Good morning, everyone, and thank you for joining us at Needham & Company's 24th Annual Healthcare Conference. My name is Ethan Markowski, and I'm a member of the Biotech Research Team here at Needham. It is my pleasure to have Gaurav Shah, CEO of Rocket Pharmaceuticals, with me today. As a reminder, any viewers who are watching through our conference portal are able to submit questions via the Ask Question feature below the video feed. Thanks for joining us today, Gaurav.
Ethan, good to see you. Thanks for having us here today.
Yeah, and before we dive in, I do want to spend a minute or two on the current market conditions we're seeing. I think they're pretty hard to ignore. Maybe if you could just start off by addressing any potential impact tariffs might have on Rocket, as well as the recent leadership changes at the FDA.
Yeah, so I'm very happy to state on the tariff side that the majority of what we do is actually sourced from the U.S. Certainly on the AAV side, which is the biggest part of the valuation, we feel pretty good about that change. I think we maybe were lucky, but we're lucky to have most of that sourced within the U.S., so I don't think there's going to be too much impact at the moment based on what we know. On the FDA situation, look, Peter Marks was a force for a long time. I would say that in general, his philosophy has already trickled through the CBER division. All of our interactions on all of our programs have been directly with clinical or CMC reviewers or their respective division heads.
The agreement on the programs was not with Peter; it was with the clinical and CMC reviewers directly. Although Peter was a big force behind the sentiment, I think that sentiment has largely infiltrated throughout the FDA in a very positive way, and I think it's here to stay. I think the new administration, at least for pediatric innovative diseases, is pretty proactive and supportive. While it's unfortunate about the big shift, timing-wise, it's not ideal and certainly affects markets, we don't think it specifically impacts any of the Rocket programs.
Okay, with that out of the way, we can start to dive really more into the company. For those who may be new to the story, would you just mind giving an overview of the company and where things at Rocket stand today?
Yeah, great. We're a gene therapy company, I would say one of the few that are remaining and moving programs forward. We focus on rare genetic diseases. We do everything from discovery to manufacturing, which we have in-house, to commercial. We're looking to correct the root causes of complex diseases. We have two platforms, both a lentiviral and an AAV platform. I think a lot of the secret of success in gene therapy is in asset selection. You have to select assets that are ideally first, best, and only in class, number one. Number two, where there's a direct mechanism of action where you're affecting the protein that's responsible for the full spectrum of disease, not a chaperone protein, not a protein in a wrong cell.
It's got to be direct and targeted, and it's got to be in conditions where they're either devastating or fatal, especially early in childhood, because that's when the value of gene therapy can be realized, not just by regulators, but by payers as well. Finally, we want to pick diseases that have a reasonable market size, so that obviously creates a nice business opportunity and a growing one over time. We're happy to say that that's how we've selected our assets in both Lenti and AAV. On the AAV side, it's the cardiac portfolio. It's increasingly become our focus and where we're putting our resources. Our lead program there is Danon disease, which is a devastating heart failure syndrome that is associated with autophagy, which is the vacuum cleaner of cells. More on that when we come back.
Danon disease is now in the midst of a pivotal trial aimed toward an accelerated approval pathway. The second program is PKP2 arrhythmogenic cardiomyopathy, which is an arrhythmia, as you can tell by the name, but it's one that's associated with defects in the desmosome and the intracardiac junction. It's actually our biggest cardiac opportunity with at least 50,000 patients in the U.S. and Europe in terms of total prevalence. Some other companies have reported as high as 100,000 or higher, so that's really a big market opportunity. The third one is BAG3, which is a dilated cardiomyopathy, and that's one where we're filing an IND pretty soon. There are only three or four buckets of cardiomyopathy. There are hypertrophic, there's arrhythmogenic, there's dilated, there's others, but we hit one of each of these biggest ones with these three programs, so we're very excited about this portfolio.
On the lengthy side, we have a hematology portfolio that is ex vivo, and these are programs that are also in pivotal and/or filing stage. Fanconi anemia is the biggest one. It's a bone marrow failure syndrome. Leukocyte adhesion deficiency is one where we're in the midst of filing with the FDA, which is a life-threatening infectious disorder. Then pyruvate kinase deficiency, which is going to enter a pivotal trial, although we're stage gating that a little bit, waiting for the rest of the programs to move forward. That's the pipeline. Like I said, we're integrated in terms of everything from discovery to commercial. We just hired a Head of Commercial and Medical Affairs yesterday with immense experience in rare cardiac and hematology diseases, so we're very excited about that.
I will say, a question you did not ask, but I think it is important to state that we are very diligent in terms of capital stewardship. We know the importance of preserving resources, especially in times like this, to extend our cash revenue as far as possible. We are disciplined in how we are spending. We are focusing on the cardiac portfolio while maximizing the upside of the lentiviral portfolio as well, including two potential PRVs. We are very thoughtful about this. In fact, I could say that with some reprioritization and focus, we are able to extend our cash runway through 2026 now, which is really good news.
Yeah, no, and I think, correct me if I'm wrong, it was previously through 3Q 2026?
Yeah, previously we had said into the second half, but I'm happy to say that it gets us much, much further now.
Yeah, no, preserving cash is definitely a very important strategy right now. You did touch on the fact that Rocket does develop both AAV and LV gene therapies, which is kind of pretty unique. What synergies have you identified by developing both types of gene therapies?
A lot of the analytics and methodologies on the assays are pretty similar, and we can read through and read across, especially things like stability and sterility. In fact, some of the learnings from LAD, for example, can be applied not just to Fanconi, but how we approach the Danon disease CMC book of work. There are synergies there for sure. I think also if we can crack the lengthy code, I believe that the AAV programs are much simpler. It's just a vector, it's off the shelf. Starting the expertise in lengthy, although it's harder, is going to make the rest of the portfolio much easier.
In terms of the regulatory interactions, it's often the same CMC and review teams across lentiviral and AAV, and we're able to leverage our learnings across in that way as well, in terms of regulatory guidance and being innovative regulatory-wise, but also being respectful of what the FDA wants.
Great. I do want to move into Danon, which, as you mentioned, is your lead AAV program. You presented some pretty remarkable long-term data at AHA last year. Would you mind just providing some of your key takeaways from those results?
Yeah, this was very exciting because for the first time, maybe in any gene therapy, but certainly in cardiac gene therapy, we're looking at protein expression out several years. Our first patient in Danon showed robust protein expression at five years. Every single patient who was evaluable at one year or longer had protein expression. They had mechanistic improvements in the vacuole, so vacuole reduction that's visible on both H&E and electron microscope. We saw improvements in the lab markers like troponin and BNP across the board. We saw reductions in left ventricular mass index, both at 12 months and long-term for these patients. I think the long-term median was in the mid 20% range, so pretty remarkable. This was sustained out to four and a half years in one patient. We saw long-term safety as well.
Also in terms of how these patients were feeling and functioning, we saw improvements in NYHA class from two to one, which means from symptomatic to asymptomatic in every single patient. The KCCQ scores, which is how patients feel and report they feel, improved dramatically in some patients with a median in the mid 20s as well, whereas most approvals in the cardiac world have been associated with much smaller improvements in quality of life. Most remarkably, some of the patients are 23-25 years old, and they're going to school, and they're working full-time.
In fact, we know that one boy who did very well in the gene therapy trial is now very fortunate and taking care of his brother who was transplanted and unfortunately is not in the same state as the treated brother is, as well as his mother who also has Danon disease. Those anecdotes really show the potential, the transformative potential of gene therapy that you can only do when you're replacing faulty DNA with correct DNA. We are very excited about those results. They were presented in the New England Journal of Medicine, and they set the stage for the phase II. I will say also that if we had looked at just the phase I, all six of six patients would have met the primary endpoint, which is protein expression plus LV mass index reduction by 10% or greater.
I'm not saying that's what's going to happen in phase II, but it sets up a nice story for phase II.
No, definitely. As you mentioned, you are in that ongoing pivotal study. One of the most common investor questions we always get is, when should dosing be wrapped up, timing of potential pivotal readout? Could you just maybe take the time to remind investors why there is a lag time between enrollment and dosing and any updates you could give there?
Yeah, absolutely. We did announce at the end of last year that patients were enrolled. The enrollment to treatment time is unfortunately a little bit cumbersome, and it's very specific to the trial. It's not going to happen in the commercial world, but that's because, number one, and there's several reasons, right? Number one, these patients require immunomodulation, so that takes time to set up and source appropriately. Number two, there's some vaccines involved in immunomodulation, which also takes time. There's a troponin run-in, number three, of three months that the FDA mandates. We have six sites going on. We want to treat patients at the different sites. Instead of putting everyone at one site, we try to stagger them. That takes a little bit of time.
Those patients who are treated at a single site, who's treating more than one patient, they want to stagger a little bit as well. All this, along with the holidays, has added the time up, which I understand is not ideal, but it is getting done. It is moving forward. We continue to anticipate an efficacy readout in the first half of 2026. I just want to make sure we're in 2025 now, so 2026 is next year, correct?
Yeah, that sounds right. I think you did mention on kind of the favorable study design that you guys got from the FDA, but can you also just remind us what requirements are there to move from accelerated to full approval in this scenario?
Yeah, one of the reasons that it took a little while for us to get the alignment with the FDA a while ago was that they actually were collaborative, and they wanted to help us understand and nail down both the accelerated approval pathway as well as the full approval. What that means is that we agreed that the same trial that's being run now could be used potentially for the full approval. They wanted to make sure that our secondary endpoints for this trial could become the primary endpoints for the full approval. They're pretty cumbersome, and that's part of the robust nature of the trial as well. In our interactions with them, using some of the secondary endpoints for the full approval out to five years was a potential path forward for full approval.
Obviously, we'll get 100% confirmation of that at the time of BLA filing, but that's been the agreement to date.
Great. You guys have also guided to providing a general update on the Danon program in the first half of this year. What kind of information should we expect to see there? What can you disclose?
Yeah, so we're very excited about that update. First of all, we will update on where we are on the trial. Number two, we'll talk about the statistical plan that we've agreed with FDA in terms of what kind of responder rate and how long, et cetera, that we need to follow these patients for. I think that'll provide some clarity. Third, we're going to get into a little bit more into how we're setting up the market for commercialization, how many patients at least that are reported, how we're thinking about the addressable patients, how we're looking in a more robust way at top-down analysis, such as frequency of LAMP2 mutations out of HCM, as well as genomics database queries.
We're going to put all that together to provide a more comprehensive look at the true Danon market than we have to date, and we're excited about providing that.
It may or may not be part of that update as well, but obviously entering closer to commercial stage here. Could you walk us through maybe what you're doing to prepare and if you'll need to expand your manufacturing capabilities at all specifically for Danon?
Yeah, I mean, it is definitely a critical topic, and I know it's top of people's minds. Danon, unlike other better-known rare diseases, is really a newly diagnosed or newly understood phenomenon. The link between the gene mutation and the triad of symptoms was really made in the early 2000s. The pickup of Danon in cardiology and even primary care and neurology and neuromuscular awareness is pretty recent. I would say as recent as the 2010s. In some ways, we have to help create the market. That's going to take time and effort for sure. It's not like there's all the patients who are out there to sort of draw down from in the early years. We have to figure that out over time.
That being said, what we're doing is we are, first of all, finding those patients now where we can get started and hit the road running right at launch. We'll have more on that to come. We think that the diagnosed patients, however, only make up a fraction of the full truly diagnosed Danon patients. We'll provide an update on what we're thinking about that. To get from the truly diagnosed to the true prevalence, there are pretty massive efforts in place that we're putting in place around genetic testing, physician education, especially outside the big academic centers. A lot of Danon is not in the big academic centers. It's in the community. Education amongst not just cardiologists and pediatric cardiologists, but also pediatricians, primary care physicians, and neurologists who see this even earlier than cardiologists do. Education there, a big push toward genetic testing.
We have a project called Mission Genome where patients can be tested for free if they suspect they have Danon disease and other cardiac conditions as well. We've started that initiative. We're also going to be working on newborn screening over time. We have a partnership with Rady's Institute, and that's going to be a big part of how do we get from the current diagnosed to the true prevalence over time. It's going to take time. I don't think that necessarily we find all the patients in the first or second year like people find in other rare diseases and rare conditions with gene therapy. I think over time we're going to build a real market and find the true prevalence. Also a lot of Danon is de novo, up to 25%.
We think that once we've addressed the top market here, the addressable market, there's going to be a long tail on this as well.
Great. There is also a concurrent natural history study that is ongoing. How important is that for regulators? When do you expect to potentially report findings from that study?
Yeah, that's a great and important question. The stats design, as we'll talk more about, is patients compared with their pre-treatment baseline. It's not compared with an external database necessarily. Now, we do need to use an external natural history database to fortify the assumptions. If they move in a different direction, that's not ideal. What we've seen so far is that these patients obviously don't have protein expression at baseline without treatment, and their LV mass tends to increase over time rather than decrease. There's a nice alligator mouth or delta between treated and natural history. We want to bring more patients onto that to further fortify that assumption. We have both the retrospective review that has several dozens of patients that have these parameters, and then we also have prospective.
We already finished a prospective series in the past that led to the current pivotal trial, but another one is ongoing, and we're excited about presenting some of those results as well. I don't think they're gating in the same way that they would be if it was a direct comparator.
Okay, that makes sense. Maybe one more on Danon before moving on to other topics. One area that's maybe less of a focus, but equally important, is the female population. What are your latest plans there, and how much of the prevalence do they represent in your estimates?
Yeah, I smile here because there's such an interest from the Danon community to start the female trial. A lot of female patients are rapidly progressing like males, and especially we think that those could be the first opportunity to expand the label early on in the life cycle. We're going to try to start that program as soon as possible. There are other females who take time, and they're a little more slower to progress. We think that, however, females could represent up to 40% of the total diagnosed cases of Danon. Obviously, in terms of true prevalence, there's more females than males because the males pass away early. This really is a big population of target, both because of unmet need and just sheer population.
In fact, the female Danon patient may be the second most aggressive type of cardiomyopathy after Danon female, even more than the others. Alongside PKP2 and BAG3, this is a pretty major initiative that we hope to start up later this year.
Great, great. I think maybe moving onward to PKP2, you guys obviously have an upcoming readout. First, can you just remind us of the unmet need in this population and what percent of these patients end up ultimately requiring ICDs?
Yeah, so our understanding is that the majority of PKP2 patients, by the time they're diagnosed, the majority of them are going to have an ICD. It's protective. It's not completely curative, but it does prevent most fatal arrhythmias, which is a great thing. It does not prevent progression to heart failure, which ultimately is fatal for a lot of patients. Two things ICDs don't do is they're still fatal arrhythmias. Not a lot, but they exist. Second is they don't prevent the right ventricular and left ventricular heart failure that ultimately is fatal. That's important to know. Despite ICDs, there's still an important role for potential gene therapy. In terms of the number of patients here, we've said that PKP2 represents about one-third of arrhythmogenic cardiomyopathy, and we've been pretty conservative on our estimates of ACM.
We end up at a number of about 50,000 in the U.S. and Europe. Other sponsors have gone as high as more than 100,000 PKP2 patients in the U.S. and Europe. Whatever that number is, it's at least double the size of Danon, and we're very excited about that program.
Great. We do have the upcoming phase I readout. What are your expectations heading into that, and how should expression data be interpreted at this early stage?
Yeah, I mean, at the end of the day, I've got a clinical background. This is the portion that I get especially enthusiastic about, data readouts. We are very thoughtful about this. We've learned from Danon not to get too excited about endpoints early on. Let's let the data develop over time before we commit to an endpoint. I think that there are several buckets of endpoints here to look at, and then I'll come back to what to expect soon. Number one is certainly protein expression and/or gene expression, which is vector copy numbers and RNA. It's also what the protein expression also actually means in the tissue. For example, in Danon, we saw that along with the protein expression, we saw a reduction in vacuoles. You can actually see visually what the protein is doing. Can we find something like that in PKP2? That's ongoing.
The second point is the second possibility is imaging, right? Because these are patients who have dilated and large, especially right ventricles to begin with. It could eventually affect the left ventricle. We've seen improvements in preclinical studies here, so we want to track these over time. I don't expect any changes here early on, but like Danon, it could take nine months to twelve months, so we'll see how that tracks. We're going to look at also importantly arrhythmias because arrhythmias, especially certain kinds, can be markers of fatal arrhythmias. What kind am I talking about? Non-sustained V-tach, certain EKG changes like T-wave inversions, and PVCs. I would say PVCs are the ones that are the most subject to external influences. Even coffee can increase the number of PVCs a person has.
A lot of coffee, I mean, I remember when we did residency a long time ago, a lot of coffee after cough can induce PVCs, right? That's sort of common knowledge amongst interns. The same thing is true here, especially if PVCs are baseline. I would be cautious to hang my hat only on any one of those, but the combination and the full spectrum of these endpoints is what we're looking for. At the end of the day, it's how patients function, feel, and survive, and it's very important to look at that. That's important for both regulators and payers, and I think it certainly should be for all of us who are investors and other stakeholders as well.
That makes sense. I know the data will sort of have to speak for itself, but from a hypothetical perspective, could you potentially foresee a pivotal study design based on maybe a time-to-event type of analysis for patients with ICDs? Have you guys started to think about that?
Yeah, everything is on the table. To come back to what to expect in this update, I would say it's early. I don't think we're going to come out and say that here's the endpoint. Certainly, safety is important. Obviously, protein is important. Whatever we can garner from another fingerprint of pointing in the direction of proof of concept is going to be important, but it is early, so we may not be there. We'll see what happens. I don't have the data yet. Now, in terms of the regulatory path, I think the win for us here would be to be able to say that we are at or close to the dose that we want to be at and that we're trying to move forward into thinking about the question you just asked on pivotal trial design.
I don't expect this pivotal trial design to be as straight. I would say, I won't say straightforward, but simple and lean as the Danon. I think Danon is a very unique case. It's the most aggressive cardiomyopathy out there where patients progress quickly and where you can see reversal quickly, right? You can see it visibly and objectively. We're lucky versus something like Duchenne, where it's much more difficult to demonstrate objective improvements. I think here with the LV mass index and troponin and everything else, there's objective markers. In Danon, we're lucky. PKP2 may not be the same thing. I don't want to guide toward what kind of trial design that'll look like, but it may be different, and I look forward to discussions with both agencies on this soon.
Okay. There are a few other gene therapy companies at similar stages, so Lexeo and Tenaya come to mind. What do you consider to be sort of the key competitive advantage for Rocket over the competitors in this space?
Okay, so to directly answer that, I think that possibly time and speed to market if we are sort of approaching the final dose here. I do not know that for sure. I think just having done this before and knowing what the pitfalls are is important. I think rh74 is a tried and true vector, certainly at this dose in the E13. In the E14 range, the safety starts to be questioned, but in the E13 range, it has been pretty good and well tolerated. We feel good about the capsid. It has the most regulatory experience and commercial experience amongst physicians and treaters, so versus a new capsid to the market like rh10. AAV9, I think, is obviously we like it for Danon. It is hard to go higher than the current dose using AAV9. The E14 range, as you know, is associated with more severe side effects.
I think rh74 is the right capsid for this syndrome. Frankly, for cardiac-specific indications moving forward, we're using it for BAG3 as well. I would say that ultimately, regardless of who is the first, I think there's room for more than one because this is a market of 50,000-100,000, and there can be a solid business case for more than one player here. While we're competitive in this, we recognize that there's a lot of patients out there to be treated who need help.
Great. Maybe one last one on PKP2 before moving on to some of the other programs. You touched on this a little earlier, but is diagnosing in PKP2 as challenging as Danon, for example? Sort of a similar line, do you plan on expanding or utilizing extra resources to help promote earlier diagnosis?
It is a lot easier. The story with Danon, and I won't say issue because it's solvable. The story with Danon is that most Danon patients present with something else. They present with learning disabilities, mood disorders. They present with muscle pain and weakness that's often peripheral, not always proximal. It's usually late in their presentation that there's a cardiac manifestation. It's often undiagnosed or misdiagnosed as something else altogether, especially with the fact that most cardiac centers in the U.S. aren't even doing genetic testing in cardiomyopathies. That's all things that we're trying to change, both in terms of education and awareness about Danon, as well as genetic testing. Danon is difficult to diagnose. As I said, as I mentioned, we'll provide an update on where we are on that.
PKP2, however, presents with a very clear manifestation of palpitations, syncope, unfortunately, sometimes sudden cardiac death. A big percentage of sudden cardiac death is genetic-related ACM. As we've said earlier, a big portion of ACM is actually PKP2 ACM. It is something that's easier to pick up. There are a lot more diagnosed cases. There's probably still a big gap between the truly diagnosed and the true prevalence, and that's something else that we'll work on with genetic testing. We're trying to synergize our efforts across our cardiac portfolio, which includes these three programs, Danon, PKP2, BAG3, but others that are coming to ensure that patients with any symptoms get tested as early as possible. Eventually, we want to make the cardiac panel available for newborn screening as we sort of build a string of pearls here.
Okay. I do want to spend some of the remaining time on your lentiviral programs. Can you maybe just start off providing an update on the current status across the lentiviral programs? Maybe could ask a couple of follow-ups there.
Yeah, sure. The first to come is LAD-1. As people know, we submitted our BLA. We did get a CMC-specific CRL last year. The questions that were asked were on three or four points focused on stability and sterility. I will say that we're still working on that. We're going to provide the response this year for sure, and we do anticipate a PRV along with that. Last year, at the end of last year, we went to the FDA, and we proposed to leverage the data set on validating some of these assays and stability and sterility using the data that we already had. We thought we had a good path there. At the end of the day, FDA sort of wanted to do things in the way that was also done for other lentiviral approvals to have consistency of programs.
Despite the fact that we thought that we could get it done last year, we ended up just agreeing with them to the point, crossed every T, dotted every I. We're doing exactly what they want and the way they want it. Unfortunately, that takes time, but it reduces the risk significantly of another issue or hiccup. We feel good about the program. The timing is later than we had anticipated or hoped for, but we still feel good about an approval as soon as possible, submission of the CRL responses later this year, and a significant PRV opportunity here. Fanconi anemia, similar situation.
We actually decided to go ahead with a rolling submission, accept the CMC, and hold the CMC until we get the LAD points at least agreed to with the PDUFA date, and then we'll press send on the Fanconi CMC module, module three, which is the quality module. We are excited about Fanconi as well, and that's also a potential PRV opportunity. The Pyruvate Kinase Deficiency Program, we've decided to just prioritize later once we get these up and running. We have agreement with the FDA, EMA for that matter, on a pivotal trial design there, and we look forward to starting that, although we can't do everything at once. It's a small biotech, so we're stage-gating things a little bit. That's the full lentiportfolio.
While it's not the focal point of our future resources, as I mentioned earlier, it's an important program, both important programs to get done from the PRV viewpoint, but also the LAD soft launch allows access for patients early on after the approval. Those are our focus areas, and we're excited about moving that forward, not letting it take air from the AAV cardiac portfolio, which is the primary focus moving forward.
Great. Thank you for the comprehensive update. You did mention that several of your lenti programs are eligible for priority review vouchers, which I think is worth touching on a little further. I found interestingly that recently PRVs have been selling at premium prices. On average, they've been going for about $150 million. Do you expect that trend to continue? I also wonder if you could comment if Rocket's programs will be impacted if Congress elects not to reaffirm the PRV program.
Yeah. So the PRV program, the current program, if you're eligible for PRV and you're grandfathered in, then we have until late 2026 to get an approval. Our hope is certainly LAD. We also hope for Fanconi in case it's not renewed. If it is renewed, which from my intel, just based on anecdotal conversations here and there and the best that we know from our internal resources here at Rocket, we do anticipate an ongoing extension of the PRV program. That would be great because then we can also get Pyruvate Kinase Deficiency, which has pediatric designation, as well as Danon. Then it becomes a long-term way to continue to get non-dilutive financing. If it's not renewed, that could increase the value of the current programs, which are LAD and Fanconi. Either way, we hope it's a win for us.
Great. Great. I will take this time to set a reminder. I know we're getting closer to time. If there are any investors or people watching that want to ask a question, please submit it through the Ask the Question feature, and I'll take a look and relay those to Gaurav. Maybe one topic which I think you did touch on in the beginning, but I think it might be worth reminding, especially like you said with cash preservation and everything. Can you just tell us again what Rocket's manufacturing capabilities are and what's being internally housed versus externally?
Yeah. The AAV programs are fully internal, and we basically build it from scratch. The only thing that we bring from the outside from the U.S. are the plasmids, but everything else is in-house. Here in Cranbury, New Jersey, we have a 100,000 sq ft total facility, but about half of it is dedicated toward clean rooms. We've built several out. We're continuing to build some more. We anticipate that this facility alone can certainly handle the full Danon market, and we're going to get started with PKP2 manufacturing there as well for a pivotal trial and potentially the BAG3 as well. We'll have a good problem if we use up all the space for those programs and we have to build additional capacity.
It really not only reduces the cost of goods substantially versus having to use a CDMO as other sponsors are, but it also helps us control our timelines. What we've learned from when FDA has questions, if you can do experiments in-house and get it done, it's so much faster and cost-effective than having to outsource this. That's been sort of a drag on the lentiportfolio because it is outsourced. While we're very proud and collaborative with the partners that we've chosen on the lenti, both the vector and the cell processing, we have to be respectful for their time slots and other clients, and it just takes time and money. That's why the increasing focus here is going to be cardiac AAV portfolio.
I do want to just mention and reiterate that we're committed to both portfolios, but the resources moving forward are AAV cardiovascular portfolio focus while maximizing the LV portfolio. With our cash runway that we last iterated was $372.3 million in the last earnings, now with their improved and refined prioritization and resource allocation, this gets us operating capabilities through 2026, so through the end of next year. That just helps us continue to grow and maintain some financial discipline.
Yeah. Yeah. I think you covered the financial question, so that was definitely one I was going to ask. Maybe I want to take this time with the last few remaining minutes. Anything else that you want to mention or programs you want to highlight for Rocket going forward?
Yeah. We continue to build the cardiovascular portfolio. There are others that are coming that, as you know, we tend to not talk about them until we're close to IND, but there are other programs that are coming. We are still just getting started, and we're very excited about being a major force in the world of gene therapy in the coming decade.
Awesome. I'm not seeing any further questions in the chat at this time, so I just want to thank you, Gaurav, for coming on, talking about Rocket, and can't wait to see what the year brings.
Thank you, Ethan. Thank you, guys.