Good afternoon. Thanks for joining us for another session at the 42nd JP Morgan healthcare conference. I'm Brian Cheng, I'm one of the Senior Biotech Analysts here at the firm. I'm joined by my associate, Sean Kim, who is also in the audience. On stage, we have the team from Editas. I'll pass the mic to their CEO, Gilmore O'Neill, for a short presentation, followed by a live audience Q&A. If you're joining us live, you can submit the questions for the team through our conference portal. Gilmore, the stage is yours.
Thank you very much, Brian. Good afternoon, everyone. It's great, and we're very grateful to have you here as we tell you about Editas, what we have done in the past year. Share an update on the data that we presented at ASH just recently for our lead clinical asset, and talk about our plans for the future into 2024. Now, before going any further, you should know that I'm going to make forward-looking statements, and the actual results may be materially different, and so we encourage you to look at our federal disclosures so that you understand the risks and factors that could result in those altered material outcomes. So 2023 was a year in which we witnessed the transformation of programmable CRISPR genome editing from a technology into an approved human medicine.
In addition, we actually saw the readout of multiple human proof of concepts from a number of different sponsors or companies, including Editas. Editas finds itself in a small field of CRISPR companies that are actively in the clinic, and our lead asset, reni-cel, an investigational gene-editing medicine with a potential to be best in class for the treatment of sickle cell and thalassemia, is in active Ruby and EdiTHAL clinical trials. In addition, Editas has some important core capabilities centered around its proprietary AsCas12a, high fidelity, high efficiency CRISPR nuclease. In addition, we have core expertise in guide RNA design and chemistry, and very importantly, at the core of our drive is a longer-term focus on creating important medicines based on in vivo gene editing.
In addition, we have scaled CMC, which places us in a very good position as we prepare for a future BLA and commercial launch. In addition, Editas holds exclusive foundational IP around Cas9 and Cas12a, for its use in the prevention of or treatment of human diseases, and is a source of potential non-dilutive capital, as evidenced by recently granted sublicenses that we gave to Vertex Pharmaceuticals and Vor Bio. Finally, but very importantly, we have actually created a new leadership team here at Editas, formed of seasoned, and I ask their forgiveness as I describe them as seasoned, but seasoned veterans with expertise and experience in the development, approval, and commercialization of medicines.
So this time last year, I stood before you and announced a new, simplified, and very focused strategy for Editas, comprising three pillars to drive us towards a long-term vision of being a leader in in vivo programmable gene editing. The first of those pillars was to drive reni-cel, formerly known as EDIT-301, toward BLA and commercialization. The second, to strengthen, reorganize, and focus our discovery organization to build an in vivo editing pipeline. And finally, to increase business development activities with a particular focus on monetizing that very strong IP that I told you about just a moment ago. So how did we do last year? Well, we actually achieved a lot, and as you look across those three pillars, I'm going to draw your attention to particular areas of focus.
We enrolled. In fact, we met our goal of enrolling 20 patients in the Ruby study and indeed exceeded that. We announced at the end of the year that we had enrolled 27 adults into our Ruby study and have continued to enroll since then. We actually provided major clinical updates from our Ruby and EdiTHAL studies, and momentarily, I will share the data that we presented at ASH just last month. With regard to strengthening and building an in vivo pipeline, we did the rebuild, the capability reorganization, and hired a new chief scientific officer, Linda Burkly, who brings three decades of experience in very successfully inventing and developing and moving new human medicines forward.
Then finally, we did actually increase our business development activities and monetize our IP, leveraging our robust IP portfolio, and as I said, a critical example was the sublicensing of our IP to Vertex in a non-exclusive way, just to, in a focused way, to enable the exa-cel launch. Now, what does the future hold for us? Well, in 2024, having built momentum with a strong focus on reorganization, redesign, and execution... We're going to continue to drive those three pillars. With regard to reni-cel, we're going to continue the enrollment, as I've said already, for adults and dosing. And indeed, we are on target to have dosed the twentieth patient in this January time frame for our Ruby study.
We are going to initiate an adolescent cohort, and I should say, by the way, in passing, that we're also going to complete adult enrollment into the Ruby study this year. We're also going to present a substantive clinical data set for sickle cell patients from our Ruby study with considerable clinical follow-up in mid-2024 and the year-end of 2024. So what do I mean by a meaningful clinical data set? Well, we're talking about 18-20 patients with at least three to five months of follow-up. So why do those two numbers matter? Well, 20 patients was the efficacy data set that was validated by the FDA for acceptance in the BLA when they accepted the BLA for exa-cel last year.
Three to five months of follow-up is really the time period in which we actually see, and you'll see this soon when I present, the data, that we see a robust fetal hemoglobin expression and correction of anemia, placing patients into a normal physiologic range of total hemoglobin. Why does that matter? Well, when you do the math and you think about that validated pathway, in the middle of the year, we can present that patient data with that follow-up, you can actually see, how that benchmark, validation path to BLA can line up, for us. Obviously, contingent on agreement and discussions with the FDA. With regard to our discovery pipeline, and particularly the focus on in vivo editing medicines, we will establish in vivo preclinical proof of concept for an undisclosed indication, this year.
This is important as it enables us to validate the formulations that we will need to deliver to target tissues and cells of interest, and enables us to, in parallel, build a, a list, of disease targets of interest to us, which we can then leverage with the plug-and-play capabilities of programmable CRISPR editing by simply modulating five to 10 nucleotides in the targeting sequence of the guide RNA to choose and expand our, portfolio of disease targets. And then finally, we are going to leverage the foundational work we did over this year and the success we had in, sub-licensing our IP, into 2024.
As you know, and as I said, as a potential source of non-dilutive, capital, indeed, actually also the tool to leverage our abilities to, partner, for complementary capabilities that advance our strategic move into in vivo, gene editing medicines. So I did promise to tell you about, our, sickle cell, data as we presented, last, year. And of course, it's an important element because a question would be, why would you invest in reni-cel? And I think that you will actually see just in a moment, why we think that this is an important medicine, with the potential to be best in class as a cell therapy, for sickle cell disease, with its ability to consistently correct anemia.
But let me pause for a moment and just remind you, and forgive me for those who are very familiar with the disease, what sickle cell disease is. This is an inherited, profoundly disabling, life-threatening hematologic disorder. It is caused by a highly conserved mutation in the beta globin gene, and it causes sickling of red cells, leading to anemia, hemolysis, or red cell destruction, as well as vaso-occlusive events. Now, vaso-occlusive events, from a pathologic point of view, are essentially events that are associated with severe and profound disabling pain, necessitating emergency room visits and in many cases, when severe, hospital admissions. But it's much more than that. It is actually also associated with end organ damage across multiple organ systems, for example, causing strokes, heart attacks, damage, acute chest syndromes with infarcts of the lungs and many other organs.
That can result in acute catastrophic organ failure or can accumulate over time, leading to progressive, organ compromise. But what does that mean for a patient? Well, the best way to tell you about that is to give you some anecdotes about some of the patients in our clinical trials. One young man, in the build-up to, joining our clinical trial and prior to treatment, experienced 100 days on average of inpatient hospitalization every year for the two years prior to, and indeed in the years prior to that, in his life, leading to, the clinical trial. And in fact, talked about how essentially, completely, and adversely impacted his dreams, ability to have a, personal and professional life. Another young woman described how those VOEs and those repeated hospitalizations for vaso-occlusive events disrupted her studies.
And then resulted in her taking nine years to graduate from college summa cum laude, but it took nine years with all those disruptions. That's what it means to patients. What we have done is taken a unique approach to treating this, you know? A validated natural history approach is to upregulate fetal hemoglobin. We are leveraging that observation and targeting the HBG1 and HBG2 promoters using our proprietary AsCas12a enzyme to essentially upregulate fetal hemoglobin robustly, correct anemia with superior red cell production and health as compared to a BCL11A targeting approach, and also reduce the risk of off-target editing using our high-fidelity enzyme, an issue that was actually discussed at the AdCom last November for the FDA. So what have we actually seen in the data? Well, an important predicate for declaring efficacy is to have successful engraftment.
And indeed, all the treated Ruby patients successfully engrafted and showed a favorable safety profile, which is consistent with that that you see with busulfan conditioning. And importantly, we saw no serious adverse events related to reni-cel. Now, in the slide, we described the demographics and the infusion engraftment data for our patient population. But I do want to draw especially your attention to the number of severe vaso-occlusive events that the patients in our trials have suffered. The average annual rate was four per year, 3.9, but four per year for the two years leading up to their enrollment in the study. And so what does that look like in the context of the study, and what was the impact of reni-cel on those vaso-occlusive events?
Well, on the left of this graph, you will see that blue, little blue lozenges represent each severe vaso-occlusive event, and you can actually appreciate visually, probably more effectively and meaningfully, what that high rate of VOEs looks like prior to treatment. What's really exciting is to look to the right in orange and see that all treated Ruby patients, followed for at least a month, had or were vaso-occlusive event-free following their infusion. So I have already said that reni-cel has the potential to be best in class. Why do I say that? Well, in addition to the effect on VOEs, we've actually shown that patients show a rapid return to normal or correction of anemia into the physiological range, in addition to clinically meaningful improvements in fetal hemoglobin levels of greater than 40%.
So let me break that down for a moment and disaggregate the data. First, every patient, every woman who was actually followed out past five months, demonstrated a total hemoglobin, which sat within the normal range for women. Every man treated with reni-cel, followed out to 5 months and beyond, maintained, achieved and maintained a hemoglobin in the normal range for men. And you can appreciate that when you actually look across at the total hemoglobins that are represented there. In addition, in orange, we highlight the fetal hemoglobin expression that was achieved by these patients. And you'll appreciate that, again, there was a rapid expression of fetal hemoglobin and then a durable effect in excess of 40%, and through the period of follow-up. So I've talked about 40%. Why does that matter?
Well, I had alluded to or previously the natural variant called hereditary persistence of fetal hemoglobin. And experiments with that, and a sort of a natural dose experiment that has occurred and been well described in epidemiological data, demonstrates that certainly at above 30% or higher, you get significant, if not complete, control of vaso-occlusive events and the complications of sickle cell disease. So turning now to thalassemia. Let me just pause for a second and tell you that thalassemia is a very serious and very severe hereditary chronic anemia that actually requires continuous and repeated transfusions from very early in life. And that is because the mutations that cause thalassemia result in a substantial and massive reduction in adult hemoglobin, specifically beta globin, and thus adult hemoglobin, which requires those transfusions.
Those transfusions are administered to enable survival. And what we saw again, was from a point of view of that preliminary or first step in efficacy, was that we saw successful engraftment and a similar safety profile as we'd seen with Ruby patients. And then what was the impact from an efficacy point of view? Well, the EdiTHAL patients had early and robust increases in their total hemoglobin above the transfusion independence thresholds. And you can see that nicely represented in a format that's similar to what I showed you with the Ruby. But let me just break down the data a little bit more. As you look at the trajectory, you can actually see that at similar time points to Ruby, we saw a similar trajectory for upregulation expression of fetal hemoglobin and total hemoglobin.
But let me also talk to you about what the definition of transfusion independence is. So nine grams per deciliter is the generally accepted threshold for declaring transfusion independence. That is accepted across the hematological field and actually has been used as an outcome measure by regulatory authorities around the world. But what does that mean for the patient? Well, I'm sure many of you have had the experience of trying to decide, are you going to spend 15 minutes waiting for an elevator to get to the next floor for your next meeting or taking the stairs? And I know from a personal experience that climbing those stairs 10 times today is actually relatively demanding. My hemoglobin is normal. I expect that most of you have a normal hemoglobin.
A hemoglobin of nine grams per deciliter would make that journey up five floors extraordinarily challenging and almost impossible. Why? Anergia, massive fatigue, and frankly, profound exercise-induced shortness of breath. That is the experience that these patients have even immediately after transfusion. You have to also remember that between transfusions, their hemoglobins drop, which means that the symptoms they have, the energy that these patients have, and their ability to carry out normal activities of daily living, are substantially reduced over time until the next transfusion. The other thing that's important is not just the level of hemoglobins that we achieved, but also the durability and the fact that it's maintained and sustained, so patients don't have to be transfused intermittently and suffer from fluctuations in their total hemoglobin.
Then it's actually worth pausing and just reiterating why it is that we see this, this potential for it to be best in class based on a differentiation around this robust correction of anemia. And it's based around two elements, but the critical element is the choice of target that we made, and we were very deliberate in making that choice. Targeting HBG1, HBG2, that is the gamma-globin promoter, versus the BCL11A target. And when we did a head-to-head study a few years ago, and I have to give credit to our researchers, I'm afraid I wasn't there, so I can't take credit. They actually demonstrated with an empirical head-to-head study, preclinical set of studies, that red cell production was superior with the gamma-globin targeting versus BCL11A, with also better proliferative capacity or growth capacity, as well as long-term, red cell health and survival.
Why does that matter? Because we expected that it would actually enable us to actually treat across the spectrum of complications of sickle cell disease, and not just target vaso-occlusive events, which are profoundly disruptive to life and are very meaningful to manage. But also to treat the anemia that's associated with sickle cell disease, and to actually even more robustly treat the anemia of thalassemia. In addition, we also chose the AsCas12a enzyme with its high fidelity and substantially reduced off-target editing as compared to Cas9, to deal with a possible risk of off-target editing, which was one of the actually was actually at the core of the discussion at the exa-cel outcome.
So these two differentiated approaches anticipated a robust effect on both fetal hemoglobin and total hemoglobin, and we saw that validated in the clinic, and we've seen it consistently validated in the clinic as we look at and present additional data sets with increasing numbers of patients and increasing follow-up. So the key takeaways from these data are that reni-cel drives early, robust correction of anemia to a normal physiologic range of total hemoglobin for sickle cell disease. It drives robust, sustained increases in fetal hemoglobin well above the threshold that is believed necessary to control VOEs. And we haven't seen VOEs to date in all our dosed sickle cell patients. The safety profile is consistent with that of myeloablative busulfan, and no additional severe events are associated with reni-cel.
And the initial hemoglobin and fetal hemoglobin responses are consistent across both the sickle cell and transfusion-dependent thalassemia populations that we have investigated in our clinical trials. So as we look forward, in summary, it has been an incredibly exciting time for us at Editas over the last year as we drive its transformation from a technology company to a commercial therapeutics company. And that excitement has been driven by a number of things. It's been driven by external events, in fact, in which we were able to play an enabling part with the first human approval of a CRISPR-edited medicine. In addition, we have actually driven the transformation of Editas as we have really delivered on our execution over the last year. And as we go forward in 2024, we look to continue that execution, but actually delivering even more.
And we are, to do that, are going to drive our reni-cel execution towards BLA and commercialization. We're going to continue and strengthen our focus on discovery to an in vivo pipeline based on substantial foundational work done over the last year, and continue our BD activities, and monetize our IP. It's a really exciting time, and we're really looking forward - I am looking forward with my new and wonderful leadership team to continue this drive as we transform Editas into a commercial therapeutics company. It must be said, before finishing, that the north star for us are our patients. They tell us where we need to go. They tell us what we need to do because they are the people who need us to deliver on the promise of CRISPR medicines.
We also couldn't do this without the support of the same patients. We could not do this without the support of clinical trial sites, the investigators, experts, our employees, our shareholders, and you. Thank you.
Great. Let's start off with a Q&A. We're joined by Gilmore O'Neill, CFO Erick Lucera, and CMO Baisong Mei. For those who are live in the audience, if you have any questions, you can raise your hands. We have a runner on the floor. For those joining us virtually, you can submit questions on the portal. So I'll start with a couple of questions. You know, commercially, first is on commercial, you know, opportunity of reni-cel in sickle cell specifically. Now, we've seen remarkable advance across some of your genetic peers in the last 12 months. We now have a CRISPR-based therapy for sickle cell. We now have a pivotal study with in vivo gene editing.
But when you look at the sickle cell space, you know, we have two products that just entered the market, right? So what are your guardrails today to make sure that, you know, we'll talk about this, that you wanna be the fast follower. You want to optimize your position once your peers kind of set the stage for you, right? So what are your guardrails to make sure that you're gonna be the fast follower to maximize market penetration?
Well, let me first say that I think you've summarized it very nicely, which we see ourselves having a product in reni-cel, which is a fast follower with the potential to be best-in-class. I think there are a couple of things we need to do. One, we need to continue to drive our execution, and that is an absolute, you know, hot focus for us. The second thing worth sharing is that I have had the privilege to develop and have approved medicines in the rare disease, as has Baisong Mei and our Chief Commercial Officer, Caren. We know from experience that there is space for a number of therapies.
I think the third point is that in granting us RMAT, the FDA recognized a continuum unmet need, and it's worth remembering that that RMAT was delivered less than two months ago, and that was within weeks of the AdCom and approval for exa-cel. One of the regulatory requirements is that an unmet need is recognized by the agency to grant RMAT. They did it after reviewing a dataset, including clinical datasets from us. Now, with regard to how we look at the market beyond that and its evolution, what we have to prepare for that, I'm going to ask Eric to talk a little bit about that.
Yeah. Thanks, Gilmore. Obviously, we'll be tracking the two launches very carefully. I think, you know, there's gonna be a number of things that have to be done to get these products to market. You have to secure reimbursement. You have to get the state Medicaid budgets in place. The centers have to sign on and be trained. So we'll be watching all of that stuff, and we'll be learning, and we'll be modifying what we're doing. And that's the benefit of being a fast follower. And assuming the data holds as it has been, we think we have a differentiated product, and we look forward to going into a market where the infrastructure's been built and having a little bit better profile.
Mm-hmm.
Any questions from the audience? I think last year, around this time, you know, I think I remember that we were in another room, but we talked about how you see the differentiation of reni-cel. There are a couple of things that jumped out. You know, there's anemia, fatigue, and organ function.
Mm-hmm.
PRO. And you're also seeing, you know, really rapid normalization of hemoglobin, right? So how do you think about, you know, just to maximize your exposure to KOLs and make sure that they know that these points are your gold nuggets compared to your peers? And perhaps, you know, maybe just to pile on this, what's been the feedback like from KOLs on those specific potential differentiation?
It's actually great to think back a year and remember that conversation when we were really talking about a good, you know, well-supported hypothesis and one patient worth of data. What has been really nice over this year is to see that we have continuously and consistently made the same observation over and over again with regard to the rapid normalization of hemoglobin. Now, with regard to actually that differentiation and its potentially best-in-class, I'm gonna ask Baisong Mei to talk to you about, you know, the experiences we've had in interacting with KOLs, et cetera.
Yeah. Thanks. I think we from clinical trial perspective, we're looking for three category of endpoints to see the potential differentiation for normalizing the total hemoglobin. One is, of course, from hematological parameter perspective, and the other one would be the end organ function. We're looking to for multiple end organ function from central nervous system, pulmonary cardiac system, and liver and kidney on that, right? So that will allow us to understand the function of the organ, whether that we actually be able to stop the damaging, actually be able to improve upon the damage on that, too.
The third category is the patient-reported outcome, and that, for example, you mentioned about the fatigue and other parameters that we'd be able to see that from sickle cell, that fatigue is really a dominant of the complaint for patients that's more related to anemia, and that has been demonstrated in other anemia clinical trials in that, too. So when we talk with the physicians, and we talk with KOLs on that, and the first one we talk to that is to say, "Okay, if you have a normal total hemoglobin of 14 grams or 15 grams per deciliter versus 10 grams or 11 grams," and they say it's a no-brainer, patient will feel better, right? So that's kind of the, that what we were talking about. When we collect the clinical endpoints, we have multiple audience in mind, right?
We have a patient in mind to see what they are concerned about. We have the physicians in mind, and we have the payers as well as regulators in mind. So we have a totality of the data to be able to demonstrate the benefit of the reni-cel, be able to normalize total hemoglobin.
You know, one of the thing... Thanks very much, Baisong Mei. I think the other thing I'd like just to highlight is that looking beyond just KOLs, you know, what is great now with our new Chief Commercial Officer, Caren Deardorf, who brings us, say, a lot of experience in the rare disease and with drug launches, and I had the privilege of working on a number of very successful launches in the past.... We're actually also have started much of that preparatory work as we think about launch, and not just from a commercial point of view, but through the medical affairs landscape.
And we're engaging not just with KOLs, but actually also engaging with patients and patient organizations. So we're actually bringing that sort of broad approach to ensuring that there is clarity and an understanding about the differentiation and mechanistically, and very importantly, and more importantly, indeed, the differentiation from the impact physiologically, and clinically.
Just to add on that point is that, you know, the differentiation, those, some of the differentiation does take time to build, right? And accumulate. How long do you think, you know, for, so for some of the indications, like cardiovascular or pulmonary functions, kidney, how long do you think it will take you to collect? And, and also maybe strategically, how do you also kinda wanna sprinkle around the medical community to make sure that, you know, you're also kind of building traction and awareness of reni-cel versus the others?
You really touched on something very important when you talk about the strategy. And, you know, what we want to be absolutely sure is that when we actually interact with the various stakeholders, that we've outlined, patients, prescribers, frankly, the, transplant centers as well as payers and, and, the regulators, we want to be very conscious that when we deliver data, it's robust, we are, very happy, with it, and, ensure that, it is, as I say, very strong and defensible. With regard to the timing and when we understand some of these outcomes are going to deliver, I'm gonna pass the to Baisong, who is really dealing with what is a very good, problem to have.
Yeah, thanks. I think we certainly look in these three category of endpoints very closely, right? So, for example, hematological parameters, you're probably to see very relatively quickly. And for the patient-reported outcome, we already see that in other incidences, you know, between six-12 months, you will see some improvement, and then you will see more after longer follow-up on that too. For end organ function, so there are two side of that. On one side is actually, although it is relatively, you know, less studied, but there are data that have been published by different groups in the sickle cell space.
We also talk with many KOLs and our advisory board that after allogeneic transplant for sickle cell patients, you'll be able to see central nervous system in function changing and improving, and you'll see the cardiovascular system, that function changing. So you can see there's an indication that after this treatment of this bone marrow transplant for sickle cell patient, and you can see not only you'll be able to potentially stop the damaging of the end organ, but also could improve the end organ functions. So that's an area we'll look into that.
I think the key thing, I think the key message here is that the problem, but it's a really good problem to have, and it's one that I and Baisong, in our previous development experiences, have encountered, which is when you bring a new high-potency treatment to a disease, which is gonna substantially change outcomes, those clinical outcome measures that you used have not had that experience. They have not interacted with that medicine, that high-potency medicine, in that patient population. And so we are collecting those outcomes, but with regard to the timing of when we will know, that is actually a matter of exploration for us as we look forward.
And obviously, as we get a clearer understanding of the interaction between a high-potency medicine and a disease previously, you know, modestly, but modestly treated and these outcome measures, we will be able to speak with more confidence and concretely about the timing of when that will declare itself.
Great. Any questions from the audience? Maybe turning to IP. I remember that I met your team, I think the last day of ASH, and then the next day, there was a press release on your sublicense of Vertex. Certainly a very interesting one. Do you see it as a validation for your IP? And if so, how do you view that as... You know, there are a lot of gene-editing companies out there. What's your strategy to, you know, make sure that your IP estate is protected?
Well, I think the first thing I want to say is I violently agree with your assessment that this was an important validation and recognition by a very important peer in this space of the validity and the value of our IP. But with regard to the strategy and how we're going to build on that, I'm gonna pass this over to Eric k.
Yeah. Thanks, Gilmore. So with respect to the strategy, taking a step back, there's a lot of companies, as you mentioned, that are engaged in Cas9, Cas12 programs, and it's quite public who they are, and we wanna be an enabler of this. We wanna be collaborative and work with these companies, but we also want to make sure that we can strike a deal to get the value of the patents that we have. These are foundational patents. We believe everyone that's working in this area is gonna have to have a conversation with us. And as we think about structuring different deals, whether it's a small company, like Vor that we did this summer, which is more along the traditional biobucks, or the Vertex deal, which is more in terms of upfront cash, we can be creative.
And structure something so that way we can have a win-win scenario to enable potential partners to use this technology, but also give us either access to technology that they have via a cross-license or give us non-dilutive capital to, you know, continue to fund our development of our products.
Okay. We have about three minutes left, so I'm gonna try to combine two or three in one.
That's cool.
Maybe really quick, short answer. So one is, you know, what should be our base case for, you know, filing for reni-cel? You know, I think you talk about how we'll, you know, that the bar is 20 patients, and so you'll be well on your way, actually, by the end of this month, right?
Yes. So I think you've actually said it right. Go ahead, sorry.
Second question is, you know, you kind of laid out the fact that there is also in vivo pre-clinical POC coming.
Mm-hmm.
What does that entail?
Quickly. 20 patients, 16-18 months follow-up. We are on track to dose our 20th patient in this month timeframe, January timeframe. So when you actually follow out, that basically, feasibly, obviously contingent on agreement with agencies, puts us in the middle to the second half of 2025, based on that base case validated by the FDA and EMA. With regard to in vivo, what that entails is us actually sharing an update that confirms our achieving of preclinical in vivo POC. I'm not going to go into the specifics of a tissue or the target. We'll actually guide to that at a more appropriate time and tell you about that at a more appropriate time. But what is important.
Sorry, not very short, but this is something that we have built a lot of foundational work to achieve, and this will essentially enable us to validate a delivery formulation into which we'll be able to, as I say, plug and play additional targets as we build out our portfolio of in vivo medicines.
Great. I think that's the end of our session together. Thank you so much for joining us for the session today. It's great to have you on.
Thanks very much.
Thank you so much.
Thank you very much.