In this episode of Biotech Breakthroughs, Matt Phipps, Ph.D., group head of biotech research, speaks with William Blair biotech analyst Myles Minter, Ph.D., about how gene editing is moving from early hype toward clinically validated therapies. They discuss lessons from the first approved ex‑vivo product and highlight key 2026 catalysts across CRISPR, base editing, RNA editing, and DNA writing.
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Transcript
00:06, Matt Phipps
Today is April 14, 2026. Welcome back to another episode of Biotech Breakthroughs. My name is Matt Phipps, group head of the biotech equity research team. And I'm joined today by my colleague, Myles Minter. Myles, thanks for joining me today.
00:20, Myles Minter
Thanks for having me, Matt. Appreciate it.
00:21, Matt P
And, today, we're going to talk about a piece that you published recently, your DECODER piece, with this one really focusing on updates in the gene editing landscape. A lot’s been going on in that field, you know. The first product was approved not too long ago, and, a lot is coming up.
Maybe, you know, I always like to think new technologies, kind of, having to go through that Gartner Hype Cycle curve, right, where there's initially some over exuberance and then there's some, you know, troughs and then, kind of, peeking out of it. I guess, you know, just, sort of, where do you think gene editing is?
And I realize it's a broad space, a lot of nuance to what that encompasses. But where do you think that technology is on that Gartner Hype Cycle today?
01:08, Myles M
Yeah, sure, so, thanks very much. It's been a while since we've done a report like this. I think 2022 was the last year that we actually put out a large deep dive like this. And coming back to that Gartner Hype Cycle, I mean, you were pretty much at the peak, right there, if not coming out of it into that, sort of, trough of disillusionment.
Yeah. I think the space had, kind of, very quickly come out of academic labs. I mean, it was every day that we were hearing about a new technology coming out of these, you know, great labs at Broad, MIT, Harvard, that sort of area. And then on the West Coast, as well. And it was in a funding environment, quite frankly, that was frothy, right?
And we were funding a lot of these companies based on their platform valuations. And I think what gives you a return on investment at the end of the day, which we'll get into a little later, is an actual product leveraging great technology that's actually making a difference for patients.
And so, you have to designate a candidate. You have to go through the clinical development of that. You have to prove out safety and efficacy and market that product. And so, I think that that's where we had a little bit of fall from grace in the sector, that it was this blue-sky promise of this could, you know, be the cure-all, just edit the genome and you’ll be fine into okay, now we have to pick those indications where there's suitable risk benefit. We've got to fund these companies to actually run those clinical trials. And so that was where that disillusionment came into. And, you know, I tracked the funding that's in the report, seems to be around about that end to 22, 23.
And, you know, more recently we've seen an uptick in the funding here. And we are starting to come, to your point, to potentially, you know, the next approved gene editing therapy, which will be in vivo, right? So, we're starting to see companies get to the end stage here. And so, I really do think that, you know, we're coming out of it, we're not yet at the plateau, because we're still emerging with the technologies that are coming through, right?
But we are starting to see actual assets being developed by those companies and making a difference in patients. So, I think that that's the key thing that's changed here versus platform blue sky valuation for a lot of these technologies.
03:35, Matt P
Yeah. No, I definitely remember the over-exuberance times. And, I mean, it did make sense that, okay we know some genes. You just got to get in there fix them. But, it turns out that's a little bit harder than it might seem or was it was able to do in a, you know, in a lab.
You know, I think one of the biggest steps for this field is obviously the first approved product.
Now, this is an ex vivo product, so maybe some, you know, an asterix there not to just discount it at all, but, how's been the commercial performance of that product? Maybe give us a little bit of details on that launch.
04:06, Myles M
Sure, sure. So, we're talking about a product here that's designed as a security therapy for sickle cell disease. So, quite a rare disease, but absolutely devastating. It's very, very painful, vaso-occlusive crisis and obviously a high degree of mortality.
This is a therapy where basically we take cells from a patient. We send them away to a lab. We use varying gene editing technologies, in this case CRISPR-Cas9, the very start of these technologies, to make a specific edit and get these products to basically over-express a fetal hemoglobin, infuse that back into the patient, into their stem cell, hematopoietic stem cell compartment. And basically, they would have a functional cure for it, for their disease, which is absolutely game changing.
And I just said that in about a minute. The reality is that there's a lot of work behind that. And there's also a lot of work in the patient journey. And I think that that's what's been reflected in the early, sort of, commercial performance for this product.
You know, this is highly burdensome on patients. There is a process and it could require two to three times, of this particular process, but it's collecting those cells. So, you have to do a mobilization or an apheresis and these are, you know, sick patients having these vaso-occlusive crisis. They're hospitalized during this process. But we need that, and we might need it three times here to actually generate enough cells that we can send away to a lab, edit those and then keep enough alive that we can transplant that back into the patient.
On the transplant part of it, as well. Obviously, you know, these cells, they have edits in them that they weren't in the body previously. We do have to do a process called lymphoid depletion for that transplant to be accepted on the back end. You know, that is a heavy preconditioning regimen with a lot of side effects, as well, for these patients.
And it is a hospital stay. It's a prolonged hospital stay throughout this process. So, you know, I think we're starting to see that reflected in the commercial performance here. There is high degrees of interest for this product, but to try and find the time and traveling to these centers that that deliver this therapy and can make this therapy is difficult for patients.
Now, having said all of this, we did actually see in the last quarter, from a revenue perspective, actually beat consensus expectations. You know, there were 111 new patient initiations. So, they have started that cell mobilization process to begin the path down this therapy, but only 30 patients were actually infused, and that's where the company actually gets paid for this product.
So, you know, I think coming to what can improve the commercial performance of this first-generation product, it's a multitude of things. One is get more patients into the funnel. I think they're actually doing that. That's why I mentioned that there's 111 new patients in the fourth quarter. Secondly, would be actually try and make this manufacturing and so collection process just more efficient, right?
If we could increase the manufacturing viability here, we would have less cells we'd need to collect. That means less mobilizations for those patients going on to therapy. That's a quicker time to actually receive the product. And, then, thirdly, I mean, it's the preconditioning regimen, highly burdensome for patients, as I explained earlier, but required to be lymphocyte depleted and accept that transplant.
So, there is a lot of work going on. And this is a little bit outside of the gene editing landscape, but there's a lot of work going on trying to improve those preconditioning regimens, to basically make this less burdensome on patients, so they're more likely to come in and demand for this therapy.
08:15, Matt P
Great. Well, it sounds like it'll be an important year to, kind of, monitor that launch. I'm sure that we’ll have some kind of read through sentiment in the space. But, I guess, what other, maybe, other key catalysts in 2026? Again, across the broader gene editing space, I guess maybe ex vivo and in vivo.
08:33, Myles M
Sure. Well, we've actually gone through two really important catalysts for the space already, but we'll get extended data from them later on in the year, as well. I want to talk a little bit about some updated data that we’ve got in alpha 1-antitrypsin deficiency. So, this is a rare disease where patients largely present with basically a deficit that they can't stop trips and activity. It eats up their lungs. They present as a COPD patient or a chronic smoker. And a smaller proportion of patients actually have a liver manifestation.
We’re starting to get to the point where these, you know, monogenic diseases, they are prime candidates for a corrective gene editing therapy. And we did actually see some follow-up data from some higher dose cohorts and follow it out for longer from a base editing application.
And we're going to talk a little bit more about all the different modalities here. But, you know, that, in my opinion, was very exciting. I mean, you're seeing correction of this protein that's mutated in vivo. So, we don't have to take cells out. We don't have to send them away to a lab and reinfuse them back into a patient. This just goes directly into the patient here.
And, for the first time in their life, they're producing functional protein that is protecting them against inflammatory insults. So, I thought that that data was, you know, very significant. I'm expecting to see additional data, more patient’s worth of that, towards the end of the year when that particular company presents at a conference.
You know, that's a base editing application, so that's a more permanent solution. There's an emerging therapy, therapeutic modality called RNA editing that I've spent a lot of time with. This is basically where we can transiently edit your genomes through RNA instead of DNA. And the advantage of this application is several fold, but one is, there's a small, oligonucleotides that that recruit machinery within you to do the editing on your behalf. So, you don't have to deliver this in a really big application. We can tune it better to where it needs to go for the tissues. There's a couple of sponsors doing trials like this. We’ll see more data from that RNA editing approach also in that alpha 1-antitrypsin deficiency disease. So, you know, you'll have one permanent solution and then one more transient solution that you might be able to tune up and down, depending on the needs of that patient.
You know, we saw some allogeneic cell therapy data, as well, and I do want to be cognizant that that we do look at CAR-T’s and looking at that ex vivo application of gene editing there, you know, moving from an autologous to an allogeneic cell therapy, you know, would obviously be a game changer. And I think we saw some interesting data on some B-cell lymphoma. But maybe you're more of an expert to talk on that than I am.
And then finally, it's, you know, it's just anything that will improve the potency of ex vivo CAR-T. And there's several sponsors that are going to have readouts across multiple indications towards the end of the year.
The final thing I wanted to touch on is moving into DNA writing. So, this is not like CRISPR-Cas9, where we're cutting up the genome and trying to repair it. It's not like base editing where we're going after one, you know, molecule of the DNA and trying to change that. This would be taking a section of the DNA and basically rewriting it, okay?
We will actually see some data from Wilson's disease, probably in, early 2027 of that prime editing modality and that really, you know, could unlock a whole new application of these gene editing technology.
12:29, Matt P
So, you know, I know over the past year, and you kind of touched on this, but some of the concerns that have come up. Things like, you know, liver injury with in vivo approach, you know, also just more broadly as you move these in vivo approaches, there's obviously this, “ Are you hitting the right cell types? And getting only the right edits that you want as opposed to something, you know, off target or something like that.”
I guess, how do you think some, you know, new technologies are trying to advance that and mitigate that risk? And, also, how do you feel investors. Yeah. Well, you know, what's the current sentiment around some of those risks in these programs or that are in clinics now?
13:06, Myles M
Yeah. So, you hit on a very important point. We actually published a separate deep dive a couple of years back looking at the delivery aspects of these genetic editors and RNA targeted therapeutics, as well. And, you know, it's a key consideration.
So, in the report, we actually go through a section where it's like, build your own game editor. And, you know, we can talk all day about CRISPR this and CAS this. And is it deaminase? Is it, you know, retaining its function? Is it a dead CAS? It's really nothing until you have a delivery modality around it.
So, traditionally, in the gene editing landscape for DNA editing that has looked something like a lipid nanoparticle, or you can pack that up into something called an AAV, an adeno-associated virus, which has packaging constraints based on size but enables you to deliver something whole as DNA would be expressing. Now, the issue with that is, obviously, you know, systemic administration, if that's what I want to do. Like, where does this go? Now, lipid nanoparticles, without playing around with them too much, tend to target really, really, really, really well to the liver, which is exactly the same reason why we see, if you do a high dose lipid nanoparticle, the first thing to show up, you know, within very near timeframes after the infusion is these things called liver function tests or transaminase levels, which is basically your liver overworking to fight and break down these fats. And it's a potential marker of liver injury.
For AAV, it's the same thing, you know. Sometimes, if you overdose on it, it's going to track to the liver, you’re going to have an antiviral response. It looks very similar there. Now, the advantages AAV have been that you've been able to use different serotypes, and based on the natural biology, they've been able to target to different tissues.
So, you know, for instance, AAV9 would be the common one that is used for delivery to the CNS, because it has tropism for neurons and glial and things like that. AAV5 has been used much more peripherally. There is a whole bunch of work going on in terms of serotype libraries and trying to engineer next generation capsids and a lot of business development around that area.
Basically, trying to find a therapeutic window for an individual tissue on the lipid nanoparticle side. Traditionally, this has been an area where there hasn't been a lot of innovation, until we have that wonderful Covid-19 pandemic, where everyone now knows what a lipid nanoparticle is. And, since that, you know, we have seen some innovations around development.
So, it's not just a standard four component fat that you put together. You can add in fifth components that might be a tissue targeting moiety or a de-targeting moiety. You can coat these lipid nanoparticles in certain conjugates that would actually target to different tissues based on cell receptors, that sort of thing.
But, at the end of the day, I mean, we're still very good at getting to the liver, we’re decent at getting to the CNS, albeit not necessarily to deep brain structures, but we do have access to the CNS. If you change up the route of administration, so you're not talking about intravenous infusion of subcu, you can do direct inhalation. So, we do have programs ongoing in the clinic for gene editing applications for things like cystic fibrosis, for instance, through an inhaled mechanism.
One other aspect that we've made leaps and bounds in is actually targeting hematopoietic stem cell compartments to try and basically access, could you do an in vivo CAR-T, right? So, rather than doing autologous or allogeneic CAR-T, which is all done ex vivo and delivered to the patient infused, then you have to do the preconditioning, theoretically. Could you just go in with a one-time in vivo CAR-T? And, that could open up agents in oncology, but also rheumatology, as well.
So, lots of work there. That's still largely pre-clinical stage, although there are some clinical trials going on at a very early stage outside of this country. So, they are things, you know, I’m looking forward to.
There was one issue that recently popped up in a cardiomyopathy patient where the liver signal that you would expect with some of these therapies, occurred much later than we originally anticipated.
And it doesn't seem to be due to something like a lipid nanoparticle, you know, being acutely processed. It happened about a month later, three to four weeks later. And so that did raise some concerns about, hey, are making edits, which, maybe there's an off target edit in one patient, maybe there's an intermediary that is being expressed and causing some abnormal toxicity.
What I'll say about that patient that, you know, the program since off clinical hold, it's just that there was a lot of comorbidities there. And they were an elderly patient, in one of these studies here. So, it's difficult to teeth apart, you know, how much of that was asset driven versus underlying co-morbidities and things. But, you know, there has since been increased screening and monitoring for these liver, you know, potential safety issues because of that particular patient.
18:43, Matt P
Yeah. Okay. Well, I mean, a lot to monitor there. I know, it's been a long time trying to find ways to get these things in the right tissue types, because that's clearly the key for some of these targets. You already touched on this a little bit, but, you know, I know people say gene editing, I think probably CRISPR pops into their mind.
You know, obviously, Nobel Prize, a lot of headlines, but the field is evolving. You touched on RNA editing, DNA writing. How do you see these approaches differentiating from one another? Are they clearly going in different directions on what diseases they can go for, or are there other aspects in how they're trying to, kind of, differentiate in their development strategy?
And, I guess, also, similarly, is it, kind of, one company has one platform, like go for that? Or are you seeing companies, kind of, utilize multiple technologies?
19:35, Myles M
If you had me on this podcast about a year and a half ago, I'd say that, you know, one company, kind of, plays within their own ring but no longer the case. I would say, when you think about CRISPR, you know, we have evolved so much since then. And so, a lot of these gene editing companies that were there at the beginning, most of them have diversified away, have included new assets that are no longer relying on just CRISPR-Cas9 and the original IP that you mentioned that came with the Nobel Prize.
We are seeing corporations of base editors, and we are also seeing various forms of prime editors and DNA writers and the report goes into a stack of them. There is a lot of freedom to operate and a lot of different modalities you could use. But it's safe to say that these companies are recognizing, hey, the gene editing field, the technologies have rapidly expanded since, even from 2018, 2019. You know, it doesn't suit us enough to just be a single modality player here.
If we are the experts in gene editing, we should be able to pick the right technology for the right indication. And so, what is that? That's a very expensive question to ask. But I think that the main thing is that the CRISPR-Cas9 approaches that we're seeing that that are in the later stage of development. That's the one where we have the, you know, greatest, sort of, de-risking, you know, we’ve been using that for a long time. We understand the off-target risks here. It has regulatory buy-in, as well, which we'll get into a little bit later.
But, you know, they know what to expect. So, there's just power in the fact that we've been using that for the longest time and, therefore, we have the most experience, you know. Base editing, we’ve seen a couple of companies now delivering vivo clinical data here.
So, I mentioned the alpha-1 antitrypsin deficiency. That would be uniquely, you know, positioned for indications where there's a single nucleotide polymorphism. You don't want to take the risk of making a double stranded DNA break and having potential integration issues or repair issues, I should say. They can cause indel formations in things. You just want to make a single, base edit change.
Not all genetic disorders, unfortunately, just snips, right? So, for instance, there are periods where you can have, like dystrophin, for instance, you have whole exons that are missing, whole exons that are skipped in the case of therapeutics, as well. So, you know, can you have therapies that just replace exons? Well, you can't do that with a base editor, you can’t do that with CRISPR-Cas9.
You need some sort of DNA writing technology. Multiple ways to do that, integrase, reverse transcriptase, those sort of things which we cover in the report. But you can start to think at, you know, larger, sort of, deletions, larger, sort, of repair needs. That's when you're starting to move into the DNA writing world. And that's, kind of, where the field is moving, because ultimately with these diseases, it's not just enough to like knock something down that went wrong.
You want to fully correct and rewrite what went wrong at the DNA level or the RNA level. So, that's, kind of, where we're moving. For the RNA editing applications, I think it's very interesting, right? Because if they're offered safe safety risks with a lot of these new technologies, you know, can you mitigate that by doing it transiently? And that's what the RNA editing offers.
Now, okay, you have to do repeat dosing, but with oligonucleotides, and the chemistries associated with them, I mean, that can be, you know, once monthly or once quarterly, or once every six months, right?
The other advantage with RNA editing is that it's not just like this is the dose that's approved for a gene editor. And this is the dose that we're going to give to every patient. If someone needs to be up titrated, if someone needs to be down titrated, based on their biomarker status, you can do that with an RNA editing therapy. You can titrate on and off. So, you know, there's some interesting advantages there. You can also introduce things that would upregulate proteins or make a constitutively active protein.
You can do a lot, lot of different things there. So, I think that there's lots of room to play for all of these technologies. I think indication selection is absolutely crucial for everyone that's playing in the space.
24:26, Matt P
On that front, your report, I thought had a really interesting section about, where does gene editing fit within a treatment landscape? A key question I have all the time is, you know, a lot of the targets that are being pursued by the gene editing companies, there's validation from RNA interference or maybe you're an antibody against the downstream protein, you know, PCSK9, like we have antibodies, we have RNA, you know, do you need gene editing?
The delay now is ongoing. Or, you know, we're seeing that, as well. So, what is your analysis there? Maybe walk us through a scenario where you think that DNA editing has that key advantage over RNA interference, which, you know, I could say is a little more validated. Given the number of approved products.
25:15, Myles M
Yeah. So, I think for indications that require, you know, an editing application to correct an underlying mutational series of mutations, you know, I do think that gene editing has a unique up on something like RNA interference. I'll come back to the alpha-1 antitrypsin deficiency example here.
You know, that is a single nucleotide polymorphisms. It's an E342K, if you really wanted to know what the mutation is. But the reason why I bring it up is because there is actually an RNA interference trial going on that just knocks down mutant protein coming out of the liver. So, it doesn't correct that protein. It doesn't restore its function. It just knocks down its protein. Now, that's going after patients that have liver manifestations only, and that trial is in a phase three. It's got a liver biopsy end point, and hopefully it kicks people off the liver transplant list. But it does nothing for the fact that you need to actually have antitrypsin activity to protect the lung.
So, if you have lung manifestations, of which most of these patients do, they have COPD, they are like chronic smokers. You're doing nothing to help them on that angle, versus if you came in with a gene editing approach, you correct the underlying snip. You do that in the liver because that's where the majority of this protein is produced.
All of a sudden, not only are you restoring the function within the lung because it's an active protein that it’s wild type is being secreted from the liver to have function in the lung there. But you're also benefiting the liver, much like you would have just knocking down a protein altogether, you're actually just restoring the wild type function and you're removing that mutant allele, as well.
So, those, sort of, indications are something where a corrective therapy that is unique to gene editing would be very, very well positioned.
The other thing I look at, as well, is anything where there's a high degree of mortality or a high degree of burden on the patient, that happens very, very early on in life. So, that can be anything from, you know, SMA and things like that when we had gene therapies like Zolgensma approved, you know, the risk benefit for those populations was immense. You were 100% more mortality at three years, unless you got this therapy, and now you're thriving into milestones and living into adulthood, right? So, where are those indications where the risk benefit is best or there's been a lot of controversy, but, gene editing for more prevalent, cardiovascular indications, right?
So, for lipid lowering, you mentioned Lp(a), PCSK9. We've done a lot of work there. ANGPTL3 would be another one, as well, that sponsors are looking at. There, I think, it's that type of patient that's had an early coronary event in their 30’s, right? So, it's rare, but it does exist. That staring at a lifelong treatment journey on lipid lowering therapies for the next 60 to 70 years of the life potentially, you know, do they want a one and done therapy where they don't have to worry about it?
And it's a one-time, sort of, risk there? Or do they want to go on that chronic therapy? And I think that that's where you can have the argument. I think if you're an elderly patient, right, and, you know, you're looking for lipid control and you're looking to add on another therapy, does the risk benefit of a gene editing therapy, in its current iteration, does it make sense? That would be on a case-by-case basis.
But I definitely think for those premature CVD patients, there is certainly an argument for one and done therapy. At least they have that option for patients.
29:16, Matt P
No, I think that makes sense. Definitely plenty of diseases that just can't be amenable or could benefit from one and done. You know, a topic that's a little bit broader than just gene editing, but I think has definitely been applicable here is the turnover at the FDA over the past year. And, you know, especially as you think about some of the rare diseases, you know, there's concerns for investors on what is going to be the new goalposts for the FDA.
You know, I guess as you've talked with investors specifically around some of these gene editing trials, any shifts, you think, to FDA guidance or maybe what the FDA is going to be looking for in terms of efficacy or safety from these modalities?
29:57, Myles M
Yeah. So, always tough to predict what's going to happen at the FDA. And there will be near-term changes for sure with the new CBER head, which, you know, sees a lot of these potential gene editing therapies across the desk. But what I would say is that the FDA traditionally has been pretty prescriptive, with their guidance and being very open to work with sponsors in this space.
You know, there are published guidance documents that helped us lay out what those post-marketing commitments are for, you know, the period that you have to monitor for potential off target effects, right, because you might not detect them at the time that you administer the therapy. Maybe they take years to manifest. So, there's a 15-year term for that. And that is a regulatory defined term.
You know, the agency helps sponsors come up with the correct assays to maintain their monitoring. Not all of those off target effects there. And also, you know, what tissues to look for and what sort of symptomatology to be reporting. So, that's all there.
Things like RMAT designation are still in place. And yes, they can apply for multiple different modalities here. But, you know, it's crucial for gene editing, and it's basically the breakthrough therapy designation equivalent to this modality. You know, I haven't seen that rescinded even through the various regulatory volatility period that we've been experiencing.
And then, finally, you know, coming back to selecting the right indication, you know, the plausible mechanism pathway that that is still talked about and potentially still valid, you know, that could be, you know, broadly applicable across many of the gene editing programs that you're seeing, at least the ones in the clinic that that I'm seeing right now, as well.
So, I do think that there's some favorable, regulatory paths here. I just don't know what I can't predict which is what's going to happen with the new CBER. And, the vast majority of these applications will be saying through CBER.
32:05, Matt P
Yep. I think everyone's in the same boat there. Okay, you know, I know there's a lot of work in the report on trends in M&A, business development across the gene editing space. I guess, any key trends that stand out to you, whether it be, you know, some of these newer technologies, like the DNA writing, or indications that have gotten the most interest recently as you've looked at some of that activity?
32:30, Myles M
Sure. So, I mean, alpha-1 has had some recent BD interest from a large biotech on the DNA writing side that utilizes reverse transcriptase. That same sponsor has also had some activity in the cardiomyopathy space from the program we previously mentioned.
But what I would say about the trends is like, if you look across, the vast majority of big pharma has fingers in this pie, across multiple development stages.
We detail the deal number for the, you know, the past five years in the space. And it's remarkable that it's not just, you know, one individual, innovative biotech that's trying to do all the deals. It's really spread across, the flavors of deals do vary.
You have some pharmaceutical companies that are looking to get in early, so they have an indication in mind, right? They have a target product profile in mind. They may not have the expertise in which to actually generate the asset that they want to take through clinical development. So, they'll go to a company that has a unique technology that is suited for that target product profile to make that asset.
So, the very, very early stage collaboration, there's, you know, a company that has been very, instrumental in the management of CF, cystic fibrosis, that, kind of, has that phenotype.
Then you've got the, the companies that, kind of, step in and say, like, you know, we're not going to apply this technology game, we're just going to apply a very asset driven game. And where we can lend our expertise is you do the technology, you designate the candidate, and we'll run the clinical program. We’ll help you do that.
And I think that that was a mistake that was really made during that, sort of, coming back to the first question, during the peak of the hype here, was that we had a bunch of platform companies that were largely academic experiments and, you know, a lack of drug developers that had actually run a clinical program.
So, what asset do you choose? How do you design a trial, all of that? That's expertise that a big pharma that's done it before can help with those companies. So, you can let them focus on the technology, which is the core expertise.
And then again, coming back to that alpha-1 and the cardiomyopathy, there is one player there that is, you know, made multiple deals, at later stage, sort of, pivotal stage assets or, you know, the one and only commercial stage, gene editing asset has obviously been the focus of a BD deal as well.
So that kind of gives you the three flavors of where we're looking at. But it's big pharma more broadly that has played across all of those avenues.
35:36, Matt P
Well, Myles, I think we're about at the end there. I want to wrap it up. Any other bold predictions for the gene editing space more broadly here over the next 12 or so months?
35:47, Myles M
I think from our report, that gene editing is really moving towards that corrective phenotype. So, I'm waiting to see the first, sort of, in vivo data of a DNA writer, okay?
So, since we republished the last iteration of this reporting in 2022, we're saying our first clinical data of base editing, right, changing one, you know, nucleotide in vivo. I think the next frontier, which is why I'm focused on the Wilson's disease data in 2027, will be, you know, can you turn that one to into three? Can you turn the three into half an exon? Can you turn that into a full exon? The DNA writing applications of gene editing is super exciting.
And then, on the other side, evolution of the RNA editing space, because I really do think that that has a role to play here, especially just the titrate ability that has been so beneficial for RNA.
Being able to titrate a genetic medicine and to do that on an editing perspective, you know, the more data we see there, outside of alpha-1 antitrypsin deficiency, and it's coming, you know, will be really important.
So, I think, I think in the next couple of years, you're going to have a DNA writer in the clinic that's writing exons.
And I think you're going to have RNA editors that are becoming much more mainstream, much like the RNA interference plays have.
37:17, Matt P
Well, Myles, thanks for joining us on this episode of Biotech Breakthroughs. I'm Matt Phipps, and we'll see you next time.