I'll take it from you if it's a distraction.
Sure. No, it's okay. I'll see you in the questions.
Okay. Well, let me do this part.
Yeah.
Oh, yeah, yeah. Please.
Yeah.
All right. Good afternoon. I'm Eric Joseph, Senior Biotech Analyst with J.P. Morgan. And our next presenting company is Rocket Pharma. Presenting on behalf of the company is CEO Gaurav Shah. And there's a Q&A after the presentation. So if you've got a question, we'll bring a mic over to you. And folks tuning in via the webcast can also submit questions there. So Gaurav, thanks for joining us today.
Hi, everyone. Welcome. Thank you, Eric. Thank you, J.P. Morgan, for having us, so I'm going to start with a quote from T.S. Eliot. "In order to arrive where you are, you must go by a way wherein there is no ecstasy." We can probably agree. Let me say it again. In order to arrive where you are, you must go by a way wherein there is no ecstasy. We can probably all agree that last year was challenging. Certainly, few of us were ecstatic last year. But we've gone by that way already, and we're arriving in 2025 with great optimism, and I believe this year holds really great promise for patients treated with gene therapy, especially those with rare, devastating diseases, and I'm especially excited about Rocket's pipeline. And I'll just get to the bottom line. Key messages for this year.
We have a host of potential milestones coming up in the first half, later in the year, and into early 2026. First of all, in the first half of this year, we will have a program update on Danon disease that has three prongs. The first is a general trial update for the pivotal phase 2 trial. The second is a trial design update. In other words, statistical plans, et cetera, moving into the top line. And the third update is going to be a patient-finding update that is both bottom-up and top-down. So that's first half. We will also have preliminary early data from PKP2, which we call Pegasus. We've completed the low-dose cohort as far as enrollment. And we'll have at least safety, if not additional information, in the first half of this year. We will be filing our BAG3 IND in the first half.
In either the second half or at some point this year, we expect an LAD PDUFA. We expect the Fanconi submission will be completed. And we'll start launch planning for Fanconi. And we will resume PKD enrollment. So that's the panoply of six programs for this year. In early 2026, we will have Danon top-line data. And I'm very excited to announce potential additional indications in cardiac gene therapy that are cooking in the background right now. So I will try to be pretty concise and move directly to some of the disease characteristics and clinical data that make 2025 exciting and the future very promising. So Danon disease is a disease of autophagy. Autophagy is like the vacuum cleaner of a cell. The underlying myocardium has beautiful designs, like the carpet here. Autophagy is needed to take trash out. Without autophagy, the carpet becomes muddied.
You can't see the designs over time. Turning on autophagy, which is what we do with gene therapy, allows the true structure of the myocardium to work so that the cardiomyocytes can contract together and have solid cardiac output. Danon disease is an X-linked condition with impaired autophagy. Unfortunately, these are boys mostly, but also females later in their life who have a lot of vacuoles building up inside the heart. Unfortunately, the boys die or need a transplant by the age of 19 or 20. The females live longer because they have some protein already there. LAMP2 is the protein involved. What we do with AAV9-based gene therapy is replace their genetically mutated LAMP2 protein. Danon disease has a prevalence of 15,000-30,000 in the US and Europe. We're going to talk more about that in a program update later.
I was going to say this semester, but later this half. These slides are new from November from AHA. Some of them are even updated since then. Protein expression using AAV9 gene therapy, LAMP2 gene therapy for Danon disease is durable and robust out to five years. This is the first time, I believe, that in the history of any gene therapy for a muscular-related disease, we've shown persistent, robust, durable protein expression here between 50% and 75% in the first patient out to five years and in all patients between two and four years. You can see more of that on the right. Those are representative IHC images. It was slide 12 for those who are following the webcast. On slide 13, Danon disease is a disease, like I said, of impaired autophagy. These vacuoles build up inside the cardiomyocytes.
You can see that on the left side, as these on the top left as the large white blobs. Over time, you can see they get reduced in number quite a bit. You can see that on electron microscopy on the bottom slides as well, so you can see histologic changes that mimic and follow the protein expression, so this is one of our favorite slides at Rocket Pharma, and I can't believe that we live in an era where most of this slide is green when it should be red. If we did nothing, every cell here would turn red between one year and five years after treatment. Everything is green, or at least light green. These are patients who are treated on the phase one study. These results were published in the New England Journal of Medicine in November.
You can see that in a disease where the median age of survival is 19 or 20 for boys. First of all, several of these boys are over 20. One of them just celebrated his 25th birthday a few weeks ago and is healthy, going back to work, and happy with his family. That's the age itself. We've already bucked the trend here. The LV mass index, which increases in Danon patients, that's why they have big hearts. Danon disease is a disease of big hearts. The heart doesn't start big. It starts normal. So obviously, it gets bigger and bigger as these boys grow up. To see a reduction in LV mass index in all six of six treated and followed patients is not expected, except with the power of gene therapy.
Moving to the right, we see similar changes in BNP, which is a marker of heart failure, massive, profound reductions in troponin, which is a marker of cardiac injury. And remarkably, every single patient, six of six, improved their NYHA class from class two to class one. That means they went from symptomatic to asymptomatic. KCCQ scores, even quality of life scores that increased by five points or more, have been associated with approved cardiac therapies. Here, the median is around 20-something with as high as a 52-point increase in how these patients function and feel. So again, we live in an era where we see green here and not red. This is the LV mass index over time in those patients.
All of those patients, you can see, first of all, that there's a sustained reduction over time compared with untreated patients, natural history, where these LV mass index should increase. It's like an alligator mouth. The bigger the difference, the more profound the impact. Now, what's interesting here is that at month 12, everyone has reduced by 10% or more. They also have protein expression, which means that if this were the pivotal phase 2 trial, the response rate would be 100%. But it was just the phase 1. We're almost done with the phase 2 now. This is another way. This is a new slide not shown before of Danon disease patients who are treated on the far right. You see an ongoing and durable, robust reduction in LV mass index over time at four years, on average 47%.
Other recent drugs that were approved for Fabry disease, amyloidosis, and mavacamten for HCM show some decreases as well, but not as profound and durable over time. Similar slide as the previous one. But this is just looking at the low-dose patients. Low dose is the one that we are moving forward into the phase 2 pivotal study, which we've done enrolling. But you can see, again, a profound decrease over time versus what would happen untreated and even versus other agents that are out there on the market. This is troponin, which is a key secondary endpoint. You see a drop in troponin to normal levels for almost all patients or near normal levels for all patients. And troponin is a marker of cardiac injury. And again, it should not decrease once this disease has started. But it does with gene therapy.
These patients, like I said, are doing well. There are cases where one sibling who was not treated with gene therapy but was treated with transplant has, unfortunately, become more frail over time. And the gene therapy-treated patient actually takes care of him and their mother. These are examples that are real and over time are going to be the basis of acceptance, I believe, in the community globally for Danon disease. The phase two trial is ongoing. We've completed enrollment as of a few months ago. We're finishing dosing as fast as possible. Again, the key endpoints here are going to be a combination of protein expression and LV mass index. Again, drawing from phase one, if we see even a partial response of phase one, this could be a positive trial. More on that in the first half program update. The manufacturing for Danon disease is in-house.
And why does that matter? It matters because we can control the cost of goods in a pretty profound way with high margins at the end commercially. And also because we control our timelines. We're not dependent on others to get the work done. And we have enough manufacturing capacity in our plant in Cranbury, New Jersey, to supply the entire Danon market. This is an expense that we took early in Rocket's history when maybe capital was easier to raise, I must say. And I'm happy we did that because it's going to translate into Danon disease, but also help with PKP2, BAG3, and other indications to come down the road. So PKP2. Now, there are several types of cardiomyopathy. There's hypertrophic cardiomyopathy. There's arrhythmogenic cardiomyopathy. And there's dilated cardiomyopathy. And I'm thrilled to say that Rocket has a hand in each of those. So Danon addresses hypertrophic.
PKP2 addresses arrhythmogenic, and BAG3 is going to address dilated cardiomyopathy. Let's talk for a second about rare disease. While each of these rare diseases may be somewhat rare, although 50,000 is not that rare, when you add them together, these are common diseases. So just these three alone add up to 100,000 patients in the U.S. and Europe, and you add on the other three that are coming down the pike, and we'll talk more about hopefully next year, and you're at 200,000. That's no longer an orphan disease using one platform. PKP2 ACM is a disease of desmosomes in which PKP2 is missing or defective. Actually, these patients are heterozygous. So they have reduced PKP2, not zero PKP2, unlike Danon disease, but these are patients who have sudden cardiac death, palpitations, especially with exercise, and it's one of the most common causes of sudden cardiac death.
Athletes who are playing sports, basketball, track, and they suddenly fall down or they faint or they could pass away. Many of these patients have ICDs in place to prevent these sudden cardiac fatal arrhythmias. This is a disease that's probably twice, if not more, prevalent than Danon disease. At some point, Rocket will be a PKP2 and Danon and BAG3 company, not just a Danon company. That's the future that's coming. 50,000 patients in the U.S. and Europe. There are others in this field. In fact, one of the others in this field has quoted a number of 140,000. We tend to be pretty conservative, not just for PKP2, but for Danon as well. In our preclinical model that was a conditional knockout model out of NYU, we showed a reduction in arrhythmias very early on after treatment with AAVrh74-based replacement of PKP2.
We also saw a reduction in RV area. These are the two most important measures of cardiac progression in patients with PKP2 ACM. Now, we believe that Rh74 is the best capsid for this disease. Why? Because, first of all, Rh74 has a lot of experience. We have a lot of experience with Rh74 in the clinic, in the commercial world with the Duchenne programs. Patients take mid-E14 range doses. And there's a pretty strong positive safety profile. This is a heterozygous disease. We don't know how far we have to go, how high we have to go. So we believe that having a capsid with the most experience in the world in the E14 range is the way to go. If I were to do this again, we'd repick the exact same capsid.
We are also starting at a dose, 8E13, which is potentially positive based on the preclinical data that I just showed, so we're not starting. This is not really a dose escalation phase 1. It's a phase 1 to potentially validate the dose that we think will work, so this program, we finished the low-dose enrollment already. This is slide 29. We will have an update in the first half of 2025. Our style and our plan has always been to move toward pivotal trials as rapidly as possible. We have an excellent iterative dialogue with the FDA going already, not just for Danon, but for these other programs. We hope to move them forward as soon as we have an understanding of the right dose to move forward. BAG3 is a very important DCM, dilated cardiomyopathy, that we inherited from Renovacor.
And BAG3, unlike LAMP2, which is associated with one function, which is autophagy, BAG3 regulates several functions in cardiomyocytes, including autophagy, but also including contractility, structural support, and apoptosis. And we have not provided a preclinical update here in some time. And we will as we move forward. But dilated cardiomyopathy is actually much more common than hypertrophic cardiomyopathy. There are more patient communities and advocacy groups that address DCM that we work very closely with. BAG3 is likely more common than Danon disease somewhere in the PKP2 range, more on the epidemiology as we move toward IND. IND submission is anticipated in the first half of 2025, as I mentioned. So three programs, three pearls, and more cardiac programs to come. Rocket started with a program called Fanconi anemia. And Fanconi anemia is a cause of bone marrow dysfunction. These are patients in which DNA repair is damaged.
And when DNA repair is damaged, well, when you have a hit to your DNA, your own DNA can't repair it. This especially affects stem cells in the bone marrow. But it affects all cells throughout the body. The standard of care for patients with Fanconi anemia is allogeneic transplant. But the problem with allotransplant is that it's highly toxic. It comes with chemotherapy. You have to give chemotherapy to these patients who already have frail bodies. And it's very hard to recover from. Also, while allotransplant can be potentially curative of the hematologic aspects of Fanconi anemia, it actually worsens the risk of cancer, solid tumors in these patients, which ultimately proved to be fatal. So patients who have been treated with allotransplant and Fanconi anemia are sitting and waiting for the cancer to arrive. So we need another option. And that is gene therapy.
We showed that without any conditioning, we were able to increase vector copy numbers in the peripheral blood and bone marrow over the course of time out to almost five years without any conditioning whatsoever. And why is that? That's because there's something called the selective or proliferative advantage that's present. If you think about it, when you get your first DNA hit in a stem cell, the stem cell goes awry and it doesn't work. But a second hit could rectify the first hit. And the second hit can't be cured either. So now you have a stem cell that works and survives. So we took this and put it and applied it to gene therapy. What if we gave these patients millions of cells with no chemotherapy?
Could they then take over the bone marrow and just allow the other cells that are going to die anyway to die out over time? It was a thesis. It was a theory, but it worked. Most of the patients treated in this trial have had improvements in what we call MMC resistance, which is the hallmark of Fanconi anemia diagnosis. The patients you see here, some are late bloomers as late as three years, but all these patients have increased in MMC resistance with no conditioning whatsoever. This is a phase two pivotal trial that's done. We've started a rolling BLA submission with the FDA. We've also submitted to the MAA, and that's under review as well. Most of the patients did have improved MMC resistance, normalization, or stabilization, and in some cases, improvement of blood counts.
Given the risk-benefit profile, the patients and community who are dealing with Fanconi anemia are asking, why not? If you have a therapy with minimal risk, there's only one grade 2 AE in the whole trial. But you can cure potentially the hematologic components of disease and not increase the risk of cancer that chemotherapy gives, why not? So we're very excited about launching this program likely in 2026. LAD-I, someone asked both Jonathan and Kinnari today what their favorite program was. And it's unfortunate that I present this as number five, number five right now. But it's actually one of the reasons why we go to work, seeing the results we see in LAD patients. These are patients who have CD18 that's absent or reduced in their neutrophils. And these patients can't fight their own infections.
They often pass away of pneumonias and fungal infections and have gingivitis and other debilitating conditions. But most patients in the world pass away by the age of two. So when we talk about devastating, this is devastating. And it's also severe. So severe LAD-I, we did a trial. We completed a phase 1/2 trial that was pivotal in this population, restored CD18 expression to slide 37 in all patients who were treated, which is nine patients treated. And this Kaplan-Meier curve says it all. It's what academic physicians and practicing physicians and drug developers like us aspire to our whole lives, 100% survival in all patients, and also a massive reduction in infections and infection-related hospitalizations after the gene therapy treatment. There's one patient that came to one of our rare disease programs. And at the time, she was a single-digit age when she came.
She said that, "My dream in life was always to ride in a van across the country or drive in a van across the country the day I turned 16." She never thought that it could actually happen. She's almost 16 now. She may be 96. This is a devastating disease. I think you can only get those sorts of results with gene therapy. That's why I'm optimistic about this year. This program is with the FDA. We're tweaking some stability and sterility issues and hoping to resolve them this year for an approval. Last, but definitely not least, when gene therapy works, it works. PKD, pyruvate kinase deficiency, this is the most common lenti program that we have. We've prioritized some of the others. We have not started enrolling the pivotal phase 2 here.
We do anticipate that we would start and resume that program later this year as LAD and Fanconi get up and running. But in this program, four out of four patients in phase one either normalized or massively increased their hemoglobins, and these patients were transfusion dependent after splenectomies and had debilitating existences and quality of life. And we're very, very happy to see how well they've been doing, and we'll move this into phase two as soon as possible. There's a picture of where we go to work most days. For me, it's about three days a week, but for some people, it's four or five here, and it's in Cranbury, New Jersey, near Princeton. It's 30,000 sq ft that are dedicated to Danon, PKP2, and in the future, BAG3 as well.
Again, this control allows for great cost of goods, but also control of timelines and the ability to negotiate quickly with regulatory agencies. Now, this little tip of the iceberg that you see, it really and continues to be above the surface. There's the larger iceberg underneath. And I think, first of all, we have at least two or three other cardiac gene therapies that are on the docket. We anticipate and hope that we are first and best in class. We can't always be only in class, given that these are exciting indications. And gene therapy could be for everybody. But we hope to be first and best in class for all these programs. We've cracked the door open here for gene therapy. And I think once we've mastered monogenic diseases, we may move to bigenic, trigenic, multi-diseases, and complex diseases.
I think there are three buckets again by which mortality happens for all of us. It's accidents or injuries. It's infections. And almost everything else can be genetically modulated. So cracking this door open is a big step forward for Rocket and for the human species. Thank you very much. I'll take questions here.
Great. And if there are questions, just wait for the microphone. But let me get just by way of getting started with Q&A. You kind of teased a series of updates coming for the Danon disease pivotal program. So Gaurav, I might ask you to just sort of unpack the setup here a little for us, which is, I mean, one is probably pretty straightforward in terms of either enrollment or treatment update. The trickier one is perhaps the trial design update with some statistical considerations. What is encompassed within that, perhaps? And to what extent are we thinking about sizing of the study or changing the size of the trial as a result?
Yeah. So to be clear, there's no change in the trial design itself. It's just clarity on the statistical analysis plan. So a lot of folks have asked, this is a responder analysis. Is it or not? Yes, it is. How many responders do you need? It's those sorts of questions that we'll be able to answer.
OK. OK, great. And perhaps on the, call it, natural history or prevalence population, patient ID side, I guess part of that would be patients identified maybe as part of the screening process, but as part of a measure of sort of priming the market to some extent when commercial. Maybe just probably orient us around that a little bit. And then how would that sort of also inform your sort of top-down estimate when thinking about the overall size of the prevalence population that you think Danon disease is, the ability to sort of refine that? And then also how your experience here is going to inform or make the ID process, patient ID identification process, perhaps a little more efficient as a commercial stage company.
So a couple of things. What we've done is we've done top-down three different methodology analysis to look at 15,000-30,000. And we've come to that more conservatively. It might be greater. But as part of the effort that we've had in identifying patients for commercial success as well as clinical trials, we've done two things specifically. One, we put an ICD-10 code in place last year. So having ICD-10 code allows for physicians that are diagnosing Danon patients to really reference them. So over time, we'll have a better idea where these patients are actually coming through. But the second thing, which is really powerful, is genetic testing. So we are making genetic testing available for free for these patients, not just for Danon, but it could actually be a platform that could be applied for PKP2 and BAG3. Why is this important?
A lot of the times, unfortunately, in rare diseases, especially in these types of diseases, it's too late by the time they get diagnosed. A lot of times what we find out from patients and families is family members passed away, and they never knew why, and now they're starting to realize that, oh, a grandson has Danon disease and find out when they test multiple family members, many of them, male and female, have Danon disease, so what we want to do is have a bottoms-up analysis of these patients and where they reside, what are the demographics, and how many patients are impacted by this, and understand their natural history, so in an effort to do that across the U.S. and Europe, we've come up with numbers center by center, so we're going to have that information be available.
And that will just get us ready to launch this product and make sure that patients have access as soon as the therapy is approved and know where to get the therapy. So that's really the key to success for us, genetic testing, having the foundation, but also working with patient advocacy so they can understand the benefit risk of this disease and gene therapy and the different options they have available. And the third thing that's really important is working with cardiology centers, not just the heart failure centers of excellence around the world, but also cardiologists that are working with pediatric population to see if we can increase the diagnosis based on symptoms they see early on.
Another just to add also on the natural history, as we've started the natural history program, sometimes when we look at other family members of the person who's actually enrolled in the trial, we find that two, four, in one case, 13 other family members also have Danon disease but just didn't know it. So the natural history efforts are also unraveling a lot of the commercial sort of pool out there that I think could be addressed with this gene therapy.
There's both the market sizing consideration related to that and also patient identification for the purposes of building the market. But also, those data sort of might incrementally inform the natural history of disease, the course of disease out there. I guess, is that sort of an expectation? And sort of will your update kind of speak to that? Any change or thinking around the level of unmet need in the particularly male Danon disease population?
Yeah, I think that as part of the update and ahead of top line, the program update is basically geared toward a better understanding of the disease, the patient journey, and also the epidemiology ahead of top line, and I think the patient journey part of this will be key and critical, and we will be able to share more about that. I think we separate that from the actual natural history efforts, which are more of a BLA filing requirement. Understanding the patient journey will also uncover the addressable market, which males are severe, who should be addressed first, who will come later, is there a population that will never be able to address in males, and then we'll do the same thing for females. That'll be part of this program update as well.
Got it. Just on PKP2, I think you've kind of touched on perhaps one of the challenges and certainly one of the things that we're thinking about in terms of the initial readout from this program, which is what the target level expression is or that you need to achieve in this patient population, just given the fact that it's a heterozygous genotype. Jonathan?
I wish I had a definite answer, and we don't. We anticipate that probably most of these patients have around 50% of normal or healthy levels of PKP2 in the myocardium. We're really not sure whether we need to get to 65% or 70% or some higher number. I think we'll start to have an indication of that as we get the phase 1 data from the initial patients, which will be part of this coming first half of the year. We'll certainly, I think, by six months after treatment, get a gauge as to what sort of protein increases we've seen, whether that protein is localizing at the desmosome, the intercellular connections where it needs to be to function, and how much of an association we see with clinical improvements with reduction in arrhythmogenic substrate with any cardiac remodeling or any other quality of life or symptom improvements.
So stay tuned for hopefully a somewhat more definitive answer on that.
Just looking at your protocol, I believe it has an exclusion criteria or specifically calls out patients with truncated mutations as eligible for participation. Just talk about sort of what that's meant to distinguish from perhaps other mutations that are present that emerge within PKP2 and sort of what the phenotype there is. I guess the question is sort of around from the population genetics, whether that might also help inform the sufficient amount of protein expression that could be corrected for these patients. Yeah.
The majority of patients with PKP2 arrhythmogenic cardiomyopathy have truncating mutations, which is important. That criteria is intended to exclude the more rare subset of the disorder where there might be a non-truncating mutation, which would then generate a protein that would serve as a dominant negative. In other words, that protein, most patients are just not making enough PKP2 protein. But there will be some rare mutations where you're making a deficient protein that's then gumming up the works, and it would then nullify the effect of the corrected protein. So we're focusing this study on the majority of the population where that's not going to happen.
OK. All right. So it's still, yeah, you can't really use those patients as a way of sort of figuring out what you can get away with. It's really to avoid those that might actually have a pathogenic protein and therefore undermine the efficacy of your agent. OK. Well, great. So I guess kind of a key question on the biomarker side there with PKP2. On the clinical side, yeah, from the biomarker, of course, we'll learn more. On the clinical side, maybe just talk a little bit about sort of what endpoints will ultimately inform kind of clinical benefit and maybe kind of leading into that, the extent of baseline information that you're gathering either from their patient histories or screening that would allow you to kind of make an assessment of clinical benefit over time.
Yeah. I mean, just briefly, I think that there are predictors of fatal arrhythmias. And there's an algorithm that predicts fatal arrhythmias. It's not single changes. It's not just PVCs. But there's a fingerprint by which we know that someone is likely to have a fatal arrhythmia. Some of them are shown here, for example, obviously having ICDs, having EPS-induced V-tach, more than three ECG leads that have T-wave inversions, a lot of PVCs. You put these together, and you get a sense of which patients are going to have more risky arrhythmias. And I think affecting any of these also affecting the heart, the RV area, which is ultimately fatal for these patients. Once you have an ICD, the ICD often protects death. But then the heart failure eventually will take over. So we don't know the answer.
I think saying that we know the answer upfront would not make any sense. One of these or two or three of these should be able to be the marker of clinical benefit and proof of concept along with protein expression, and we look forward to figuring that out as we go. I think the update in the first half is really geared toward just updating safety, maybe protein and localization. Obviously, if we see a footprint or fingerprint, we'll talk about it, but that may take a little bit longer to unravel.
But one thing that's, I think, pretty definite for a lot of the people that are dealing with this disorder is if we're able to reduce the incidence of the ICD firing relative to the one-year or two-year period prior to coming on study. That's going to make a tremendous impact on the lives of these patients because you do have a number of these individuals who they have the ICD implanted, and it fires and saves their lives sometimes on a monthly basis, sometimes several times per year. But it's lifesaving, but it's incredibly unpleasant. It's like getting kicked by a horse. And when it happens, it's obviously disturbing. Even when it's not happening, they're living in fear of it. And it creates a tremendous amount of sort of psychosocial impact on well-being and health.
They're also, for the most part, told to avoid any sort of strenuous activity in the hopes that that would diminish this. The goal is really to give them freedom from the firing, a very different outlook on life, and hopefully a situation where these things are really not going off and maybe over the long term as part of life cycle, not even necessarily needed once gene therapy is administered.
Maybe last question. Just thinking about the lentiviral products potentially launching in 2026, just how to think about the prioritization of those commercial launches relative to everything that's going on on the AAV side. And then maybe I'll just try to sneak in to the question about the extent to which you've had interactions with payers and potential pricing considerations of both the LAD1 and Fanconi anemia gene therapy.
Maybe I'll start off with the second question first. Most of the diseases, especially the LAD disease, Danon, and PKP2, there are no other treatment options available. So our pricing is really going to be based on value-based pricing. If an LAD patient, Danon patient, passes away by the age of two, and if we can give them a normal life, that's a huge difference. If a Danon patient, instead of passing away by the age of 20 or having a heart transplant and complications, if they can live. So that value-based proposition pricing is how we're thinking about it from designing the clinical studies to also having the experts that are treating these patients actually talk to the payers and understand what kind of data they need. I think New England Journal of Medicine data that came out for Danon disease shows the durability.
And I think that's one of the questions that will really help with the pricing perspective when we think about commercial launch. At this point, our time and effort are really focused on Fanconi anemia launch because for us, we know there are patients out there. Fanconi anemia, many of you guys that are physicians, pharmacists, you've learned about this in pharmacy and medical schools. It's easy to diagnose. There's foundations available across the U.S., Europe, and around the world. So we know where the patients are. Patient and patient communities and the treating physicians have bought into the benefit risk, the why not of this therapy. So our goal is really to set up the QTC or treatment centers for these patients.
So as soon as we can get an approval, hopefully soon after, we can get these patients enrolled into the study. I'm sorry, not into the study, enrolled into the QTC centers and get treated. The beautiful thing about Fanconi anemia is for Rocket, Fanconi anemia is a starting point for our launch for commercialization. The learnings we'll have by launching it in the U.S. and Europe will actually be applied to Danon when we think about the bigger markets. And hopefully, that learning will be applied to PKP2. So as a whole, we believe that Rocket has this beautiful fortune of diseases that have high medical need, science and data that makes sense, patients that are awaiting therapy and they have the urgency. And every step of the way, we've brought the health authorities and the payers and the treating physicians along.
We think this could lead to a quite meaningful commercial opportunity, not just for the lentis, but application of it to the success of Danon and the cardiac programs.
Great. All right. We'll leave it there for time. Thanks so much, guys.
Thank you, Eric.
Thank you.