Solid Biosciences Inc. (SLDB) Earnings Call Transcript & Summary

So good morning, everyone. Thanks for joining us this morning at the Citi Healthcare Conference. Max Riso here with the Citi Healthcare Investment Banking team. Very excited this morning to have with us Bo Cumbo, CEO of Solid Biosciences joining us. Solid Biosciences is advancing a portfolio of neuromuscular and cardiac programs, including SGT-003, the differentiated gene transfer candidate for the treatment of Duchenne Muscular Dystrophy; AVB-202, a gene therapy treatment for the treatment of Friedreich's Ataxia; AVB-401, a gene therapy program for the treatment of BAG3-mediated dilated cardiomyopathy and additional cardio assets. Look forward to having Bo with us, and I will pass the mic over to Bo. Thank you, Bo.

Thank you, Max and Citibank. I really appreciate the opportunity to present. I'm going to be making a number of forward-looking statements today. Please take time to look at our forward-looking statement page on our presentation. Many of the events I talked about today could differ from -- materially differ from the outcomes, which could be out of our control. So please take a look. For those of you online, there is a presentation. Well, it's [ found ] on the presentation page on solidbio.com. With that said, I really do appreciate everybody taking the time to come and talk to us today, and talk about Solid Biosciences and our key investment highlights and our progress that we've made on our program so far to date. On Page 3, you'll see that we're going to be talking a little bit about our management team and proven experience from the management team as well as our diverse pipeline, our manufacturing capabilities and our capsid library formation. On the next page, in your presentations, you can see on Slide 4, the management team. We have a very, very experienced precision genetic medicine management team that has been around companies, not only in biotech, but also mainly genetic medicine and more specifically in gene therapy. Leading with myself; Ty Howton; Kevin Tan, who's in the audience today is our Chief Financial Officer; Jessie Hanrahan, who comes from Bluebird previously and AavantiBio. And then Chief Scientific Officer, Dr. Jenny Marlowe; Paul Herzich, who's our Chief Technology Officer, who was the Vice President of CMC at BridgeBio prior to this running their gene therapy programs. So from a CMC perspective, we have excellent manufacturing team. And then Shu, Dr. Kulak, our Head of BD. And I've just recently started hiring a CMO, and we'll have that CMO in place very shortly. That CMO will be a cardiologist, with cardiac background. And you'll see why because our pipeline is so robust within cardiac genetic medicine. On the next page, you can see a strategic outline of what we're building and what we're working towards. This is on Page 5. And you'll see that we have a very differentiated platform than many other companies. At the top, you can also see that we put delivery at the forefront of everything that we're doing. So it's not just novel capsid and a capsid library, but it's also the CMC platform. CMC is the drug, and so everything that we do revolves around CMC and our process. And then once you look at our programs, you can see very quickly the strategic nature and how we're developing this, and we're leveraging our capabilities and knowledge that we have in Duchenne and FA and moving it over to our cardiac pipeline. Today, we'll be talking a little bit about Duchenne, FA as well as BAG3, and we're announcing another program today that I'll be discussing very soon. We're also working on our capsid library and we're excited about our capsid library. We just had another capsid that was came out of this library this recent week, and we'll be using these capsids for non-dilutive financing down the road. So on Page 6, you'll see the pipeline that we're working towards. I just talked a little bit about it, and we're going to go through in depth these programs. So the next section is the neuromuscular programs, and we'll start with DMD. And many of you know DMD because it makes headline news from other companies that are in the space. Obviously, it's caused by a mutation in the dystrophin gene that leads to a lack and absence of this protein, and we're trying to replace this protein with a functional mini dystrophin or microdystrophin. Now, we talk about Duchenne a lot from an epidemiology standpoint of 10,000 patients in the United States. But realistically, when that's the ocean of Duchenne and realistically, when you look up under that ocean and there's subpopulations. And every population needs to be treated a little bit differently. So when you look at Page 9, you'll see the different sections that actually make up Duchenne from our early ambulatory phase. You have about 3,500 to 4,500 kids that are somewhere in this age, 1 through 8. You have about 1,500 of those children that have antibodies to drugs prior to being dosed. So they'll have antibodies to rh74 to AAV9 to AAV8 to other capsids. It's just somewhere along the way, they've been exposed. And we have to find a way to treat these children. We have to find a way to lower their antibodies down to a specific threshold and then dose above that. You have older children, they're sort of late ambulatory, they're partially ambulatory or they're nonambulatory. This represents roughly 50% of that population as well. And unfortunately, many of them -- 15% to 20%, have antibodies for these capsids as well. So we have to -- we have not only the adult -- older population who's weaker, they typically have a fat fraction of greater than 80%. This is why they're nonambulatory. They have fatty livers. They have decreased pulmonary outcome, decreased ejection fraction. These children are fragile. And you're going to have to find ways to get the drug to them. And most likely, you're going to have to find ways to get the drug to them over and over in the heart, in the diaphragm. So redosing is going to play an important role. And then unfortunately, there's going to be a subpopulation that's going to be dosed with drugs that are currently coming to the market and these are going to be suboptimal durability for one reason or another, either the child didn't get enough exposure or the child has a breakdown of muscles. And unfortunately, these drugs are not in the stem cells, the satellite cells and over time, as the muscle breaks down, they're going to regenerate dystrophic muscle. And so we're going to have to think about redosing these children. And the population, if these drugs are working, and I believe they are, if these drugs are working to increase survival over the long haul, Duchenne gets bigger. It actually doesn't decrease. It actually becomes a bigger, more urgent population. Now as we're developing these drugs and we're thinking about the next generation, we have to consider all this and think about the redosing and what it takes to redose and it starts with the actual construct itself, including the capsid and the manufacturing. So on the next page, on Slide 10, you'll see that we have now what we believe has created the next-generation Duchenne program. We use this transgene that has domains to R16, R17 in it without Hinge 2. So the Hinge 2 -- without Hinge 2, you provide increased flexibility as well as with R16, R17, you are recruiting for alpha-syntrophin as well as nNOS, that's very important proteins for the microdystrophin. We've changed our capsid. We're using a new capsid that comes out of our capsid library called SLB101. I'm going to talk about why this capsid is so important. And then we've changed our manufacturing process. And we've made improvements to our manufacturing process, not only in Duchenne, but our future programs, and we're on the cutting edge of really transforming the way we think about gene therapy manufacturing, not only from a yield standpoint, but also from a plasmid and purity. So when we think about Duchenne, it starts with transduction speed, especially in a new world where we have to lower antibodies and we have a short period of time to dose, transduce and express. And on the next page, you will see that this capsid, this SLB101 along with our transgene has done something that we actually have never seen before in Duchenne, where at Day 4, we're seeing expression in the MDx mouse. And so what does this mean? This means that we have dosed, we have transduced, and we now see an expression at day 4. We've repeated this study and we can see expression all the way down to day 2. At day 29, all the animals regardless of the dose, at the doses that are listed here, they're all at roughly 100% across the board whether it's the heart, the diaphragm, the quadriceps. The heart and the diaphragm is extremely important, especially in the nonambulatory boys because that's all they have left. You don't have peripheral muscle. So you're looking at trying to get to the diaphragm and the heart. And this capsid in our nonhuman primate study increases microdystrophin expression, around 3- to 5-fold in the nonhuman primates in our GLP tox study in the diaphragm. And that's extremely important when you think about pulmonary function in the nonambulatory patients. The following slide, we look at the mdx mouse and we compare it to Solid's original program which is -- which was called 001. How did this program -- this next-generation program compared to Solid's original program. And you can see from a biodistribution standpoint, expression standpoint and from a reduced CK levels it's significantly different than Solid's original program. In nonhuman primates, on the next page, Slide 13, you can see that this -- what we saw in the mouse did translate over to these nonhuman primates. Now this is with luciferase. It's not with microdystrophin, it's luciferase. We looked at skeletal muscle, looked at cardiac muscle and looked at liver. We have completed our GLP tox and that study started in December. We took down the animals in March at the end of study, all animals from start of trial to the end of the trial did very well. And there were no unscheduled takedowns until the end of the trial was completed. We're in the process of writing our IND. We're on track to file the IND in early Q4. And then hopefully, we will be screening and dosing patients shortly after IND acceptance, which should happen roughly 30 days or 40 days post submission. Now the following program. The next program I'll talk a little bit about is Friedreich's Ataxia. That's Slide 14. We're very committed to the patients in Friedreich's Ataxia. If you don't know a lot about FA, what I'll tell you, the most important thing is to understand that it is a multisystem -- multi manifestations of the disease and cardiac as well as neuromuscular and CNS. And it's extremely important to pull all the patients along when you're treating these patients. It's a very large unmet need. And what we decided to do, we have a lot of data on cardiac and we feel very comfortable with the levels in the expression and the outcomes we're getting in cardiac. However, we've determined that it is not sufficient to move forward cardiac only and that you really need to make sure that you address the CNS and neuromuscular manifestations of this disease. And when you talk to patients, and the patient advocacy organizations, they will tell you, hey, listen, it's wonderful if you can cure a cardiac. But if you lose the ability to walk, see, speak hear, quality of life really does matter. And so what we're trying to do is address all the manifestations of the disease by doing dual route of administration. And why is that important? Because it's not just the amount of frataxin expression, it's actually the distribution of that frataxin. It's across the DRGs, it's the base of the cerebellum, it's the Purkinje cells, it's the heart. And so that's what we're trying to accomplish. On the following page, you can see why we're so confident about cardiac. We've started at the very top and find that sort of toxic levels of frataxin and then we work our way down to get to the lowest level -- the lowest dose with the best outcome. And you can see that we're already down to 6.0E12. We can change ejection fraction, we can change survival. And just as importantly, on the far right-hand side of the screen, you see SDH activity, that's succinate dehydrogenase, in patients that have FA when, unfortunately, they pass away and you do autopsies and you look at the mitochondria in the heart, the DRGs, you see succinate dehydrogenase is completely depleted. You see the mitochondrial chambers. And so what we want to know is while we're restoring frataxin, is it functional and we can restore succinate dehydrogenase level and which is a surrogate for the mitochondrial health right back to normal levels. We can also, as I mentioned, change ejection fraction, change survival. So now we want to know, can we do this in the CNS model? Can we get to therapeutic levels without creating a toxicity event in the heart? And so on the next slide is a very large Slide 16. It's a very large nonhuman primate study, 40 nonhuman primates. This study was done of 6-month period of time to help us understand, can we get into dose therapeutic ranges in the heart as well as the DRGs. And the answer is yes. We looked at multiple different doses and multiple routes of administration. IT only, IV only, IT and IV combination dual therapy with multiple doses. And the outcome is that we can elegantly increase expression by manipulating the dose and the route of administration. So we can get to therapeutic levels in the DRGs as well as the heart. Now what are we going to do next? We're going to take these learnings, and we're going to look at our nonhuman primate study to understand, are we getting enough frataxin in the cerebellum to make a difference? We're going to be talking to KOLs about this. We're also working on a CNS mouse model to understand can we change the outcome in the CNS model similar to what we did with the cardiac mouse model. And more to come on time lines to an IND for this program, and we'll -- I'll update you further as we move forward. Now I'm very excited to start talking about cardiac programs. You saw in our first 2 slides, we have a whole host of cardiac programs that we're building in a very strategic manner. Our first program is called BAG3. And -- well, let me talk a little bit first about Slide 18 of why we're going into cardiac. Really, when you think about cardiac, you look at CNS, you look at neuromuscular, you look at all the companies that are out there are really attacking these rare and fatal diseases, cardiac has been largely untouched. This is going to be a huge opportunity for companies that are in the space. Projected societal cost is going to be $1 trillion by 2035. And heart failure rates are 40% -- heart failure mortality rates are 40% after 5 years, even more so when you think about some of the programs that we're trying to tackle. These are big disease states, dilated cardiomyopathies, BAG3 more specifically, which is our first program is 29,000 patients, CPVT, and I'll talk a little bit about that, 33,000. And then hypertrophic cardiomyopathies can be up to 600,000. These are huge opportunities, and we'll talk about why we're going into them later. BAG3 is our first program. If you don't know BAG3, you should get to know it. It's called -- it codes for this BCL-2-associated athanogene 3 protein or BAG3. When you have reduction in this protein, you're going to end up in cardiomyopathy and you're ultimately going to end up in heart failure. Males decline a lot faster than females. And once you're symptomatic, your quality life is shot. You're in heart failure. You're not moving around. You're not active. Eventually, heart failure sets in, and mortality sets in, 25% in the first year once you're symptomatic, 50% in 5 years. Now we're delivering -- we worked on multiple different constructs. We took our time with this to understand constructs, promoters, the combinations plus manufacturing and we landed on rh74 with a very specific cardiac promoter. -- with triple transfection manufacturing platform that we're using across all of our programs. And we have a great collaborator and Dr. Eric Adler, out of UC San Diego. We're working with him. And you can see on the next slide, Slide 20, we're utilizing his mouse model. And this is a great time-dependent mouse model that's very predictive. And this will be the program that we're working on. We've also created a construct that is very specific for the heart, as you can see on the right-hand side of the page. It goes to the heart, it doesn't go to the gastroc, doesn't go to the quad, it doesn't go to the liver. So we're very excited about this. On the next page, you can actually see on Slide 21, how 2 of our constructs that we created compare against each other, one rh74, one AAV9, with a ubiquitous promoter on AAV9 and the cardiac-specific promoter that we haven't disclosed. And you can see we're getting to the heart. We're getting to the endogenous levels that we actually need to. However, it's not going to the liver. It's not going to other muscles. And so this is going -- we're very excited about this program. Equally important on Slide 22, when you look at cardiomyocytes. When you actually look at the heart muscle, you can see that 80% of the cardiomyocytes are going to be positive for BAG3. This is huge. We don't believe that we need anywhere near 80%. However, our drug is getting to the heart and getting to the hear well. Now our next program, we're very excited about this program, and we're working with a collaborator that's one of the world's leading experts in this. It's a Catecholaminergic Polymorphic Ventricular Tachycardia, CPVT. It's a fatal disorder. It's actually 2 disorders. It's called CASQ2 and RYR2. And this program, we're extremely excited about. It's a large population. Now CASQ2 is relatively small, about 2,000 patients in the United States. However, RYR2 is 20-plus thousand patients in the United States and extremely fatal. When you look at RYR2, and that's going to be our lead program with the CPVT. When you look at RYR2, these kids are diagnosed young, age 7 to 12. They end up with ventricular tachycardia , many of them die, present with their first instance of ventricular tachycardia, they end up unfortunately passing away. You see this in a lot of sports kids that are playing sports that unfortunately eventually fall down and die or have to be treated. Also, when you think about the program -- what they are on, there's really no drug to treat the underlying cause of this disease. And we believe that we have an approach that we'll do that. And so we'll talk a little bit about this. And what we're planning on doing is delivering and overexpressing the CASQ2 transgene and overexpressing calsequestrin. And this will inhibit the -- it's on the next couple of slides, but this will inhibit the arrhythmias in RYR2, and we have the animal model to prove it. Now on the next slide, Slide 24, you can see, as I mentioned before, how big of a population this is. And I already told you that BAG3 is going to be roughly 30,000. Our next program is going to be roughly 20,000 in the United States. It averages -- so 80% to 90% of the mutations will be adaptable to our program and there's no therapies currently. The current options for these children are exercise restrictions, so you just tell them, you can't move, can't run around, can't do anything. Beta blockers, but the beta blockers are very important to take on a very timely manner if they miss a dose, they could have an event and they could unfortunately pass away. And so the outcomes are not very good for this. And so this is a perfect disease state for us to go into. And I believe we're the only company that is doing this currently. So on Slide 25, you can see what we're trying to accomplish here, the normal signaling on the far left, what's actually happening in the RYR2 mutation in the middle. And then following the over expression of the CASQ2 or the calsequestrin, we're binding up all the calcium and this gives us a normal rhythm in the RYR2 channel. On the following slide, you can see our results on Slide 26. And we have 2 different mouse models. We have the RYR2 mouse as well as the CASQ2 mouse. And you can see both in the low dose, the medium dose and the high dose that we can pretty much eliminate all arrhythmias in this mouse model. So we're very excited about this program. We're going to be moving this from a research-grade material into normal FDA-approved type of material getting away from the low empty defaults. We're also going to be moving this over to a capsid that we're working with internally. More to come on that, we'll announce which capsid in short order. But we're very excited about this program, and we're going to be moving it forward very quickly. So the next slide, we'll be talking about our platform, and it's extremely important if you're going to be a next generation or a genetic medicine company in the gene therapy space, you really need to focus on delivery and delivery comes in multiple forms. And the 2 most important forms are capsid delivery and CMC. So let's focus on capsid delivery first. We're in the middle of our building out of our capsid library. I already mentioned that we're using SLB-101 for our Duchenne program. That was our first capsid that came out of our capsid library. We actually have another capsid, we're working on internally called SL34 and it bypasses the liver all together and it goes specifically to the heart. We're very excited about this new capsid that's coming out of our library, and we'll be moving that forward and talking more about that in the future. We're already through our second round of nonhuman primates as well as pigs and our overall capsid library. We'll be taking -- we've already taken down the animals, and we'll be working on identifying 10 capsids to move into further rounds of nonhuman primates and pigs as well as mice. More to come on that. The next slide is around manufacturing. We do -- everything we do revolves around manufacturing. We have the ability to scale up to 500 leaders in-house. So in our process development team in Charlestown right down the street, about 100 feet from where I sit, we can go from a shaker fast to 10 liters to 50 liters to 250 liters to 500 liters. Feel free to come by and we'll give you a lab tour. You can see the labs yourself. It's extremely impressive. We also have a vector core down in North Carolina that makes all our small-scale materials. So any material that's for nonhuman primates or mice, can be built out of our vector core while we hone in our process at the 500-liter level in Cambridge. We've also made improvements. We've already made improvements for our Duchenne program. We're making improvements for our BAG3 program. We're moving from a triple plasmid to a dual plasmid platform, which was not many companies are doing. And we believe that we can make multiple other improvements that we've seen in the labs in our process development team right now, and we can scale up and increase yields by about 15x to lower the COGS, increase purity. And we definitely focus on the full to empties. And most of our drug is close to that 80% true full. So it's very, very important. The next 2 slides is really driving to the future. So Slide 31. The overall, what is our foundation going to be built upon. Obviously, I mentioned its new management team that came in during the merger of Aavanti with Solid. We now have a very diversified but strategic platform and pipeline that you can see we can build off each other. So the knowledge that we gained from BAG3 or CPVT or FA can really work all synergistically together. And you also saw that we have multiple other cardiac programs that we'll disclose in short order and these programs continue to build. It also provides a lot of confidence for the KOLs that we speak to that they know that we are committed to the space. And I think it will be a major driver and player in the cardiac space. We have our next-generation Duchenne program. That IND is on track. And so in the next -- so it's September I said early October, we'll have [ IND ] early Q4. We'll have an IND. And so we feel very comfortable with the time lines there. And we'll be dosing patients as soon as we can get IRB approvals and screen patients post IND acceptance. We have thought leaders that are working with us around the world, not just the University of Florida but we have Wisconsin -- University of Wisconsin is working on one of our cardiac programs. UC San Diego, as I mentioned, Dr. Adler is working with us on our BAG3 program. We have [ Iowa ] that's working on one of our dilated cardiomyopathies. We're working with Phlox on a rare dilated cardiomyopathy. And we have -- so we have leaders across the world that have collaborated with us now. We're really building a great network. And then as I mentioned before, platform where you have a lot of capsid library that is already pulling out multiple capsids that are very unique. And then we have our CMC platform where we can scale up to 500 liters in house, really refined product trying to focus in on that 80% true full, not partials true fulls and it's extremely important, CMC is the drug. And so what are our anticipated milestones is the last slide on Slide 32. As I mentioned, Duchenne, coming real soon to you for an IND capsid library dosed already nonhuman primates and pigs, second round. Third rounds coming up at the end of the year or early next year. AVB-401, that's our BAG3 program. Dose-range finding is underway that we'll move into GLP tox now the preclinical studies early next year. And then for CPVT, we have CASQ2 as well as RYR2. Drug candidate selections underway as well as preclinical studies will happen early next year, and then we'll move forward with that. We have $160 million in cash as of June 30, that will take us into 2025. And with that, I'll turn it over -- back over to Max. But thank you very much for your time. I really do appreciate it.

Thanks. Maybe we can have a seat and have us sit down, would like to answer. Well, congratulations to you and the team on all the progress you've made and exciting to hear about the new program. What I thought we do for the remainder of the time here is maybe just spend a few minutes peeling back the layers of the onion in some points you made during the course of the presentation. Maybe to kick us off, I was just wondering if you could comment a little bit more on how you see the SGT-003 program being positioned in the Duchenne market.

Yes. Yes. As I mentioned, Duchenne is a -- unfortunately, it's a traumatic disorder, and it's very complex. And every patient is different. It's a very heterogeneous population. As I tried to show you on that one slide, but really that slide doesn't give it justice. You have so many patients that are sort of early ambulatory, late ambulatory late ambulatory has multiple layers within it as well because these kids are fragile. You have a decent amount of kids, 15% to 20%, sometimes it could be greater, depending on the capsid that have antibodies before they've been dosed. Duchenne is going to be here for a long time. If these drugs, by the way, work, and I believe they do, Duchenne is going to get bigger. And we're going to have to find ways to redose, as I mentioned, for the -- not only the kids that already have antibodies, but for the older children that can really only get to the heart and the diaphragm. You have to have a capsid that really drives expression in the diaphragm and the heart. And then unfortunately, you're going to have a lot of children that don't get the response that they truly need, and you're going to have to find ways to lower antibodies and get to them. I think what we have done is we've created a next-generation program that can get to and transduce and express very quickly in that 4-day window. So once we're able and once the scientific community is able to lower antibodies down to a certain threshold, and we're working on that internally right now then we can dose transduce and express. We also know that this capsid gets to the diaphragm a lot better than AAV9, our old program. And so -- and that's what you're going to have to have for these older kids, right? Get to the diagram in the heart. It was already expressed in the heart very well AAV9 does. But the diaphragm is always lower. This program in our nonhuman primate is getting about 3 to fivefold higher than AAV9. And so we feel that it's positioned well. And unfortunately, these kids -- these drugs, all drugs, so it doesn't matter which one you want to talk about. They really don't get in to the satellite cells, these stem cells. And unfortunately, these little boys start acting like little boys again. And they're going to run around, they're going to tear down the muscle. You can see the CK rise. And you know that microdystrophin drug muscle is going to turn into dystrophic muscle after it's regenerated. So we're going to have to find ways to get to it. So I think it's positioned very well as a second-generation drug, where we can get to older population as well as the younger population, naive as well as redosing. Now we've got to work on the science to get the threshold, antibodies down to a certain threshold so we can dose, and that's what we're going to do. Unfortunately, Duchenne is going to be around for a very long time.

Moving over to cardiac, and I know you touched upon this a little bit in the presentation. But anything else to note on why you've chosen -- you and the team have chosen cardiac as the next phase to make an investment. But more importantly, the indications that you selected?

Yes. I think one, we are one of the leaders in cardiac space. And we started off working on cardiac multiple -- many years ago at Aavanti. And Aavanti was actually purchased by Solid Bio last December. And so we brought over all the programs from Aavanti. So we've been in the cardiac space from the day Aavanti started and why is the next frontier. And you can actually see some follow-on -- where companies jumping into the space recently because they know that we're on to something. This is going to be a huge market, neuromuscular and CNS. They were the first and the -- and genetic testing started really ramping up 2010, 2011, 2012, I ran a genetic testing program for one of my previous companies I worked for, for a very long time. And you saw the increase in genetic testing. You did not see that in cardiac. You're seeing it now. Cardiologists are jumping on to it because they're realizing there's so many reasons for these ventricular tachycardia. I mean, arrhythmias for dilated cardiomyopathies, hypertrophic cardiomyopathies and they're jumping on and now they're dosing -- they're genetically testing over and over. So these populations are going to explode and no one's in the space or not many companies, just a handful and Solid is leading the frontier. You don't have a lot of turnover in the muscle. So -- and you can use a promoter drive expression right to the heart, as I showed you with CPVT, as I showed you with BAG3, so we can go right to the heart you're not having turnover. There's other reasons. It's huge unmet need, $1 trillion by 2035, as I mentioned. And I think what's also important is you see our strategic pipeline and how we're building it, we can piggyback our knowledge off of it. So our program 401, 501, 601, they all start to add up. And so I think strategically, it is the right move and it's positioned very well. And importantly, when we talk about rare disease, I'm still in rare disease, but now I'm into patient populations that are 20,000, 30,000, 50,000. And so from an investor standpoint, when I hit a milestone, it's going to be significant. And I think that's very important as well.

The capsid library, very important part of the Solid's story. Can you just explain to us a little more about the value of the work you and the team are doing there?

Yes. Look, it's everything. We believe everything in gene therapy and precision genetic medicine is going to revolve around delivery, and delivery takes sort of multiple roles, right? It's the CMC, making sure you have a very clean and pure product and try to drive the yields up. It's also been delivery from a capsid and promoter standpoint. And we focused in on capsids because there's a lot of capsids that have been tried and true. But they have some flaws and we think that we can do a better job of delivering specifically to the heart and then try to bypass the liver. And so we've been building these capsid libraries for an extended period of time. We're actually starting to see major results. So obviously, our first capsid came out of it, it's SLB-101. That's the capsid we're actually using in our Duchenne program. We have another capsid that literally we just received the data on this past week called SL34, and it completely bypasses the liver. We're going to use this. We're looking at using these types of capsids for our cardiac programs in the future. But this capsid goes straight to the heart, bypasses the liver almost all together. And what we want to accomplish is we want to build a whole portfolio of capsids like this. So we're already through our second round, as I mentioned, nonhuman primates and pigs and our capsid library. We did first round was mice and nonhuman primates only. Second round was mice, nonhuman primates and pigs. Now we're going to take it down to about 10 or so, 10, 20 capsids and then really develop those capsids. We'll use these capsids for our own pipeline, but we'll also use them for nondilutive financing for people who want to work with them. So very excited about the capsid library. And if you really want to be a precision genetic medicine company leader in the space, you've got to get delivery down, and that means CMC as well as capsid/promoter.

Great. And on that end, you and the team have also made significant investment on the manufacturing end of things. Can you talk a little bit to us about what you've done there and why you view this so important?

Yes. So we have a full process development, analytical development team in place. And as I mentioned, in Charlestown, where Kevin and I said about 100 feet our process development team could scale up from Shaker flask all the way to 500 liter. So they go for shaker flask to 10 to 25 to 50 to 250 to 500. So we locked down our process -- and we modify our process. We continue to modify our process for future programs. Once you lock down your process like Duchenne or BAG3, it's locked. Don't make any changes because the FDA won't like that, and it will just send you back but you continue to modify your process, make your own process. By the way, that is proprietary to us and how we did 2 things. And then we believe we -- based on the data that we have, that we can potentially get up to 15x greater yields by making tweaks along the way. And we're going to continue to do that, modify it, follow our patents and keeping it as a trade secret. We're already moving away from triple plasmids to dual plasma design. That increases our yields as well as decreases our COGS. And so process development is everything. Now we have a vector core down in North Carolina. So they're full-time busy, and they're backed up for like months just working on making material for mice as non-human primate studies. So our PD team stays there and then our vector core. It's more -- very important for you all to know that we have 2 other teams that not a lot of companies have. We have a CMC regulatory team that's embedded in everything we do. And so these are regulatory experts that focus on CMC only. So they're not regulatory strategy type of teams. There are people who understand CMC from the very smallest of molecule and changes and how that affects regulatory they're embedded. We have a medical manufacturing science and technology team. And why is that team very important? Because when you create your own process and then you have a CDMO that's making your commercial grade material for you. You don't want them to mess around with your process. You don't want them to make changes. So we embed this MS&T team in the CDMO. They sit on the floor. They're solid employees, but they're embedded in that CDMO and so you don't make any changes. It's extremely important if you want to get it right.

I know we're coming up on time, but maybe if there's any questions from the audience. Okay. Great. Well, Bo, on behalf of Citi, it was a pleasure having you up here today. congratulations again on all the progress and look forward to seeing more.

Yes. Thank you, Max. Thanks, Citi, for the invitation. I really do appreciate it. Thank you.

Thank you.