Well, thanks for joining us, everybody. I'm Terence Flynn, the U.S. Biopharm analyst at Morgan Stanley. Before we get started, for important disclosures, please see the Morgan Stanley Research Disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative. With that out of the way, we're very pleased to have Editas joining us this morning. Today, from the company, we have Gilmore O'Neill, who is President and CEO, and Baisong Mei, who is Senior Vice President and Chief Medical Officer. Thank you both so much for being here today. Maybe to start us off, just kind of a high-level question here. You know, back earlier this year, you announced plans to reprioritize the company's pipeline. Maybe just give us an update on kind of where that stands and the latest strategy for the company? Yes, thank you very much. It has been a very exciting year to date. Having rolled out the strategy, we have really focused significantly on execution. As part of our focusing, we terminated or divested some assets, and we were very delighted to have Shoreline take our NK oncology asset, and that has been a very exciting collaboration and/or divestiture, and they're very happy with how things are going, I'm pleased to say. From an internal point of view, we redeployed a substantial amount of our resources and capital to our CMC support for our EDIT-301 autologous ex vivo hemoglobinopathy therapy, both as a CMC and to clinical execution. Baisong's leadership has actually been very important here, in that he really has transformed the development organization. By the end of May, we were able to share that we had already doubled enrollment, indeed, had enrolled 20 patients into the RUBY study. Why did that matter? Because as part of our strategic refocusing on execution of 301, narrowing and sharpening our focus on in vivo, and then changing our business development posture, we also said that we would shoot for 20 patients dosed in the RUBY study by the end of this year. That matters because we felt that would likely be a meaningful efficacy cohort, and indeed, that hypothesis was validated when the FDA accepted the BLA filing for exa-cel, which includes about 17 patients in their efficacy data set, with 15 months-18 months of follow-up data. Overall, we've done, you know, very well there. We, I'm actually delighted to say that from the second part of the pillar, it was wonderful to announce just a few weeks ago that Linda Burkly had joined as our new Chief Scientific Officer. She will be an important catalyst in driving the building of our in vivo pipeline, which we believe ultimately is going to be the way to truly unlock the full potential for editing as a technology for therapeutics and actually also for Editas. I think the other piece that's important, and it's always hard to do, but when we described or reset our strategy and sharpened our focus, we did reduce our headcount by 20% in a very targeted manner, where we took out capabilities that we no longer required. And then we read out data, as we had promised, in June of this year at the European Hematology Association, and shared exciting data for our sickle cell program, where we demonstrated not just a robust expression of fetal hemoglobin in our first two patients who had at least six months data, where they crossed the threshold and maintained a threshold above 40% fetal hemoglobin, but actually also both corrected their anemias and returned total hemoglobin to physiologic ranges by the fourth or fifth month, which is a very good timeline because that's when you tend to see plateauing, but also full washout for any donor blood that had been given to the patients during their transplant. So all in all, a very satisfactory outcome, even more so when we looked at the third and fourth patients with shorter follow-up, but where the trajectories of fetal hemoglobin expression followed and were very consistent with what we saw in the first two patients. And indeed, in a webinar a few days after EHA, we shared not just those data, but the data from our first thalassemia patient, EdiTHAL, who again had a very similar trajectory with their fetal hemoglobin. And then on the heels of that, we were able to raise a net $117 million. And what that basically means is between the reorganization, the redeployment of the capital, our cash runway extends out into Q3 2025. And that matters because with that 20-patient cohort that we will have, we are on track to dose by the end of the year. If you consider that the efficacy data set 15 months- 18 months, with 17 patients is kind of the range of efficacy data set, and obviously this is contingent on our discussions with FDA and their agreement, then you could actually see that, it's plausible, that we would have a similar data set, 15 months-18 months with 20 patients by the middle of 2025. Yeah. Okay, that's a great overview, great place to start. The other one, you know, there are kind of two subsequent ones I want to follow up on, but the first is just differentiation. Obviously, you know, the pace of genetic medicine is accelerating here. There's a number of new technologies, new platforms that are being rolled out across the landscape, and so maybe just help us think through where you think you can differentiate most in terms of the CRISPR platform that you guys are working towards, and again, this pivot towards in vivo. So I'd be delighted to do that. I think a key thing, and one of the things that actually drew me to Editas, was, Editas's proprietary AsCas12a enzyme, CRISPR enzyme, which actually we believe is meaningfully differentiated. And indeed, in our hands and the hands of others, it is both more efficient in editing than Cas9 and actually, more specific, or higher fidelity. And indeed, again, across a number of labs, including our own, where we submit the two to a sort of global, genome screen for off-target, we cannot detect, with AsCas12a whereas we can with Cas9. This is obviously within the constraints and limitations of the detection systems, but that's still a meaningful difference from off-target. And then from an efficiency point of view, we actually see real benefits. Indeed, we were very happy with the efficiency of editing that we actually were able to share in June for the drug product from 12 patients enrolled in both in the RUBY and the EdiTHAL study, so 9 + 3, respectively. So that's the one, I think, critical differentiation because that has an impact on dosing, risk, et cetera. From the point of view of how we would then apply that as we move into our in vivo pipeline, I think that's that differentiation or separation for the technology itself is meaningful. I think the other thing is actually also our approach, and we've talked about that before. One of the reasons I'm so happy to welcome Linda is that she and I, and frankly, Baisong, and the senior leadership in the organization share a very similar mindset about the criticality of, you know, differentiation at a commercial side. But obviously, you want to focus on areas with a high probability of technical translational, and this is important and probably one of the reasons I'm so happy with Linda, who is not just a successful drug finder and biologist, but somebody who has actually had the experience of and the understanding of the criticality of understanding the patient and the translation experiments before you even select the target. And so that translatability and obviously the regulatory path. So as I say, our philosophy will very much focus on going to areas where we believe our technology or our focus is going to create a meaningful difference, for patients. We're not necessarily interested in going to an area where there are lots of technologies we can leverage and lots of players already there. So I think that's a critical part of how we are thinking about that future of in vivo. Okay. We look forward to sharing more about that in appropriate time. Great. You know, as Linda just has her feet under the desk. Can you give us any early insights in terms of like, where, what types of diseases you might be interested in? I think it's too early to say. What we did say when we rolled out at JP Morgan was that we did articulate our indication interest already in targeting hematopoietic stem cells in general, and specifically the gamma globin promoter, which we believe is a meaningfully different choice for editing. And indeed, we believe that's one of the reasons that we're seeing that correction of anemia with our 301 product. So we believe that actually bringing that to the in vivo space is really very valuable and meaningful. And then from the point of view of looking beyond that, we're gonna talk about that more in the future. Okay, understood. One of the other challenges broadly for genetic medicines is delivery, and, maybe just, again, high level, you know, where are you now? And as you think about this pivot to in vivo, is this gonna be an internal, effort, or are there external opportunities to bring technologies in? Again, I know, you know, a lot of different technologies out there, but maybe where are you most focused as you think about that? Well, when we rolled out the strategy in January, we sort of said there were three pillars, and I just recapitulate the 301 focus, the in vivo focus, and then the business development. That third business development posture had two elements. One was sub-licensing, but the other was a shift in posture, that we don't actually have to invent everything. That we are actually, in fact, actively looking for those capabilities, or technologies that would complement ours to drive and realize our in vivo vision. And so that is a, you know, an exercise we're going through. With regard to something more specific, what we did do was take out AAV. So when we actually refined and narrowed, sharpened the pipeline, we stopped any further ex vivo cell therapy discovery. We actually also stopped the use of AAV and have looked beyond to non-viral delivery for in vivo. And, you know, our, I think the nanoparticle space, general and the lipid nanoparticle space specifically, is an area of significant interest. We do have in-house expertise, but we are actually looking to make sure we get the right balance and complementarity and leverage where, you know, the terms and the lineup or the matchup and synergies would actually be most effective. Why remove AAV from the equation? What was kind of the key piece of it? Well, I think there are a number of elements. I think the key piece was that you want to really focus on areas where you have true and a deep expertise. I think there are other, you know, some other additional challenges, but that was the key one, which is, you know, we are an editing company. That's where our expertise sits, that's where we should actually focus. Okay. And as you think about the BD licensing side, do you feel like there's enough opportunity out there? And is this mainly among private companies? Is it among academic institutions? Like, where does the opportunity set lie in terms of these delivery technologies? We actually see it as sort of a broad set of opportunities across that spectrum you outlined from, you know, academic to private to public. And obviously, what we will finalize in those kind of set of situations would be the ideal and the optimal balancing of our capabilities and those capabilities. And also, you know, how we think about the degree of investment. Okay. Okay, great. Maybe Baisong, I'll pull you into this. You know, do you, y ou know, Gilmore touched on some of this, but just the data that was presented at EHA, w hat else stood out to you? As, again, I think investors are trying to differentiate as we think about two other products potentially coming to market early next year. What is most encouraging about the data that you saw as you think about that differentiation piece? Yeah. Yeah. I, I think Gilmore touched upon it a bit, but I think happy to follow up on it, but more on that. Maybe back up a little bit. When we designed the EDIT-301, this is a rational design, we actually did head-to-head comparison between the Cas9 enzyme versus the AsCas12a enzyme, between the BCL11A target versus also the HBG1/2 target in that too. Then Gilmore touched upon the enzyme, and to add on that is actually for the target. When we do the head-to-head comparison between the BCL11A and the HBG1/2 promoter, we found that the HBG1/2 promoter targeting give us more red blood cell production, red blood cell health, and, and more efficacy process. Then when we last December, we see our very initial data, we find, wow, this first patient actually go to, did go to normalized total hemoglobin, which is very exciting to us. Then in the June, we see additional patients go to normal range, and also the other two patients go to the same trajectory. This is very important to us because I think that the two molecule ahead of us, and they also have very good results, for example, preventing the VOEs, which is great. But when we look in the rare disease eyes, you know, when we have very limited choice, when we first get into the to target the most severe symptom, which is, in this case, for sickle cell. But the sickle cell symptom is way beyond VOEs. So we are looking to that. One of the important thing is actually to be able to have normalized the total hemoglobin to correct the anemia. Sickle cell is actually called sickle cell anemia also in it before. So when we see this data, we feel there's good chance for us to actually to be able to differentiate, to beyond correcting the VOE, then we'll be able to normalize total hemoglobin that is related to the end organ function and the quality of life. Right. So that's kind of three category direction we're looking to that. One is, of course, we're looking the lab value, the symptomological parameter. But we're also looking to the end organ function, for example, cardiopulmonary, we do cardiac or pulmonary function and liver function and kidney. Then the patient report outcome is very, very important in this case. For example, one of the important complaint from sickle cell patients is the fatigue, and that fatigue is very much related to the anemia. We all actually experience that, too. When we go to high altitude, we feel fatigue, we're short of breath. So that's kind of the direction we're looking to. When I look at that is, generally, I've been working in rare disease for 20 years, more than 20 years now. And when you go to the rare disease, you start with the severe symptom you're trying to address. Now we're trying to together elevate the standard of care for those patients. Hopefully, that's kind of the direction we're getting to. I'm sure there'll be, you know, more things behind us in the future to be able to even further elevate. I think that's the direction we're going to treat the disease. Okay, great. What, and I know we had the presentation at EHA, and Gilmore, you kind of walked through the highlights there. But as we think about the next update, I think you guys have said sometime by the end of this year. I'm assuming ASH is probably one of the key venues, just given, you know, that's where we typically see data. Is that a fair assumption? Then anything more you can share in terms of obviously beyond longer follow-up on the existing patients? I'm assuming more patients, but again, anything else you can tell us about kind of how you're thinking about that updated data disclosure? Yeah, so two things. First, we're maintaining our optionality for the end of the year. It could be ASH, it could be webinar, it could be a combination, because we have that optionality. We obviously share that sooner and closer to the time presentation. You're absolutely right. The minimum data we'll be showing will be those five patients, one thalassemia, four sickle cell patients that we've already presented with longer follow-up. And indeed, all those patients would have had more than 6 months of follow-up, which will be very interesting. Indeed, we have patients with more than a year of data, and we would have additional patients. And what we would share, without going into specifics, again, we will talk more about that closer to the event, will be again our hematologic parameters, safety, VOC, VOE data. Yep. And do you think, again, you talked about this a little bit, but in terms of the other two products, let's say they do secure approvals later this year, early next year, does that change the bar in terms of what FDA, how FDA approaches unmet need, I guess, in this space? Meaning maybe they hold you guys to a somewhat higher bar than they did before because you already have these two other products. And have you talked to FDA at all about that in the event that that does happen? So I go to perhaps some remarks, and then Baisong can talk a little more about that. We don't necessarily see that. I mean, the interesting thing is, Baisong and I have worked in the rare disease space on the development side and have been on point negotiating with regulators, and that's actually not the path that we see. I know people talk about it a lot, but in fact, sometimes it's actually the opposite. You know, as comfort is, you know, developed, precedent with the technology or a rare disease, or indeed some of the outcome measures, indeed, you can sometimes see things going the other direction. And so that's obviously... I'm not saying that's going to happen, but I think that's one of the things that we're certainly well used to and accustomed to discussing with the agency and other agencies as well. So we don't see that as a bar. Indeed, we actually see it as an opportunity. Baisong, I don't know if you want to add to that. Yeah, absolutely. Just continue on the last topic, is that, and last point is actually, as an opportunity, we really feel that these two molecule are recent [sub-VOE] is an opportunity for us. T hat help the agency and help the community understand better the technology, right? Both technology, actually, chromosome gene editing, right? So gene, gene, chromosome editing on that, too. So then, we will see that how the—how do we know this technology, how safe it is, and how well this can actually change patient life. So that's gonna be very positive for us. And in terms of the, you know, fast follower, whether you have a higher standard, and as Gilmore said, there are many examples, as long as your drug is safe and efficacious, and all the players or stakeholders here, from agency to the patient community, to the HCP, and to the payer, they'll all welcome choices, right? So we feel EDIT-301 will be a competitive product and potentially differentiated product, as Gilmore just mentioned earlier. So I feel this will be very well welcomed by the community and by agency, by payers. And we already, our investigator has been, you know, very experienced in many different trials before, without specifics. They very much welcome that we have this EDIT-301 molecule, we have different approach. They appreciate that, and they're actually very excited, especially now we see actually be able to correct anemia, and they're very excited to see before. They say, "My patient was much lower before in a different trial." Yeah. So that's kind of we see. Yeah. So, you - Baisong used the term fast follower and differentiation. I'd say that fast follower with potential difference is potential, strong potential for differentiate. We believe actually puts us in a very good position, because frankly, this is a new space that's been developed. And we appreciate the, you know, the, that the trailblazing work done by our friends at bluebird and ultimately Vertex and CRISPR. Yeah, and I think that will actually enable the sites who have to make an adjustment, both from a capacity point of view. The development of autologous ex vivo basically means that whereas currently, a patient with sickle cell or thalassemia has to find a highly matched donor, 10%-20% probability, at best, they will find them. So we're going to increase that eligible patient population 5-fold to 10-fold. So those sites are going to have to, you know, expand and lean in to support that, patient, growth. And then obviously, they're going to move from what is essentially a reimbursement model around, which is largely procedural, to one that is a combination or hybrid of both procedure, transplant, harvesting of cells and transplant, but actually also the pharmaceutical reimbursement component as well. And then obviously, the patient community is going to have to, adopt. So what we're actually looking to and expect to see is, you know, really, a very, you know, compelling set of work done by, exa-cel or bluebird and, and, Vertex. But that will enable us, really, I think, to both, exploit that work, but actually also take all the lessons learned as we, move through. As we said before, we anticipate the vast majority of patients. We, we estimate there are between, or you know, I think the, the analyst consensus is somewhere between 20,000 patients-35,000 patients who will be eligible in this country from the prevalent patient population. That's not counting incident. Prevalent patient population that'll be waiting for this therapy, and the vast, vast majority of those patients will be waiting by the time of our approval. As I say, you know, we can't make promises. We have to agree with the agency, but as I say, if we have 20 patients dosed by the end of the year and we're on track for that, then we're in a very good place, you know, by mid-2025. Okay, great. Two follows on that. So any perspective on the Ad Com coming up? So, you know, one of those two companies has an Ad Com, one of the products doesn't. Any idea on why that would be the case, and, you know, how you think about some of the key questions FDA might be considering for that Ad Com? I think there are two big areas to consider with the Ad Com, and it'd be very interesting for all of us to actually see that on the 31st of October. One is around CRISPR. This is the first time that our CRISPR technology is being approved. And I would think a big focus of the questions will be on CMC and other pieces on just essentially the risk. I will say that from risk point of view, I think much of the discussion around risk on the clinical side will effectively be discharged by the lentiviral discussion last year when there was an Ad Com for bluebird's thalassemia program. And then on the sickle cell disease side, I think the benefit risk, you know, once they have talked about the CMC, will actually be quite clear. We'll have a good sense of that, but I think it'll be very interesting for us all to see, to get a breakdown of what additional clinical data has been collected, and actually also to get, maybe get a more granular view of what the fetal hemoglobin and hemoglobin responses were. But I anticipate this will be a, I think we expect this to be a positive one. We actually feel that the Ad Com was very clear last year when they recommended unanimously the approval of bluebird's product for thalassemia with the benefit risk, and that was actually with some myelodysplastic syndrome outcomes in some of the patients. So I think overall, I think this is a, I think we will learn a lot, and that's how I think we see it lining up. Okay, great. And the other question you mentioned, site capacity. That's something else I know from just following the CAR T space, has been pretty important. There are some centers that, you know, have been investing to build out their capacity, because they don't have a number of beds, so et cetera. So what have you learned so far about thinking about that aspect, the site capacity? Because you talk about 20,000 patients-30,000 patients, but do the sites, are there enough sites to, you know, work through that there? So the broad question, and I'll pass, Baisong will have some granularity about the next part. There are two pieces. I'd say the one is, I think overall, we actually anticipate that investment will be made. Okay. The other piece, which is of more immediate interest to us, okay, so what happens with the balancing capacity with our ongoing study and with a commercial drug? And we actually anticipate, in fact, our experience today has been that this is not going to be a challenge for us. Based on maybe you could build on your conversations with the sites and the investigators. Yeah, yeah, certainly. I think it's great you actually mentioned the CAR T experience. That's kind of what we see in this space, too, and every stakeholder in this space, when you have learning and building up space. So that's where feel the early uptake will be relatively slow and taking time and eventually will be to all the systems there. And many of our sites actually have both CAR T and this hematopoietic stem cell transplant experience. And they've - they, several of them, as you mentioned, they actually already expand the space of hematopoietic cell transplant because they see a lot of demand in there, too. So I think the general thinking is to see - you will see this entire space grow. It's like the CAR T cell space and going there, too, because then people appreciate the value of that, appreciate how this strategy can do. That's also validated by ICER's report, right, to see how this space grows. So we're optimistic, but I think it will be taking time and to get all those set up on that, too. So I think that what you get opportunity to really catch up to, to actually be able to help the patient. I think the other point, of course, is for the near term, is that we do not see a challenge with, you know, commercial launch for our recruitment. And you can- Yeah. You know, based on conversations. In fact, the ongoing uptick in interest, we saw an 8-fold to 10-fold increase in inbound from patients, and indeed, the investigators continue to recruit into both studies, and really have articulated desire to continue that. I think, based both on the data they've seen, but frankly, actually also that where a patient is eligible in a clinical trial setting, it's actually easier than, you know, certainly working out, sort of going through the teething of that sort of, you know, early launch of this new type of therapeutic. Okay, great. Maybe just in the last couple of minutes, you know, the IP portfolio is, again, another part of the company's strategy here. Mm-hmm. Maybe just walk us through on kind of plans to monetize that and, and how should we think about next steps, milestones to be on the lookout for? Yeah. So very quickly, Cas12, we can license both Cas12 and Cas9. Cas12, we don't have any Cas9 products ourselves, so we're focused on our Cas9 sublicensing. We have ongoing sublicensing to developers. Indeed, Vor was one company that announced just three weeks ago that they had, and sorry about that. And then, excuse me, with regard to programs that are later, they will need our IP. We have a robust set of foundation IP sublicense from Broad, Harvard, MIT. Some, not all, of the IP that's required by others is subject to interference. I know there's been some confusion about that, but we have other IP, not subject to any interference, which also people will require. And so we, you know, expect that we will be doing licenses for that IP for these programs as they come close to approval and launch. Okay. Okay, would any specific products you can point to? Well, obviously, the first one that will be coming up for approval is Vertex CRISPR exa-cel. Have you had any conversations with them yet that you can tell us about, or? So we, we will share if and when we have an agreement, but obviously, we're looking to what we, what we philosophically embrace is the idea that we want to make this technology available to patients and therapeutic developers, and obviously do it at terms that are meaningful and valuable for both parties. Okay. Well, thank you very much, Gilmore, Baisong. Really appreciate the time today. Thank you. Thank you.