My name is Bo Cumbo, representing Solid Biosciences. Thank you, Goldman Sachs, for the invitation. We're going to be making a few forward-looking statements. Please take time to look at the cautionary note regarding forward-looking statements and take the time to read through it. As you know, this is science; it's hard to predict sometimes, but I'll be doing my best to look forward. Here's the pipeline of Solid Biosciences. It's a very robust pipeline. We're going to be up top Duchenne and FA, followed by a maturing cardiac pipeline that is one of the deepest cardiac pipelines in the gene therapy space. Today, I'm going to be really focused in on Duchenne and FA, and I'll touch a little bit on our platform. As mentioned before, we have two programs that are already in the clinic, as well as our capsid platform and manufacturing platform. Our novel next- generation program for Duchenne called 003 is currently in phase III clinical trials, as well as we're continuing to enroll in our phase I/II INSPIRE program, which we've already dosed 51 patients as of this week. We also have the first in-class dual route administration for Friedreich's ataxia, targeting not only the cardiac, but CNS manifestations of the disease, is another program, just like the 003 program for Duchenne, that we're extremely excited about. Our capsid library is becoming very robust. We do have Polaris out there. We have 50+ agreements with academic labs as well as small companies. By the end of the year, we should have multiple other capsids that we can license out that are going through non-human primate studies currently. Let's spend a little time on 003. We believe at Solid when we think about Duchenne, we look at the disease from a muscle integrity standpoint. Realistically, we look at both acute and chronic biomarkers of muscle integrity, and that gives us a lot of confidence to understand the long-term clinical benefit of this drug as we enter in the phase III double- blind study. We'll take a little look at, in a second, of all the data that we've accumulated to date and why we feel so confident that we are providing these young boys with clinical benefit long term. Our next- generation program consists of really three important factors. Realistically, there's a fourth factor that's not up here, and that's the manufacturing. We look at our program in three buckets right now: a novel microdystrophin transgene. This is a unique transgene compared to all the other programs that are currently out there, and that's because the inclusion of these repeats of 16 and 17, repeat 16 and 17, which is this nNOS binding domain. When you have nNOS, you get increased blood flow, decreased inflammation, decreased induced ischemia, as well as muscle injury. We believe that this is one of the benefiting factors of our program. We use a CK8 promoter. It's highly active in both cardiac and skeletal muscle. We have this next- generation capsid called SLB101, now named POLARIS-101. Just for background, why did we rename Polaris? It's because, as I mentioned before, we're talking about our capsid platform. By the end of the year, we plan on having multiple other capsids available. We want to make sure that we brand them, distinguish them from the others. This capsid that we're using in our Duchenne program, in pre-clinical studies, it was really quite amazing. It was liver de-targeting in both the mouse as well as the non-human primate. We got better distribution depending on the muscle group. We could get the diaphragm up to 10x better distribution than AAV9. You can get 3x to 4x better distribution in the quad or the gastroc. As far as cardiac goes, when we compared it to AAV9 and looked at actual expression in human cardiomyocytes, we saw about 20x better expression in the heart. That's due to these RGD peptides that are scattered across this capsid, really binding to integrin receptors and driving quick and rapid expression. It has an increased binding capacity over all other capsids that binds, transduces, and expresses very quickly. Of note, we've also learned that this capsid clears the human body very, very fast, and we are seeing it in our human subjects, our current patients in the INSPIRE trial. It's 90% cleared out of the whole blood by day four. We think that that's really going to make a difference when you think about safety, especially from an aHUS or TMA standpoint. As the body is looking to clear the antigen, it has already left circulation. I think that, that's going to play an important role down the road. We take a look at our human data in multiple different ways, not only from an expression standpoint, but also from a muscle integrity biomarker standpoint, and from the acute phase to the chronic phase, and then toward this satellite cell preservation phase, which is embryonic myosin heavy chain. It all starts with transduction and expression. Some people really enjoy Western blot, others like to talk about mass spec. I personally like to talk about positive fibers. We provide it all. That way, every single person can get what they want from this. I think I get asked all the time what is my favorite point about our data, and truthfully, it's the consistency of it. It does not matter what you want to look at. It's very consistent across the board and very linear. I think that should give you a lot of comfort that no one is cherry-picking data here. It's all our data, and it all looks roughly the same. Western blot, mass spec, positive fibers look good, but we don't stop there. We take a look at the dystrophin-sarcoglycan complex, which is critically important to these young boys. If you look at the individual proteins that make up the complex, whether it's beta sarc, delta sarc, gamma sarc, alpha sarc, et cetera, each one of these proteins provides a benefit for these little boys. We quantify that data. As you can see here, very consistent with the expression data for dystrophin. Beta-sarcoglycan is in the 60% range at day 90 and 69% at day 360. We also look at eNOS, and as I mentioned before, eNOS is very important for blood flow, decreased inflammation, decreased oxidative stress. This is an activity assay, so it's not like a Western blot. This is actually an activity at the sarcolemma level, and you can see a 35% activity of this very unique protein. Then we take it a step further. We know that we're producing protein, and so what happens on the next phase? This is the muscle integrity part. Remember, Duchenne is a muscle integrity disease. We look at acute as well as chronic biomarkers, and it does not matter what you want to look at. CK, ALT, AST, lactate dehydrogenase go to the chronic phase, sort of titin. Some will say ALT, AST is a liver enzyme. Not in Duchenne. In Duchenne, the entire skeletal muscle is breaking down. These boys, and I'll show you in a little bit, these boys' baselines are 300, 400, 500, depending on the ALT or AST. When you bypass the liver, when you have liver de-targeting like our capsid does, you can actually take a look at how the muscles are reacting to our drug, and you see a decrease in ALT and AST as well as lactate dehydrogenase, and then ultimately titin. This leads us to what we believe is one of the most important biomarkers, is embryonic myosin heavy chain. Remember, it's a disease of regeneration degeneration, and that is what is going on in Duchenne. When your muscles are breaking down in the degeneration phase, satellite cells are activated and proliferate, and then they're trying to repair or generate new muscle fibers. That's where embryonic myosin heavy chain comes in. In Duchenne, you have a lot, and that's the baseline up top. You have a lot of these embryonic-positive fibers that are activated. You don't want that. Normal, healthy subjects should not have embryonic myosin heavy chain at baseline, and we're trying to drive that down. We are seeing a decrease of roughly 44% in our little boys, and that gives us a lot of confidence at Solid that ultimately we are going to see a clinical benefit in these young children. That's how we design our clinical trial. We also take a look at cardiac care. When you look at how these little boys unfortunately pass away, it's due to mainly cardiac failure cardiac and respiratory failure. It's very important to look at cardiac benefit right from the beginning. Some of the naysayers will tell you, "Hey, you don't have dilated cardiomyopathy or low ejection fraction at this early age." One thing is very important to know. You also don't wake up at 12 years of age and end up with an ejection fraction of 50% or 55% with a diagnosis of dilated cardiomyopathy. That happens over time. There is a drift that happens, and a lot of the physicians have become numb to it, and so they don't really talk about it all the time to the parents. One day, the child will come in, it's ejection fraction of 65%. The next year, it could be 63%. The following year, 61%. That is significant over time, that drift that happens. We're tracking all our little boys. What you see is you see while the vast majority of them will have normal ejection fraction at this age, you do see a subset of patients that are at least one standard deviation below normality, that is somewhere right around the 58%. We have 12 boys, 12 little subjects that have an ejection fraction in the high 50s. Of those 12 boys, each and every one of these little boys got right back to a normal level or had increases. I think that is very important. We believe we have a hypothesis on why, there will be more to come as we release more data. It all comes back to the unique construct design that we have, the proteins that we can recruit for, and the amount of protein we can recruit for, and that sort of combination we think is what is driving some of this. Now, this is the liver and data that we look at. There has been a lot of emphasis on the liver. This is 24 subjects, but if you ask me what all 50, 51 of the boys look like, it is going to look the same. We will update this slide pretty soon. Now, on the right-hand side, I think it is important to understand, as I mentioned before, we look at ALT and AST as muscle integrity biomarkers. That is because look at how high they are at baseline. You are looking at 400, 500 at baseline, depending on the biomarker, and these decline relatively fast. That tells you that you are doing something; not only are you bypassing the liver, but you are also shoring up the muscle. Now, GGT is flat; that is exactly what GGT should look like. It should not look like an EKG. It should be very flat. You can see the whisker bars in there; you can feel confident that to date, knock on wood, this drug has been safe. We have had no drug-induced liver injury, no aHUS, no TMA, no myocarditis as I stand here today. This is our overall safety profile. As you can see, as I just mentioned, it is looking very good. Of note, we did have a little boy that had to be admitted to the hospital for a lab abnormality. He was given two different types of doses of antibiotics, only antibiotics, and was discharged. He is in the database here. Overall, our safety profile continues to look better and better as we accrue more patients. These are the overall results that I just went over. It does not matter what you look at or which biomarker you like the best. They are all extremely consistent, whether it is expression, whether it is muscle integrity, embryonic myosin heavy chains, CK, ALT, AST, et cetera, all the way down to troponin, which troponin decreases over time, and the ejection fraction increases or stays stable. Now I will switch gears too. This is our Friedreich's ataxia program. Extremely excited. This is another high unmet need, not only in the U.S., but also in the EU. There are 5,000 to 7,000 patients in the United States. It is a big founder effect out of Europe. You see a much larger population in Europe of about 25,000. That also incorporates the Middle East as well. I think what's important when you think about FA is that you not only have cardiac manifestations, you have CNS manifestations of the disease as well. When you talk to families, they'll say it's wonderful if you can treat the heart. If you can't walk, see, speak, swallow, cough, et cetera, quality of life really does take over. We want to make sure that we create a drug that meets the patient where they are and that can treat both manifestations, both the CNS and cardiac manifestations. We take a very unique approach to this. Now, before I go into our approach, I just want to talk about mFARS, and I think this is critically important to understand what we can look at and what we can't based on the patient population we're currently dosing. mFARS is really broken down into four groups; you can see the actual maximum scores in the first column in blue. Basically, you look at lower limb coordination, upper limb coordination, upright stability, bulbar function, and this is how they're measured. I think one thing that's critically important to think about, mFARS, is as you progress with this disease, mFARS always goes up. It always moves up and to the right, and that is not a good thing. It's very progressive in nature. Even when you're in the most severe patient population, which we are, we dose all these patients in the sort of dark orange to bright red, where they've already lost most, if not all, of the lower limb and upper limb coordination and most of the stability. You continue to progress over time until, unfortunately, you pass away. We're going to be taking a hard look at mFARS over the course of our program. Now, what we're trying to do is solve for this by treating both aspects of the disease that you mentioned before, without pushing the dose up. We're doing this by this very elegant way of dual route of administration. By doing dual route of administration, you can get to the heart of the matter of the disease, which is really the dentate nucleus of the cerebellum, the spinal column, and the heart. By separating the route of administration, you're able to give a much lower dose. Our total dose for the intradentate administration is in the E9 range, and that's the total dose. That's not done by weight. Of course, the dentate nucleus is very small. By doing that, we can actually give a lower dose in the IV and get to the spinal column and the heart, that is a vg/kg dose, and that is in the low E12 range. Now, why is this important? As I mentioned before, you want to get to the patient wherever they are in the disease. I want you to think about three different types of patients. You could have a 30-year-old patient who has already lost lower limb mobility, upper limb mobility, most of his or her stability, can barely speak, can't read, can hardly swallow. Realistically, you're probably not going to do that much in the spinal column, the dorsal root ganglia. I'm sure you can help in the heart, but it's very important to get to the dentate nucleus and try to restore that individual. Some of the ability for that individual, or at least slow down the disease. That's why we do this dual route. You also have a 20-year-old patient. This 20-year-old could have the ability to walk, maybe not. Might have dilated cardiomyopathy, maybe not. You don't exactly know where they are, but by doing this dual route administration, you can treat that patient. You think of a six-year-old little girl, six or eight. She just got diagnosed. She really doesn't know what her future's going to be. You want to make sure that you can treat them, that little girl as well, before the disease really takes over and slows her down. Dual route administration will get to the heart, the spinal column, and the dentate nucleus for that little girl as well. This is the only drug I know of its kind that can treat all different populations of the disease, wherever they are within their disorder. This is the dentate nucleus. What we're trying to do on the left-hand side is coat about 15%-20% of the dentate nucleus. That's what we believe you need for coverage before you can start really seeing meaningful clinical benefit. On the right-hand side is our dose. You can see we use this enhancement agent. It's MRI-guided dual route of administration right to both dentate nucleus with an enhancement agent. We were shooting for 15%-20% coverage, and you can see here, while we didn't quantify, we did quantify it internally, we've just haven't disclosed it. It's significantly more than 15%-20%. This is patient one, by the way. Patient two was even higher coverage of the dentate nucleus. What we're going to do the rest of the year, we're hoping to dose somewhere in the range of five or six patients by the end of the year. We can hopefully have a readout early next year and provide all the clinical as well as safety data. I don't have a safety slide in here, but I'll tell you both patients did extremely well. Second patient had no AE at all, like no headache, no nausea, no vomiting, no fever. The first patient only had a headache that resolved with Tylenol. Both patients did extremely well. We've never had an SAE, knock on wood. The only AE that we've had was a headache, as I mentioned. Later on this year, we're going to continue to dose. Eventually, we're going to go down to ambulatory, we'll do some ambulatory, non-ambulatory study if we want to. Anticipated milestones. This is going to be a pretty exciting year for the company. This year and next year could be transformative, and really, with multiple inflection points this year, either through regulatory interactions as well as data readouts. We've already met with the FDA twice in the last six, seven months. Once on the phase III clinical trial design, the double-blind, placebo-controlled trial for Duchenne. Obviously, that trial is underway. We got approval from the FDA. We're still working on approval with European countries, and that should happen relatively soon. Currently, Australia, as well as Canada, is open, and we'll have more sites open later on this year. We also met with the FDA to establish whether there was an unmet need in the disease state and to talk about a couple of other things. Even with a commercial- approved microdystrophin gene therapy, the FDA still states that there's a high unmet need in Duchenne. They have also acknowledged that our program is unique. We create a unique protein. We also have a unique capsid that has these RGD peptides on it for better expression and distribution, or at least better distribution. They do acknowledge that our program is unique, as well as there is a high unmet need. One thing we talked about is our trial; do we have enough data for a surrogate marker for clinical benefit? The answer was no, because we've actually never shared clinical data with them. It turns out you need clinical data to get that clinical benefit; we have not even looked at it. We're going to look at this in July and August, and start doing all the analysis internally. Currently what we're doing right now, we're updating the stat plan. We're going to submit their stat plan this month in June. Once we do that, Dr. Brooks and his clinical team are going to start looking at all our data and doing the analysis over July and August. Then we'll request a meeting to go over the data for the first time with the FDA later this year. I do think it's important to understand that the FDA has told us in the past, this last meeting, they're really not that fond of open- label studies with functional endpoints, and I don't think that's a surprise to anybody. In the same breath, by the way, they acknowledge, "Hey, thank you for starting our double-blind, placebo-controlled trial," and we've already dosed our first patient. We're working very hard to try to eliminate any bias that's in the INSPIRE trial, and we're going to provide data to them later on this year. I also give an update on the enrollment for the placebo-controlled trial, and hopefully find a path forward with the FDA. We're very excited about the discussions with them. Of note, safety never came up in our discussion. We did provide them somewhere between 30 and 38 patients' worth of safety data at the time of the meeting, and no discussion around safety whatsoever. For FA, we're going to dose somewhere right around five patients, hopefully by the end of the year. If everything goes well with safety and potentially efficacy in these patients, we'll reach out, we'll set up a meeting with the FDA at the end of the year to early next year and talk about a path forward, whether it's either with a natural history study or some kind of other study where we can gather additional data and think about how we can move our program into future studies down the road. For CPVT, we have multiple patients that are trying to get into the trial. We have been going slower with CPVT, and we've been doing it on purpose. We're only 130 full-time employees, and we have two programs in the clinic with three different trials with INSPIRE and Duchenne. We are going a little slower with patients later this year. We also have another program called TNNT2, dilated cardiomyopathy, and that will be ready for an IND later this year as well. With that said, I will say thank you very much to Goldman Sachs. I appreciate your time and thank you for letting me present. I have a question. You mentioned that there was talk about the transgene. One of the discussion points that came up, for anyone who's online, the question was around the unique transgene and any discussion points around it. The transgene has this repeat domain called R16, R17. When you have R16, R17, you can actually recruit for a protein called alpha-syntrophin. When you have alpha-syntrophin, that actually is a binding spot for another protein called nNOS. nNOS is needed, and there's studies out there that show that patients with nNOS and without nNOS, patients with nNOS actually walk longer. They do better in life. This protein has been shown to increase blood flow, decrease fibrosis, decrease oxidative stress, decrease inflammation. We're the only program that has this repeat domain of R16, R17, and that gives us the ability to discuss this with the FDA. This protein, combined with the sarcoglycans, especially beta-sarcoglycan and delta-sarcoglycan, can really provide a lot of benefit, especially on the cardiac side. I think that that's one of the reasons that we are seeing a difference in some of the cardiac output right around that. The commercial landscape has continued to evolve. Unfortunately, there's a lot of patients that need help, and I think it was important that the FDA did state that there is a high unmet need for Duchenne, even with the products that are currently on the market. Unfortunately, I say unfortunately because I wish the disease didn't exist, but the gene therapy-naive market is unfortunately getting bigger. Why do I say that? Every single year, there's about 400 patients who are born with Duchenne, so roughly 100 patients are born with it a quarter. Currently, with the gene therapy that's on the market, of course, this is my guesstimation, is the majority of patients are being dosed in the 340B clinics, and that puts the net price, my math, not anyone else's, somewhere right around $2.8 million. When you really break it down, that's about 40 to 44 treatment regimens per quarter. That means each quarter, there's about 60 little boys that are born that are not being treated. The actual incidence population or the prevalent population is increasing because the rate of therapy is actually slowing down. I hate to put it in these types of terms, but unfortunately, there's about $1 billion-$1.2 billion annualized that is born in Duchenne each year, and if you're treated with a microdystrophin type of program. The market is getting bigger, and that's an opportunity for us to come in with the next-generation gene therapy. As I mentioned in the presentation, we've dosed somewhere right around 51 patients. More to come very soon. The drug looks relatively safe with a safety profile with some of the highest expression out there. I think that this long-term is going to provide a clinical benefit, and hopefully, we can take the majority, if not all, the market share in the disease state. Thank you very much. I appreciate your time.