Okay, great. Good morning, everybody. Welcome to day two of Cantor's Virtual Muscular Dystrophy Symposium. Really excited to be hosting REGENXBIO this morning, very timely for them as they just announced some data for Duchenne. So from the team, I'm joined by Ken Mills, the President and CEO, and Dr. Olivier Danos, the CSO. Thank you both for joining us today.
Thanks, Kristin, for having us.
Thank you.
Yeah. So as part of this symposium, we've asked every company to focus on a theme, and for the REGENXBIO team, that is, what evidence supports the key elements required for building transgenes to truly differentiate. So maybe to start, we all know REGENXBIO as an early gene therapy pioneer. You've even seen your technology platform utilized in a commercial setting. So maybe can you take us back to Gene Therapy 101 and really help us understand the important elements that are required to build a gene therapy?
Want to start with that?
Yeah, I can start with that. So in general, before we focus on DMD, gene therapy is about reprogramming cells, you know, with a therapeutic goal, right? So how do you do that? For that, you need a modality that allows you to reprogram the cell, and that can be. Today, there are, we have access to many ways of doing that. You know, the old way is just to express a new protein, so you, one way or the other, you add to the cell genome new information that codes for a protein, and this protein, for instance, compensates the absence of its absence in a genetic disease, or it has a therapeutic property of its own.
You can also use as a modality something that will act on mRNA and remove the mRNA, like, you know, anti-sense, siRNAs, or modify the mRNA one way or the other. You can also, obviously, get into the cell, anything you need for gene editing, right? And that's another modality of modifications of the genome and gene therapy. So if you're listening to me, I mean, I've said several times, get into the cell. So how do you do that, right? You have all these modalities. You can do all these beautiful things very precisely on the genome. You can do it basically whatever you want, right? How do you bring all this into a cell? And this is where you need the famous vectors.
So what we call vectors are, you know, little engines that are made to carry genetic information into the cell and maintain it into the cell. So, you know, to make a long story short, it's been 40 years of development of vectors, and, basically, people have been guided through this development by viruses because viruses are precisely doing that. They're invading the cell. They're expressing new genetic material, their own. It's not to cure the cell in this case. It's more to destroy it. But they do that, right? We can modify viruses. We can make them harmless, remove all the bad stuff, and keep all the properties that allow them to get into the cell and express new genetic material.
So at REGENXBIO, we've been focusing on one particular type of virus, that's called adeno-associated viruses. They're kind of cryptic viruses. They're very small viruses, that haven't been associated to any disease in humans, but they're everywhere. They're there. And in a weird way, they are parasites of other viruses. They're not even themselves real viruses. They tag along other viruses to invade the cell, but they have everything to get in, right? And so we're using all that to develop our vectors. And those AAV adeno-associated vectors are today probably the most used, the most popular in gene therapy. And we've, you know, we've advanced several of those viruses, us and our licensees, to the clinic and to the market.
Well, there are now products, derived gene therapy products, that are from AAV, and we're, you know, currently developing our own in ophthalmology, and as we'll discuss today in Duchenne muscular dystrophy.
Okay, thank you for that. So as we know, dystrophin is one of the largest known human genes, which puts it at a bit of a disadvantage as it won't fit in the AAV capsid. So why theoretically should a microdystrophin be able to still work?
Okay, so, you know, basically, we've been learning from nature. We've been learning from observing patients with different forms of muscular dystrophy, more or less severe forms, and looking at the kind of mutations they have. Some of these patients that have a mild type of disease called Becker Muscular Dystrophy actually have shorter dystrophins. That's the problem they have. They have a dystrophin which have, you know, pieces are missing. Therefore came the notion that, you know, you could probably remove parts of the dystrophin and still conserve most of the functions. I'm saying most because, obviously, this is not a truly fully functional dystrophin. But, you know, it allows this patient to walk and enjoy a happy life until the age of 60-65.
After that, they need a cane, and they're getting, you know, weakness much faster than other people. But still, you know, it's nothing compared to the real Duchenne, where boys start to have symptoms, you know, after the age of 3 or 4, and it's devastating. I mean, I'm not going to get into the whole description, but it's very severe, right? Anyway, nature tells us that there are forms of dystrophin which are deleted, and we can probably make more compact proteins that would fit into our AAV vectors, and that's what we – when I say we, this is REGENXBIO, but it's also the whole field. I mean, it's been, again, 25, 30 years of research around that.
Then, as you know, many other companies are developing microdystrophins, which are, you know, there are different ways of doing that, and that's what we're going to discuss, I guess.
Great. Thank you for that. From a high level, we know the role of naturally occurring dystrophin. But what work did REGENXBIO do to really understand the different parts of this, to understand what's going to be most critical for driving most of the effect in healthy humans?
Yeah. You know what? We don't fully understand exactly the role of dystrophin, and that's I mean, I don't want to sound too negative, but there are a lot of things that are not fully understood, right? And again, we go step by step, and some of what we're doing is empirical. We have models where we can measure the function, some of the function of dystrophin. What we know about dystrophin is that this is a molecule. It's a big protein. And as all these big proteins, it's made of an assembly of different domains, right? So it's the little parts of the proteins are they're linked together in a larger molecule, and each of these domains have different functions.
In dystrophin, there are two very important domains at each end of the molecule. The first one at the so-called N-terminal domain, the first domain that you find in the protein is the one that binds what's called the cytoskeleton. And the cytoskeleton, in a cell, it would basically hold the cell together, and it has very important mechanical properties. In a muscle cell, the cytoskeleton is what contracts, basically, the myosin filaments slide over each other, and it's a complicated process, but it makes the muscle contract. Dystrophin binds this, right? So dystrophin feels and knows when the cytoskeleton contracts. Dystrophin also binds the membrane of the cell, right? The envelope, the outside envelope of the cell. And it binds it through a protein, a pretty complicated protein complex that's called the dystrophin-associated protein complex.
And it binds this through its C-terminal domain, right? At the other extremity of the protein, there is a domain that links dystrophin to the membrane. So dystrophin senses what's happening with cytoskeleton and relays that to the membrane. When the muscle cell contracts, what's happening is that a huge amount of stress is being generated at the membrane level, right? And what happens is that constantly you have tears and destruction locally of this membrane. And there's a whole process, a natural process that makes all this get repaired constantly. But this is complicated. It needs to be coordinated. It needs to be checked and held in place. So all this complex plays a very important role in repairing the membrane.
So when you don't have any dystrophin, you don't have this constant check about lesion of the membrane, and you keep not repairing the membrane the way you should, and then it becomes a catastrophic thing for the cell, and the cell dies. So you have those two domains which are very important for linking dystrophin, and in between, you have something that links them. And in the protein, this is very long, and it has properties. This is where we're getting into the kind of the unknown. Exactly how is it structured? How does it relay the information? The whole biophysics of it is not exactly worked out, but we have good ideas, although so this is where you have a lot of things.
It's a repeat structure, and we know we can remove a lot of these repeats. So this is where you can shrink the protein and make mini or micro proteins that are still active. They still link the cytoskeleton to the membrane. They still do that. But the way they transmit the signal might be a little altered, right? So they work. They may work a little differently. And that's where we need to test them. That's where we need to get into models, cellular models, animal models. And that's the work that we've done in developing our own microdystrophin. Sorry, I've been a little long, but it's actually a long story.
Christine, does it sound like there's not much known about dystrophin after a little bit about it? I have to say, though, that you know since Olivier's come to Regenx and built a team and colleagues that have been involved in really the you know the industrialization, I think, of microdystrophin into AAV. The benefit has been not only of his knowledge and growth and understanding, but just we've been doing it in a more modern day, you know, just in the last several years, and our observations and commitment to microdystrophin improvements, including focusing on adding elements and domains that weren't in preexisting you know, or existing now therapies as well as reagents and constructs.
Came from the fact that it did seem like there was this huge gap that there had been like 10 years or more, maybe 15, where people were working with the orthodoxy of what was understood about dystrophin, you know, in academic labs years ago. And a lot had been, you know, grown and developed since then that we thought could be applied. So, you know, RGX-202. While Olivier may, you know, posit that there's still more to understand about dystrophin, I think it really is standing on a more modern understanding of features of dystrophin. And the challenge and the exciting challenge was, how do we get more of what's understood into a compelling product candidate?
Yeah, that's very true. So again, you know, we know a lot. We don't have a 100% coherent knowledge, but that's true of anything. So let me not get into that. But so, it's just that in the long history of engineering microdystrophin and mini-dystrophins, people have started, you know, in a pretty brutal way. They said, well, you know, we can let's reduce the size. Let's chop it off, and that's it, right? And doing that, they've done. They stumbled on structures that were, you know, not good, actually, and they discarded it. And some of them, you know, kind of work, and let's move along with them. Now what we've been doing for the past, you know, 10 years or so.
And that includes a lot of our work was to, you know, try to be more precise. You know, how do we make sure that those little domains are correctly phased together? How do we make sure that we don't exclude information that, you know, we thought we're not important, but actually is probably important? And that's where I come to, you know, the piece that we've added in our microdystrophin that distinguishes this from others, what we call the C-terminal domain, which is an added domain at the end that we believe does a better anchoring into the membrane and a better aggregation of this complex that repairs the membrane. So that's where and that's what Ken was saying. So this is a more learned approach, and we benefit from, you know, 25 years of work.
We come a little bit after the early developers of microdystrophin in the clinic. But I think we benefit from a lot of knowledge, and we've, you know, incorporated that into our construct for sure.
Yeah, absolutely. It's sometimes not best to be first so that we can really learn from others. And you know, even though you had a very detailed response, I still think dystrophin is a bit complex. So you're right. The more we can gather and learn, the better. So thank you for that background. And I would love to talk a little bit more about what differentiates you. But first, can you tell us what parts of the transgene that you and your peers all include in the constructs before we talk about what differentiates you?
Yeah. So I mean, basically, I mentioned it. It's about you need to have the N-terminal domain that binds actin, what's called the actin binding domain. You need to have a first region called H1 that just after this and that that's it all microdystrophins. And then you need to have the distal part, the a piece of the C-terminal called the cysteine-rich region with another hinge called H4. So ABD H1 H4 cysteine-rich is what everybody has. And in between what's in between varies a lot. And so we have our own blend of what's in between. And we add a larger C-terminal domain, a larger piece that connects to the membrane. That's how we do it.
Okay. And to get to the fun part about what differentiates you. I think, you know, on one end, whenever you introduce a couple new variables, you sometimes question if one of them in particular is driving some of the greater benefits or, you know, if they kind of all work in concert to do this. So when we think about REGENXBIO, you have a different AAV serotype. You have a different promoter. And of course, the most distinguishable attribute, perhaps, is that functional element of the C-terminal domain. So thinking about that question, I guess, you know, do you believe it's one thing in particular that may be driving your effects or again, are the new variables that you introduce relative to your peers kind of working in concert to do this?
Well, I think there's a strong rationale for the C-terminal, but it's difficult to tease out the effect of the C-terminal from all the other things that you mentioned. We're using another capsid. We're using another promoter. We're actually very importantly something we haven't talked about is that our transgene is encoded by a DNA sequence where we excluded the famous CpG dinucleotides, right? So those are sequences which are present in that are recognized by the cell as foreign DNA, right? And I think that's probably a key element.
Those are things for which we, you know, we do have data in preclinical showing that could be part of the safety of our vector. So if you have a better safety, then you have a better expression of your protein. You have a better transduction. You know, just this alone can have consequences in terms of efficacy at a clinical level. If in addition, you have a microdystrophin, which is more potent because it has, let's say, the C-terminal, then, you know, it's a double win. That's the way we're looking at it. But having said that, I agree. We will never make the experiment where we'll just change one variable and test that on patients and another variable and test that on patients.
You know, we take a chance of putting them all together and hoping for the best.
But I think, you know, my observation was of the science that was done. Again, it was a very rigorous and industrialized process, you know. So while there were a lot of variables and even more variables at deeper levels where the team was looking at, you know, genomic stability and how that was going to communicate with stability of translated protein and things, frankly, that you know, I just think again are more modern for the field of AAV gene therapy, even when they were, you know, in the origins of like ZOLGENSMA, for instance, Christine, you know, I mean, these are things now that I think have become really sophisticated because the drug development at REGENXBIO is so much more learning. You know, it's from clinical experience and from preclinical and research experience.
It's sort of intertwined. So I mean, literally the goal was certainly to lean heavily on, you know, the C-terminal domain as a significant differentiator, but leave no stone unturned in terms of stabilizing and, you know, optimizing expression of that C-terminal domain based microdystrophin. And you know, I mean, so far we've been really encouraged about how that's translated clinically. But it was based on a ton of work preclinically and even it had meaningful contributions to yields in CMC, for instance, you know, which I think is a you know, the product is the process here, right?
Okay, thank you for that. So yeah, let's get to the data. You've had four patients. They were at different age ranges. And across the board, you showed robust microdystrophin levels in addition to CK level reductions. And look, it's obviously very difficult to make cross-trial comparisons, but how do you believe that these data demonstrate your differentiated construct build is essentially what's been able to drive some of these effects?
Yeah, I mean, we've been incredibly pleased and enthusiastic about this early data. But because it does stand on top of, you know, a few other data sets, including one based on a, you know, accelerated approval product where a lot of data has been shown. You know, we're okay making some comparisons, you know, particularly because, you know, some of the ideas we had about designing, you know, improvements into the construct would mean that it could be intended for a broader range of boys with, you know, different things going on in Duchenne. It might be different mutations. It might be, you know, where they are in the progression of the disease. So, you know, like others. And I think even more so than others, we embraced a really broad enrollment criteria in our first-in-human study.
Because we had the confidence that the C-terminal domain and these other improvements were likely to show things based on what we saw in animals in boys where in other cases things hadn't been shown. So, you know, on an age basis in particular, Christine, for us right now, both the first result at dose level 1 as well as the more recent result at dose level 2 in an age range of 8-11, which has largely been unexplored or left behind by Sarepta and Pfizer in terms of either pivotal designs or the ability to share more data transparently or otherwise has been a major focus area for us in terms of prioritizing enrollment. We showed dose response. We showed consistent levels of microdystrophin expression, and we showed a very high level of microdystrophin expression at dose level 2.
And, you know, we were even able to, you know, with an investigator presentation at the MDA, you know, show some evidence of, you know, early anecdotal improvements and stabilization on functional assessments that we'll be doing throughout the studies. At the same time, we hit the mark in the, you know, the age ranges, if you will, that are more familiar to all of us in terms of larger data sets. I mean, we're entirely comparable. And I think I have the opportunity to continue to show, you know, the effects of the improvement of the design of RGX-202 with the C-terminus and otherwise. So, you know, so far we continue to be really enthusiastic about the translatability of what we intended, what we could show in the animals.
The initial evidence that we have here, while early, you know, as encouraging as I think, you know, we and others could have expected in the experience here with an improved microdystrophin.
Yeah, and I think your comments about the age group being left out are probably something that's really underappreciated. And you know, we've been having a number of conversations with KOLs this week. And you know, I think you can make the argument for a lot of indications that the later you treat, the harder chance of success. But you know, one might argue for DMD that that's an even tougher ask. So I think it's, you know, a good point you bring up. So then can you talk about why it's essential for the Microdystrophin to be?
I think for the reason I gave before, this is really a protein that acts at the membrane when it's linked to the membrane, what's called the sarcolemma in a muscle cell. So it has to be there inside the cell, but linked to the membrane. So if it's floating around somewhere else in the cell, it just doesn't have any purpose. So that's why everybody looks when you express microdystrophin or dystrophin in any form of dystrophin in the cell. First thing you look at is does it correctly goes where it should be at the sarcolemma? That is and you see those staining in histological sections around like that like delineating the section.
It's not only a dystrophin, but everybody looks at the complex that is supposed to form with the dystrophin, the dystrophin-associated protein complex. That also needs to be localized at the sarcolemma. It's essential for the function, basically.
Okay, thank you. So Sarepta failed their primary endpoint on NSAA. And we saw that the data and the effects were different depending on the age of administration. So you guys have actually aligned with the agency on using microdystrophin expression as a surrogate endpoint. So just, you know, frankly, why do you believe that this is a better choice for you?
Yeah, I think we've been working, you know, with the agency for years as REGENXBIO on the use of biomarkers in rare diseases that are reasonably likely to predict clinical benefit. Christine, you know, we actually through individual experiences and you know, we have a whole program in a lysosomal storage disease where we've also done important characterization of the product candidate biology in a biomarker and showed its evidence of its correlating to benefit in also it's an excellent disease. So boys, you know, in Duchenne, we actually helped form with our colleagues at Solid and others a development consortium that we called Pathway Development Consortium. Held many meetings specifically and including on Duchenne and the use of microdystrophin.
Look, we're open to looking at other potential biomarkers as well with many, many stakeholders around the table to talk about the high unmet need, the need for acceleration and how biomarkers can play a role in that. And look, it works. I mean, I think we are incredibly pleased about the agency's focus and approach in these areas, including on Duchenne, including in MPS II and other areas. And I think we're seeing, you know, I mean, real meaningful advancement for rare diseases specifically in gene therapy, but I think it has an opportunity to fan out to other areas as well. Importantly. Microdystrophin, you know, I think as you and Olivier have been discussing, it's a little bit different than our MPS II experience where we're literally expressing the full length protein there, right?
And then we're actually interrogating a substrate of that full length protein, which is an enzyme. And that's known in nature to have that interaction. And you know, obviously the microdystrophin question, you know, raised everything that you started with, Christine, was, well, you know, is it really like dystrophin or how do you prove to me that it is? But there's actually a tremendous amount of, you know, as Olivier said, 25, 40 years of development, you know, whether it was brute force approaches or the more, I think, thoughtful, industrialized, engineered approach that Olivier and the team took. These things work. They clearly work. The question is, to what degree are they getting closer to the full function of a natural expressing dystrophin?
And you know, that's, I think, what we would posit is that some of the first generation reagents are things that got there, got above a certain threshold. And you know, now you might be able to see that, you know, clinically, they show benefit relative to that certain threshold. They certainly are. I think the FDA has supported it. I think there's evidence out there of it doing so. I just think that RGX-202 is better. It's engineered to have something more. I think it goes above that threshold. And I think therefore its application in more boys, you know, whichever part of the matrix you want to explore, where they are in the progression of the disease, that could be based on age, that could be based on weight.
Maybe it's going to be things that are based on the types of mutations they have and what that does to contribute to safety or the development of the disease. It's just something that is an improvement that I think has the opportunity to get more coverage. But the NSAA point, Christine, I mean, I can tell you. As soon as we started to talk with stakeholders about the use of microdystrophin as a surrogate to predict clinical benefit, at least to my ear, you could already understand that NSAA had some deficiencies as kind of a, you know, broad spectrum tool for assessing clinical outcomes. And Duchenne, is that because it's rare? Is that because, you know, it of certain limitations that, you know, I'm sure you've gotten into with other people in the last couple of days. It almost doesn't matter.
What I will say, though, is that, you know, many, many people who have experience with it are guiding the agency to look at it and more things that are real-world evidence of improvement for Duchenne boys and for, you know, function and strength and the evolution of the disease relative to what's known in natural history. I think that as long as you do that in a thoughtful way and on a pre-specified way, you know, then I think you have a lot of the opportunity to build a development program that just like we did with science and preclinical can be better maybe than what people have been able to achieve before.
But at the same time, I look at, you know, some of the results that came from the earlier regimens, including the approved product, and go, but we are also better. We also have an argument for better biology. So, you know, we do not rule out we will be studying North Star. We are studying it in our first-in-human study. We will be studying it as long as we study RGX-202. And we don't rule out that the impact of the improvements in RGX-202 can show different things in North Star, not just on individual domain basis, but on the full scoring of a North Star Ambulatory Assessment that's different than, you know, what the precursors have done. And I think that's, you know, that's where we are with our development, how we're thinking about things.
How the dialogue goes for us. But the focus on microdystrophin is because it's a validated endpoint right now from an accelerated approval perspective. And I think FDA feels the urgency to be able to bring, you know, frankly, more treatments, especially treatments that are new innovations to the Duchenne community as we talk about in these areas of older boys and boys with different mutation statuses. But even in the boys where the accelerated approval exists today. If something can be better, they want to accelerate it there. And I think that alignment on the use of microdystrophin right now is very real.
Yeah, I would love to go into that in more detail. Obviously, the FDA has been very outspoken about this. So on one hand, we do respect and understand that there is a dire need for these patients. But on the other end, gene therapies are tricky, right? They're one-time therapies and sometimes that can mean that if you get on that drug, it could preclude you from potentially receiving a more therapeutic option down the line. So I guess how do you think there's a fair way to kind of balance and weigh those decisions in light of that? You know, time is muscle, but obviously there could be future drugs where patients can benefit even more.
Yeah, I'll start. I know Olivier has some to add here. I look, I think that what the FDA has done in the last year or so has been, I think, a really thoughtful and I think, you know, I attribute it to really listening to stakeholders, especially the experts, the physicians, and the families in the community about what they feel the need is and what they're willing to accept. And then balancing that with real science and areas where there is real data. Even if it's not perfect, because I do think that perfect could be the enemy of good here. But it still needs to be good in that sense.
I think that an area that I think is emerging is going to be are there areas that, you know, where you don't have data and you're going to try to think about how to extend observations clinically and scientifically to areas where, you know, you don't have literal data, whether it's on an age basis, whether it's on a mutation status basis. You know, that and I think that's what's coming. The first instance was pretty straightforward, right? I mean, they had data. They had data in 4-7. There were at least two pivotal trials running in 4-7, two different sponsors. They stratified it a bit more by 4-5 and 6-7. A lot of healthy dialogue, a lot of public dialogue. A decision was made based on that orthodoxy and that stratification.
It didn't necessarily, you know, was it universally accepted by everyone that that was absolutely the right thing to do? Of course not. You know, I mean, these are complicated, but also important. I would argue public health decisions, but I think it was really founded strongly, though, in at least a data set that people could really sink their teeth and hands into and mold and manipulate and sort of share it with everyone. That's why I think it's been so important for us to think about both the improvements of RGX-202, bringing that forward when we study it clinically. Looking at where gaps in understanding are about the microdystrophin class in general. And running, not walking to those areas to develop new data sets. To inform the whole field and all stakeholders about benefit there.
And I feel very strongly, you know, and not everyone will agree with me, but I think that, you know, that's why it's great that we all have our opinions and, you know, and share these as stakeholders. You know, thinking about the mapping of approvals in areas where you have data and then you have sort of a vacuum of less data, I think is something that we would think twice about, you know, and I think that we would be expecting for an agency, for frankly, stakeholders, physicians themselves and families to want to see that data before, you know, a decision was made about looking to include or expand or apply that.
You know, we're not asking to sort of take it out of the hands of the experts and the decision makers, but I think, you know, as a public health issue and as a community issue, we're expecting there to be data. You know, and I'm not just talking about the light peripheral adjacent areas that, you know, I think are covered by, you know, certain areas of study. I'm talking about things that would be. It's basically a different part of the disease process. It's further enough along where there's both evidence that other things haven't done the job. They're starting to be emerging evidence that new things might not only have improvements, but literally deliver things that haven't been seen before. I think that's been, you know, the entirety of the reasoning behind the innovation of RGX-202.
You know, we feel enthusiastic that we're finally able to contribute. We've been, you know, Christine, since we've started dosing patients. We've been incredibly transparent about sharing all of our data that is coming forward with patients. You know, I think that is also an important feature of, you know, how this whole process works with stakeholders.
Okay, thanks. And maybe the last question for me is if you can recap for us any upcoming milestones specific to this program. And then I know REGENXBIO is not just a DMD company. You have many other programs, but when you think about your stock in general, why do you think you're undervalued just on the potential for this DMD program alone without even thinking about everything else going on?
A lot to fit in or unpack in a couple of minutes there, Christine. I think that, you know, yes, we have a tremendous amount of enthusiasm for where we are in Duchenne. And what's coming next. You know, we've guided to the fact that by the middle of the year, we plan to pick our pivotal dose. We're already in healthy discussions and continue to have a plan for initiating a pivotal trial in the second half of the year, which means, you know, as the data comes out, we're just using it to build on the foundation of the communication that already exists with the FDA. And that's not just based on the clinical data. It's also based on our manufacturing. We've said that, you know, we built a facility and we've been using a facility here for all of our programs.
I mean, we'll be on one hand filing a BLA this year for Hunter syndrome. You know, we're working in late stage development with AbbVie on our eye care partnership and Wet AMD and diabetic retinopathy. And Duchenne is actually our third program in terms of stage of development, but one that we're looking to accelerate into pivotal by the end of this year. All of them stand on top of a very robust, high quality, high yield manufacturing facility and process. That's literally just above our heads here in Rockville. So we're already having discussions with FDA on those features as well because we want Duchenne to and we think we've moved quickly, frankly, on things like Hunter syndrome from, you know, first patient dose to timing of a BLA.
Being as far ahead as we are in Wet AMD with our subretinal program for, you know, pivotal phase development with thousands of patients. But we actually want Duchenne to be the absolute strongest and fastest development program that this company has ever had. And that's the goal to kind of complete the remainder of this year is report more data. Share that data. Finish the engagement with FDA on very efficient microdystrophin based accelerated approval. And then talk about how we're going to be filling in those gaps of understanding in areas that I think we view as important, whether it's older boys, you know, even improvements in areas where existing labels exist. New mutations. You know, just new data that can be collected is a major priority for us to contribute. We think that should translate into value.
We think it has translated into value already, but we think our role in Duchenne is important to us. It's a major commitment. It has been for years. We feel that, you know, having that clinical data now has been able to let that concept breathe a little bit more for investors. But we want to keep reinforcing that. We think that the breadth and depth of RGX-202 in terms of its market potential. And look, I personally believe that those areas where there is not data right now, where we're generating data is a highly accessible and rich market for us to approach.
As well as we generate more data about safety and sort of the influence that we have overall with RGX-202, I think, you know, that the ever-present incident population and the decisions that will occur there clinically and commercially with the type of innovation that RGX-202 represents is an incredibly important and a creative aspect of our clinical and commercial plan as well. So we've got older boys. We've got, you know, boys with different types of mutations maybe that to this point haven't been able to benefit from earlier candidates and exploration. We've got, you know, the ever-present incidence.
There's still a lot to be done for Duchenne, and I think RGX-202 with its scientific advancements and now some of the clinical evidence and support to me is as exciting as anything we have in our pipeline, and really exciting for the Duchenne community.
Great. Well, Ken and Olivier, thank you so much for the very thoughtful discussion around Duchenne. Really appreciate it and wishing you guys all the best as you think about that pivotal trial.