Editas Medicine, Inc. (NASDAQ:EDIT) J.P. Morgan 42nd Annual Healthcare Conference January 9, 2024 7:30 PM ET
Company Participants
Gilmore O’Neill – CEO
Baisong Mei – CMO
Erick Lucera – CFO
Conference Call Participants
Brian Cheng – J.P. Morgan
Brian Cheng
Good afternoon. Thanks for joining us for another session at the 42nd J.P. 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. [Operator Instructions]
Gilmore, the stage is yours.
Gilmore O’Neill
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 have 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.
And 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 center 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 place 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 AsCas12 or AsCas12a 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 asked their forgiveness as I described them as season, 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 edging 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 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 human medicines forward.
And 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 nonexclusive 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 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 20th patient in this January timeframe 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 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 are talking about 18 to 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.
And 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 physiological large 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 when we 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 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 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 sublicensing our IP into 2024, as you know and as I said, as a potential source of non-dilutive capital.
Indeed, actually, it was also a 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.
Vaso-occlusive events, from a pathologic point of view, are essentially events associated with severe and profound disabling pain, necessitating emergency room visits, and in many cases, 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 syndrome 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 buildup to joining our clinical trial and prior to treatment, experienced 100 days on average of in-patient 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 it 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. And what we have done is taken a unique approach to treating this, a validated natural history approach is to upregulate fetal hemoglobin. We are leveraging that observation and targeting the HBG1 and 2 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 describe 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 4 per year, 3.9, but 4 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 see visually probably more effectively and meaningfully what that high rate of VOEs looks like prior to treatment. What’s really exciting is to look at the right in orange and see that all treated RUBY patients filed 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 the 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 bring that and disaggregate the data. First, every patient — every woman who has 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 five 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 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 about 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 and 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’ve 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 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 timepoints to RUBY, we saw a similar trajectory for upregulation expression of fetal hemoglobin and total hemoglobin.
But let me also talk to you about the definition of transfusion independence is. So, 9 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 a patient? Well, I’m sure many of you have 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 9 grams per deciliter would make that journey up five floors extraordinarily challenging and almost impossible. Why? Energy, massive fatigue and frankly, profound exercise-induced shortness of breath. And 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.
So, the other thing that’s important is not just the level of hemoglobin that we achieve that, 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.
And then it’s actually worth pausing and just reiterating why it is that we see this potential for it to be best-in-class based on the 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 empiric head-to-head study, preclinic set of studies, that red cell production was 7superior with a gamma-globin targeting versus BCL11A, with also better proliferative capacity or growth capacity as well as long-term red cell health and survival.
And 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 a 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 from — 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 — 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. But we also couldn’t do this without the support of 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.
Question-and-Answer Session
Q – Brian Cheng
Great. Let’s start off with a Q&A. We’re joined by Gilmore O’Neill; CFO, Erick Lucera; and CMO, Baisong Mei. [Operator Instructions]
So, I’ll start with a couple of questions. Commercially, — first is on commercial opportunity of reni-cel in sickle cell specifically. Now, we have seen remarkable advance across some of your gene editing 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 editor.
But when you look at the sickle cell space, we have two products that just entered the market, right? So, what are your guardrails today to make sure that — we’ll talk about this that you want to 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 going to be the fast follower to maximize market penetration?
Gilmore O’Neill
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 hot focus for us.
The second thing is worth sharing is that I have been — had the privilege to develop and have approved medicines in the rare diseases, as has Baisong; and our Chief Commercial Officer, Caren, and 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 continued 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.
And one of the regulatory requirements is that an unmet need is recognized by the agency to grant RMAT. And they did it after reviewing a data set, including clinical data sets, 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 Erick to talk a bit about that.
Erick Lucera
Yes. Thanks Gilmore. Obviously, we’ll be tracking the two launches very carefully. I think there’s going to 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 has been built and having a little bit better profile.
Brian Cheng
Any questions from the audience? I think last year, around this time, I think I remember that you were — 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. There’s anemia, fatigue, and organ function [Indiscernible]. And you’re also seeing really rapid normalization of hemoglobin, right?
So, how do you think about 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, maybe just to upheld on this, what’s been the feedback been like from KOLs on those specific potential differentiation?
Gilmore O’Neill
It’s actually great to think back a year and remember that conversation when we were really talking about a good, well-supported hypothesis and 1 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 potential to be best-in-class, I’m going to ask Baisong to talk to you about the experiences we’ve had in interacting with KOLs, et cetera.
Baisong Mei
Yes. Thanks. We — from a clinical trial perspective, we were looking for three categories of end points to see the potential differentiation for normalizing their total hemoglobin.
One is, of course, from hematological perimeter perspective and the other one would be the end-organ function. We’re looking for multiple end-organ function from central nervous system, pulmonary cardiac system, and liver and kidney on that. So, that will allow us to understand the function of the organ, whether we actually be able to stop the damaging, actually be able to improve upon the damage on that, too.
The third category is a patient reported outcome. And that, for example, you mentioned about fatigue and other parameters that we’ll be able to see that for sickle cell, that fatigue is really a dominant complaint for patients that’s more related to anemia and that has been demonstrated in other anemia clinical trials in there, too.
So, when we’re talking with the physicians and we talk with KOLs on that, and the first, when we talk to that is to say, okay, if you have a normal total hemoglobin of 14 or 15 gram per deciliter versus 10 or 11, and they say that’s a no brainer, patients will feel better. So, that’s kind of — that what we were talking about.
When we clinical — 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 to be able to normalize total hemoglobin.
Gilmore O’Neill
One of the thing — thanks very much, Baisong. I think the other thing I’d like just to highlight is that looking beyond just KOLs, but it’s great now with our new Chief Commercial Officer, Caren Deardorf, who brings us a lot of experience in the rare disease and with drug launched that 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 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 differentiation and mechanistically and very importantly, and more importantly, indeed, the differentiation from the impact physiologically and clinically.
Brian Cheng
Just to add on that point is that the differentiation, those — some of the differentiation does take time to build, right, and accumulate. How long do you think — for some of the indications like cardiovascular, pulmonary functions, kidney, how long do you think it’ll take you to collect them?
And also maybe strategically, how do you also kind of want to sprinkle around the medical community to make sure that you’re also kind of building traction and awareness of reni-cel versus the others?
Gilmore O’Neill
You’ve really touched on something very important when you talk about the strategy. And 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 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, I’d say, very strong and defensible.
With regard to the timing and when we understand some of these outcomes are going to deliver, I’m going to pass it to Baisong, who is really dealing with what is a very good problem to have.
Baisong Mei
Yes. Yes, thanks. I think we certainly look at these three category of endpoints very closely, right? So, for example, hematological parameters, you probably could see it very — relatively quickly. And for the patient reported outcome, we already see that in other incidences between six to 12 months, you will see some improvement and then you will see more after a longer follow-up on that too.
For end-organ function, so there are two sides of that. On one side is actually, although it is relatively less studied, but there are data that has been published by different groups in the sickle cell space.
And we also talked with many KOLs in our advisory board that after allogeneic transplant for sickle cell patients, you’ll be able to see central nervous system function changing and improving and you’ll see the cardiovascular system that functioning changing.
So, you can see that the indication that after this treatment of this bone marrow transplant for sickle cell patients, and you can see not only be able to potentially stop the damaging of end-organ, but also could improve the end-organ functions. So, that’s an area we’ll look into that.
Gilmore O’Neill
I think the key thing — I mean the key message here is that — and the problem, but it’s really good problem to have, and it’s one that I and Baisong in our previous developing experiences have encountered, which is when you bring a new high-potency treatment to a disease which is going to 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 — modestly — but modestly treated on these outcome measures, we will be able to speak with more confidence and concretely about the timing of when that will declare itself.
Brian Cheng
Great. Any questions from the audience? Maybe turning to IP. I remember that I met your team, I think, at 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 that as a validation for your IP? And if so, how do you view that as — there are a lot of gene editing companies out there, what’s your strategy to make sure that your IP assay is protected?
Gilmore O’Neill
Well, I think the first thing I want to say is that I valiantly agree with your assessment that this was an important validation of — 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 going to pass this over to Erick.
Erick Lucera
Yes. 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 want to be an enabler of this. We want to 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 going to 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 BioBox, 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 continue to fund our development of our products.
Brian Cheng
Okay. We have about three minutes left. So I’m going to try to combine two or three in one.
Gilmore O’Neill
Let’s go.
Brian Cheng
Maybe a really quick short answer. So, one is, what should be our base case for filing for reni-cel? I think you talk about how exa-cel — the bar is 20 patients. So, you’ll be well in your way, actually by the end of this month, right?
Gilmore O’Neill
Yes. So, I think you’ve actually said it right. Go ahead, sorry.
Brian Cheng
And second question is, you kind of laid out the fact that there is also in vivo preclinical POC coming. What does that entail?
Gilmore O’Neill
Quickly, 20 patients, 16 to 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 exa-cel.
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 at the 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.
Brian Cheng
Great. I think that’s the end of our session together. Thank you so much for joining us for the session today. It was great to have you on.
Gilmore O’Neill
Thanks very much.
Brian Cheng
Thank you so much.
Gilmore O’Neill
Thank you very much.
