Welcome to the last fireside for Day 1 of the 2025 Bank of America Healthcare Conference. Thanks for joining this session with Editas . My name is Alex Stranahan. I'm Senior Biotech Analyst at BofA , covering Editas . I'm pleased to be joined by Amy Parson, Chief Financial Officer at Editas Medicine, as well as Christy Barnett, Senior Vice President of IR . Thanks for being here.
Thank you for having us.
Yeah. Maybe we can start with the focus or the refined focus to in vivo development. Sort of walk us through the thought process there and how you see the field evolving over the next few years?
Sure. I think refined focus is actually the right word. Since Gilmore joined Editas, Gilmore, our CEO, joined Editas about three years ago, we've been sort of targeting to be the premier in vivo gene editing company. We were able to generate some very exciting data that we've shared recently this year and then even today. We are sort of moving forward on that promise. We are excited about what we've been doing there.
OK. Maybe we can double-click on that and talk about sort of the landscape in thalassemia and sickle cell. What are sort of the weaknesses or, I guess, things that might be desired in the currently approved options? How is in vivo really kind of the right solution for these patients?
Great question. I mean, obviously, there's a ton of potential. Even with the ex vivo therapies, we're seeing patients benefit from gene therapy and putting it to use in that setting. The great thing about in vivo gene editing is it really expands the market, expands the potential, while also helping to lessen the burden on patients and even the infrastructure. We're excited about being able to enter in vivo gene therapy for sickle cell disease. We think that's an exciting place to be. It's the right place. It's the right treatment for the disease. It's the right way to use the technology.
Do you see the bar for activity here maybe being slightly lower for in vivo, just given it's so much more convenient? Or what's sort of the yard marker folks should use to compare these different modalities?
Yeah. I mean, I wouldn't say the bar is lower at all. I think about the ability to reach a much larger patient population and still be able to have the impact that we've seen on the ex vivo side. I would actually say it's on par, if not better.
Yeah. I mean, you guys have sort of had to blaze the trail when it comes to direct editing of stem progenitor cells within the bone marrow. It's not an easy tissue to get access to. Sort of what's the optimization that's been going on and maybe walk folks through the preclinical data we've seen there?
Sure. I will do my best. I am the Chief Financial Officer and not the Chief Scientific Officer. We're making strides there. I mean, we have a lot of learnings that we've had from Reni-cel in the ex vivo program. We're applying those in our in vivo program. We've clinically validated the target, the HBG1/2 promoter. That's the same approach we're taking. Now we're really focused on the delivery. Our proprietary tLNPs are showing, in the preclinical setting at least, great potential.
OK. OK. Maybe thinking about sort of the BD priorities for this year's updates, assuming the focus will be sort of on building upon that editing efficiency and, I guess, any plans to maybe also disclose biodistribution or safety in animal models?
Yeah. I mean, we're certainly willing, one, to be disclosing that data. Probably there's maybe a press release tomorrow. So we will share that data sort of as it continues to emerge.
OK. OK. Given the reprioritization of the pipeline, do you think that's released some dry powder for additional investments? I guess, how do you see expansion of the pipeline over the next couple of years?
Yeah. I mean, I think we're using the great tools that we have. We have two fantastic programs right now that, again, we're showing some great data with. We want to continue to leverage those. We're very excited about our proprietary tLNP platform that we're using on the HSC side. On the liver program, we're seeing great results there as well. That's another tool in our toolbox to be able to deploy as we look to expand and build on our pipeline.
OK. OK. I guess, as you're thinking about growing the in vivo pipeline, you have, obviously, the editing technology. You have expertise developing Reni-cel. Would you ever expand the scope, I guess, of applications internally outside of sort of liquid tumors? Obviously, you've got the liver program as well. Maybe just talk about sort of the disease areas or what kind of scope you see the technology being able to?
Right. Our strategy is to upregulate functional proteins. We are very thoughtful in the targets that we are thinking about and the indications that we are thinking about. Both of the programs that we are progressing right now are aligned with that strategy. From a scientific perspective, that is sort of where we are aiming. The other thing we want to do is target to be the best in class, first in class in vivo gene therapy. We will let those two principles guide how we are thinking about future programs and pipeline.
OK. Do you think there's room to partner or out-license, given you do have sort of a patent estate around sort of a key editing approach? Is that maybe a way to bring in some additional non-dilutive financing?
Yeah. I mean, the markets are tough right now, right? Bringing in capital in any way that we can is definitely important. I think we're sort of open to all avenues right now. Yeah.
OK. OK. I guess, in terms of additional BD, maybe just walk us through sort of the other particular indications that you'd want to target. Would you want to be the first mover? Does maybe the scale of what a clinical study or the speed that you could get to approval? How does that sort of factor into your thought process?
Yeah. It's a good question. I think right now, like I said earlier, we're really focused on generating exciting science and showing the potential of gene editing and gene therapy. That is really driving how we're thinking about programs. We want to make sure that we're generating, we're creating programs that generate value for patients, for our shareholders. That is kind of how we're thinking about the pipeline coming together. If that is attractive, that's great. It should be, right? Great science is always attractive.
Right. Right, right. That makes sense. I guess maybe we can talk about the cash position, runway into 2Q 2027, which is a decent, it gives you some decent breathing room to get in vivo a little bit closer to the clinic and probably into the clinic. How do you think about staying capital efficient, just given the current perpetually difficult financing backdrop?
Yeah. Obviously, the best cash that you can get is the cash you have, right? We are being really thoughtful about how we deploy our capital and making sure that it is congruent with the strategy that I just outlined. You are right, Q2 2027 is a great cash runway, but it catches up faster than you think. We are being really thoughtful about that. I am in a unique position where I am fairly new in the role as CFO, but I have been with Editas for almost three years. I am leveraging my experiences of being in the company to help everyone sort of think through how to do things in a more effective way.
Yeah. I imagine that kind of perspective is kind of invaluable through the restructuring. You can kind of identify those areas where you can maybe trim the fat while also retaining the ROI. Are there any sort of examples that you see that? Or is most of that kind of in the rearview?
No. I don't think that's in the rearview at all. I also think leveraging relationships with people at Editas and understanding what we need to execute on to sort of hit all of the externally stated and even internal goals is important. I kind of understand that having been on the ground with folks for a while. I think every day there's something that kind of pops up that we can think through and be helpful through.
Yeah. Yeah. That makes sense. Maybe just talk about sort of the OPEX expectations, whatever you said publicly. If there's additional room to go, I'm kind of trimming that.
Yeah. I mean, so publicly, we're still in the Q2 2027. I think we're cognizant of that. I think we're working to make sure that we're able to achieve that. We are also being pretty capital efficient around some of the wind-down costs that we had for our former ex vivo program. That's also sort of helping to provide that cash runway through. I think we're well positioned.
OK. OK. Maybe circling back to sort of the license agreement side of the business. I think that there's probably room to grow that. Does the, I guess, CAFC decision sort of impact any of your current licensing agreements or the way that you're thinking about that?
Sure. I'll take that one. No. The short answer is no. Those license agreements, the current decision, which you're referencing, is that the CAFC vacated the decision back to the PTAB to relook and make sure that they correctly applied the law. We still feel very confident. We've already prevailed, or Broad has prevailed there three times, three times in this situation already. While it's going back to the PTAB, the IP is still in full force. We can still license it. All of our current agreements are still in effect. I think the other important part is that the interference only covers a small subset of the IP, not all of the Cas9 IP that you would need to actually make a CRISPR- Cas9 medicine. We remain confident that, and we're still open for business if people want to license it.
Yeah. OK. OK. That's, I guess, reassuring. Maybe thinking about upcoming data, I think you have a couple of presentations at ASGCT in a few weeks. Maybe walk us through sort of the scope of what those updates could entail to start.
Yeah. So actually at ASGCT this week, today we unveiled some posters around our liver program. We're excited to show that we've achieved maximal editing on the gene target level and have also were able to reduce a disease biomarker by greater than 80%. That was the press release today. We're excited to share that. Obviously, we haven't disclosed the indication yet. We will at a time that sort of makes sense. On the in vivo HSC side, we're continuing to show follow-up data there and editing level data there. There's actually, I think, an oral presentation tomorrow where we'll share more on that program. Like I said, we're progressing. We're showing some strong science and data.
Yeah. I guess on the liver program, is that kind of, does that kind of hit your internal metric for the editing efficiency? I mean, it's not the easiest, I guess, tissue to target with an LNP. But sort of walk us through your own internal metrics and if you're seeing preclinically it gives you confidence to maybe move things forward.
Yeah. I'll reference our Chief Scientific Officer again, who feels fairly confident, very confident in moving things forward there. I think we're seeing the editing levels that we were hoping to see. We're continuing to progress there.
OK. OK. And maybe in the next minute or so that we have, if you could sort of maybe wrap up the catalyst flow for folks to look out for. We've got the data this week at ASGCT. But any sort of other updates that you'd direct investor attention to?
Sure. So we've announced that we'll be declaring two drug candidates for both of those programs at some point. We're in mid-2025. Then subsequent to that, we hope to file one IND for one of those candidates next year in 2026 and showing potential human proof of mechanism at the end of 2026 and the beginning of 2027. Like I said earlier, two years, a year isn't far out. We're generating inflection points there to keep people excited, so.
OK. Do you think there's a point that you'd ideally love to reach under your current runway assumptions? Is that like first patient dose in a clinical program, maybe one or two for HSC or liver or both? Or what's sort of the end goal that you think you could stretch to?
Sorry. With the current.
With the current runway.
Right. I mean, so we're obviously thinking through that. We want to be able to make it to show proof of concept and then go beyond. I mean, to answer your question, we're there. We understand that we need to continue to generate value and sort of finance those programs through those, so.
OK. OK. Definitely looking forward to the updates this week. That's something we'll be paying attention to for sure. And the updates for the rest of the year. I think with that, we'll have to end it there. Amy, Christy, really appreciate the conversation. Please join me in thanking them for being here.
Thank you.