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Good day, and welcome to the Rocket Pharmaceuticals Investor Program Update on Dana Disease and the RPA501 Pivotal Phase II Trial. As a reminder, today's program is being recorded. I will now turn the program over to Meg Dodge, Senior Vice President and Head of External Affairs. Meg, please begin. Thank you, and Welcome to everyone joining us for today's Dana Disease Program Update. Today, we will share new Phase 1 findings and preliminary Phase 2 clinical observations for RPA501, including the initial safety experience of the recalibrated dose. We will also review the pivotal studies definition of efficacy success and our plans to complete dosing, followed by an update on the Danden population and potential commercialization opportunity. You'll hear from Chief Executive Officer Dr. Gaurav Shah, our Chief Medical Officer Dr., Syed Rizvi, and our Chief Commercial Officer, Sarbani Chidhuri. We are also pleased to include perspectives from Dr. Eric Adler and Dr. Barry Greenberg, whose work has helped advance the understanding of Dana disease and the clinical evaluation of RPA501. Following prepared remarks, we will move into Q&A with our management team, including Gaurav, Syed, Sarabani, and our Chief Science and Gene Therapy Officer, Dr. Jonathan Schwartz. Before we begin today's discussion. This discussion includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements concern our development plans, clinical and regulatory milestones, population estimates, and potential commercial opportunity. They involve risks and uncertainties, and actual results may differ materially. Please review the disclaimer in today's presentation and the risk factors in our filings with the Securities and Exchange Commission. These sentiments speak only as of today, and Rocket Undertaker takes no obligation to update them except as required by law. RPA 501 is investigational, and its safety and efficacy have not been established. The revenue estimates we will discuss are internal modeling scenarios subject to clinical success, regulatory approval, and commercial assumptions. And with that, I'd like to now turn the program over to Gaurav. Thank you, Meg, and thank you to everyone for joining. Before turning to today's program update, I want to highlight the financing we just announced after market close. Under our new Hercules facility, we received an initial $35 million, which together with our existing resources and based on our current operating plan, is expected to fund operations into the third quarter of 2028. Additional borrowings under the facility could also extend our cash flow into 2029, subject to applicable borrowing conditions and depending on the timing and amount of future draws and our operating expenditures. This additional financial capacity supports our focus on completing the RPA 501 pivotal program and advancing toward a potential BLA submission. Let's start with the bottom line up front. First, today we are sharing additional Phase I data, both longer-term clinical observations and biopsy findings, which show evidence of disease reversal or stabilization through 36 months and up to seven years post-treatment with RPA501. Second, and completely new, preliminary clinical data from the initial four Phase II patients who were treated before the recalibrated dose and immune regimen and did not receive C3 inhibitors further support the efficacy of RPA501. These findings build on evidence of restored myocardial LAMP2 expression and changes in cardiac structure while extending our understanding of patients' clinical courses over several years. The original Phase II experience included serious safety events, including the tragic loss of a patient in the cohort evaluating an immunomodulatory regimen containing a C3 inhibitor. Together with input from global external experts, we responded by recalibrating the dose and modifying the treatment protocol. So the third takeaway here today is that the first three patients treated under this final revised approach have completed the initial observation period without clinical or laboratory evidence of thrombotic microangiopathy, capillary leak syndrome, or other significant toxicities. These early observations are encouraging, and follow-up continues. SIAD will review the safety experience and the changes we have made. Number four, we have aligned now with the FDA on a pivotal efficacy population of 12 males receiving commercial-grade RPA501 at the recalibrated dose. New today is that the efficacy target is at least seven patients meeting both the pre-specified responder criteria at 12 months, which are improvement from baseline to at least grade 1 in myocardial LAMP2 protein expression and a reduction of at least 10% from baseline in left ventricular mass index, or LVMI. This gives us a clearly defined responder analysis. The first three patients count toward that population, leaving nine additional patients to be dosed. We expect to complete dosing by mid-2027, and I will discuss the pivotal design and our intended accelerated approval pathway later in the program. And finally, today we will share new analyses that substantially strengthen our understanding of Danone epidemiology and the diagnosed patient population and reinforces our confidence in a potential $1 billion-plus global peak annual commercial opportunity in the initial indication in Dan and males alone. And we have already engaged with FDA in expansion to females, which would increase this market much further. We conducted two comprehensive independent epidemiology analyses, one genotype-based and one phenotype- based, and both converge on approximately the same estimate, which is 10,000 to 11,000 people with Dan and disease in the United States, and more than 20,000 if you include Europe as well. Importantly, we have also identified on a cumulative basis more than 900 U.S. patients with a confirmed Danone disease diagnosis, establishing a patient level baseline while highlighting a substantial diagnosis gap. And Shabani will take you through this evidence behind the findings where these patients receive care and how these insights provide a strong foundation for accelerating diagnosis as we move toward launch. Now, to put both the clinical need and the opportunity in context, we first need to understand the disease itself from the underlying biology to its aggressive clinical course and, importantly, the limitations of current care. And for that, I'm pleased to turn this over to Dr. Eric Adler, an internationally recognized cardiologist, expert in Dannon disease, and academic founder of the Dannon Program alongside Rocket. Eric will take us through the biology of Danone, how the disease progresses, why patients remain underdiagnosed, the burden and limitations of heart transplant, and finally, why LAMP2B gene addition is so directly aligned with the underlying disease mechanism. Well, thank you, Gaurav. As you know, I'm a heart failure cardiologist who's cared for Danone patients and studied Danone in the clinic and laboratory for many years. I still remember the first Dannon case I saw nearly 20 years ago and the hopeless feeling I felt staring at that hypertrophied heart and knowing there's not little I had to offer. How far we've come. Dannon stands out among inherited cardiomyopathies for its near-complete lifetime penetrance in males, early and aggressive cardiac course, multi-system involvement, and the potential to affect multiple members of the same family. Dannon is called by pathologic variants in LAM2, which encodes glycosome-associated membrane protein 2. It is X-linked and can affect successive generations, but de novo variants also occur. An absent family history does not rule it out. A hallmark is severe thickening of the heart muscle. Ejection fracture can remain relatively preserved despite substantial hypertrophy, myocardial injury, and electrical abnormalities. By the time it falls, the cardiomyopathy may already be advanced. Importantly, hypertrophy, and specifically cardiac mass measured by MRI, is a very important tool we have for prognosticating patients. We know that when the estimated LV mass is abnormal in patients with cardiomyopathy, their prognosis is worse than those with normal LV Mass. The clinical course differs for down in males and females. My focus today will be primarily on the males. males, cardiomyopathy often begins in childhood or adolescence and progresses rapidly. By the time many inherited cardiomyopathies are only beginning to manifest, a young man with DADN may already have progressed to advanced heart failure, transplantation, or death by his early 20s. Females generally develop cardiac involvement later in a more heterogeneous course. Emerging natural history data shows that the majority are clinically affected by their 30s, while some have developed life-threatening heart failure at a younger age. Despite its severity, data remains significantly underdiagnosed. We know the clinical signals that should raise suspicion. A young patient, particularly a male, with unusual severe unexplained hypertrophy. DKG abnormalities, including ventricular pre-excitation, elevated muscle or liver enzymes, or manifestations outside the heart are sometimes but not always present. In my opinion, any unexplaining cardiac hypertrophy or dilation in a young person should warrant a genetic test. You may be saving their life as well as their family members. This is the best way to diagnose data as well as other genetic cardiac disorders. Unfortunately, genetic testing remains low in the broader community and findings across organ systems may not be connected. Essentially, any young person with cardiac hypertrophy should get an evaluation. This can save lives for the patient but their family members as well. In short, in my opinion, any unexplained cardiac hypertrophia or dilation in a young person should warn a genetic test. You may be saving their life as well as their family members. This is the best way to diagnose DADN as well as other genetic cardiac disorders. Unfortunately, genetic testing remains low in the broader community, and findings across other organ systems may not be connected. Earlier diagnosis supports disease-specific cardiac care, timely planning for advanced heart failure and transplantation, and evaluation of potentially affected family members in DADEN. And time matters because progression to heart failure can be remarkably radical. Today, there's no approved disease-modifying therapy for Danone. Currently, current care is largely supportive, cardiac surveillance, arrhythmia treatment, including ICD, management of heart failure complications, and as disease advances, chemical surgery support and transplant evaluation. When our patients progress to end-stage heart failure, heart transplant is the only rescue we can offer from imminent death. But getting a patient a transplant becomes a race against both the disease clock and donor availability. A donor-hearted SCARES. It's not something I can simply prescribe when my patient needs it. Patients must be referred, evaluated, and listed, then remain alive and clinically suitable until the right donor becomes available. Across published damning cohorts, only about 20 to 25 percent ultimately receive a transplant. As a heart failure and transplant cardiologist, I think it's important to put these outcomes in context. Transplantation can be life-saving, but keep in mind that an average survival post-transplant is roughly eight to ten years. On a young person that gets a heart transplant, their lifespan is still significantly reduced after transplant. And for those who survive, transplantation carries a substantial lifeline medical burden. Patients require lifelong immunosuppression and rejection surveillance with ongoing risks of serious infection, organ toxicity, graft failure, and potentially another transplant, even in best-case scenarios. That's why transplant-free survival is such a meaningful outcome in data. More time alive with their own functioning heart without dependence on a scarce donor and without the mortality risk, lifelong burden of transplantation. That is why transplant-free survival is such a meaningful outcome in Danone. More time alive with our own functioning heart without dependance on a scarced donor and without the mortality risk and lifelong burden of transplantations. For gene therapy, the opportunity is to change that trajectory, to preserve their own heart for longer and delay or potentially avoid the need for transplantation. Data in biology provides a direct rationale for LAMP2B gene addition. Pathogenic LAMT2 variants can leave cells with absent or markedly reduced LAM2B, a lysosomal membrane protein needed for normal cellular clearance. When that recycling process is impaired, material accumulates, contributing to heart muscle injury, hypertrophy, and advanced remodeling. Essentially, cellular garbage builds up in these hearts. The result is thickened and abnormally functioning heart muscle. We know from stem cell, mouse, and more recently a clinical trial that when you replete LAM2, you need to improve clearance of this garbage as well as size and function of the heart muscle cells. RPA501 is an investigational gene therapy designed to deliver a functional LAM3B coding sequence to heart muscle cell cells through a single infusion with the aim of restoring protein expression and addressing the underlying cellular defect. What makes this approach compelling is the direct connection between the genetic defense, the protein we aim to restore, and the cellular process we aim to improve. We can test that hypothesis by measuring myocardial LAM2B expression, cardiac mass and remodeling, markers of heart muscle injury, and clinical status. With that, I'll turn it over to the Rocket team for their program update. Thank you, Eric. This is Gaurav again. So that clinical context explains the urgency of developing a treatment that addresses the underlying cause of Dana disease. So let's now turn to clinical evidence supporting our development strategy. Before reviewing the findings as shown on this slide, I want to distinguish the different patient populations. Seven males received RPA501 in phase one across two nominal dose levels. Six patients received the original phase two dose of commercial grade product, four in the initial cohorts, and two in the subsequent cohort that included the C3 inhibitor. Finally, three additional patients have received the recalibrated dose under the modified protocol. So together, these groups represent 16 treated patients. The three patients treated at the recaliberated dose are the first members of the 12-patient pivotal efficacy population, as noted on this slide. This would be cohort four and is the final pivotal cohort. The phase one principal efficacy analysis includes six patients. A seventh patient entered the trial but had advanced systolic dysfunction and underwent heart transplant approximately five months after treatment. The phase two, sorry, the phase one biopsies provide evidence of myocardial LAMP2 expression in all six patients in the principal efficacy analysis at the assessments summarized around the 12-month time point. So today, we share additional biopsy findings that extend that evidence. As the box shows on this slide, both low-dose pediatric patients demonstrated robust LAMT2 expression at their 36-month assessments. Patient number two also demonstrated robust expression at a 60-month assessment in long-term follow-up, and these findings provide additional evidence that myocardial LAM2 expression can persist for years after a single administration of RPA501. The updated Phase 1 analyses use a July 31, 2026 data cutoff and includes follow-up extending to seven years with assessment timing varying by patient and parameter. The red outlined columns here highlight LV mass index reductions, one of the two co-primary efficacy measures in the pivotal phase 2 trial, and troponin reduction, which is a key secondary measure. As summarized, all six evaluable phase 1 patients met the numerical thresholds for both pivotal responder criteria at approximately 12 months, which are at least grade 1 myocardial LAMP2 protein expression and at least a 10% reduction in LVMI. At the latest available imaging assessments, LV mass index remained below baseline in five of the six patients with reductions of at least 10% in four of them. The magnitude of these reductions observed is notable in the context of other cardiomyopathy therapies, while stabilization over multiple years also differs from the progressive increase in cardiac mass observed in DANA natural history. While that's true, NT-PROBNP, which is a marker of heart failure, and troponin, which is a marketer of cardiac injury, were reduced from baseline in all six patients at their latest follow-up. We also continue to see encouraging findings in how patients feel and function. Significantly, five of the six patients are now asymptomatic from heart failure symptoms, having improved from NYHA Class 2 to Class 1, while 1 remains at Class 2. All six patients reported an improvement of at least five points, and in most cases much more, on the Kansas City Cardiomyopathy Questionnaire, a measure of patient-reported health status. Patient 6 completed the Phase 1 study and demonstrated phenotypic improvement in the years after infusion. After the last data cutoff for this presentation, we have learned that the patient underwent cardiac transplantation approximately 4.8 years after the RPA501 infusion. This patient had been lost to long-term follow-up, and efforts are ongoing to obtain additional information and evaluate the explanted heart for transgene expression. Across the six evaluable Phase I patients, five remained transplant-free. Kaplan-Meier estimated transplant-free survival was 80% at 58.05 months. Median transplant- free survival was not reached, and median follow-up was 5.7 years with the range of 4.3 to 7.1 years. Now, turning to long-term biomarker observations, the analyses performed from data from the International Dan and Disease Registry indicate that LV mass and BNP levels increase with age in untreated males due to disease progression. In contrast, patients in the Phase I study demonstrated reduction or stabilization in BNPs and LV masks following RPA501 treatment. And of note, the prospective natural history study has now enrolled 58 patients and counting. The Phase I findings support the rationale for the pivotal endpoints as they demonstrate a clear association between cardiac protein expression and improvements in hypertrophy. All six patients in the principal efficacy analysis demonstrated myocardial LAMP2 expression of at least grade 1 and an LV mass index reduction of at last 10% on the assessments in this summary. These assessments were predominantly at 12 months with some LV mass index measurements extrapolated from staggered visits due to COVID-19. At approximately the 12-month assessment as summarized here, all six patients in the Phase 1 efficacy analysis met the numerical thresholds used for the pivotal responder definition in the phase 2 trial. Longer-term follow-up now extends from 4.3 to 7.1 years, thus providing additional context on the durability of myocardial LAMP2 expression, cardiac structure, biomarkers, and clinical status after treatment. These longer-term observations add evidence regarding the course of these responses over time, and together, they give us confidence in the biological and clinical rationale for the pivotal study, which will evaluate response at the recalibrated dose at the pre-specified 12-month assessment and beyond. Now, turning to Phase 2, six patients received the original dose of 6.7 times E to the 13 genome copies, or GC per kilogram. The preliminary clinical findings we will focus on today come from the initial four patients treated in Cohorts 1 and 2 who received an immunomodulatory regimen of rituximab, sirolimus, and steroids. And then Syed will review the safety experience and how it informed the modified treatment approach. So today, I'm excited to share these preliminary observations that extend from approximately 18 to 36 months after treatment in the initial four phase two patients. At the latest available assessments, three of the four patients in cohort one and two had LV mass index reductions of at least 10% from baseline, ranging from approximately 30 to 36 percent, and in those three patients, other measures of cardiac structure, biomarkers, and clinical status showed a mix of improvement and stabilization. The third patient had an approximately 9 percent increase in the LVMAS index, but reductions in posterior wall thickness and NT-proBNP reductions and an improvement from N-way HA Class 3 to Class 1. These preliminary findings show variable cardiac responses and should be interpreted in the context of each patient's age, growth, and disease stage. Symptoms in patient-reported health improved or remained stable across all four of these initially treated patients. So these findings provide encouraging evidence of clinical activity in the early Phase 2 trial. The pivotal efficacy analysis would be based on 12 patients treated in cohort 4 at the recalibrated dose of 3.8 E13 GC per kilogram. So now I will ask Syed to review the safety findings and how they informed the modified treatment approach. Thank you, Gaurav. The safety experience has directly informed the current treatment approach, TMA, kidney injury, and other complement-mediated complications are recognized risks of systemic AAV gene therapy. This is why the trial includes pre-specified monitoring and management protocols. In our study, TMA occurred in two of the initial four Phase II patients. Given the frequency and clinical significance of these events, we revised the protocol to include a C3 inhibitor as a component of the immunomodulatory regimen in cohort 3 to mitigate the risk of complement-mediated toxicities such as TMA. C3 inhibitor is a type of targeted medication that blocks the complement system protein C3 to potentially stop immune reactions and inflammation, although this appears to have other untoward consequences when combined with other trial-related treatment. Following RPA501 infusion, both patients who received the C3 inhibitor containing immunomodulation experienced severe adverse events, including capillary leak syndrome, resulting in the death of one of the patients. The death of a patient is a profound loss. Following these events, treatment was paused. A comprehensive root cause analysis was performed. It is likely possible that the C3 inhibition resulted in decreased opsonization of AAV and increased uptake by vascular endothelial cells with resultant vascular injury. In conjunction with global immunology and gene therapy experts, we reassessed the dose, immunomodulatory regimen, eligibility criteria, and monitoring plan. In agreement with the FDA and IDSMC, the C3 inhibitor was removed from the immunomodelatory regimens and the dose was recalibrated. These changes were intended to reduce the observed risk while retaining a dose supported by the biological and clinical evidence. We resume treatment in the pivotal cohort 4 at 3.8 times 10 to the 13 genome copies per kilogram compared with 6.7 times 10 to 13 in the original phase 2 cohorts. The phase 2 study uses in-house commercial grade products with a higher proportion of full capsids carrying the therapeutic genetic material. Because empty capsids add to total viral exposure without delivering the intended cargo, so a better full to empty capsid ratio allows a lower total dose to deliver a biologically comparable amount of functional vector. We therefore recalibrated the phase 2 dose to 3.8 times 10 to the 13 genome copies per kilogram with the goal of preserving the therapeutic profile seen at the effective Phase I dose while reducing total capsid exposure. Ultimately, the recalibration incorporated analytical characterization of the commercial grade product, including full particle content, together with the earlier clinical experience and non-clinical bridging work. The overall body of evidence, including updated Phase I clinical efficacy data as well as the non-clinical data supports the potential of rpa501 at this pre-calibrated dose to modify the underlying disease process in dannon disease furthermore today we are also reporting new data from a denon disease mouse model as they recalibrated 3.813 dose, commercial-grade Phase II material produced robust cardiac LAMP2B expression, mitigation of disease-related enzyme abnormalities, and showed vector biodistribution, comparable to the seen at a higher mid-E13 dose. These findings, considered alongside the Phase I experience, support dose selection. In addition to dose recalibration and removal of C3 inhibitor, the protocol modifications include rituximab given over three doses versus two and enhanced monitoring for TMA and capillary leak syndrome. The protocol specifies thresholds for intervention, including the use of eclosimab. Eligibility screening includes a comprehensive evaluation for underlying predisposition to TMA, including genetic testing. The first three enrolled patients in cohort four under the modified protocol were treated sequentially with at least four weeks between infusions. Each of these patients completed the required initial observation period. As of the September 4, 2026 data cutoff shown on this slide, the platelet and complement marker data extend to approximately three months after treatment. Over this period, there was no clinical or lab evidence of TMA, CLS, or other severe complement-mediated toxicities consistent with the objectives of the protocol amendments. The platelet and complement marker assessments that were part of the enhanced monitoring plan remained largely within normal limits. One patient had a transient decrease in platelet count at approximately weeks 8 to 9 without symptoms or signs of platelet dysfunction. The investigator and sponsor did not consider it clinical significant. Subsequently, it fully resolved. Reported SC5B9 values should be described only in relation to the assay range and the absence of associated clinical findings. These initial observations are encouraging. Follow-up is ongoing, and additional patients and longer observations will be important to characterize safety under the modified protocol. After reviewing the initial safety data from these three patients, both the Independent Data Monitoring Committee for the trial and the FCA confirmed that enrollment and dosing could continue under the modified protocol. I will now turn to Dr. Barry Greenberg to discuss the clinical significance of the findings and what he will be looking for as the pivotal study progresses. Barry, thank you for joining us. Thank you. What stands out to me as a clinician is the findings that we had from the initial patients that we treated between the changes in LAMP2B protein, the deficient protein in Danone disease in the heart, and the changes that we saw in cardiac structure. We performed endomyocardial biopsies over the first three years that patients had been treated with gene therapy, and what we were able to show was really remarkable because we could demonstrate that we were getting an adequate number of genes to the heart, that is the deficient gene in Danone disease, and even more importantly, that this gene was functional and producing protein. We provided evidence that the gene was producing this protein for at least three years. That's the duration of time that we did these endomyocardial biopsies And then we paired up this finding, this observation that we're now getting the deficient protein in the heart along with measures of cardiac structure, in particular, the amount of thickening of the muscle mass or cardiac hypertrophy. That's a hallmark of Dannen disease, and we were able to show that virtually hand-in-hand with the production of the Lab2 protein, we were able to see a reduction in cardiac hypertrophy. This is particularly important because we know that Dannen disease is a rapidly progressive disease for which there are no curative treatments available. At present, only cardiac transplantation is available for patients with end-stage cardiomyopathy, but that's associated with significant morbidity and mortality. As a treatment, gene therapy with RPA501 has the potential to eliminate or delay the need for cardiac transplant and it represents a significant benefit to these patients if that could be accomplished, especially when the therapy can be administered with limited or manageable toxicities. Under the modified treatment protocol, we've treated now three patients. These are in phase two, cohort four, and they were all treated safely without any evidence of TMA or complement-mediated adverse events. Taken together, the longer-term phase one clinical data that I described and the most recent safety profile that we're now seeing in phase 2 cohort 4 indicates that the value of gene therapy as a potential for Danon disease is considerable. We're looking forward to continuing this study and seeing the final outcome in these patients. At this point, I'm going to turn the floor over to Gaurav, who's going to explain the pivotal analysis and the plan to complete the study. Thank you, Barry. Throughout the development of RPA501, we have remained in close collaboration with the FDA to align on the regulatory path toward a potential BLA for this important therapy. Most recently, we received FDA agreement to complete enrollment in our updated pivotal phase 2 study with a total of 12 patients treated at the recalibrated dose. To provide a brief overview, this is a global, single-arm, open-label, registrational study with an efficacy population consisting of 12 male patients receiving commercial-grade RPA501 at the Recalibrate Dose. The clinical efficacy target is defined as at least 7 out of 12 patients meeting both pre-specified responder criteria at 12 months. Those criteria are, one, myocardial LAMP2 protein expression of at least grade 1, and two, improvement from baseline and LVMI reduction of at last 10%. A responder must meet both. We intend to use these 12-month pivotal results as the primary efficacy basis for a BLA seeking accelerated approval with priority review and, upon approval, eligibility for a pediatric rare disease priority review voucher. Any FDA approval would be based on the agency's comprehensive benefit-risk assessment of the totality of the clinical evidence and overall data package, which includes safety, efficacy, manufacturing quality. The first three patients mentioned earlier count toward the pivotal efficacy population, thus leaving nine more patients to enroll and treat. We have six sites prepared to dose with the potential to add more, and we expect to complete dosing by mid-2027. Each patient will contribute a primary assessment at 12 months after treatment. Our immediate priority is completing enrollment and dosing these patients safely. Our natural history study combines retrospective clinical history with prospective follow-up, which now has 58 patients enrolled, collecting cardiac imaging, biomarkers, heart rhythm, major clinical events, and quality of life information through a common approach across centers. Following both males and females helps us characterize the broader disease and can inform future development in additional Danone populations. As I mentioned earlier, in parallel with this clinical work, we have substantially strengthened our understanding of the Danone population itself, both the underlying epidemiology and the real-world diagnosed patient population. Shabani will now take us through the comprehensive analyses behind our epidemiology estimates, what we have learned about where diagnosed patients are receiving care, and how these insights are informing our strategy to accelerate diagnosis as we move toward launch. Thank you, Gaurav. Gannin disease represents a meaningful initial opportunity in males with HCM. A broader opportunity exists across males who are earlier in the disease course. Beyond males, exists a female patient population with high unmet need. To size that population rigorously, we approach the question from two independent directions, one starting with genotype and the other with clinical phenotype. Both approaches converged on approximately 10,000 to 11,000 people living with Dynan disease in the U.S., about 4,000 males and 6,000-7,000 females. Let me start with the genotype analysis. We have conducted the most comprehensive molecular characterization of Dynon disease to date, integrating more than 450 patient records with pathogenic or likely pathogenic LAM2 variants across six complementary data sets. This provides the foundation for a genotype-based prevalence estimate. We started with classic loss-of-function variants, which are commonly reported in Dynan disease. These variants are well represented among approximately 315,000 individuals in genome 80, giving us a strong population genetics anchor. But they account for only 62% of pathogenic or likely pathogenic LAM2 variants identified in confirmed dannen cases. We therefore use a molecular characterization to scale from this anchor and estimate the full dannen genotype positive population. We then approach the same question independently from the phenotype. Here, we built a comprehensive sex-specific natural history model informed by more than 700 Danone patients, as well as analysis of real-world Danone disease patient data. We selected HCM as a population anchor because it is the predominant cardiac phenotype in Danone Disease, and robust population-level HCM data are available. We applied age-adjusted estimates of Danone prevalence within HCM from published studies separately for males and females to estimate the Danone population with an HCM phenotype history. But HCM does not capture the full Danone population. A significant proportion of patients have not yet developed cardiomyopathy, while others may have progressed to dilated cardiomyopathic phenotype. We therefore use real-world Danone patient data and a natural history model to estimate these additional patients and scale from the HCM anchor to the full Dannon population across all stages of disease. So, two independent approaches, one anchored in population genetics and one in clinical phenotype, converge on approximately 10,000 to 11,000 people living with Dannon disease in the U.S. Applying the analysis to the United States and EU5 supports an estimated population of more than 20,000. These are model estimates that we will continue to refine. Our initial commercial focus is narrower. Danone males with HCM consistent with the population being studied in a pivotal program. Treatment eligibility within that population will depend on clinical evidence and approved label. Now, let me move from estimated prevalence to patients we can already see in the real world. On a cumulative basis, we have identified more than 900 patients with confirmed Danone diagnosis across approximately 300 U.S. institutions. This is a deduplicated unique patient count compiled across multiple sources, including claims and electronic health records, genetic testing data, HCP reporting, and our field intelligence. Most were captured over the last five years. As a cumulative count of diagnosed patients, it includes those who have undergone heart transplantation, as well as those who may have subsequently died. Importantly, we know not only how many patients have been identified, but where they receive care. The top 100 institutions account for approximately 60% of identified patients. Yet, these 900 patients represent less than 10% of our estimated U.S. Dianin population. The significant diagnosis gap is consistent with the low rates of genetic testing in cardiomyopathy today, ranging from only 1.6% in adult HCM to approximately 14% in pediatric cardiomyopathie. We also see evidence that underdiagnosis is greater among females. Females represent 55% of identified patients, a lower proportion than we estimate in the underlying Danone population. So the patients are there. We already see a meaningful diagnose base and a clearer understanding of where they have been treated. The opportunity now is to build from this foundation, expanding into the broader cardiology community to identify the many patients who remain undiagnosed. To narrow the diagnosis gap, a strategy directly addresses the barriers that Dr. Adler described earlier. First, patients are dispersed across the broader cardiology community, where genetic evaluation is not yet systematic. We need to educate and activate cardiologists to increase testing in young HCM patients and to recognize the Danone signature as a trigger for heightened urgency. Second, genetic counselor capacity and peer requirements can create barriers to testing. ROCKET's Mission Genome Program helps simplify the path to genetic confirmation through no-cost testing and counseling. Third, we want to empower young patients with unexplained cardiomyopathy and their families to ask, could this have a genetic cause? And advocate for evaluation. And fourth, cascade testing can be a force multiplier, with each diagnosis creating an opportunity to identify additional affected family members. I'm encouraged by the broader environment. HCM guidelines increasingly support genetic testing, while targeted therapies and genotype-driven trials are making genetic diagnosis increasingly important. Our strategy is designed to build on that momentum and identify patients earlier and more systematically. Our initial focus is males with HCM, with U.S. as our first commercial priority. We estimate this initial indication could support more than $1 billion in global peak annual revenue, reflecting both the addressable patient opportunity and the potential for premium value in a catastrophic rare disease with no approved disease-modifying therapy. That estimate incorporates diagnosis, treatment eligibility, access, and achievable annual treatment volume. This also indicates targeted expansion into priority international markets with launch sequencing informed by access and market opportunity. Beyond the initial indication, there is potential to reach additional down in populations over time, including females with earlier onset HCM, males before HCM develops, and females with later onset cardiomyopathy. any expansion into these populations will be guided by supporting clinical evidence and regulatory alignment we are already laying important groundwork for these potential opportunities our natural history work is deepening our understanding of disease trajectory and helping inform patient selection and future clinical development the epidemiology and patient identification work that we've done to date allows us to understand where the currently diagnosed patients are or have been managed. We also have an understanding of the broader HCP community who are likely to manage Danone patients not yet diagnosed. Together, these potential populations could expand the global peak annual opportunity to more than $2 billion. With that, I'll turn it back to Gaurav. The long-term clinical data from the Phase I study demonstrating sustained LAMP2 protein expression and improvement or stabilization of cardiac hypertrophy, biomarkers of cardiac injury and stress, and heart failure symptoms are indicative of RPA501's potential to transform the natural history of Danden disease. The early efficacy data from the Phase 2 study further corroborates RPA501's therapeutic potential. The initial experience in the first three Cohort 4 patients treated under the modified protocol is encouraging. No DMA, no capillary leak syndrome, and no other severe toxicities through the current follow-up period. These findings are consistent with the intended effect of the safety optimizations, although additional patients and longer follow-up are needed to characterize the safety profile fully. Most recently, we aligned with the FDA on the updated Pivotal Phase 2 study, which will include a total of 12 patients treated at the recalibrated dose. As mentioned, the efficacy target is at least seven responders at 12 months, with each responder required to meet both criteria of protein and LV mass index reduction. We plan to complete treatment of the remaining nine patients by mid-2027. That is our immediate priority at Rocket as we work toward the pivotal analysis and a potential submission seeking accelerated approval. Our commercial work has always so advanced. We have a clear understanding of the damage population, the institutions caring for patients, and the steps needed to connect diagnosis with potential treatment. That work provides a focused basis for commercial preparation as development progresses. So, the evidence shared today reinforces our belief that RPA501 has the potential to alter the course of data disease. Phase 1 results show sustained myocardial LAMP2 expression and improvement or stabilization across measures of cardiac hypertrophy, injury, stress, and heart failure syndromes. Preliminary Phase 2 findings support continued clinical activity, and the initial cohort for safety experience supports continued enrollment under the modified protocol. As announced today, based on our current operating plan, our existing resources, together with the initial $35 million funded under the Hercules facility, are expected to fund operations into the third quarter of 2028, with additional borrowings under the facility extending our cash runway into 2029. Thank you especially to the patients and families participating in these studies, to the investigators and study teams, and Drs. Adler and Greenberg for their perspectives today. And we can now open the call to Q&A. Thank You very much. Certainly. Everyone at this time will be conducting a question and answer session. If you have any questions or comments, please press star 1 on your phone at this time. We do ask that while posing your question, please pick up your handset if you're listening on speakerphone to provide optimum sound quality. And once again, if you have any questions or comments, please press star 1 on your phone. Please hold while we poll for questions. Your first question is coming from Andrew Tsai from Jefferies. Your line is live. Hey, this is Matt Barkus calling in for Andrew Tsau. Congrats on the Danon update. Just a couple questions from me. It's pretty clear that with this new efficacy target of only needing seven of the 12 patients to respond, I'm just curious if there's a chance for you to provide a rolling update to the top line. For instance, like if the next seven patients achieve the target, are you able to announce study success early, or are you going to have to continue to wait for all 12 patients to have the 12-month follow-up. And then also, if you can just talk a little bit about your natural history study, is this going to be a formal external comparator to the filing, or is this just a supporting context? Thanks. Thank you, Matt. For the rolling update, I can tell you what we've done in the past, certainly in former trials like with Fanconi anemia when we hit five out of 12, we did announce the top line. However, that's a discussion that we will have with the FDA as to exactly what top line means. So we will wait on answering that until further conversations and as the trial progresses. And regarding the natural history study, yes, the external natural history study is important to verify and authenticate the assumptions that form the statistical basis for this pivotal design. In other words, do patients get worse with left ventricular mass index and troponin and other markers? Just as we've seen in the first few patients, we're going to confirm this with the larger group of patients. But it is not an external comparator. The comparator for these patients' outcome is the patient's pretreatment baseline of LV mass index, protein, troponins, et cetera, right? So the natural history study as a fortifier, but not an external comparator. Very clear. Thanks. Thank you. Your next question is coming from Rowana Ruiz from Learing Partners. Your line is live. Hi, everyone. Afternoon. A couple from me was wondering if looking at the, I think you were talking about phase one patient parameters for up to seven years. Could you elaborate a bit about that one patient that underwent cardiac transplantation? I think it was around 4.8 years after treatment. I know it might have been lost to follow-up, but any additional color there? What do you think might have happened and any implications on durability there? I'll pass this over to Jonathan. Thanks for the question. We are still gathering information regarding the clinical deterioration So, it's very premature to state anything conclusively. We do know that this patient had a significant arrhythmic component to his Danone presentation, which may have exacerbated some aspects of arrhythmia tracts or heart failure that were not necessarily reversible by the gene therapy, although that's quite speculative. We do know that this patient had improvement in a range of parameters and very extensive symptom resolution during the initial years following therapy. We do intend to update this more significantly. I think this emphasizes that, you know, there is likely to be a spectrum for the duration of benefit, and this patient in all likelihood represents the short end of that spectrum. Obviously we have the majority of patients who continue to do well multiple years after therapy, and we will obviously have much greater information with respect to the overall bell curve for durability, which is likely five years at the low end, and we still are awaiting the remainder. Median progression-free survival, median transplant-free survival has not been reached, and that's really about all we can state at this juncture. Okay. That helps. And I want to dig into the, I think you mentioned the 900 patients diagnosed across 300 US institutions. Any thoughts on what proportion of them may have already had heart transplants or might be too far along in disease to potentially receive gene therapy? Hi. As I mentioned, this includes those who have already undergone heart transplant as well as those who may have subsequently died. These have been cumulatively diagnosed over the past five years. We only at this point know the male versus female breakdown. As as I mentioned, we don't have the full status of the heart transplantation. I think what we can say from this analysis is there are real dead end patients that are being diagnosed on an ongoing basis. We know where these patients are being treated or have been treated. We're also building the foundation for sustainable long-term patient finding efforts. Considering the rapid disease progression, some of the patients identified today may deteriorate to advanced heart failure, unfortunately may succumb to the disease, and some may be transplanted in the future. So our main focus as we ramp up our efforts is to educate on Dannon, accelerate diagnosis, so we see identified patient numbers grow up, grow, and leading up to the approval and sustaining the growth from initial launch to peak. Understood. Thanks a lot. Thank you. Your next question is coming from Josh Shimmer from Kantor. Your line is live. Thanks so much for taking the questions, and congrats with all the progress, both clinically and commercially. A couple questions. First, for the efforts in the female Danone patient population, what are the gating steps before you're able to start a clinical trial? In that population, do you have to have a certain safety experience from the trial in the boys, or is it on a different trajectory? And then if you're projecting peak sales north of a billion for the Danone program, I just want to clarify first that's just in boys, number one. And number two, given the cadence at which you've been identifying patients thus far, it would seem like you'd have to have quite a significant step up to be able to treat, say, 400 to 500 patients in any given year. So maybe you can talk a little bit towards those efforts and what gives you the confidence that you'll be ableto really ramp up the patient identification efforts as substantially as you've indicated. Thank you. Thank you, Josh. So I'll answer the first question and then hand it to Shabani. So for the female Dannon population, we know that there's a very high unmet need. We have a lot of inbound interest from the community. I will say that the FDA has already been engaging with us on developing a female trial. We'll probably start in a more severe population that mimics men or boys really with Dannon disease. And in fact, the FDA in their correspondence has indicated proactively that we should start a female trial as soon as possible and even look at pre-symptomatic males. So, I think for this disease, the FDA is super supportive of the development as fast as possible. Regarding how we get to the opportunity, Candidate Shabana. Hi. Let me just also clarify about what we mean by peak and how we get there. So first of all, the RPA 501 revenue model is based on a sizable prevalent patient pool with a treatment window of several years. is not a birth incidence-based model, nor will it be dependent on any type of newborn screening. We estimate the revenue curve to be a gradual time-to-peak with a steady adoption curve, a flatter trajectory at peak over multiple years before a gradual ramp down to diagnose incident patients. So when we think of the gene therapy treatable poll over the next several years after launch with a study adoption curve it's going to come from three places. Existing prevalent Danone males with HCM who have preserved ejection fraction and the disease is not yet at end stage, which, as I mentioned, gives us a treatment window of several years. Fresh pool of Danone male transitioning from pre-HCM stage to HCM stage each year. And third is increasing diagnosis rates that enable identification of the above patient in addition to those who are already diagnosed. A rocket plans to intensify the efforts on Dannon education and genetic testing to drive an order of magnitude increase in Dannon diagnosis rates. We have external industry benchmarks as well as cardiac ecosystem tailwinds such as what we've seen with ATTR as well other analogs that will act as a catalyst on our Dannon diagnosis mission. In addition, potential regulatory approval of disease-modifying treatments will also meaningfully accelerate Dannon diagnosis. And we have already started the efforts, because we know where current diagnosed patients are. We have a very good idea about where future patients could lie, mainly in a very select group of pediatric cardiologists, and that's the work that we plan to take on over the next couple of years leading up to the launch, so we can sustain not only an initial bolus, but also over time. Thank you. Thank you, your next question is coming from Patrick Dolezal from LifeSci Capital. Your line is live. Hi, thanks for taking the questions. Is it your expectation that manufacturing is the primary driver of the meaningful safety improvements we're seeing in cohort four and can you help quantify the magnitude of those improvements with respect to full capsid proportion? And then second question, what are the biologic drivers behind the improved manufacturing process at the go-forward dose level driving similar transduction and biodistribution as at the prior dose level, which was higher on an absolute basis? I think you commented on that in some of the preclinical studies. Thanks. Yes. So I would say that the improvements in safety are a combination. Well, first of all, removing the C3 inhibitor, which was together with the torrential rain of AAV9, is problematic really for endothelial cell injury. And in removing that, we certainly were able to remove the capillary leak issues that we saw earlier. Now, with regard to the TMA that we see, saw in the earliest part of the phase two trial, I would say that the recalibration to account for higher potency in the phase 2 product versus phase 1 that was predicted is really the recalibration that did the trick. Our ratio of full empty improved from less than 50% to over 70 percent consistently and reliably. And I would say also, Patrick, that the conviction that we have that the recalibrated dose is truly a recalibrated dose and not just a dose reduction is threefold. One, based on the clinical data that we saw in the treated patients and the three patients treated safely mimics and mirrors the phase one data in the patient population that had a 100% response rate, as we indicated earlier. Secondly, Cyan mentioned some preclinical evidence in a mouse model that compared the 3.8 E13 new dose with the older low dose, and that comparison confirmed equal impact in mice at those two different levels or two different products. And finally, manufacturing enhancements increased the proportion of full capsids, as we mentioned, resulting in greater biological activity in laboratory testing compared with the earlier clinical material in Phase 1, especially at higher dosing levels. And while these potency assays are not designed to predict clinical effectiveness, the findings are consistent with the improved quality profile of the Phase 2 product. And that last point I made is based on new information that has emerged in our internal analyses recently. And I think that should address both of your questions, correct, Patrick? Yep, super helpful. Thank you. Thank you, your next question is coming from Tara Bancroft from T.D. Cowan. Your line is live. This is Ekenna on for Tara. Thank-you guys for the presentation. I had a couple of questions. You know, you guys mentioned that the PIVOTL study will use Lab 2 expression and a 10 percent reduction in LVMI as a key endpoint. What data or clinical rationale support that selection of the 10 percent LVMi reduction threshold? And will the assessment for LVMy be evaluated by a central imaging core? And then I'll follow up after that. me ask Jonathan to answer this one so I'll start with the I'll start with C last part of the question all of the imaging as well as immunohistochemistry will be evaluated at a central facility in in a blinded fashion by independent readers, and that's true both for echocardiogram and MR on the imaging side, as well as obviously the lab two histology. I think our phase one data in which really all of the six patients who did not have reduced left ventricular ejection fraction and stage cardiomyopathy at baseline really met those endpoints at 12 months and that those 12-month milestones were then indicative of sustained improvements. In most cases, patients continued to improve beyond 12 months for most parameters. So we believe that these thresholds really are indicative of improvements that are sustained and likely to continue. Additionally, we also have accumulating natural history data that the progression of hypertrophy in males continues, it doesn't, it really, these hearts thicken at a demonstrable rate per year, and so any degree of stabilization or even modest improvement represents a substantial deviation from the relentless progression of the hypertrophy that has been well documented and we'll continue to further document in male individuals with DEN and disease. All right. Thank you. And I had a really quick follow-up on the rolling disclosure. You guys noted that the timing and definition of the top line are under discussion with FDA. Could you share any more color on what the specific options are that are being discussed and what would need to be true to report results before all 12 patients reach that 12-month assessment. So what we described today was a protocol-defined efficacy target of seven patients out of 12. We are in the process of finalizing a statistical analysis plan, which will mimic that, but a lot of these definitions are in discussion, so I can't really answer further. I will note that based on recent FDA feedback, we're going to be very thoughtful about how we roll that answer out, given that the SAP is so critical to the final outcome with the FDA. So right now, the protocol-defined efficacy output is 7 out of 12, but further information once we finalize the SAP and other details of top line, et cetera. Thank you. Thank you, your next question is coming from Tess Romero from JP Morgan. Your line is live. Hi team, thanks so much for this presentation and for taking our question. I wanted to double click here on just what the feedback is that you're receiving from the Dannon community on RPA501, their interest in receiving gene therapy and specifically their comfort around the safety profile. How do they think about what would be an acceptable level of risk here? And, you know, how do you think about it? Thank you. The Dannon disease community has been very courageous and very patient as we go through this development plan. I would say that, in fact, the support level from the Dannon Foundation and parents and other caregivers has been unprecedented in our programs. I think that Danone disease is such a rapidly progressing disease with guaranteed fatality if these boys are not transplanted. And the community is willing to take more risks than they would otherwise if it weren't such a devastating fatal disease. So even with the safety events we saw last year, we saw the community rally around continuing the trial. Today, even though we only need to enroll nine more patients, I should use this opportunity to explain that there are way, way more patients that are waiting, so we need to get this done as soon as possible. In general, they've been enormously supportive of the trial because the alternatives are bleak. Thank you. Thank you, your next question is coming from Richard Law from Goldman Sachs. Your line is live. Hi, this is Jane. I'm for Rich. Thank you so much for taking my questions. So my first question is for the KOL, and then I have two questions for the management team. Hi, Dr. Adler. Do you believe there's a threshold in LAMP2 expression that is needed to have clinical benefit? And for patients whose LAMPs2 expression decrease over time to low levels or back to baseline, how durable could the initial clinical effects last? Yeah, so Dr. Adler is not on the Q&A here, so this is Gaurav. I'll answer. So for the LAMP2 expression, what we call a grade 1 increase, in other words, a visual increase by grade 1 or more, is not an exact percentage, but in general, for the patients who have responded at the grade 1 level, it's been about 10% to 25% protein expression. Grade 2 is approximately 25% to 50%. Grade 3 is 50% to 75%. And grade 4 would be up to 100%. So, in terms of how durable this effect should be, I would say I would divide it up between protein expression, LE mass index, and other lab biomarkers. Protein expression should remain intact for many years to come. We see it intact for at least five years as we showed earlier in two patients, the second patient being new information. The cardiomyocytes that we all are born with are mostly the cardiomyocytes that we keep the rest of our lives. We don't recycle them. So the episomal expression should remain for long term in the heart and it has and I was happy to report that today. For LV mass index, remember that these hargs grow, and on average, we've shown that they grow about 8% per year. So we expect reductions in the first year, but over time, even if we just see stabilization over two, three, four, five years, that's much better than the expected increase we would see, you know, 1.08 times 1.28 times 1,808 year after year can be additive and or multiplicative, right? So versus that, even stabilization alone is a big win. And I think the FDA has recognized that in how we're thinking about the long-term follow-up and the full approval pathway here. For the troponins and the BNP, the lower the BnP, the better the heart failure symptoms, and the lower of the troponen, the less cardiac injury. And we've seen both of those sustained three to five plus years as we showed on the tables today. Okay, thank you. So for the second question, I want to follow up on earlier discussions on how many patients qualify for treatment. Let's use the 900 patients that have been identified. Can you please walk us through, like, how many of these patients would be qualified for treatment and the qualifiers you would use, like gender, age, within treatment window? And then also, like, how many patients do you believe will require C5 complement therapy in the commercial setting? Thanks for the question. So to be clear, the 900 patient is a record. It's a recorded count in an internal database. We don't have all the information there. This is done merely to find patients for the clinical trial and know where the sites are where patients can be found. So it wasn't a big commercial endeavor to find these patients. It was pretty straightforward and simple. And the reason I say that is the answer to your question is something that we'll provide a little bit closer to launch. We're not quite ready. But as Servani had said earlier, there's slightly less than half males, slightly higher than half females, and we don't have the transplant status yet. So addressable market, et cetera, is something that we will talk through as we get closer to BLA. The goal today was merely to identify the patients out there and basically to reiterate that while Danon diagnosis, truly diagnosed rates are rare to find, they're out there, and they're quite prevalent in the U.S. more than folks might have thought. Got it. Thank you. Yeah. Go ahead. Oh, my last question is, like, why did you set the efficacy target as 7 out of 12 responders? Is there any reason to believe there are subpopulations not able to benefit? Thank you. Based on the phase 1, we saw all patients respond. I think that this gives us some room. It's just how the assumptions differ from treated patients versus natural history. If you look back at, I think, slide 10 on the presentation, we know that LVMAS increases year after year, and we've seen this in the retrospective and prospective cohorts that we've studied. And we knowthat LVMAs index in treated patients goes down here at least year after year. We see that sustained over time, sort of creates this alligator mouth, right, top versus bottom. And the 7 out of 12 target is based on what it takes to read out as a positive trial, and that's based on these assumptions. This is Jonathan. Yeah, the null hypothesis is very stark. Patients will not spontaneously express LAMP2, and patients will not simultaneously have hypertrophy stabilize or regress. So, the 7 of 12 provides us with very good statistical robustness. Obviously, we hope that everyone benefits, and everything that we've been discussing today and everything we've doing up until this day is to optimize the potential for the largest possible benefit. I see. Thank you, and congrats again. Thank you. Thank you! Your next question is coming from Jason Zeminski from Bank of America. Your line is live. Great! Congrats on the program, and thanks so much for taking our questions. I guess if I could ask one on the patient of the original Phase II cohort whose LV mass index increased by around 9%. Have you identified anything that differentiated the patient from the three with the 30% reductions? I know you mentioned age might be a factor, but just in terms of trying to understand whether there may be an underlying phenotype, I know you're not ready to comment on the addressable population, but does this change what you've learned about how broad a treatment this could ultimately be? great question so we have not done a full analysis of either genotypic or phenotypic difference differentiation of responders versus the couple who didn't respond so far first of all this patient at the most recent visit which was two years out was 15 years old which means he started at 13 a time of heart growth rapid heart growth so that could be part of it, we do see reductions in left posterior wall diameter, for example. So, you know, the other thing, though, I would say is that this was the earlier phase two before the recalibrated dose and also with a different immune modulation regimen. And we believe that the new recalibrated dose plus the revised regimen is going to be better both for safety and is going to hopefully recapitulate the efficacy that we saw in phase one. That's why we designed it that way. So while this is preliminary, it's early phase two data, and it's reassuring in the sense that we're continuing to see responders with gene therapy, but it's not an early read on the pivotal phase two in any way, shape, or form. Great. Thanks for the color. And then maybe just a quick one on enrollment. I think you previously discussed about having identified possible candidates for the nine remaining patients for the study. Maybe what are some of the gating factors to getting these patients into the trial? Is there a reasonable scenario where mid-2027 could be a conservative estimate? Our official guidance is mid-27. There is a three-month troponin run-in, as there was, you know, previously, so we do have to do that for all patients. There's logistics, there's vaccines, and there's just coordination across a limited number of sites. So we are moving as rapidly as we can, but the guidance is mid-2027. Perfect. Thanks for answering the questions. Thank you. Your next question is coming from Yeon Song from Wedbush Securities. Your line is live. Hi. Good afternoon. Thank you very much for taking the questions, and congratulations on the update. A follow-up question on the initial four patients. Although I understand that the dose was different from the recalibrated dose, I wanted to confirm that the material is the same commercial-scale manufacturing material. So is there any way that those data from those four patients could potentially contribute to the final statistical analysis if for any reason you are not able to reach that seven patients out of 12 patients to be responders. And also, the second question is, given that the Phase 2 study is a global study, and can you remind us, have you talked about your plan for XUS and maybe your discussion with the FDA on the requirement of full approval, please? Thank you very much. Great. Thank you. So the official pivotal analysis is going to be based on cohort four. Everything else is supportive, right? So I think it's hard to extrapolate beyond that statement. The phase two pivotal study is cohort four with 12 patients. The responders in both phase one and phase two form the larger data package for both safety and efficacy and will be considered as a totality of evidence but is not part of the pivotal population. For X-U.S., I would say that right now we're focused on a U.S. launch and approval, as Sirbani outlined. We have talked to the EMA in the past, but really as a small company, our focus is strongly going to be in the U.K. We have enrolled and will enroll from Europe, but U. S. is the focus. Thank you. Thank you. And our final question comes from Mike Oles from Morgan Stanley. Your line is live. Good afternoon. Thanks for taking the question, and thanks for all the updates as well. Maybe just a quick follow-up on some of the safety questions. Can you just remind us when you typically saw the TMA and capillary leak syndrome from the prior cohorts, and then just how that compares to the follow-up you have so far in the cohort four. Thanks. Yeah, thanks, Mike. We saw TMA and capillary leak, as you mentioned. This is a very different experience, what we're seeing at cohort four, it's much more similar to what we saw in phase one. There certainly is evidence of transduction. You saw in the graphs that we showed that SC5B9, for example, does go up, so we do know that the virus is getting in. And just following some of the early cardiac and liver markers, we're not giving water. There is a torrential rate of AAV9 that these patients are experiencing, but it's a different experience from the early phase two where we saw TMA and capillary leak, so much more reassuring. Great. Thank you. Congrats again. Thank you, Mike. Thank you. This concludes today's program. Thank you for your participation. You may now disconnect.