't screen until we get IRB approval.
We're gonna get IRB approval, hopefully this month, maybe early next month, but then we'll have multiple patients to screen at that time, and then we can say, you know, we've enrolled or not.
Can you comment a little bit about the age of the patients that are gonna be enrolling in the first couple?
Yeah, it's 4-5, and this cohort is 4-5 years of age. And, we're not gonna have trouble enrolling, if that's the question.
Well, how are you thinking about scenarios around the Elevidys label expansion and how that will either impact your near-term or long-term development of SGT-003, whether it's from a clinical or enrollment perspective?
Yeah, that's a great question. You know-
I know a little something about that compound.
Yes, and look, I think everybody at Sarepta, Solid, you know, Pfizer, we're all shooting for the same thing, to try to help these kids. So, you know, let's start there. You know, I don't think... I think what we're doing is we're gonna go into the United States, we're gonna go into ex-US. It's gonna be a global trial. There's a lot of demand. All the families are always looking for things that are gonna provide a true clinical benefit for their child. So, you know, whatever happens with Elevidys here in the United States really doesn't impact our plans. We know that we have significant demand in the States already for our clinical trial.
We're gonna be filing CTAs, as I mentioned, throughout this year in multiple countries. Obviously, Elevidys will not be in outside of the United States, immediately, you know, if at all. We just don't know. And, there'll be plenty of patients, so it really doesn't affect us. We've got our game plan. Double-blind, placebo-controlled trial will be coming. We'll pick a different endpoint, we'll focus in, and, we'll be ready.
Questions from the audience?
Thank you for the- Hello? Yeah. Thank you for the nice talk. So you mentioned that you use the dual plasmid system for the production, so 90-90% full ratio is really high. I mean, that's really good. So can you disclose your productivity? We know that dystrophin is pretty big, so usually we have very low yield. So do you have data for, like-
the productivity? So-
Yeah. Yeah, so that. It's a great question. So let's start with the manufacturing process. We've made about 5 changes over the course of the last 12-15 months that have really dramatically changed not only the yield, but the purity of the drug. Now, for Duchenne, what you don't wanna do is you don't wanna change your manufacturing process in the middle of trials, especially when you're trying to go straight into a double-blind placebo-controlled trial after, you know, this cohort. So you wanna hold your process tight. We already had a pretty robust process, especially from a full-to-empty ratio. We were using triple plasmids prior, and so the Duchenne program was using triple plasmids. We were getting significant yields with and a high full-to-empty. We won't be altering that process.
Now, everything I mentioned before about changes moving to dual plasmid, that will be implemented in our cardiac programs right from the start, and as well as the high full-t o-empty ratio. As Duchenne will wait to post-marketing to implement the changes in manufacturing. If you're talking about yields, you know, obviously, you have to scale up. So everything that we're doing in our PD labs, if it's scalable to 1,000-liter, you're talking high E17s, low E18s at that level. So it's significant. From a COGS standpoint, I would have to think it's one of the lowest COGS out there, some of the lowest COGSs out there.
Oh, you mean that you are still using triple plasmid transfection for the DMD?
For DMD-
Okay
... we're using triple transfection-
Okay
... triple plasmids until post-marketing, and then we'll do a bridging study.
Understand. Yeah.
Yeah.
So how-
For cardiac, we'll use dual plasmid.
How about the dual plasmid system? What's your recovery?
Sixty-five percent.
65%. Okay, great. Thank you.
Additional questions? Bo, there was a question.
Oh, sure.
Malcolm.
So when we look at day 4 expression... Oh.
Okay.
Day four, yeah.
When we look at day 4 expression, zero zero three, better across the board. But I notice a little bit of a lag when it comes to cardiac. Is that because cardiac expression takes longer, or is zero zero-
... three just, you know, more targeted towards the large muscle groups?
Yeah, it's a great question. So let's- I'll come back to that. What we saw just overall. Well, it's really SLB-101, so it's the capsid, right? This really is, but it is part of 003, which is the program. So the capsid is very similar in the heart compared to the parental capsid, which was AAV9. So the heart was really relatively the same. It was the other muscles that were dramatically different. The gastroc, the quad, was roughly 3-5 times greater biodistribution with this new capsid compared to the parental capsid, which was AAV9. The diaphragm was dramatically different. It was 5-10 times greater biodistribution than non-human primate than the AAV9. The heart was roughly the same, you know. And AAV9 gets to the heart really, really well. So it's fine.
You know, we're not looking. And we were above 100%. If you look at some of the data, you know, once at day 29 and at day 90, when we thought, you know, we were gonna take the biopsies, you're at 100% in the heart. Now, we did some really cool, unique studies, because when we were creating the capsid, we were thinking about a world where you're probably gonna have to dose, bind, transduce, and express in the face of antibodies. And so we wanted to know, can we create a capsid that really, you know, transduced in the face of antibodies? So we actually did a really cool study, and we did it with some other capsids, competitor capsids as well.
And by far, this was a very unique outcome. In the face of IVIG at 0.5, 1, and 2 grams per kg, this capsid, using luciferase, was expressing at a greater percentage than other competing capsids without any IVIG at all. We also noticed that we were seeing expression at day 2, and at day 4, it was significant. We then did another study. That was 5E13. We then did another study at 1.95E13, pretty low dose, and looked at 30 days, and it was significant across the board, very different than other capsids looking at expression. So this capsid is acting different, and we're very hopeful to see if it plays out that way in patients.
So there's just more incremental ability to make a difference in the larger groups is-
Yeah, it seems like it.
The heart was relatively the same.
Yeah.
It was the quad, the gastroc, the other muscles, the diaphragm especially, really changed. It could be because we added a peptide for a specific integrin receptor. So, you know, it could be that just the skeletal muscle and the diaphragm have more of these integrin receptors than the heart.
Awesome. Thank you.
We've got a question in the portal: How do you think about the potential to partner aspects of your platform or other cardiac gene therapy programs to accelerate timelines to clinic for your pipeline?
So when you think about partnering, you can have, you can partner programs, and you can partner platform, right? So let's talk about platform first. So we're highly focused on partnering the platform out, but we're not taking the Voyager approach. I'm taking the approach where I wanna get this to the masses. I wanna get our capsids to as many institutions and as many small companies as I can. 'Cause I believe that once we get these capsids, and we have multiple capsids. I showed you the snow leopard capsid, the 134, that bypasses the liver altogether, you know, well, 1,000-fold change. And then we have SLB-101 that I'm talking about in Duchenne. We have another capsid that we're working on. I wanna get these to the masses.
I wanna get to these all small companies that really can't afford the $20 million upfront, but get it to them. 'Cause they'll work through their programs, and they'll ultimately end up in the big pharmas or the big bios. I wanna get into every institution I can possibly get in. I want the dual plasmid that you're talking about, get into every single company as well. So we have set a strategy, these two right here, have set a strategy, of doing that. And so it is a different approach. It's not the same approach that other companies take. And but I believe if you're playing the long, long game, this is, this is how to get it to everybody. Now, as far as programs, you know, data is king, and I... value goes up.
Can I take these programs across the globe? I could try, and I've done it in the past, but odds are good I will need a partner. And it's all about when you get that partner, what data you have, and what can you get for it. So right now, we were very, very fortunate for any capital overhang, it's been eliminated. We've raised $109 million that we're very, and very fortunate with great, great investors. So we can now focus on our trials without needing a partner, and then we can get a partner down the road.
Questions from the audience? Is it fair to say that you've kind of prioritized CPVT now over, say, Friedreich's ataxia?
And is there a sort—because Friedreich's ataxia was kind of like the second- lead after DMD.
Can you talk about-
Yeah, I'll talk.
... the strategic switch there?
Yeah, well, and we're still working on FA, but I, but I'll be very open about it. So we made a very deliberate choice early on. We had great data on the for FA; we're talking FA now. We had great data in the heart, and we felt like we could go into an IND in the heart immediately. However, when you talk to the patients and the families, they will tell you, "Look, it's wonderful if you can cure the heart, but look, if you lose the ability to walk, see, speak, hear, quality of life really does matter." So we were very focused on the CNS neuro side of the business. We did a large animal study. It was 40 non-human primates. It was 6 months. We looked at multiple doses.
We've got great expression in the DRGs, all through the spinal column, and we did not, and we got great expression in the heart, and we did not cause tox event from overexpression. But then we looked at the cerebellum, and we looked at Purkinje cells from those non-human primates, and it was relatively low expression, sub 5%. And so we don't know what that means. Is sub 5% enough, or should we try to create a better drug? So we're tinkering around with it, looking at promoters, looking at capsids, looking at route of administration, because if you're going to try to change the course of the disease, really make sure that you have the best program possible. And we, and we did good science, and it came down to we just didn't know if we had enough expression in the cerebellum.
So here comes CPVT, another fatal disorder, children, 40% mortality in 10 years, not a single drug out there. I don't know of another gene therapy company in the space. Twenty thousand patients in the United States, more ex-US, and we had unbelievable data. Let's go prioritize that. Let's put money where there's green space, and let's see what we can do. That's why.
Any final questions?
Okay, thanks, Bo.
Thank you.
Oh, oh, yeah, whoops, sorry.
I'm sorry.
Go ahead.
Yeah, you mentioned that FDA does not have, like, any, like, rejection, like, for the new AAV capsid. So how do you think about it? I mean, that's... Usually, I think the people may think that a new capsid may have a lot of risk, you know, take a long time to verify it's safe for, like, in viral delivery, so.
Yeah, no, it's a great question. Look, I think this is why we're doing the studies that we're doing. We have an extremely robust preclinical data set Mm-hmm.
And all of that made its way into the IND. We did not receive any safety questions from the FDA in our IND at all. Now, obviously, we've got to do the, do the human studies and see, you know, if that translates to human. That's why we're taking the approach we're on. The FDA requested us to go into a double-blind placebo control trial right from the beginning. However, I wanted to make sure that we had a safe product, to your point, so that's why we're doing four patients. We're gonna see what we get. If we have expression, I'm gonna pivot, and we'll be aggressive.
Okay. So these capsids are selected from animal model, from mouse-
No, no
... or NHPs?
It started with AAV9. So we have a whole different platform. We have multiple capsids that come from different directions. The capsid that you're specifically talking about is called 101. This capsid, we took AAV9.
Okay.
We looked at-
... we looked at skeletal muscle and integrin receptors on skeletal muscle, and then we created peptide, and inserted that peptide for those, integrins, integrin receptor. And so that is how this capsid... It was more of a handcrafted capsid approach, rational design, you know, made with, made for a specific reason. We have other capsids that are going through directed evolution, different parental capsid, multiple rounds, pigs, non-human primates, et cetera. And, those capsids, you know, have different reasons. They're actually focused on cardiac, cardiac only-
... and liver de-targeting. And so you'll see more of those capsids in the future. And, again, to my point, I'm gonna try to get these capsids in literally everybody's hands.
All right. Great. Thank you.
Thanks.
Good luck.
Thanks, everyone.
Thank you.
Thanks, Bo.