Good afternoon, everyone, and thank you for joining me at the second day of the Needham Healthcare Conference. My name is Gil Blum, and I'm a Senior Biotech Analyst here at Needham, and I cover the cell and gene therapy sub-sectors. It is my pleasure to have with me today Bo Cumbo, the CEO of Solid Bio. Bo will start with a short presentation, following by a Q&A session. Bo, you have the floor.
Thank you so much, Gil, and thank you to Needham for the offer to present today. We really do appreciate the time. Oh, I'm having a little already difficulty here on getting the slides. Today, we'll be going over a lot of data and answering a lot of questions. Obviously, we're in a unique time from a regulatory standpoint as a company. Please look for the forward-looking statements disclaimer and take time to read through them as I'm going through the data and going through question and answer sessions. This is our pipeline. Overall, very proud of the pipeline. We've come a long way in the last three years.
Today, I'm going to spend all my time on Duchenne and FA for this presentation, but realistically, we have multiple cardiac programs, and we're finally getting our first cardiac program out of the Mayo Clinic collaboration that we're already talking about internally. It's going to stay undisclosed, but it's a very large cardiac indication. We're very excited about the data that's coming out of the Mayo Clinic collaboration. Today, though, we'll spend our time on 003 and on 212, which is for FA. I'll spend a little time on this slide. Many of you have seen this before, but I want to make sure that we go over why it's so critically important to talk about not only the microdystrophin transgene, but also our capsid makes it a very unique program. On the left, this program is unique for many reasons.
One, it only has two hinges, so it's one of the most flexible microdystrophin proteins out there. It also has this R16, R17 binding domain. This unique inclusion of the nNOS binding domain, it's with the goal of preventing induced ischemia and associated muscle injury. It also increases blood flow, decreases fibrosis, decreases oxidative stress, and it is very critically important for normalizing NO production, which improves calcium homeostasis and enables contraction and relaxation in the heart. Another aspect of this that's not even on this slide is we also recruit for what's called cavin-4 protein in the heart, and it acts as a structural component and signal regulator in the heart. We believe the combination of this nNOS plus this cavin-4 tied to high levels of beta-sarc, delta-sarc, alpha-sarc, gamma-sarc, are what is making the ultimate difference in these little boys' lives.
Now, why am I honing in on this? I think it's extremely important for you to know that one of our foundational structures that we actually have to have to ultimately end up with an accelerated approval process is we have to work with the FDA on getting feedback and guidance around, one, there being an unmet need in Duchenne muscular dystrophy. That's going to set the main foundation. The second thing, the second piece that you have got to have, is you've got to have a unique program. Because of this nNOS binding domain, recruiting for R16, R17, recruiting for nNOS, recruiting for alpha-syntrophin, recruiting for cavin-4, we feel that hopefully we'll be able to qualify for both, one, a high unmet need in Duchenne, second, a novel protein, and that's why I go over it so much.
What also makes this program unique is this capsid. This capsid is called POLARIS-101. It used to be called SLB-101. By the way, we named it POLARIS-101 because with multiple capsids coming down the pike. Before this presentation started, Gil and I were chatting about how many capsids we have at the end of the year, and many of which look like from a skeletal muscle and cardiac or liver de-targeting, even superior to POLARIS-101. We'll have a whole host of capsids coming out at the end of the year that we're going to provide to other companies through licenses, and we'll also create a 3rd-generation Duchenne program eventually, so we can continue to hopefully increase our market share in this space over time. POLARIS-101, it is a rationally designed capsid.
It is designed to have increased binding capacity to both skeletal and cardiac muscle. We have RGD peptides that are scattered around the surface of the capsid for these integrin receptors. This is why it binds so quick, transduces so high, and actually produces microdystrophin protein very, very quick in animal models up to basically two days and four days. We also noticed that it is unique from clearance because of this capsid. It clears out of the body within four days, from the blood out of four days, in our patients that we are dosing right now, all due to this unique structure. It provides beautiful data as well. As you can see here, I'm not going to go through every bit of the numbers here.
I think what's most important is when you look at Western blot, mass spec, positive fibers, or when you look at beta-sarc as well as nNOS activity, look how consistent the numbers really are. From the Western blot, mass spec, immunofluorescence, or beta-sarc, those are all either assays. They're for protein or positive fibers. The nNOS is an activity assay, so it's not an assay measuring protein, it's actually measuring the activity of the sarcolemma. Because of all of this, we feel that this is going to be one of the most comprehensive data sets that have been put together, because we can fundamentally look at from transduction all the way down to expression to nNOS activity, and the numbers are relatively high, some of the highest, if not the highest in Duchenne.
We don't really stop there, because what we wanted to make sure that we felt as we're dosing these little boys, that once you dose, transduce, and express, that you can take it to the next level and actually see the muscles shoring up from a muscle integrity standpoint. Realistically, the first four columns here ultimately lead to column five, and so when you're looking at membrane stability, sarcomere preservation, reduced tissue injury, reduced cellular damage, those four we can use biomarkers such as left ventricular ejection fraction. We can look at troponin, lactate dehydrogenase, titin, CK, ALT, AST, and that gives you a very good picture of the muscle shoring up.
That leads to the last one, which is this satellite cell preservation and muscle fiber stability, and ultimately that's going to lead to the muscle fibers maturing, and hopefully that's going to lead to clinical benefit. When we look at the first four, we see very good, robust responses across the board. Once again, whether you're looking at membrane stability, sarcomere preservation, reduced tissue injury, or reduced cellular damage, each one of these biological correlates come back to that. That leads to this embryonic myosin heavy chain. The reduction of embryonic myosin heavy chain of 44% tells you one thing. We have slowed down this degeneration, regeneration process that you're seeing happen in Duchenne. That's what happens over and over and over. The satellite cells, the muscle fiber is damaged, the satellite cells start to proliferate.
You see embryonic myosin heavy chain scattered throughout the muscle fibers, which you see at the top middle part of the slide. Ultimately, this is a futile development because the muscle is going to continue to degenerate. Satellite cells, the pool's going to eventually deplete, and that's going to lead to kids ultimately off their feet when their fibrosis score fat fraction's at 80%. This measure right here tells you everything. It tells you it's slowing down the degeneration of that muscle, and so we're very happy to see it. We also take a look at cardiac function over time. Now, the naysayers are going to say you do not have cardiac dysfunction at ages five, six, seven. Roughly correct, but that's not ultimately true.
The majority of kids are going to have normal left ventricular ejection fraction, but there's always subset kids that are more severe. I can tell you one thing that's 100% factual. These kids, all these boys in Duchenne, don't just wake up at age 10 or 12 and then overnight end up with ejection fraction in the 50% or cardiomyopathy diagnosis. It happens over time, and it's a drift, and the physicians are actually, unfortunately, used to this drift now. They see the patients, they see ejection fraction at 65. That's the normal range for these little boys at this age. The next time they see him at 63, they don't really think anything about it, 61 the following, and then ultimately end up in the 50s. The parents are shocked when they ultimately get a diagnosis of cardiomyopathy and ejection fraction in the 50s.
It didn't happen overnight. It happens over time. You have different kids at different stages. What we're trying to do is keep the normal kids, the kids that are in the normal range, stable for longer, and any child that is in the lower to low normal range, help them increase back into normality. That's exactly what we show you here. If you, on the left-hand side, all our boys, all 39, if you start normal, you stay normal. For the little boys that are one standard deviation below what they should be, well, we can actually, 12 of 12, there was 12 boys, so we can bring them right back to a normal range. Now, there's a lot of talk about the importance of liver health.
I think this is why a lot of people are using sirolimus, eculizumab, steroids for longer, all thinking about liver health. These are all our little boys that have gone out 90 days. Actually, just to let everyone know, we've dosed in almost to mid-40s. It's like 44 children as we sit here today. I think when we presented this data at the MDA conference, it was roughly 39 kids. We're at 44 right now. The safety profile has stayed the same. We do not have any kids that have drug-induced liver injury. We do not have any kids that have myocarditis, any kids that have aHUS or TMA. If you ask about the little boys that are not included in this, because this is end of 2024, this was all the boys that have made it out to 90 days at the MDA conference.
If you ask about the other boys, they're going to follow the same pattern. You can actually see that we do have the whiskers in here, GGT, you can barely see them, but they're there. This is what a good liver function looks like. I think it's very important. Take a look at ALT and AST. I don't know of many companies that dose gene therapy that can look at ALT, AST as a skeletal muscle biomarker. For us, we can because we see a very quick decline of ALT and AST, and that's caused by the skeletal muscle breakdown. Because this capsid was liver de-targeting both the animals, mouse as well as monkey, we believe it's liver de-targeting here in the human as well, and this is why we're seeing this decline. Overall, our safety profile looks very good. It's staying very consistent.
As I mentioned, we've dosed now 44 patients, and they all fall within this range, and we don't have any new AE to tell you about. It's extremely important for everybody to realize that when we dose up on our steroids, we start tapering at day 30. We taper faster than any other company in the gene therapy space that I know of. We start tapering at day 30, and by day 60, they're back down to normal steroids. That's critically important when you think about, especially when you think about your day 90 data points. You have to question, if you're still on long-terms or high-dose steroids at day 90, you have to ask yourself, are you seeing a steroid effect, or are you seeing the real drug effect? All our little boys are down to baseline by day 60.
By day 90, you can feel very comfortable that the data that we're presenting is data from the drug. This is the overall picture here. It really does not matter what you look at. I think one of the great things about the drug is directionally, it's all consistent. That's what I'm the most proud of, is that we don't have to talk about one biomarker and then sort of avoid another. They're all very linear in process and in thought here. It makes me have a lot of confidence in the drug. Now, this is one of our first little boys we dosed. We have a video here. I'm not allowed to give his age because it de-identifies the young man. This is day 360 and baseline.
I'll show you the video here, and this is part of our clinical trial, and I'll talk a little bit about that in the next video. Take a look at his feet, and you can actually see it's called a Stride Velocity 95. There on the feet. We're monitoring everything, all his movements. On the left-hand side is clearly baseline. On the right-hand side is one year post-dose. It's the same vehicle that his parents take the video in. You can see that he gets into the car with a lot more ease after one year of treatment. We track all these videos, and we'll have more going forward. Now, this little boy, once again, I can't give his age. This is six months post-dose, and he's going to go up 12 stairs on the left-hand side.
As he goes up these 12 stairs on the left-hand side, you can watch. It takes 14 seconds to go up the stairs. He has to literally put his hand on the knee on 11 of the 12 stairs. He has to hold on to the side with the other hand. This is typical in a boy with Duchenne. It takes a long time for him to get up. The next one is 20 steps, and he gets up these 20 stairs in 10 seconds. He doesn't touch his knee once. He doesn't touch the side of the rail a single time. He really, if you could actually hear the video after he runs up the stairs, he says to his dad, "Dad, I ran up the stairs." It was really quite cute.
Now, the great thing about these videos, they're all part of our clinical trial. They're incorporated in our plans. The FDA knows that we're going to be submitting videos. We use two different vendors. We have one vendor that does all the videos inside the clinic. As they're doing baseline and day 90, day six months, day 360, a four-stair climb, et cetera, they actually get that monitored. I have another vendor that does these home videos. The parents, they sign waivers. They take videos of daily activity. That's why one was getting in the car. This one is getting in the stairs. They video baseline, and then they video at certain time points, six months, nine months, 12 months, et cetera.
All of these videos go to a third party, and this third party does all the analysis, looking at walking and the gait, how they use their hands, how they compensate, and that is going to get submitted to the FDA. We're very excited. While you have some of these videos that are overwhelming, you have others where the gait changes, how they have to hold themselves change. All of that is going to be important to these families and these children. This is a little boy. This is the one that actually the physicians love the most. Why? Because you don't have stomach muscles. Unlike most people, not myself, but unlike most people, they have stomach muscles. Duchenne kids, they actually don't have core muscle, and definitely in the neck and head region. To be able to do headstands like this is really exciting.
Now, for 212, I'll talk a little bit about 212. I'm really getting more and more excited about this program as well. We're the only company that I know of in this space that does this dual route administration straight to the dentate nucleus, as well as to the heart and the spinal column. Because we're able to dose this elegant way, this dual route administration, we're able to lower the doses pretty low. In the brain, the total dose is going to be in the E9 range. That's total dose. It's not a vg/kg type scenario. Its total dose is E9. In the heart, it's a E12. That's dual route, so it's spinal column as well as the heart. You're talking vg/kg, and that's in the E12 range. Now, keep in mind, very different than Duchenne. Here, normal, without a phenotype, you might have 50% protein.
You could be walking around without a phenotype of FA, a carrier at 50%. Realistically, you're just trying to get the protein of that individual patient up closer to that 45%-50% range. You want to get to the heart of the matter, which is the dentate nucleus of the cerebellum. Because the dentate is going to send commands and info to the motor and premotor areas of the forebrain, and it regulates voluntary movement planning as well as initiation and control, alongside cognitive and your visuospatial functions. It's extremely important for reading, speech, swallowing, coughing, et cetera. Now we wanted to get to 15%-20% of the dentate nucleus. This is patient number one. You can see on the left-hand side, we sort of showed you where the dentate nucleus is, and we want to get to 15%-20%.
We were able to get to a great deal more than 15%-20%. We're very excited about this. We've already dosed our second patient, by the way. Both patients are doing great as of today. This is actually the adverse event profile for patient one. We haven't updated it for patient two because we only dosed him a little bit ago. We need a little bit longer before we update this table on patient two. Realistically, as we sit here today, this table is going to look the same with an N of two, and so we're very excited about it. With that, I will stop the presentation and turn it over to Gil, and I'll be glad to take any questions.
Well, Bo, thank you for the presentation. Really hard to go by my normal order. I'm going to have to go with the video. It's a little too notable. I'm going to go back in a capsule back in time to early days of gene therapy here. We all remember the 2018 presentation from Sarepta. Very exciting data from those first four patients. One thing that kind of came up in discussions over the years, and this is my question, patients don't regain function, right? That was the main takeaway, and I'd like you to maybe touch on that decisively, especially because of the two videos we showed.
Yeah. That's the unfortunate thing about this disease. I was asked earlier, obviously I'm doing one-on-ones at your conference here, and I was asked earlier about can we hope for a day with these type of therapies to regain function, especially in the older children? Unfortunately, the answer is no. Once you lose muscle, and it happens relatively quick, these drugs are not going to regain, rebuild muscle, which is, by the way, critically important of why embryonic myosin heavy chain is critically important, because this is where muscle regeneration is going to happen from the satellite cell proliferation. What we have to do is we have to, unfortunately, get to the kids as early as possible, try to slow down the disease progression. You can actually even see in our data that we can only shut down basically this sort of degeneration, regeneration process roughly 50%.
44% was what I showed on embryonic myosin heavy chain. You're not going to be able to regain. What you can hope you can do is you can slow down the progression, and maybe halt it in certain areas, like the heart, you can hope because of the fact they won't turn over as much. Other than that, none of us can claim that they should regain function. FA, you might be able to, ironically. FA, you might be able to because of just the nature of the disease and the CNS. FA's going to act a little bit different than Duchenne.
This is also an old question that I'm repurposing here. If patients feel better and have higher function.
Yeah
Doesn't that necessarily lead to more muscle damage? They're more active.
Yeah. It's a great question. This is like you can see CK bounce. As the kids feel better, they run around, CK will bounce. This is why you hope that you need a protein that's very flexible in the muscle that as the kids are moving and running around, jumping, and the muscles are contracting, that the protein is able to flex with the muscle. Because we're taking a very long protein and we're shrinking it down into a micro protein, is it flexible enough to move with the muscle contraction instead of breaking and losing it? Because once you've lost it, you're going to lose it forever because these are not going to really be in the satellite or stem cells for multiple rounds of regeneration.
Yeah. You probably remember this too, but there were a few cases of rhabdo.
Yeah.
Is that also a risk in this situation?
Yeah, it could've been, but it's probably part of the myositis type cases. We had one case of myositis that we didn't know about a hotspot, and it was a very large deletion. This was acknowledged, I guess, sometime last year. We had one, it was deletion 51 through 70 something. It was a very large deletion. We've removed that, so we've not had any cases like that. We've not had any rhabdo. We got very lucky with that case because it was basically his lips swole up, and his eyelids swole up, and he resolved on steroids. I think other cases have had rhabdo, and I think most of them come from myositis type of scenarios. We've tried really hard to avoid them by carving out the hotspots. This last one was a hotspot that no one knew about.
It's now in the label, I believe, for the commercial approved drug, and now it's excluded in ours. I'm not real sure about other programs, if they've excluded that mutation. We've made it known so people can exclude it.
Right. Now circling back to the clinical data that you provided. Very comprehensive biomarker picture. Any guesses why no one else does this?
No. I don't have any guesses, and I don't want to guess. I will tell you that we thought it was critically important because we're sitting here investing the investors' money, and as operators, we try our best to make sure that we're putting the money to a program that's going to hit multiple inflection points and hopefully return on investment, and we can help kids in the meantime. When we put together this biomarker panel, we were a $38 million company, so ironically, and we had to make sure that every dollar went to the best place. What we wanted to feel is we wanted to feel like there was a lot of confidence that the drug was shoring up muscle. We knew that you would see transduction, you would see expression.
You didn't know exactly how much in the time back in the day, but that's only the first part of the story. Ultimately, you guys and everyone, including the families, want clinical benefit, and you're not going to be able to see clinical benefit at day 90. What can you do? You can actually look at the muscle integrity. You can look at this membrane stability and sarcomere preservation, and that's going to give you a lot of confidence that from transduction to expression to the dystrophin complex coming together to ultimately muscle integrity, the muscle shoring up, and then satellite cell preservation. I have a lot of confidence that this is doing something in the human body, and I know my heart of heart, I know that this is going to provide clinical benefit.
Now it's about how big of a benefit, how long you'll wait, when will you see this benefit? That's the next step, and that's probably going to be your next question.
I do want to spend a second on something that at least we consider very important, and that's the consistency of expression across individuals dosed. You guys show about 60%, and if you notice, the error bars are a lot smaller than some of your compatriots in the space. It's a two-sided question. Why do you think you are getting more consistent expression? Maybe conversely, why are they not?
I think gene therapy in the future is delivery. It's all delivery. Delivery comes in multiple sort of flavors. It's your capsids, it's your promoters, it's your purity of manufacturing. I talk about this as like a F1, Formula One racing car. It's not one aspect in gene therapy that's important. It's like a hundred little things, from the engine to the tires, to the team, to the airflow. It's the same way here in gene therapy. It's the capsids. What integrin receptor are you binding to? How pure is your medication? What's your promoter look like? All these little aspects add that sort of incremental change. Instead of swinging for the fence here and waiting for one capsid that's 15x, 20x better, we believe that this incremental progress in each process and each standpoint is what makes the difference.
I fundamentally believe that that's what we're seeing here. The capsid has these integrin receptors, these RGD peptides for integrin receptors. We have a promoter that's specialized for muscle. We have high purity for manufacturing at 75% full-to-empty ratio. That matters not only for safety but also efficacy, and you don't realize it until it's too late. Let's say you're dosing 1 E14, and it's 50% full. Really half of your capsids are going to bind to receptors, and they're going to have no active cargo, and they're going to block the full capsids if the empties get there first for that receptor. All of this makes a difference. I think all of this is why this program is next generation.
The capsid makes it the next-generation, and then the delivery of manufacturing and the cargo, and then the uniqueness of the protein even add on top of it. I think that's a long-winded way of saying multiple reasons on why the error bars seem to be tighter.
Digging in a little on the safety side, there are some reports of cytopenias. Any commentary around there?
I'm sorry. You broke up for half a second.
Cytopenias.
No, we haven't seen anything. Everything is very stable from the safety profile. Our safety profile after we adjusted our steroid regimen, and how we adjusted our steroid regimen was we moved the steroids back three days. We used to dose all our increased steroids at day of, and once we moved our steroids back, our safety profile's been shoring up and getting better as we go along. What we're seeing now is very consistent, and we aren't seeing anything. I think, ironically, safety is going to be the one thing that really leads us to probably the highest market share out of the group. I talk to all the physicians, and I can list off 10 different scientific reasons of why I believe that this is the best program to the physicians.
Ironically, when they tell me, they were like, "Ease of use is going to take the market share." These ladies and gentlemen are dealing with a lot of things here. They dose SMA, they treat SMA, they treat limb-girdles, FSHD, Charcot-Marie-Tooth, and then they get to Duchenne, and it's just very challenging. You have to now use sirolimus or eculizumab. They have to be on vaccines. They have to be on antibiotics for an extended period of time because the kids are going back to school. They have to stay around the hospital for a while, depending on which therapy they're being dosed. Here, it's an outpatient setting, two hours. They dose, they go home. They're on steroids only, and then they taper off at day 30.
When I talk to the KOLs, it's ease of use that's going to end up being the main driver for this drug.
At what point do you think we can start getting excited about the cardiac data, like as it relates to follow-up?
I'm excited now, but I know the data's early. All right. I think it's very important to understand that we're tracking it from day one because we know long-term, and when I mean long-term, 10 years, it can be a long time before you start seeing mortality and actually in the group that we're dosing. What you want to do is you want to track it from day one. Our finding is very unique. We didn't realize that we had 12 of 39 boys that we looked at had at least one standard deviation lower. We use echoes, and every one of the kids went right back to normal.
What we've done as a company is now we're working with a hospital, and we're taking all our echoes, and we've blinded them, and we're going to scramble them and reread them and do a mini trial with the patients that we already have dosed. I think over time, as this data matures, if that data comes out and it says, yeah, the reread and the blinding and everything, the scrambling, shows the same results, and then as we follow these kids, if they stay normal for longer and/or if they're one standard deviation low, and they all go back to normal as they did already, I'll start getting pretty excited. There's mechanisms of why, from a scientific standpoint of why our drug is acting different. This cavin-4 recruitment, it's a structural component and signal regulator for cardiac care.
nNOS in combination with high levels of beta sarc and delta sarc are critically important. You can go back and look at a patient that has full-length dystrophin. They're a limb-girdle, and they have full-length dystrophin, but they're missing beta sarc or delta sarc. Look at the importance of those proteins for cardiomyopathy, and ultimately heart failure. Very important. You tie that to nNOS and this cavin-4, and all of a sudden you have something. More to come, Gil. I'm not banging my chest yet on cardiac, but we're tracking every bit of it.
That is very helpful. As it relates to how investors are assessing the data, when do you think you'll have a more comprehensive conversation around the functional results?
Yeah. Yes. Hopefully, late this summer, we're going to be analyzing. We haven't analyzed the data yet. All right. Everybody can feel very confident that I have no functional data that I've seen personally. We have to work with the FDA, make sure that the stat plan that's already been submitted months and months and months ago, that they acknowledge it, and then we can start analyzing the data, and we'll submit it to them over late summer. That data, we'll have a discussion with the FDA about the data and compare it to natural history and look and see if there's directional functional benefit tied to some of the biomarkers that we have that I showed you today. If that's the case, then hopefully we can get some guidance and some feedback from the FDA on a path forward for accelerated approval.
If that's the case, then we'll share all the data until the FDA really says, "Okay, you've got enough data," because we do have an avalanche of data that's going to be coming in. We've dosed 44 kids, and that data's just going to mature. As we present that to the FDA, and they sort of say, "Okay, you have a decent package now," I'll share it with everybody. I just want to treat the FDA with the respect that they deserve, and right now, we're in decent graces with them. We have a very cordial professional relationship with them, and I would like to keep it that way.
Well, I could absolutely spend another hour on Duchenne, but I do want to spend a second on ataxia, especially because you've provided an interesting update here. How do you think this is going to play out, your dual administration? We know Lexeo really focused on the cardiac side of things. We know other companies focused on the neurological. Are we looking at, like, a composite?
Yeah. We'll look for mFARS, and mFARS is really broken up into four major categories. You have the bulbar function that is a 0-5 rating on mFARS. You have your upper limb function, that's going to be 0-36. You have your lower limb function, that's going to be 0-16. You have upright stability, and that's 0-36. mFARS is broken out into four categories. Because we're attacking or going after the CNS manifestations of the disease, we're going to be able to use mFARS, and we're going to be able to look at these different functions, milestones that people can hit. Now, we're dosing really, unfortunately, very severe individuals. If we see a benefit out of these patients, it's a miracle for them, for the family. Put it in perspective. It's unlike NSAA.
Remember, NSAA, 33, 34, those are the best numbers you can get. That's what you want in Duchenne. A 93 in FA is the worst. It's the worst of the worst, so you can't get any more severe than 93. The patients that we're dosing are in the mid to high 80s. Really sick. They can't move, they can't really read, they can't swallow, they can't speak. They have a hard time coughing. These are really severe patients. I can tell you that we're really excited about what we're seeing in patient one, and I've said this publicly. He's reading again for the first time in years, and he's able to speak even though it's very, very mumbled. Before you couldn't even comprehend anything, and now he's speaking again, and he's starting little movements in his hand.
When you asked a question about can you regain anything in Duchenne, I said no, but FA is surprising us already. We've dosed our second patient in FA. It's a little too early to say he's in the clear, but as of right now, and he's a couple weeks in, he's doing great. No AEs from the drug as we sit here today. These are really sick people. They have a lot of arrhythmias. They're pretty severe. Hopefully this drug changes their life. I think this could be an AveXis type drug for the FA community. I don't mean mortality in any way, but I mean the AveXis program really changed the way we think about SMA.
I think this program has that ability to change the way we think about Friedreich's ataxia and how to treat it, and I hope that it plays out that way. We're going to find out over the next couple of months. Our game plan is to dose five or so patients in the next couple of months, and then have a readout for you at the end of the year to JPMorgan-ish type time frame.
Excellent. We have maybe one more minute here, if there's anything you'd like to highlight for our investors.
I want to thank you for all your support and as well as Needham. I would like just to say that we've come a long way in three years. Three years ago, this company was a preclinical company only. As we sit here today, I think we have the next generation, the best Duchenne drug that's in gene therapy that's hopefully going to make its way to market over the next year to couple years. We have a program that's so unique in FA, that I hope it changes the disease state personally forever. We have multiple cardiac programs. We are trying to go a little slower because it's hard with a small company to juggle more than two programs, but we have multiple cardiac programs. We just got our first cardiac program coming out of the Mayo Clinic. That's a huge program for us.
We have all these capsids. I was telling you, Gil, before, we have capsids that'll be coming out at the end of the year that are 3rd-generation, 4th-generation type capsids, and we're going to give them to all the small companies to hopefully make gene therapy investable again for not just Solid, but for multiple disease states that we're not going to go into. I'm really grateful for every investor that has supported us and got us here because we've come a long way in three years. Thank you.
Thank you, Bo.
Thank you.