Hello, everyone. Welcome to the last day of the J.P. Morgan Healthcare Conference in 2024. My name is Cindy Xu. I'm an associate at the J.P. Morgan Healthcare investment banking team. Today, it's my great pleasure to introduce you, Faraz Ali, the CEO of Tenaya Therapeutics.
Thank you, Cindy, and thanks to the organizers for the opportunity to present the Tenaya story to you today. I heard from Cindy and the organizers that they actually like to save the best for the last day, so glad to occupy that slot. Tenaya is a publicly traded company. I'll be making forward-looking statements, and these are some of our standard disclosures. Tenaya was founded in 2016 to pursue a bold and noble purpose, and that is to just transform and extend lives through the discovery, development, and delivery of potentially curative therapies that address the underlying causes of heart disease.
These are pictures of actual patients who are affected by both rare and prevalent forms of heart disease, and what is common to all of them is that none of them have a treatment option to address the underlying cause of their disease. Tenaya is ideally positioned to lead innovation in this space, and partly just because of focus. We have a singular focus on heart disease. That's all we do. We don't dabble in other modalities, and we have assembled the leadership and the capabilities and the expertise to pursue opportunities in heart disease. We have three clinical stage programs at this point, one small molecule, two gene therapies. We'll talk about all three of them today. This is a potential big year for us, with the anticipated release of data in the second half of this year for our lead TN-201 gene therapy program.
In addition to our clinical stage efforts, there's a lot of exciting things happening beneath the surface in our early stage programs. We won't have as much time to talk about today, modality-agnostic drug discovery. Within that, though, we have a particular expertise in AAV gene therapy, and some markers of our expertise is the more than billion capsids and promoter and regulatory elements we've screened in our own in-house efforts, and the ability to scale up to the 1,000-liter scale for AAV gene therapy manufacturing in our own GMP facility. And with everything that we're doing, we do have cash to get us into the first half of 2025.
I'd like to make the argument that we are at the cusp of a revolution in the field of genetic medicines for heart disease, and that's not only on the back of some outstanding science that we hear about and read about every day, but increasing clinical and regulatory validation for both precision medicines as well as gene therapies. On the precision heart medicine side, the approval of Vyndamax back in 2019 for ATTR cardiomyopathy and CAMZYOS more recently for hypertrophic cardiomyopathy gives good clinical validation and regulatory validation of the ability to achieve approvals with pivotal studies that were measured in the hundreds of patients, not the thousands of patients, and not requiring large outcome studies.
And in fact, that was predicted and supported by the FDA draft guidance of 2019, indicating that the FDA was willing to consider feel and function endpoints as a basis of approval and didn't always require large outcome studies. We're also very encouraged by the amount of genetic insight we now have in our field. More than 100 genes have been tied to genetic cardiomyopathies, and we're very encouraged to see that the medical societies in both U.S. and Europe now recommend genetic testing as part of the normal diagnostic workup and long-term management of patients with heart disease, many forms of heart disease. So that's very encouraging. Frankly, even more encouraging is what's happened in the field of AAV gene therapy. We now have 7 approvals, including 2 last year alone, ROCTAVIAN for hemophilia and ELEVIDYS for Duchenne muscular dystrophy.
A statistic that many don't appreciate is that at this point, more than 5,500 patients have been treated with AAV gene therapy in more than 50 countries. That's a growing... Every day, there's a growing database of safety for and efficacy for these therapies, and that's exciting backdrop for us. Our friends at NEWDIGS at Tufts Medical Center published some really interesting analysis toward the end of last year, showing that on average, AAV gene therapies for rare diseases have a 2- to 3.5-times higher likelihood of success in clinical development, going from phase I to phase III.
The blending of both the left and right comes in the form of now we have early proof of concept for clinical efficacy in AAV gene therapies, particularly from our friends at Rocket in Danon disease, but now also early evidence from 4D MT and Lexeo, and that's very exciting for our field. Taking a step back, what does this revolution look like? We can draw some lessons from the field of oncology. We no longer just talk about breast cancer. We talk about breast cancer with particular mutations. We don't just talk about a particular, you know, you know, multiple myeloma, but with a specific receptor on the cell. That's where the field of heart disease is going.
We don't just talk about heart failure; we talk about heart failure due to dilated cardiomyopathy, or DCM, hypertrophic cardiomyopathy, or HCM, myocardial infarction, HFpEF, HFrEF. But even more important, we can now go to the next level down. We now have genetic insight with the and the gene pathways and the regulatory mechanisms involved in these diseases. So we talk about HCM due to MYBPC3 mutations, dilated cardiomyopathy due to the phospholamban mutation, and even within that, specifically the R14del mutation. So we really are getting down to the underlying driving causes of these different forms of heart disease. And with that, there's the opportunity that Tenaya pursues to follow the science and pick the right tool for the job. So if the problem-...
is a lack of protein because of a genetic mutation, gene transfer is the solution, which is what we're doing in our TN-201 and TN-401 gene therapy programs. If the problem is that phospholamban mutant protein has a gain-of-function mutation, the only way to fix it is to silence or gene edit and, and correct it, then gene editing is the solution. If the, if the problem is that you've had a heart attack and lost cells of your heart, the solution is to create new cells, and that's what Tenaya does. We follow the science and pursue things in a modality. This is why we talk about modality-agnostic drug discovery, is to deliver on this opportunity for precision medicine and heart disease. We have a deep and uniquely deep and diverse pipeline.
Three clinical stage programs that we'll talk about today: TN-201 and TN-401 gene therapies, TN-301 small molecule. Substantially, we'll focus most of my comments on the gene therapy program 'cause that's where most of the interest and excitement is this year with the intended release of data. We are pursuing both rare and prevalent forms of heart disease. You can see that for our clinical stage programs, these are not small indications. You see millions of patients with HFpEF, but even the gene therapies are going after large indications. TN-201 is going after 115,000 patients alone. That's a large orphan. This last year or last year and a half has been a period of high execution for Tenaya. We cleared 3 INDs within 13 months.
We dosed and completed the dosing and release data on our TN-301 small molecule program at HFpEF, and we dosed the first patient in the world for the TN-201 gene therapy. So we not only have bold vision, but we also have strong execution. How do we do all of this? It is on the back of integrated capabilities that we've internalized many years ago. So we have all the tools that we need to do modality-agnostic drug discovery, including in vivo models, in vitro models, engineered heart tissues. We can do phenotypic screening, AI machine learning, drug development. In fact, that has been published. Almost everything that we've done has been published. And then on the right-hand side of this equation, we have now the tools, capsids, promoters, physical delivery, manufacturing, gene editing.
So we can do things very quickly, very robustly, and develop products that are not only have the opportunity to be first in class because of the speed at which we can move, but potentially best in class because we now know better how to target the heart and even specific cells in the heart, and to get a robust expression of the desired therapeutic candidate. With that, I'll transition to talking about TN-201, which is our lead gene therapy program intended to address MYBPC3-associated HCM. Hypertrophic cardiomyopathy is a devastating disease. MYBPC3 is the leading genetic cause of hypertrophic cardiomyopathy, and it accounts for about 19% of all HCM. That translates to about 115,000 patients or more in the U.S. alone. It is a severe and progressive genetic heart disease.
The hallmark of the disease is the thickening of the ventricles and leads to a variety of symptoms. These patients have an elevated risk of sudden cardiac death and heart failure, significant functional impairment, and diminishment of quality of life. Patients with this specific mutation or mutations of the sarcomere in general have more severe disease, higher burden of disease, and often early onset of the disease. And Gabe is the poster child for that, a young boy in Florida who required an ICD at the age of one and ended up on the heart transplant list before the age of 10, and there's nothing that current medicine can do for him. TN-201 may be the first thing that can do something for somebody like him. So TN-201 is the first gene therapy being developed for MYBPC3-associated HCM.
The good news is we really understand this gene and this disease quite well. We understand that MYBPC3 is a structural protein that is critical component of the sarcomere, and mutations of the gene leads to a deficiency of this protein. On average, the heterozygote patient has 60%-70% of the wild-type protein, and that diminishment of 30%-40% is enough to have overt and severe disease. What we are doing here at Tenaya, our approach is very precise. We are delivering a working copy of the MYBPC3 gene. That working copy of the gene now produces protein. We need to produce 30%-40% of the missing protein in these severe heterozygous patients.
We utilize AAV9 to deliver that gene, well-validated from other gene therapy clinical studies, to be able to do that, and we have a novel promoter that we're using that allows for robust cardiomyocyte-specific expression. And in fact, in our preclinical models, we produce 100% of the wild-type protein when what we need in the clinic is 30%-40%. What might be the advantages of approach like this? Well, one, TN-201 would be the first therapy to actually address the underlying cause of this disease in a way that no current surgical procedure or small molecule could.
The potential product profile here is that a single dose with a durable effect that can replace the missing protein, can halt the progression of the disease, improve heart function, reverse the symptoms of the disease, and eventually improve quality of life, and hopefully extend lifespan for the worst affected patients. What's our reason to believe in this compelling vision? Well, it is a very robust, comprehensive body of evidence from our preclinical efforts. We do all our work in a high-bar homozygous knockout model, and we've been able to demonstrate in many different experiments and many different doses, that we can get reduction of heart mass, we can get improvement in heart function, and 100% survival in this animal model when 100% of the untreated animals die.
Importantly, we're able to demonstrate this at doses as low as 3 × 10^13 vector genomes per kilogram. Those of you who are familiar with the space know that in general, there's a drive to avoid higher doses in the 10^14 range, and so we're very pleased that we're able to get near maximal efficacy at the 3 × 10^13 dose. And in fact, we're very pleased that the FDA, this is the IND design of our clinical study. It's called the MyPEAK-1, Phase 1b clinical study. The FDA has indeed, in this study, allowed us to initiate dosing at the 3 × 10^13 vector genome per kilogram dose that is predicted to have near maximal efficacy.
We're very excited to have announced last year in October, the first patient dosed in the world with TN-201 gene therapy, done in collaboration with our friends and collaborators at the Cleveland Clinic, a world-leading institution with a long history of innovation in heart disease. This is a simple design. We intend to dose three patients at the 3 E13 dose cohort, and after that, after consultation with DSMB, we have the opportunity to either escalate up to 6 E13 or to continue dosing at the 3 E13 dose. It is an open-label study, multicenter, where site activation is going quite well here, and we're quite excited. There's a lot of patient enthusiasm for this study. Initial focus is on non-obstructive patients, symptomatic, severe adults who have this mutation.
This is designed to be a very data-rich study, and this graphic is intended to try to give you a sense of how much that we're gonna be capturing from each patient. Of course, the focus is on safety and tolerability. But importantly, we're gonna get a biopsy for every patient at both the 8-week mark and the 52-week mark. That biopsy will give us measures of vector copy number, RNA expression, and protein expression. We're also gonna be looking at circulating biomarkers, including NT-proBNP, as well as cardiac troponin I. We will be looking at, of course, echo parameters, the size of the heart, the mass of the heart, LV wall thickness, dimensions, and of course, the performance of heart function as measured by ejection fraction and diastolic dysfunction. A lot of echo parameters.
Functional changes measured both by the six-minute walk test as well as CPET, cardiopulmonary exercise test, and then symptom improvement and improvements in quality of life as measured by the Kansas City Cardiomyopathy Questionnaire. So each patient, we're gonna get a lot of information, and so we're very excited to be in the position to provide some data updates from the first handful of patients in the second half of this year. Why do we think TN-201 is positioned for success, and why this might be a breakout year for Tenaya on the back of early data? Well, some of it just comes from what I've shared with you so far. We were only able to share a tip of the iceberg.
We would just have a mountain of really strong preclinical evidence in multiple models, both 2D and 3D and in vivo models, showing reversal improvement in almost all the hallmark features of this disease. We also have a very, well-designed clinical study, data-rich, as I mentioned, designed for safety, taking all the learnings that have accumulated over the last couple of years and applying them to ensure patient safety in this study. We have activated, at this point, 3 sites, and more are coming, some of the best sites in the world, and we have very high patient engagement. Last year, we announced a serostatus study, interim results from a serostatus study. We're checking for seroprevalence of antibodies against AAV9. And at that point in time, when we reported in October, more than 70 patients had submitted blood samples, indicating their interest in participating in this study.
But in addition to the work that we're doing that supports TN-201, we can't ignore the fact that there's now clinical cardiomyopathy experience in the broader field, and there's 6 companies that are listed here that have all made some advancements in this field, and we think that de-risks clinical development for TN-201. Through the work of Rocket, we have seen that AAV9 does a great job of transducing the heart and expressing in the heart and durable expression out to 3 years. And through the work of all of these companies, we now have seen in the heart in multiple forms of cardiomyopathy improvement in circulating biomarkers, LV wall thickness and mass, heart function, New York Heart Association class, quality of life, exercise capacity, the very things that we are measuring in our phase 1b study.
Importantly, through the work of CAMZYOS, the folks at MyoKardia, now BMS, we have now validation that you can get an approval based on relief and function endpoints, including things like peak VO2 that we're already measuring. And through the announcement of Rocket last year, we have now evidence that the FDA is willing to consider accelerated approval in severe pediatric patients on the basis of a surrogate endpoint in a small number of patients, presuming that the data is good. So overall, this paints a great picture for TN-201, both in 2024 and beyond.
So just wanna make the point that while TN-201 is initially focused on a very precise population, but there's a lot of phenotypic heterogeneity here, and we believe that TN-201 can address all of it because the entirety of this heterogeneity is driven by the same thing: mutations in the MYBPC3 gene, which is what we are addressing. We have infants, peds, adolescents, adults. We have homozygous patients, heterozygous patients. You see patients with obstructive cardiomyopathy, non-obstructive cardiomyopathy. Our initial study design is very focused on adult heterozygous, non-obstructive patients with ICDs Class two and three. But from here, once we have established some early signs of efficacy and safety, we intend to relax the criteria and explore this entirety of heterogeneity and see how the product performs in different populations. This will help us assess our path through pivotal studies...
In anticipation of further development in peds, we launched the MyCLIMB Natural History Study back in 2019. At this, we don't talk about it a lot, but at this point, 29 sites have been activated in more than 4 countries, 4-5 countries. We have more than 100 patients who have been enrolled. We're learning a lot about the natural history of this severe pediatric disease, and the data we collect here may provide the control arm for a future pivotal study in infants that might go even faster than adults. Now, I'll transition to speaking about TN-401 gene therapy. TN-401 gene therapy is addressing the leading genetic cause of arrhythmogenic right ventricular cardiomyopathy, or ARVC, due to the PKP2 mutation.
This is the leading genetic cause of ARVC, accounting for about 40% of patients, and we estimate that translates to about 70,000 patients in the U.S. alone, so another large orphan. Similar to the previous indication, this is a severe and progressive, life-threatening genetic heart disease. The hallmark of disease is different. There is enlargement of the heart, but typically in the right ventricle versus the left ventricle, and the name of the disease comes from the fact that there is severe arrhythmia. In fact, for the majority of the patients, they present in their twenties and thirties with severe arrhythmia. And one startling statistic is that for almost a quarter of the patients, their first presentation is sudden cardiac death. This is a genetic disease that runs in families.
You can see Tracy and her daughter, Ava, who both have the disease. Ava has a severe form and presented in her teenage years. There is nothing that addresses the underlying genetic cause of this disease, and that's why we're excited to have an opportunity to pursue drug development and provide some hope to this patient population. Like TN-201, we have a mountain of evidence supporting preclinical efficacy in a very severe model for TN-401, including arrhythmia prevention, prevention of the enlargement of the right ventricle, and improvement in survival. I'll call your attention to the little graph on the right with arrhythmia. At the top, you see a normal sinus rhythm.
In the middle, you see. You don't have to be an electrocardiologist to see that is a highly disorganized, an abnormal, ECG. After a single dose of AAV gene therapy, TN-401, you can see an ECG that looks very much like normal ECG, and that's what we're hoping to achieve for in humans. Like, TN-201, we have a very robust study. It's called the RIDGE-1 Study, Phase 1b, IND cleared last year, and we anticipate dosing in the second half of this year. And like TN-201, preclinical evidence suggested that we achieve all that wonderful efficacy in the model at doses like 3 × 10^13 vector genomes per kilogram associated with near maximal efficacy.
We're again very pleased that the FDA is allowing us to dose at 3 × 10^13 vector genomes per kilograms for the initial dose cohort. From there, we will escalate to 6 × 10^13 with DSMB review. Like the TN-201 study, the study design here is data-rich. We will collect biopsies at baseline, 8-week, and 52-week, of course, looking at safety, looking at circulating biomarkers. Another important PD or biomarker here are changes in PVCs, or premature ventricular contractions, and NSVTs, non-sustained ventricular tachycardia. We'll be looking for frequency of ICD shocks. That's actually one of the really terrible things that happen to these patients. They have a high degree of shocks, terrible shocks that you know impact their quality of life.
We'll be looking for improvements in quality of life, and of course, we'll be looking with echo for all the kinds of things that we mentioned for TN-201: size of heart, heart function, dimensions, thickness of the walls. So another data-rich study. Anticipate to begin dosing in the second half of this year. Again, as in the case of TN-201, we're very pleased with the progress we're making with site activation and very pleased with the level of patient engagement we're getting. These are patients who are really looking for an option. And last, but by no means least, TN-301, small molecule HDAC6 inhibitor for HFpEF, heart failure with preserved ejection fraction. This is not a genetic disease, and it is not a rare disease.
This is actually one of the leading causes of heart failure today, accounting for about 50% of heart failure cases, more than 3 million patients in the U.S. alone. The hallmark of this disease is diastolic dysfunction, which is the inability of the ventricles of the heart to relax and fill properly, and that contributes to the morbidity and eventually the mortality seen in this disease. This disease co-presents with, particularly with obesity and then diabetes. So to the extent that those have become an epidemic, HFpEF is also becoming an epidemic. One startling statistic to capture the severity of this disease is that 75% of people who are hospitalized with HFpEF die within five years. Again, this is not a disease that will be addressed by gene therapy. Very complex pathophysiology involving multiple pathways.
You can see a metabolic dysfunction, inflammation, oxidative stress, fibrosis, thickening of the left ventricle, you know, autophagy, all of that leading to the eventually the diastolic dysfunction. What is neat about the HDAC6 target and inhibition of HDAC6 is that we have a multimodal mechanism of action that we've been able to demonstrate in multiple models that addresses all of these pathophysiological pathways, and we have been able to demonstrate that we can reverse them, which is quite remarkable for a small molecule against a single target. So that is part of why we're excited about the potential for TN-301 and HFpEF. We successfully completed our phase I study last year and presented the data at a major conference at the Heart Failure Society of America. 70 patients were dosed.
There were no dose-limiting toxicities across a very wide range in both the SAD and MAD arms of the study. We dosed acutely up to 700 mg, and 14 days of dosing up to 300 mg. All the AEs were GI-related. No difference in the frequency of AEs here versus placebo, and no increase in those AEs with dose. So really, quite a remarkably clean safety profile. The PK and half-life supported, and the plasma exposure supports once-daily dosing, which in this field is actually important in product attributes, so we're pleased with that. What was actually really exciting to us is that we are able to demonstrate target engagement. So we're able to measure target engagement even in the blood of healthy volunteers through something called tubulin acetylation. That's downstream of HDAC6 inhibition.
We were able to show target engagement at even very low doses in the clinical study, and importantly, we were seeing target engagement at levels that exceeded what was associated with maximal efficacy in our preclinical models. So that bodes very well for once this molecule goes into the patient population of choice. So target engagement is something you don't always get with a first-in-human healthy volunteer study, but we have it, and we think that significantly de-risks the program. This is an exciting program, but this is something that we're excited to partner with somebody. We could develop this on our own, but that will cause a lot of funding. The next step here would be a phase IIa. That could be 50 patients, it could be 100, it could be 200, depending on who's doing it.
We think this is exactly the kind of molecule that belongs in the hands of a big pharma company or some other syndicate to advance it robustly through later stages in development. We have high engagement from many parties here, and so we're excited to continue those discussions. Another reason why this belongs in the hands of somebody else is that, frankly, this target is quite relevant in many other indications. And so, if you have a clean and safe molecule that can address this, this has relevance in many other inflammatory disorders, including outside of the heart failure field. There's a lot of published literature that supports it. So it's a true potential pipeline in a pill.
We're gonna marshal our resources on TN-201 and 401, where we can make a difference and get to the next value inflection point quite efficiently. But we are very excited by what we've been able to demonstrate in this program. So I'll close with what I was just saying, which is we're really, really focused on executing on TN-201 and 401. This year is all about dosing more patients in TN-201, and then generating data in the second half of this year, which, per my earlier comments, we have every reason to believe that that can be positive early data from the work we've done and the work of others. TN-401, continue site activation and begin dosing in the second half of this year.
And then we gonna continue our our long history of excellence in research, as well as in manufacturing, and selectively release some of that data at conferences over the course of this year. Importantly, we have about $120 million in cash as of the end of Q3 2023, and that's sufficient to fund our planned operations into the first half of 2025. So with that, I wanna thank the organizers again, thank the audience for being here. I wanna thank the patients who have been supporting everything that we've done so far, as well as our external collaborators, and of course, thank the employees of Tenaya Therapeutics, who are the workforce that drives our innovation and is gonna carry Tenaya into the future.
With that, I'm happy to take any questions, either from the audience or from online.
Happy to kick off the questions.
Thank you, Cindy.
First one, can you talk a little bit about how your TN-201 is differentiated compared to, for example, CAMZYOS or aficamten, which has reported positive phase 3 data in December?
Mm-hmm.
What do you think, TN-201 is differentiated?
Yeah, I mean, first of all, we're very happy for patients that they now have an approved small molecule for hypertrophic cardiomyopathy. That was a great innovation, a lot of respect for the work that they're doing. We think we might have the opportunity to do better, right? Because we're targeting the underlying genetic cause of disease in a way that the myosin inhibitors are not doing. And so the potential for a single dose with a durable effect, that's targeting the underlying genetic cause. One thing that we sort of glanced or mentioned is that the majority of the patients over here are non-obstructive. 70% of the patients with this mutation are non-obstructive phenotype, 30% obstructive.
So far, none of them have been approved for the obstructive phenotype, and we're still early days to determine whether they will be able to demonstrate efficacy in the non-obstructive phenotype. So time will tell, but even if they do, we still think that we have the opportunity for a greater magnitude effect and duration effect that truly halts the progression of the disease because it's going after the underlying genetic cause in the way that the myosin inhibitors cannot. So I think there's room for both those molecules to address the other, you know, forms of HCM that are not genetic, and plenty of room and opportunity for TN-201 to have a differentiator profile and robust uptake.
Frankly, we're seeing that enthusiasm from the sites and the patients, including patients who have access to the small molecules, and including from physicians who are involved. With the most of the sites that are working with us are the same sites that were involved in the approval of mavacamten for HCM. So I think there's room for both molecules.
... Yeah, that makes sense. Maybe a follow-up question, speaking of gene therapy, can you talk a little bit about the rationale of building your in-house capabilities versus outsourcing with a third party?
You're referring specifically to manufacturing?
Yes.
Yeah. You know, that's, thank you for the question. You know, this is manufacturing, we have learned, as a field, unfortunately the hard way, of how important manufacturing is to, in this field in particular of gene therapies, whether it's AAV or otherwise, and, the process is the product. So we made a deliberate choice early on that we wanted to have total control in creating the, the, the best possible process that would give us the best possible product attributes. We did that first for non-GMP, and now we're able to do it at GMP. So that decision was-- And also because we are working with different, capsids and promoters, that's gonna change the product attributes, and we wanted to be able to really design the process around the novelty and the innovation that we're advancing.
Frankly, we're very excited that we're sitting here in a position that we have produced 100% of all the material we need to support both the TN-201 phase 1b study and the TN-401 phase 1b. Both of them are approved to dose up to 15 patients. We have more than enough material to dose that many patients, and then some, and we're able to do that within our own facility. You know, if we have positive early data and we wanna accelerate and go rapidly, it is much easier for us to then get the next couple of batches going and to race towards a pivotal study if we control our own process in our own facility.
So we're glad that we made that investment, that strategic investment a few years ago, and I think the co-location with our research and our non-GMP facility really allows for rapid innovation cycles and rapid learning cycles. So overall, we're very pleased that we made that investment, and that we made it here in California, close to our other facilities.
I see. Okay, I guess maybe one last catch-all question for you is: Is there anything else that you feel like the market is still lack of appreciation, or you still think a lot of your manufacturing platform capability is still underappreciated by the market or investors?
Well, we don't know what the market is appreciating or not right now, so we're feeling underappreciated in general, but I think we're not alone in that. I think that, going back to where I started, I don't know if everybody fully appreciates how we are at the cusp of something really special, right? The idea of applying precision medicines to the leading cause of death in the world, and the fact that we are, to our knowledge, the first and only company that has been purposefully built from the bottoms up to pursue this exciting new opportunity, that is not a small opportunity, it is a very large opportunity.
And that in every single thing that we do, whether it's capsids or promoters or scalability of manufacturing to address large populations and having, first-- potentially first-in-class and best-in-class, both clinical stage assets as well as preclinical stage assets in small molecules, gene therapy, gene editing, and cardiac regeneration, all with 140 people. We're pretty, pretty happy with what we've been able to accomplish, and I think that's not fully understood and valued. We spend most of our time... We didn't talk about a single early pipeline asset, and there's a lot of things that are exciting there that we tend not to talk about investors. Because appropriately, people are focused on get the lead, the program to clinical catalysts, and then, you know, and then people are very interested in funding, that progress.
But beneath the surface, there's so much going on that's so exciting and really differentiating. And so with Tenaya, you get not just that first horizon of catalysts, of the clinical stage assets, but right behind that, there's the second and the third and the fourth wave that's already being baked in and maturing. So I think that's something that's not fully understood, that is, embedded in the Tenaya story. Yeah. Thank you, everybody. Any questions? Sorry. Okay, thank you very much.