Editas Medicine at BofA Securities 2025: In Vivo Gene Editing Focus

On Wednesday, 14 May 2025, Editas Medicine (NASDAQ:EDIT) presented at the BofA Securities 2025 Healthcare Conference, emphasizing its strategic pivot towards in vivo gene editing. The company aims to expand market potential and reduce patient burdens, while also navigating financial challenges and capital efficiency.

Key Takeaways

• Editas is focusing on in vivo gene editing for thalassemia and sickle cell disease.
• The company has a cash runway extending into Q2 2027, prioritizing capital efficiency.
• Editas plans to declare two drug candidates by mid-2025 and file an IND in 2026.
• Licensing agreements remain unaffected by recent legal decisions.
• Upcoming data presentations will showcase advancements in their liver and HSC programs.

Financial Results

• Cash Position: Editas has a cash runway extending into Q2 2027, focusing on capital efficiency.
• Capital Strategy: The company is exploring partnerships and out-licensing to secure non-dilutive financing.
• Operational Costs: While specific operational expenses remain undisclosed, Editas aims to manage costs effectively, especially concerning the wind-down of the Exvio program.

Operational Updates

• Pipeline Focus: Editas is concentrating on in vivo gene editing, leveraging its TLNP platform for hematopoietic stem cell (HSC) and liver programs.
• Strategic Goals: The company plans to declare two drug candidates by mid-2025, with an IND filing for one candidate in 2026.
• Preclinical Success: Recent data from the liver program show maximal gene editing and significant biomarker reduction.

Future Outlook

• Drug Development: Editas aims to demonstrate human proof of mechanism by late 2026 or early 2027.
• Value Generation: The company is committed to creating value for patients and shareholders through innovative science.

Q&A Highlights

• In Vivo vs Ex Vivo: In vivo gene editing is seen as expanding market potential while reducing infrastructure burdens.
• Bone Marrow Access: Insights from the ex vivo program are being applied to improve in vivo strategies.
• Licensing and Partnerships: Editas remains open to partnerships and licensing opportunities to enhance financial flexibility.

Readers are encouraged to refer to the full transcript for a detailed account of the conference call.

Full transcript - BofA Securities 2025 Healthcare Conference:

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Welcome to the the last fireside for day one of the twenty twenty five Bank of America Healthcare Conference. Thanks for joining the session with Editas. My name is Alex Stranahan. I’m senior biotech analyst at BofA covering Editas, and I’m pleased to be joined by, Amy Parson, chief financial officer at Editas as well as Christy Barnett, senior vice president of IR. Thanks thanks for being here.

Amy Parson, Chief Financial Officer, Editas: Thank you.

Christy Barnett, Senior Vice President of IR, Editas: Thank you. Having us.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Yeah. Well, maybe we can, you know, start with the the the focus or the refined focus to to in vivo development, you know, sort sort of walk us through the thought process there and how you see the the field evolving over the next few years.

Amy Parson, Chief Financial Officer, Editas: 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 editing gene editing company. So, you know, we were able to, generate some very exciting data, that we’ve shared recently this year and then even today. Mhmm.

And so we’re sort of moving forward on that on that promise, and we’re excited about what we’ve been doing there.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Well, maybe we can, you know, double click on that and talk about, you know, sort of the landscape in thalassemia and sickle cell. What are sort of the the weaknesses or, you know, I guess, things that might be desired in the the currently approved options? And how is in vivo really kind of the the right solution for these patients?

Amy Parson, Chief Financial Officer, Editas: Great question. So, 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 lessen the burden on patients and even the infrastructure. So, you know, we’re excited about being able to, you know, enter in vivo, gene therapy for sickle cell disease.

We think that’s, you know, 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.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Do you see, you know, the bar for for activity here maybe being slightly lower for in vivo just given it’s so much more convenient? Or, you know, what sort of the, you know, the the the yard marker folks should use to to compare, you know, these different modalities?

Amy Parson, Chief Financial Officer, Editas: 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. So I would actually say it’s, you know, it’s on par if not better.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Yeah. And, I mean, you guys have sort of had to place the trail when it comes to direct editing of, you know, stem stem progenitor cells within the bone marrow. It’s not an easy tissue, to to get access to. Sort of what’s the optimization that’s been going on and maybe walk folks through through the preclinical data we’ve seen there?

Amy Parson, Chief Financial Officer, Editas: Sure. I will do my best. I am the chief financial officer and not the chief scientific officer. But we are you know, we’re we’re making strides there. I mean, we’ve got we’ve have a lot of learnings that we’ve had from runicel in the ex vivo program, and we’re applying those, in our in vivo program.

You know, we’ve clinically validated the target, the h b t one two promoter, and so that’s the same approach we’re taking. And now we’re really focused on the delivery, and our proprietary TLNP, are showing, you know, in the preclinical setting at least great potential.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Okay. And maybe thinking about sort of the the BD priorities, you know, for this year’s updates, assuming the focus will be sort of on the, you know, building upon that that editing efficiency. And I guess any plans to maybe also disclose, you know, biodistribution or safety in in animal models?

Amy Parson, Chief Financial Officer, Editas: Yeah. I mean, we’re certainly willing wouldn’t be disclosing that data. Probably, you know, there’s maybe a press release tomorrow. So we will share that data sort of as it continues to emerge.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Okay. And and, you know, given the the reprioritization of the pipeline, do you think that’s released released some, you know, dry powder for additional investments? Or, I guess, how do you see expansion of the the pipeline over the next couple of years?

Amy Parson, Chief Financial Officer, Editas: 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, and we wanna continue to leverage those. We’re very excited about our proprietary TLNP platform that we’re using on the HSC side. And then on the liver program, you know, we’re seeing, great results there as well, and and that’s another tool in our toolbox to be able to deploy as we look to expand and build on our pipeline.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Okay. And I guess, you know, as you’re thinking about growing the in vivo pipeline, you have, obviously, the editing technology. You have expertise developing ReniCel. Would you ever expand the scope, I guess, of applications internally outside of, you know, sort of liquid tumors?

Or, obviously, you’ve got the liver program as well, but maybe just talk about sort of the disease areas or what what kind of scope you you see the technology be.

Amy Parson, Chief Financial Officer, Editas: Right. So our strategy is, to uprate upregulate, you know, functional proteins. And so we’re very thoughtful in the targets that we’re thinking about and the indications that we’re thinking about. Both of the programs that we’re progressing right now are aligned with that strategy. So from a scientific perspective, you know, that’s sort of where we’re aiming.

The other thing we wanna do is target to be the best in class, first in class in vivo gene therapy. So we’ll let that those two principles kind of guide how we’re thinking about future programs and pipeline.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Do you think there’s room to, you know, partner or or out license given you do have sort of a patent estate around sort of a key, editing approach? Is that, you know, maybe a way to to bring in some additional nondilutive financing?

Amy Parson, Chief Financial Officer, Editas: Yeah. I mean, the markets are tough right now. Right? So, you know, bringing in capital in in any way that we can is definitely important. I think we’re sort of open to all avenues right now.

So yeah.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Okay. And I guess, you know, in terms of additional BD, you know, maybe just walk us through sort of the other particular indications that you wanna target, would you wanna be the first mover, Or does maybe the the scale of what a clinical study or the speed that you could get to to approval how does that sort of factor into your thought process?

Amy Parson, Chief Financial Officer, Editas: Yeah. It’s a good question. I think, you know, right now, like I said earlier, we’re we’re really focused on generating exciting, you know, science and and showing the potential of gene editing and gene therapy. And so, you know, that’s really driving how we’re thinking about programs. And, you know, we wanna make sure that we’re generating we’re creating programs that generate value for patients, our shareholders.

And so that’s kind of how we’re thinking about, you know, the pipeline coming together. And if that is attractive, you know, that’s great. But, you know, it should be. Right? Great science is always attractive.

So

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Right. Right. Right. That makes sense. And I guess, you know, maybe we can talk about the cash position, you know, runway into 02/1927, which is a decent it gives you, you know, some decent breathing room to get in vivo a little a little bit closer to clinic and probably into the clinic.

You know, how do you think about staying capital efficient, you know, just given the current, you know, perpetually difficult financing backdrop?

Amy Parson, Chief Financial Officer, Editas: Yeah. So, you know, obviously, the the best cash that you can get is the cash you have. Right? And so we’re being really thoughtful about how we deploy, you know, our capital and making sure that it’s congruent with the strategy that I just outlined. Mhmm.

You know, you’re right. Q two twenty twenty twenty seven is a great cash runway, but it catches up faster than you think.

Christy Barnett, Senior Vice President of IR, Editas: So Mhmm.

Amy Parson, Chief Financial Officer, Editas: We’re being really thoughtful about that. And, you know, I’m I’m in a unique position where I’m fairly new in the role as CFO, but I’ve been with Editas for almost three years. So I’m, you know, leveraging my experiences of being in the company to help, you know, everyone sort of think through how to do things in a more effective way. So

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Yeah. I imagine that kind of perspective is is kind of invaluable through the restructuring. You can kind of identify those areas where you can maybe trim that while also retaining the ROI. Mhmm. Are are there any sort of, you know, examples that that you see that, or is is most of that kinda in the the rear view?

Amy Parson, Chief Financial Officer, Editas: No. I don’t think that’s in the rear view at all. And I also think leveraging relationships with with people at Editas and understanding, you know, what what we need to execute on to sort of hit all of the externally stated and even internal goals is important, and I kind of understand that having been on the ground with folks for a while. Mhmm. So I think every day, there’s something that kind of pops up that, you know, we can think through and be helpful through.

So

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Yeah. Yeah. That that makes sense. And and maybe just talk about sort of the OpEx expectations, whatever you said, you know, publicly. And, you know, if if there’s additional room to go, I’m kinda tripping that.

Amy Parson, Chief Financial Officer, Editas: Yeah. I mean, so publicly, you know, we’re still in the q two twenty twenty seven. I think we’re we’re cognizant of that, and we’re we’re working to make sure that that we’re able to achieve that. We are also being pretty capital capital efficient around some of the wind down costs that we had for our former Exvio program. And so that’s also sort of helping to provide that cash runway through.

So I think we’re well positioned.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Okay. And maybe circling back to, you know, sort of the the license agreement side of of the business, and I think there’s probably room to grow that. But does the, I guess, c a f CAFC decision, you know, sort of impact the any of your current licensing agreements or the way that you’re thinking about that? Sure.

Christy Barnett, Senior Vice President of IR, Editas: I’ll take that one. No. The short answer is no. Those license agreements the 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. We all of our current agreements are still in effect. And I think the other important part is that the interference only covers a small subset of the IP, not all of the Cas nine IP that you would need to actually make a CRISPR Cas nine medicine. So we remain confident that and we’re, you know, still open for business if people want to to license it.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Yeah. Okay. Okay. That that’s, I guess, reassuring. You know, maybe thinking about upcoming data, I think you have a couple presentations at ASGCT in a few weeks.

May maybe walk us through sort of the scope of of what those updates, couldn’t tell to start.

Amy Parson, Chief Financial Officer, Editas: Yeah. So, actually, at ASGCT this week. So today, we unveiled some posters around our liver program, and, you know, we’re excited to to show that we’ve achieved, maximal editing on the gene target level and, have also we’re able to reduce a disease biomarker by greater than 80%. So that’s that 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. And then on the, in vivo HSC side, you know, we’re we’re continuing to show follow-up data there and and editing level data there. And there’s actually, I think, an oral presentation tomorrow where we’ll share more on that program. So like I said, we’re progressing, and we’re showing some strong science and data.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): I guess, on on the liver program, is is that kind of what what is that does that kinda hit your internal metric for for the editing efficiency? I mean, this is it’s not the easiest, I guess, tissue to to target with an LNP, but sort sort of walk us through your your own internal metrics and, you know, if you’re seeing preclinically, it gives you confidence to to maybe move things forward.

Amy Parson, Chief Financial Officer, Editas: Yeah. I’ll reference our chief scientific officer again who is who feels fairly confident and very confident in moving things forward there. And I think we’re seeing the editing levels that we were hoping to see. And so we’re we’re continuing to progress there.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Okay. And and maybe, you know, in the next minute or so that we have, if you could sort of maybe wrap up the the the catalyst flow for for folks to look out for you. We’ve we’ve got the data this week at ASGCT, but any sort of other updates that you direct, investor attention to?

Amy Parson, Chief Financial Officer, Editas: 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 twenty twenty five. And 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 twenty six and the beginning of twenty twenty seven. So, you know, like I said earlier, two years, a year isn’t far out, and we’re we’re generating inflection points there to keep people, excited.

So

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Okay. Do you think there’s a point that you’d ideally love to reach under your current runway assumptions that that, like, first patient dose in in a clinical program, maybe one or two for HSC or or liver or both? Or what’s sort of the the end goal that you think you could stretch to?

Amy Parson, Chief Financial Officer, Editas: Sorry. With the current

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): With the current runway.

Amy Parson, Chief Financial Officer, Editas: Right. I mean, so we’re we’re obviously thinking through that, and we wanna be able to make it to show, you know, proof of concept and then go beyond. And so I mean, to answer your question, we’re we’re we’re there. We understand that we need to continue to to generate value and and sort of finance these programs through those.

Christy Barnett, Senior Vice President of IR, Editas: So Okay. Okay.

Alex Stranahan, Senior Biotech Analyst, Bank of America (BofA): Well, definitely looking forward to to the updates this week. That’s something we’ll be paying attention to for sure and, you know, the updates for the rest of the year. So I think with that, we’ll we’ll have to end it there. But Amy, Christy, really appreciate the the conversation, and please join me in in thanking them for for being here.

Amy Parson, Chief Financial Officer, Editas: Thank you.

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