Editas Medicine at Cantor Global: CRISPR Shift and Financial Resilience

Published 04/09/2025, 19:44
Editas Medicine at Cantor Global: CRISPR Shift and Financial Resilience

On Thursday, 04 September 2025, Editas Medicine (NASDAQ:EDIT) presented at the Cantor Global Healthcare Conference 2025, showcasing a strategic pivot towards in vivo CRISPR editing. The company emphasized the promising potential of its lead asset, EDIT-401, while addressing paused programs and financial stability. Despite some challenges, Editas expressed confidence in its future outlook and market strategy.

Key Takeaways

  • Editas Medicine is focusing on in vivo CRISPR editing with EDIT-401 as the lead asset.
  • EDIT-401 aims to reduce LDL cholesterol by 90% in preclinical models, with human trials targeted for 2026.
  • The company paused its in vivo sickle cell program to optimize assets.
  • Financially, Editas has a cash runway extending into Q2 2027, with $179 million in cash.
  • The company is exploring additional liver targets and a new undisclosed tissue target.

Financial Results

Editas Medicine reported a strong financial position, with a cash runway extending into Q2 2027. The company ended Q2 with $179 million in cash, providing sufficient funding to advance EDIT-401 through human proof-of-concept and beyond. Additionally, resources are allocated for discovery work, including the optimization of the hematopoietic stem cell (HSC) program and identifying an additional target by the end of the year.

Operational Updates

EDIT-401:

  • Selected as the lead asset, targeting LDLR.
  • Achieved a 90% reduction in LDL cholesterol in preclinical models, including non-human primates.
  • Human proof-of-concept is targeted by 2026, with Phase 1 trial sites under exploration.

In Vivo Platform:

  • Focused on in vivo delivery via simple infusion for durable, lifelong benefits.
  • Utilizes a scalable, off-the-shelf lipid nanoparticle (LNP) manufacturing process.

Sickle Cell Program:

  • Advancement paused to optimize the asset, including payload and delivery mechanism adjustments.
  • Open to partnership opportunities for further development.

Pipeline Expansion:

  • Plans to announce a third tissue target by the end of the year, distinct from liver and HSC.
  • Identified over five potential opportunities within the liver space.

Future Outlook

Editas Medicine is set to continue its development process for Good Manufacturing Practice (GMP) manufacturing. The company plans to conduct a formal dose range finding study and advance its off-target editing package. An Investigational New Drug (IND) or Clinical Trial Application (CTA) submission is expected by 2026. The commercial strategy targets patient groups with higher baseline levels of LDL-C, starting with refractory heterozygous familial hypercholesterolemia (HEFH) patients, and seeks partnerships for broader population studies.

Conclusion

For a detailed account of Editas Medicine’s presentation and strategic plans, please refer to the full transcript below.

Full transcript - Cantor Global Healthcare Conference 2025:

Eric Schmidt, Biotechnology Analyst, Cantor: Okay. Good morning, and welcome back to day two of the Cantor conference. My name is Eric Schmidt. I’m one of the biotechnology analysts at the firm, and I’m delighted to host our next session with Editas. We’re gonna be doing a fireside chat with Gilmore O’Neill, the president and CEO, as well as Amy Patterson, SVP and CFO.

So thanks, guys. Very timely chat. It is coming off some news earlier this week, but maybe before we get to the the very latest on your product development plans, Gilmore, just give us a quick overview of where Editas stands, including your cash, your cash burn, etcetera.

Gilmore O’Neill, President and CEO, Editas: Yeah. Thanks very much, Eric. Great to be here. So Editas is actually nicely positioned, we have made significant progress in completing our pivot to being a fully in vivo CRISPR editing company. Over the last year, we had planned or outlined that we would advance a number of programs in our portfolio, mid year make a selection for a prioritized program, but in additionally also announced near the end of the year a third tissue building on our targeted LNP platform and technology.

As you know earlier this week probably coincidentally leading into the investor conference season, we announced that we had selected zero one as our lead asset that will go into the clinic. It is an LDLR targeting program that substantially and to an unprecedented level of 90% reduces LDL cholesterol and it enables us to get to human POC in a meaningful time frame and generate human value by the 2026 within our current cash runway and our cash runway extends into Q2 twenty twenty seven.

Amy Patterson, SVP and CFO, Editas: Well said.

Eric Schmidt, Biotechnology Analyst, Cantor: Okay. Well let’s talk, let’s maybe just high level back up to how we got here. What Editas has been around now for many years as a as gene editing pioneer. What is special about your gene editing technology and and now obviously you’re applying it in a different direction, but but from a core principle standpoint, why is why is Editas differentiated?

Gilmore O’Neill, President and CEO, Editas: Well I think let me start with one point which is I think CRISPR editing is differentiated from all other therapeutic technologies in that it has demonstrated a probability of technical success in translating from the pre clinic to the clinic at a level that’s not been seen for other platforms worth saying. Then within that context what differentiates Editas is a know how that we have consciously built around mechanistically differentiating how we use CRISPR to functionally up regulate or increase the levels of proteins that have therapeutic benefit. And I think that is a critical part of our secret sauce. I think the other thing is that philosophically sort of at a corporate level, we have determined from an early stage that while CRISPR can do many things, we should really focus and choose to go after areas with an efficient use of capital that will get to human or demonstrate human value as quickly as possible.

Eric Schmidt, Biotechnology Analyst, Cantor: Okay. Two points in there I guess, one is the decision to go after upregulation of biology and the second is the decision to maybe go in vivo. So let’s, tease apart those two. In terms of upregulation of biology, why is this not intuitive or or or maybe a little bit off the beaten path relative to other CRISPR companies and how many scenarios where have you looked at where you identified biology that can be up regulated? I think that what

Gilmore O’Neill, President and CEO, Editas: differentiates it is or further what enables it is that we live in 2025 and that means that we don’t have to go back and say well has this target been pharmacologically validated? We now have the luxury of working with very well curated large data sets that have genotype data and phenotype data in sort of large populations from around the world and that enables us to substantially de risk targets using human genetic data as opposed to pharmacologic data. That immediately opens up the possibility that you can do things that no other technology can do. So I think that’s how we kind of went to say well how can we be different from others and so that’s how we manage to do that while derisking it and I think edit four zero one is a nice example of that because it leverages a genetic human experiment and we’ll come back to that in a second.

Eric Schmidt, Biotechnology Analyst, Cantor: I know you want to talk

Gilmore O’Neill, President and CEO, Editas: about it. No, no, worry, don’t worry. But I think that was the important point. Think you had, there was a second

Eric Schmidt, Biotechnology Analyst, Cantor: part What about in vivo?

Gilmore O’Neill, President and CEO, Editas: Why in vivo? Oh yes and why in vivo? So I think the in vivo approach really is demonstrated again by many of the issues we’ve learned from dealing with cell and particularly autologous cell therapies. The cost of goods is high and the cost of development use is high and the complexity of use is high. Going to in vivo means basically taking our machinery delivering directly to the human cell of interest through a simple infusion.

So idea is and what we’re all trying to drive to now is a point where a patient can go in and have an infusion, it maybe spends two hours and $10 in the hospital fire park which is an important these days and enables them actually then get a meaningful and lifetime and durable benefit from that simple infusion, which is totally different from having cells collected then going through a transplant and spending a month in a high dependency or ICU situation to recover from transplant. That’s the first thing and that’s kind of obvious even with The United States but also means that we can ubiquitously use this technology around the world because it’s much simpler and easier to use. The cost of manufacturing is much lower, the margins are better and ultimately it’s going to be easier for healthcare systems to really understand how to use these. And I think very importantly and I want to summarize, we have found we believe an economic model whereby with that scalability, with that ease of use and with the durable lifelong benefits provided, you can actually create substantial benefit for patients, meaningful savings for healthcare systems and payers, but also ultimately also create value for our shareholders.

Eric Schmidt, Biotechnology Analyst, Cantor: So putting the two together, Gilmore, up regulation genes with an in vivo targeted approach. Anyone else doing that?

Gilmore O’Neill, President and CEO, Editas: It’s I don’t don’t think so. I want to be always careful about that.

Eric Schmidt, Biotechnology Analyst, Cantor: I’m not I’m not aware of this.

Gilmore O’Neill, President and CEO, Editas: We’re aware

Eric Schmidt, Biotechnology Analyst, Cantor: of that. How many different applications or opportunities have you identified where that strategy could

Gilmore O’Neill, President and CEO, Editas: could make sense? So if I was just to say within the space of the liver because that’s an area of liver, we’ve identified in excess of five and more potential opportunities there and I would say higher end for a point of view of just technical opportunities. We haven’t really explored with as much rigor as we will in the future looking at other tissues but even within the liver we actually see significant opportunity there.

Eric Schmidt, Biotechnology Analyst, Cantor: Okay. We can get to four zero

Gilmore O’Neill, President and CEO, Editas: one now.

Eric Schmidt, Biotechnology Analyst, Cantor: So you recently unveiled this as your lead program, EDIT four zero one. Yeah. Just why don’t you walk us through what it is and why it’s of interest?

Gilmore O’Neill, President and CEO, Editas: Well we’re interested because we think that it basically can transform the hyperlipidemia treatment paradigm not least because we’ve seen robust preclinical efficacy with 90% reduction in LDL cholesterol across not just nonhuman primates which have demonstrated a high positive predictive value in translating interventional cholesterol lowering studies from the preclinical space to the clinical space. But we’ve actually also seen it across multiple models including models with high baseline LDL C and that was a mouse model with a high fat diet and we’ve actually also seen it in a model of heterozygosity for the LDLR mutation which essentially reflects and is a good reflection of the heterozygous familial hypercholesterolemia genotype in humans. So that’s the first thing from an efficacy point of view and why does that matter? Well I’ll have a slide later but the point is it is an unprecedented level of reduction that hasn’t been seen and doesn’t exist in the current standard of care. As I’ve already alluded to, with an in vivo delivery, it’s a potential one time treatment.

You go in for your two hours, you have your infusion, you go home and you don’t have to worry about taking an injection or a pill for the rest of your life. The market potential is sizable. There are multiple segments within that market from refractory heterozygous familial hypercholesterolemia to high risk patients with existing cholesterol or high levels of cholesterol who are not meeting targets and then beyond. I’ve already alluded to the economics and how that would benefit, but the business model is further enhanced by the scalability off the shelf nature of the LNP, which means we get to margins that can actually create meaningful value for us while enabling healthcare systems to realize the savings that should accrue from such a clinical benefit. And I think the final thing is again with that positive predictive value non human primate and our ability to move into the clinic quickly, we could get to human proof concept by 2026.

Eric Schmidt, Biotechnology Analyst, Cantor: So for those who want to go a little bit deeper into science, how does the upregulation occur and what’s sort of the genetic basis that supports this approach?

Gilmore O’Neill, President and CEO, Editas: So an Icelandic kindred was identified some years ago, seven members of that family across three generations had very low LDL cholesterols of about 17 to 30 milligrams per deciliter, which is equivalent to about 0.4, 0.5 per liter for those in the SI world. And this was associated with excellent health outcomes. It was determined or identified that they had a large two and a half kilobase deletion in the three prime untranslated region and we able to look at that target and say, okay, how can we optimize around that? So we actually, our discovery team basically did a massive optimization mapping experiment and essentially identified a deletion that they could create, which spans a number of regulatory domains, which ultimately results in the production of a message RNA that is more stable and allows the synthesis of large levels of LDL receptors. So we’re seeing at least 6x or six fold increase in LDLR levels at the doses we’ve treated or tested to date in nonhuman primates and indeed have seen higher levels of regulation in murine models as well.

Eric Schmidt, Biotechnology Analyst, Cantor: So the genetic association that pointed you in this direction was just out there in the public domain and It

Gilmore O’Neill, President and CEO, Editas: was in public domain, but again, was a nice validation of something we’ve leveraged before in that we use the hereditary persistence of pheochemoglobin to determine we should go after the h p g one two promoter. I think the other thing it validated was a strategy we have internally of working with existing databases and indeed identifying non publicly disclosed targets with this same natural variant.

Eric Schmidt, Biotechnology Analyst, Cantor: There are additional variants of the same nature that

Gilmore O’Neill, President and CEO, Editas: are not publicly disclosed beyond this cohort of seven? Sorry. I I thank you for correcting because I realized how that could be misconstrued. I wasn’t talking about hyperrestrial immune, I’m talking about the general more our general strategy for identifying natural variants that would point

Eric Schmidt, Biotechnology Analyst, Cantor: to targets that can functionally upregulate. Okay. So just remind us of the development path and milestones going forward So on four zero

Gilmore O’Neill, President and CEO, Editas: the key elements that will have to be executed and we’re focused on is to continue the process or not the process optimization, but the development process for GMP manufacturing, a formal dose range finding study, which by the way we will also leverage to further look at lower doses because we have yet to determine a minimally efficacious dose in the non human primate and obviously we are actually well advanced in developing our off target editing package which looks good to date and by the way sits in the hands of a very seasoned group that has built previous off target editing packages that for have met the needs of regulators. So those are the things we have to get through and that drives us towards a human INDCTA in the 2026. I would say by the way, we’re already looking at and exploring countries and sites where we can find the patients for our phase one.

Eric Schmidt, Biotechnology Analyst, Cantor: So is the next announceable event the filing of that submission CTLA or IND submission?

Gilmore O’Neill, President and CEO, Editas: Is possible but there may obviously we are sharing more non clinical data in between those now and then. We have a number of scientific meetings that we would present and obviously we’ll share the venues and dates at a time that’s allowed to us.

Eric Schmidt, Biotechnology Analyst, Cantor: Okay. So you spoke to a ninety percent reduction in non human primates of LDL C. Just put that into context relative to other therapies that are out there and why is lower better? Yes. So first,

Gilmore O’Neill, President and CEO, Editas: and this is highly illustrative. We’ve taken out the numbers for various reasons just to keep it simple, but the current highest standard of care would be the mean reductions of sixty percent seen with PCS inhibition either through the use of monoclonal antibodies or an antisense approach. We are substantially higher. So what does that mean in real terms? Well, a lot of genetic studies and importantly interventional long term outcome studies have demonstrated that a reduction of 40 milligrams per deciliter or one millimole per liter of LDL cholesterol is associated with a twenty percent reduction in cardiovascular risk at five years.

So when you actually look at the onset of action of time for us and the ninety percent reduction, that translates into meaningful meaningful differences. I just use illustratively, that’s not to say anything about the type of patient but just for illustrative reasons. If you think of a patient with a LDL of 180 per deciliter, a 90% reduction that translates into multiples of that forty milligram reduction and it appears those multiples do translate into risk reduction. So that matters from the efficacy point of view. From a safety point of view, I think this sort of leads to the next question, well is there a floor to how far you go?

Is LDL cholesterol, is it like a cigarette where there’s no amount that’s good for you or is it like blood pressure where you need a minimal, there’s a floor that you need that is compatible with a good health even if there is a ceiling that is way too high. And we would say that the genetic data and experience to date and subgroups with very low LDL cholesterols, the order of medians of 25 milligrams per deciliter in long term interventional studies, outcome studies like Fourier or Odyssey would all point to the idea that there is no floor yet identified for LDL cholesterol and it is important to say we’re not LDL, we’re not removing cholesterol. Cholesterol exists and circulates in many forms and in many particles of different densities. And remind us, when you

Eric Schmidt, Biotechnology Analyst, Cantor: look at your 90 reduction relative to baseline, that’s independent of where base baseline starts in terms of your higher fat or lower fat models?

Gilmore O’Neill, President and CEO, Editas: Yes, it is and thanks for your reminding of that. We have demonstrated that across multiple levels. So the healthy non human primates had relatively low levels of LDL C, 90% reduction in mouse models where we had high fat diet or they were heterozygous for LDL cholesterol and had high baseline LDLCs, they saw a similar reduction. So I assume your initial commercial strategy would be directed at patient groups that have higher baseline levels? I think our development and early commercial strategy, and I think it’s worth highlighting that from a commercial development point of view, there are sub populations within the hyperdipidemia blanket population that we could actually develop to ourselves.

Obviously as our ambition for public health impact increases, we could expand the size of this population that would include larger studies, longer outcome studies and would necessitate partnerships, but from a commercial development point of view starting with that, starting with those refractory HEFH patients, certainly a very viable path and frankly also even commercially would create meaningful value for us, but obviously maximizing value is what we want to do and that might necessitate partnering.

Eric Schmidt, Biotechnology Analyst, Cantor: And what does proof of concept look like? I know you’ve guided to that by the end of next year.

Gilmore O’Neill, President and CEO, Editas: Yeah. So proof of concept means that we will see substantial reductions in the LDL cholesterol. The wonderful thing and I should say, the wonderful thing about this space is that the LDL cholesterol biomarker has been very well validated over the years. It has a regulatory precedent as well, not just for use of advancing into larger studies, but frankly for regulatory approvals. I will say that one of the ways that we chose diseases to prioritize was not just mechanistic differentiation, efficacy differentiation, but actually also translatability to humans including the use of rapid readout, easily accessible biomarkers.

So the LDL C biomarker really gives us that opportunity. From what do you the size of the study, when we look at what I would say the precedent that’s been demonstrated by CRISPR editing, you see large outcomes in small numbers of patients with the effect size that we’re seeing here in the non human primate, with the early onset of action we’re seeing forty eight hours post infusion these levels, you can anticipate how you can work out and think about how that effect size can translate into smaller numbers of patients required to see the signal as well as a shorter follow-up to see that signal.

Eric Schmidt, Biotechnology Analyst, Cantor: Is there a cohort size wise that you’d like to have read out by the end of next

Gilmore O’Neill, President and CEO, Editas: year? I haven’t locked that down yet. We’re actually thinking very actively about that. But you know, there is a good set of precedent we’ve already seen within the PCSK9 development space across monoclones right through to CRISPR editing. And I think that points to a number of things and highlights a number of things.

First of all, the point I’ve made about effect size and the size of the cohorts. The second is about where dose expiration is likely to go. And the third is that there is a well validated path from a regulatory point of view in a number of jurisdictions. And frankly, also highlights that those particular patients, refractory patients are readily available and it actually also validates the time lines that we’re talking about.

Eric Schmidt, Biotechnology Analyst, Cantor: And you mentioned earlier you had yet found

Gilmore O’Neill, President and CEO, Editas: a lower limit of activity. There a lower dose cohort that you expect to begin at or have targeted? We haven’t identified that yet. What I will tell you that the precedent to date and the FDA guidance suggests that when you’re scaling an in vivo CRISPR formulation in an LNP from non human primate to humans, you basically reduce by about two thirds or divide by three. Indeed the presence we’ve seen to date would put you there.

I also want to emphasize that we haven’t identified a lower starting dose because we haven’t found the minimally efficacious dose yet, we will continue to explore downwards. Okay. So there’ll be additional preclinical data that we would be seeing,

Eric Schmidt, Biotechnology Analyst, Cantor: you would expect even before you start these studies?

Gilmore O’Neill, President and CEO, Editas: Well, will be generating more, yes. And what would be

Eric Schmidt, Biotechnology Analyst, Cantor: the form for those preclinical results to be shared?

Gilmore O’Neill, President and CEO, Editas: We haven’t determined yet. Some of the preclinical data that we already have is in upcoming scientific meetings and obviously as that data evolves, as we select more meetings, we will keep people informed, keep you all informed.

Eric Schmidt, Biotechnology Analyst, Cantor: Could it be as early as this year?

Gilmore O’Neill, President and CEO, Editas: We will give the we will give timelines and venues at you know, as know, soon before those meetings. Okay.

Eric Schmidt, Biotechnology Analyst, Cantor: Maybe let’s shift to commercialization or commercial considerations for EDIT four zero one. You’re moving from an area, sickle cell disease, where you would have expected to be charging millions of dollars per patient to one in which the standard of care is a few thousand dollars per patient. So how do you think about the commercial cost considerations considerations here? Here?

Gilmore O’Neill, President and CEO, Editas: Yeah, so we’ve been doing a lot of work to understand sort of the pricing paradigms that sit around here. I think there are a couple of things. First of all, we see a substantial change in the effect size. We think that creates value. We have a durable effect as well, which doesn’t require redosing or certainly lifetime use and so that also creates value.

That combination along with the fact that we have an off the shelf scaled manufacturing process that gets margins and cost of goods much, much lower than what people have seen to date with cell therapies, etcetera, basically puts in a place where we can actually create a competitive pricing and access paradigm or agreements with payers while maintaining value for ourselves.

Eric Schmidt, Biotechnology Analyst, Cantor: You’ve talked about pharmaceutical margins.

Gilmore O’Neill, President and CEO, Editas: Yes. What does that mean? Well, what that basically means is that we’re not talking about cost of goods of $600,000 patient, what it basically means is that you go into a world where much of your cost will reflect what you’ve done or what you’re realizing and returning for your development and IP as opposed to sinking it into manufacturing for individual patients. So correct

Eric Schmidt, Biotechnology Analyst, Cantor: me if I’m wrong, but I think the piece the anti PCSK nine drugs are on an annual basis somewhere in the $5,000 or $6,000 per year range today.

Gilmore O’Neill, President and CEO, Editas: Is that ballpark? I think their list is higher, and I think they’re probably effective to fact it was about that. Yes.

Eric Schmidt, Biotechnology Analyst, Cantor: So you’ve got a bit you think you’ve got a more potent effect that deserves a bit of a premium and then you think of course you’re gonna capture a multi year. I don’t want to put words in your mouth but

Gilmore O’Neill, President and CEO, Editas: Yeah. We can capture a multi year and I think the other thing I just want to say is that as payers look at this and by the way we have had some preliminary discussion with payers, they are excited by and acknowledge and recognize the potential transformational nature of these data should it be replicated in humans and they’ve already identified patient populations which they feel that this would be very appropriate to use upfront. I think the final thing I want to say is that as we think about payers, the payer spectrum is different around the world. But what we believe is with what I’ve outlined from a manufacturing cost, from a potent durability and from an effect size cost, we can deliver two different timeframes, the savings they want in the near term, some payers in The United States have to worry about cycling of patients, so they need to see a near term benefit, but obviously then we have to have long term and durable benefit. And with that and with our margins, we believe that we can actually realize those value propositions for those different payer needs and requirements while actually giving ourselves value.

Eric Schmidt, Biotechnology Analyst, Cantor: Okay. So bear with me while while I I do some math. Sure. You’ve got the cost of a PCSK9 that you get $5,000 or per year, and and you’ve got a better drug, so we’ll we’ll give you twice that value. We’ll give you $10,000 a year.

And as you say, payers wanna get a fairly rapid return on on their investments. So four years time, we we would expect a a payer to wanna recoup the the the cost of therapy. If you’re charging $40,000, four years times $10,000 benefit per year. Can you have pharmaceutical margins?

Gilmore O’Neill, President and CEO, Editas: Without committing to a price, I would actually say that you you actually have got yourselves in. Your calculation would put us in a Farmers margin.

Eric Schmidt, Biotechnology Analyst, Cantor: So and what else needs to happen to scale to that level? Are you needing to put in place any manufacturing considerations or any facilities or or brick and mortar or anything like that?

Gilmore O’Neill, President and CEO, Editas: Actually, a great question. These are things we would always consider. I think the good thing about the state of the LNP manufacturing space is that owing to unfortunate events five years ago and the pandemic, the ability, the bricks and mortar etcetera and sort of I’d say generic process for manufacturing LNP at very reasonable costs has actually been done. So we’re basically able to leverage off that substantial experience and dare commoditized supply chain.

Eric Schmidt, Biotechnology Analyst, Cantor: Same with the RNA component as well? Largely so. Yes. Yeah. Yeah.

Okay. So we know that you back burner to some extent well, shouldn’t I don’t want put words let let us know what happened to your in vivo sickle cell program. How would you communicate the status of that?

Gilmore O’Neill, President and CEO, Editas: Yeah. So the status of that is that we had achieved reached a point where it was achieving more than the minimum product profile that we had set, a threshold of efficacy that was meaningful. Any of you who have worked in the clinical development space or have sat with discovery researchers always know that they do not want to hand that child over just yet because there’s one more thing they can do to make it better. And so what we are actually doing and we have that critical mass in our discovery is to enable and allow and exploit this opportunity of dare I say the pause or rather the non advancement to high cost and spend to development to further optimize the asset. So it’s still getting resources?

So it is getting resources too for optimization, yes. And what would you be trying to further optimize? Well, you could always do better as they say, and are there so we can actually work across the gamut of there’s some elements of the payload that could be tweaked and some elements of the targeting and delivery that can be tweaked. I mean

Eric Schmidt, Biotechnology Analyst, Cantor: the challenge here I assume would be delivery to stem cells as a

Gilmore O’Neill, President and CEO, Editas: Yeah. I would say from a profile point of view, we’ve been very happy to where we’ve gotten to date. And, you know, frankly, if we had the luxury and the assets to both, we would advance, you know

Eric Schmidt, Biotechnology Analyst, Cantor: So is this now a partnership candidate or are you active in discussions or do you want to do the further optimization prior to investigating those?

Gilmore O’Neill, President and CEO, Editas: I I think we’re agnostic, you know, frankly. As I say, the perfect can be the enemy of the good, but when an opportunity arises to optimize something, you take it. And so I think we’re in a very good place from the point of view of any of those options.

Eric Schmidt, Biotechnology Analyst, Cantor: Okay. And what’s the outlook for a potential partnership? Are folks interested in in this opportunity more broadly speaking? Well, with the going without going into specifics, what I

Gilmore O’Neill, President and CEO, Editas: would say is is that this is a the space for targeting LNPs is actually has been getting more and more exciting.

Eric Schmidt, Biotechnology Analyst, Cantor: Is that interest driven by business development transactions we’ve seen or anything in particular? I think

Gilmore O’Neill, President and CEO, Editas: I think one can look at what’s happened in the business development space to sort of really get a sense of that excitement.

Eric Schmidt, Biotechnology Analyst, Cantor: Okay. And then just remind us again the cash and cash burn that we’re looking at between here and the end of next year. I know this is of excruciating importance to investors.

Amy Patterson, SVP and CFO, Editas: So to reiterate, we have cash into 2027. As Gilmore mentioned, you know, we’re excited to be able to fund at 04/2001 through to human POC and beyond with our cash runway. Our cash runway, you know, we ended q two with a 179,000,000 of cash, which is a good, you know, measure of how the math kinda works to get us to q two twenty seven.

Eric Schmidt, Biotechnology Analyst, Cantor: So And in addition to April progress, you’re funding modest investment preclinically or or even Yeah. Research wise into sickle cell. Anything else getting funded?

Amy Patterson, SVP and CFO, Editas: So we’re excited about our pipeline. We’ve announced that we’ll have an additional target by the end of this year. So, you know, we do have some funding allocated to some discovery work both as we optimize the HSE program and, for this additional target to

Eric Schmidt, Biotechnology Analyst, Cantor: ship. And that’s a liver target, I assume.

Amy Patterson, SVP and CFO, Editas: We haven’t Yeah.

Gilmore O’Neill, President and CEO, Editas: We haven’t said what we did actually guide to at the beginning of this year is that we would talk have a third tissue. So we have liver, we have HSC, and we would announce the third target tissue.

Eric Schmidt, Biotechnology Analyst, Cantor: So the third target will not be liver or HSC. But it’s a target tissue. It’s a tissue. It’s it’s not it it is a tissue. Great.

Well, we are out of time. Thank you very much, team. Appreciate the updates. Thanks very much, Irish.

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