Good afternoon. I'm Eric Joseph, Senior Biotech Analyst with JP Morgan. Our next presenting company is CAMP4, and presenting for the company is CEO Josh Mandel-Brehm. There's a Q&A after the presentation. If you have a question, we'll bring a mic around to you before asking your question. If you're joining online, feel free to submit questions via the portal. So with that, Josh, thanks for joining us.
Thank you, Eric, and thanks, JP Morgan, for hosting us. CAMP4 is the last camp before the top of Everest. For us, it represents the opportunity to pioneer a new wave in innovative medicines to address the hundreds of diseases where upregulation could provide a meaningful clinical benefit. To accomplish this, we use antisense oligonucleotides to specifically increase the expression of genes. And we do this by targeting a novel set of RNAs called regulatory RNAs. These are RNAs that arise out of the non-coding genome, specifically enhancer and promoter regions. And we know, thanks to our work and the work of our founder, Dr. Rick Young, that all the protein-coding genes in our body rely on their own unique set of regulatory RNAs to control their expression.
Using our RAP Platform, we've been able to catalog in over 10 different cell types all the regulatory RNAs and the protein-coding genes that they correspond to. That allows us a very rapid way to identify targets, to then drug these regulatory RNAs with antisense oligonucleotides, and force a deliberate increase in a target gene underlying a disease. Our current focus areas are in the metabolic and CNS space. In the metabolic space, our first program that's in the clinic is for a rare, life-threatening genetic disease known as urea cycle disorders. We have a healthy volunteer study ongoing that is leveraging a biomarker, and that's set to read out in the second half of this year. Behind that, we have our first CNS program for a genetic haploinsufficiency called SYNGAP, which I'll talk about later in the presentation.
That program is set to start GLP tox studies this year as well. Our team has expertise ranging from gene regulation through preclinical development, chemistry, clinical development, manufacturing. This is all needed to be able to scale our platform and be able to bring our drugs into the clinic. As I mentioned, our urea cycle disorder program is already in an ongoing study. The SAD portion of that study has already been complete, and we're more than halfway through the MAD portion of that study that's set to read out in the second half of this year. Behind that, we have our SYNGAP program that's on track to start GLP tox studies. And we very recently nominated another program, GBA1 for Parkinson's disease, which is a more prevalent disease, bigger disease opportunity. And we have some really exciting data that's starting to emerge from that program.
Other earlier activities that are ongoing will feed into our future pipeline, but also in business development as a platform company. That's an important area for us. We also recently announced a collaboration with BioMarin. And although we didn't disclose the tissues, these are outside the metabolic and CNS area. In this collaboration, we are able to identify regulatory RNAs as well as the ASO candidates against targets that they nominate. Then we collect milestones and royalties as they develop the products further into the clinic and towards commercialization. To talk a little bit about the biology that makes us really special, what I'm showing here is a loop of DNA. What I'm showing in the center of it is a regulatory RNA that's emerging out of an enhancer. This is our target space.
Thanks to the work of Rick Young, what we now know is that activators, repressors, transcription factors all come together and form around these regulatory RNAs to control the expression of a nearby protein-coding gene. What we've discovered at CAMP4, if you look to the right, is that by taking an antisense oligonucleotide and putting it in a very particular spot on the regulatory RNA, we can very specifically displace a repressor, and that allows for more gene expression to come out of the protein-coding gene, thus allowing us to go after many different types of diseases where you wish to increase the gene expression in a very specific way.
The first step in our platform and our special sauce is being able to use next-gen sequencing mixed with machine learning capabilities that allow us to "map cells." This creates an in silico database that allows us to be able to identify all the regulatory RNAs and the protein-coding genes that they control. Once we have a target gene in mind, we can very rapidly pull out the sequence of the regulatory RNA that controls that gene and screen standard ASO chemistry against that regulatory RNA to get optimal upregulation of the target gene. The last step in this is that we can optimize those leads, meaning we can tinker to make sure it has the most upregulation. We can make sure it's as safe and specific as possible, and in the case of our urea cycle program, we can conjugate it to GalNAc so it has subcutaneous delivery.
Or in the case of our SYNGAP program, we can use intrathecal delivery to get it to the regions of the desired brain regions. Very importantly, we get asked a lot about how we choose diseases. The simplest way to explain that is the first stop is that we look for diseases where we have high conviction that the gene that we're targeting, a modest increase in it, would lead to a high impact in patients. Typically, those fall in haploinsufficient disorders like SYNGAP or in partial loss of function disorders like urea cycle. We then look to translation and drug ability, meaning we want to make sure that the desired drug can get to the regions safely and effectively. We put a lot of weight in our preclinical data. We tend not to move programs into the clinic unless we have high conviction based on the data.
From a clinical regulatory perspective, we like to work on diseases that have approvable endpoints but also have biomarkers. Urea cycle is an example of this, and then lastly, we tend to look for opportunities that we can build businesses around, so those tend to have high unmet needs. Many times, those are life-threatening, and we make sure that we're differentiated from competition. As I mentioned a bit earlier, we are developing our own pipeline, but for a company like ours, business development is very important, and this slide is just meant to articulate that there are many prevalent diseases or larger genetic diseases that are going to be of interest to partners, and these are highly applicable to our platform. We're not showing the gene targets here. We have that at CAMP4.
But each and every one of these diseases has a target that we believe if you were able to upregulate a modest amount, could have a huge impact on the disease. Our first program that's in the clinic is for a disease called urea cycle disorders. On the left, I'm showing a picture of the active urea cycle. It's six enzymes that work together to convert toxic levels of ammonia down to non-toxic urea. If you have a mutation in any one of these enzymes, here I'm showing the OTC mutation, the most common subtype. Although you retain partial enzymatic activity, it's not enough to prevent the buildup of ammonia, which is a very toxic molecule. When you have the buildup of ammonia, it's called a hyperammonemic crisis. This is a life-threatening circumstance. More so, it can lead to permanent neurological damage over time.
These patients have absolutely no disease-modifying treatments broadly. They are basically on a very restrictive protein diet that borders on malnutrition, or they use nitrogen scavengers such as Ravicti, which are useful but not disease-modifying and don't prevent the attacks from happening. Very importantly, we've guided people to say there's about 4,000 severe patients in the U.S. alone. However, more recently, through our own work and discussion with KOLs, we've also identified an additional patient population, that being the OTC female carriers, who are sometimes asymptomatic but many times do have symptoms as well, so this disease is in need of disease-modifying treatments. Our approach to having an effect on this disease is by targeting CPS1, which I'm showing here. This is the first enzyme in the urea cycle, and it has two very important properties that we care about.
The first is that when we increase CPS1, it immediately starts the conversion of toxic ammonia to non-toxic byproducts that can be taken up by the urea cycle, including the partially active enzymes. And the second feature is that when we increase CPS1, all the downstream enzymes can also increase. Taken in totality, by increasing CPS1, we can boost the urea cycle in a mutation-agnostic way, which allows us to make the claim that we believe our drug could potentially be used for more than just OTC and go after the other subtype populations, thus calling it the pan-UCD approach. Just to level set on how we think about the patient population, there is a spectrum in this disease. And it's through that heterogeneity that we know a modest increase can lead to a big impact. There is a small percentage of the patient population that's called early onset.
Unfortunately, these patients have less than 5% enzymatic activity, which is not compatible with life. And they need a liver transplant. Otherwise, they will not live past a month of life. We are focused on the severe patient population, which I'm showing here has more than 5% activity but still has hyperammonemic attacks. But we've also now started to focus on the OTC female hets who are symptomatic as well. So the patient population is not just severe, but it's also the female carriers. I mentioned before the standard of care is pretty limited. There is a very restrictive protein diet that patients are on. And then there's also a drug called Ravicti. This is highly burdensome, but it is useful, although it does not prevent the hyperammonemic crisis from happening. There are also two other modalities that are in development. One is a gene therapy.
The other is an mRNA approach. Both these approaches are limited to just OTC and patients 12 years or older. Our approach, which I'm showing on the left, would be a once-monthly subcutaneously delivered product, so it'd be an injection into the stomach. As I said, it would be for more than just OTC, so 90% of the severe patients, and importantly, we believe we'd be able to use our drug in a very young age, which is quite important because you want to prevent these risks from happening because they cause that damage over time, so we're really focused on trying to get to the underlying mechanism of the disease, prevent the hyperammonemic crisis from happening, being able to relieve them of their diets, and get them off of these scavengers. I have a few slides of data.
But in summary, what gets us so excited about this program is that we're actually able to upregulate the CPS1 gene in both healthy hepatocytes as well as disease patient cells. There is a mouse model replicating the disease where we showed that when we gave our drug, we could actually bring the mice back to a wild-type state in terms of their ability to clear ammonia. And we also took our drug into wild-type monkeys and used the same assay in monkeys that we're using in the clinic and showed that even in healthy monkeys, we could increase their urea cycle by using our drug. On the left, what I'm showing is a very simplistic view of our platform. What I'll point you to is the CPS1 gene. And then what you'll see are five red boxes. Those are enhancers.
The one with an asterisk is where the regulatory RNA is arising from. This is our target. This is what's shown on the right. We are drugging that target with our clinical candidate. And in blue, you can see a nice dose response against CPS1 increasing. There is a mouse model that's well-trodden. It is an OTC-specific model. And in these mice, they have about 10% residual OTC activity compared to wild-type. And what you do is you challenge these mice with ammonia, and you create a state of hyperammonemia. So it's very similar to what a patient experiences. Now, if they do not have any drug, what you'll see is on the left in gray at the top, they are not actually able to clear the ammonia. So they stay sick. If you're a wild-type mouse, you're able to clear the ammonia.
What you can see in the orange, teal, and blue lines, which represent different doses of our drug, is that when we give these mice our drug and we challenge them with ammonia, over the course of a few weeks, they're actually able to clear the ammonia as well as the wild-type mice. I should also point out that these concentrations correspond to the same concentrations that we're using in the clinic. These are in the therapeutic range. Last slide before I get into the clinical development plan. On the left, what I'm showing is what's called labeled sodium acetate. This is what we're actually using for our ureagenesis rate test. It is a surrogate for ammonia. It is completely harmless. Either patients or healthy volunteers can take the sodium acetate. It gets taken up by your urea cycle.
And it allows us to measure how quickly it can be converted into urea. What I'm showing on the right is we actually gave this to monkeys in conjunction with giving them our drug. And very happily, what we saw is in orange, we could see a 40% increase in their active urea cycle compared to PBS using the same URT. So we've taken that URT, and we've actually built it into our Phase I study. What I'm showing in the lower left is the SAD portion of our study. There are four cohorts, 12 participants in each, randomized three to one. So nine participants get drug, three get placebo. It ranges from 0.2 mg per kg all the way up to 4 mg per kg. That has been completed.
We've also completed the first two cohorts in the MAD portion of the study and are now on the third MAD cohort, so we are on track for our second half of the year readout. We recently announced the safety data. Said simply, it's unremarkable. We're really happy with what we're seeing. It does not even have a maximum tolerated dose, meaning we could dose higher if we needed to. We have no safety trends of concern, and the only treatment emergent events that we've noticed so far are grade one or two, so headache and nausea. And as I mentioned, we've already been given the green light to start cohort three by the safety review committee. As we look forward to what comes next, we want to go into patients, and we want to move forward quickly, given the unmet need.
We do anticipate the opportunity to potentially start a phase two, three registration study. From this study, we're not only going to get safety. We'll get pharmacokinetic data, but we'll also get that URT data, which will help us optimize the future studies. One other point that I will say, and I mentioned earlier, is that ammonia is actually an approvable endpoint. So that's good. We already have a way to get this approved. But we also now know we can use our URT test to basically work out other endpoints with the FDA where we can liberalize the patients from their diet or get them off those nitrogen scavengers. So we have multiple shots on goal to be able to get this approved for patients. We'll, of course, be looking at ureagenesis as well as other clinical events, which are very relevant for this patient population.
Shifting gears, our next program that I mentioned that we're very excited about is for a disease called SYNGAP. This is a haploinsufficiency. So you have one allele that's still healthy, and the other allele is now dysfunctional, meaning you're 50% of what you need to be healthy. Although based on human genetics, we think that if you increase it about 30%-50%, you'll probably have a meaningful effect on these patients. What happens in this disease is you don't make enough healthy SYNGAP. Therefore, you get hyperexcitability at your synapse. This causes all types of problems for children and adults. You have intellectual disability. You have seizures. You have sleep problems. And there are no approved disease-modifying therapies for their patients. This is a rather large genetic disease. There's at least 10,000 patients in the U.S. today.
Our approach is, of course, to use an antisense oligonucleotide to act at the transcriptional level to boost or increase the expression of SYNGAP1. What we're showing on the left is the way that we do this, and by increasing SYNGAP1, we believe we can restore healthy function between the synapses, lower the hyperexcitability, and allow these patients to reduce the seizures and hopefully correct learning disabilities and other autistic problems. In terms of the treatment landscape, as I mentioned, there's no disease-modifying therapies available for these patients, and more so, there's not really anything in the clinic to date. We think we may be one of the first companies that would be able to enter the clinic with what would be a disease-modifying therapy, so a bit of data on why we're so excited about this program.
On the far left, what I'm showing is how we use our platform. In the middle, you can see the SYNGAP promoter on top of that in orange. That is actually the RNA that we're drugging. If you look in the middle region, this is number two. This is our form of target engagement. So what we're showing in ABC is transcriptional activity after having drugged the regulatory RNA with an ASO. Control 1 and Control 2 are transcriptional sites at other nearby genes and housekeeper genes. So this is basically showing we get target engagement and at the same time, we don't get any off-target activity. What that corresponds to is what I'm showing on the right. We took SYNGAP patient iPSC-derived cells, and what you can see is if you have a perfectly normal person, they have one in terms of their SYNGAP mRNA full-length.
If you have a SYNGAP patient, there are exactly half of that, which is shown in light blue. If we treat those SYNGAP patient iPSC-derived cells with our ASOs, ASO7 and ASO9, we can restore function back to the normal state. So that was very encouraging. But what about in vivo? So what I'm showing on the left, these are two different mouse studies. The first one is showing a dose response increase on the x-axis. This is increasing concentrations. On the y-axis, this is SYNGAP mRNA. And here, we've developed a mouse-specific SYNGAP ASO that we've used to show increasing concentrations. And what's very encouraging about this is the upregulation that we are seeing in this mouse model is what would correspond to clinical doses that we know we can get to in the actual intrathecal delivery. So that's encouraging.
On the far right, this is more recent data that we published on. We took a human SYNGAP gene and transplanted that into mice, and then we took our human clinical leads and tested that. And what you can see is in different regions, we get statistically significant upregulation of SYNGAP in regions that we believe are critical for treating this disease, and again, these are concentrations that we know we can achieve in the clinic safely through the intrathecal form of delivery. Lastly, just a snapshot of our newest program. This is GBA1 for Parkinson's. It affects about anywhere from 5%-15% of Parkinson's disease patients in the U.S. So that corresponds to about 100,000 patients, give or take. Why we're particularly excited about this program is GBA1 is a haploinsufficiency. So that gives us a good starting point.
But it could have applicability to the more broad sporadic Parkinson's patient population. In the third row right here, what you're seeing in the lower right is just a snapshot of the dose response data as our starting point. We're very excited with what we're seeing there. And we're starting to move this program forward as well. We also get to benefit from already existing clinical development biomarkers and scales that we can bring to bear when we get this towards the clinic. So in conclusion, there are many different prevalent diseases as well as rare diseases where upregulation could be the difference between being healthy and sick. Most of these have no approved treatments. We are the leading company for using ASOs for upregulating gene expression. Our current focus area remains in the metabolic and CNS space.
We have a few major readouts coming later this year, the most important being around our CMP-CPS-001 program for urea cycle, which we'll read out in the second half of this year around our biomarker. Thank you very much.
We got time for questions. I think for the CMP-CPS-001 program, obviously a key focus point for investors and for you guys as well, obviously, will be the MAD data later in the second half of this year. Given that it's a healthy volunteer study, what sort of ureagenesis do you think is observable in this patient population as you kind of look at flux and such that it would be de-risking for or support proof of concept and also be de-risking for a patient study?
Yep. We get asked that a lot. And we, in turn, have spent a lot of time talking to the clinicians that treat these patients and the experts around that. And the feedback that we received was, on one hand, if you see any activity showing that you could target CPS1 and that biology corresponds to an increase in ureagenesis, you have to get this drug in patients as quickly as possible. I think that comes from the fact that they understand we're going after the root cause of the disease. Ureagenesis is completely correlated with severity. So any amount of increase in ureagenesis that you can offer to this patient population can be meaningful. Of course, we're really hoping to see great activity. But we also understand that these are healthy volunteers, and they have fully functional urea cycles.
There is some precedent showing that using the URT, you can see an increase in human ureagenesis. We're hoping to see that. But we also know, just given the unmet need in this population, that the bar is set really low. Patients are waiting. The doctors are really hoping that we move this forward as quickly as possible into patients.
What's the dose interval, a range of dose intervals that you're evaluating in the MAD study portion? And to what extent have the completion of the SAD study portion been incrementally informative or kind of adjusted at all to influence the dosing parameter in the MAD study portion?
Yep. Right now, based on the PK, PD, and all the preclinical studies, as well as the SAD, we continue to dose on a monthly basis. So you asked about the MAD. That's three doses over three months, once per month. The SAD was actually informative for us in not only just making sure that it's the right dosing frequency, but also just adjusting how we use the URT assay, because what we're really focused on is the MAD, because they're getting multiple doses. If you recall the primate data I showed where we saw a ureagenesis increase in those healthy primates, that was after two doses as well. So the SAD was also very helpful for helping us optimize how we were using the URT and the timing at which we were measuring that.
Yeah. I guess just theoretically, I think maybe you answered it in the first question. But I'm just wondering just if there is a meaningful difference in the ability to upregulate CPS1 in a healthy versus OTC disease setting. I mean, yeah, I guess how do you, I mean, is there anything from the preclinical data that sort of informs this question?
Yeah. I mean, we believe, based on the disease mechanism, that actually, I didn't answer this exactly how you had asked. If we see activity in a healthy setting, we believe it will be demonstrably more impactful in a patient setting, and the reason I say that is a couple of things. One is there has been a precedent by some other drugs where they saw a little bit of activity in a healthy volunteer setting using the URT that translated to a significant reduction in ammonia in, for example, NAGS deficiency, so that's a Carbaglu example, so there's precedent to show that a little goes a long way. These patients don't have fully functioning urea cycles, so there is sort of reserves there.
So we do expect that it won't be one to one, but it would be in the favor of what we want, which is a little in healthy can translate to a much bigger one. And I think the other point is we know from talking to KOLs; you're probably not going to max out a healthy ureagenesis cycle too much more. So it's very likely that if we see a little bit, it could be a lot more impactful in these patients, based on the precedent, based on the mechanism.
You've laid out what could be a potential registration program, at least loosely. To what extent have you engaged regulators regarding that plan? And if none, I guess, when would such interactions be anticipated?
Yeah, so we've had initial conversations previously. But to the point you're asking, we are planning to have those conversations this year, and so we anticipate that there'll be flexible ways to move this forward. We've looked at other programs, which is sort of how we've come up with that perspective, but it's going to be important that we have those regulatory interactions this year.
In this setting, do kind of particular drug designations kind of facilitate or, in addition to sort of orphan drug designations, kind of help facilitate interactions? Are those designations something you'd be seeking along the development process?
Yeah, so thank you for asking that. We do have orphan drug designation, and we do have a rare pediatric designation. Of course, we hope that that gets renewed. We do anticipate that that, as well as the patient community and the frameworks that the FDA has been putting forward, for example, around rare diseases and moving things forward more quickly, will work in our favor. I should say Ultragenyx is ahead with an AAV approach using the URT, and I think it has been doing really nice work with the FDA on paving the way for other endpoints aside from just ammonia reduction as well, so we get to benefit from that too and their learnings.
Your expectation about ammonia reduction kind of being, yeah, right, a primary endpoint or a pivotal endpoint, is that would that support full approval? Or do you anticipate sort of like a two-pronged?
Yeah.
More meant to, where you kind of have to do first on the basis of accelerated approval and then you'll see full approval.
Yeah. Prior products, it's a clinical, have been approved on ammonia reduction. So the challenge is that some proportion of patients will enter the study with already pre-existing higher levels of ammonia. So that's pretty straightforward. None of us in here have high ammonia levels. And if you come in, you can ask the question, is the ammonia lowered or not by virtue of your drug? And so that is an approvable endpoint. Some patients, though, will come in with perfectly normal ammonia levels because they have it in check. But it could spike at any moment through getting sick, getting pregnant, relieving yourself of the diet. And that's called a hyperammonemic crisis. It's hard to predict when those are going to happen.
And so I think the innovation here that I was referring to is the ability to use the ureagenesis rate test as a way of measuring that your drug is working. And then you can create a responder analysis that asks the question of, if my drug's working, can I loosen up the diet, which is a dangerous thing to do. If it's not working, can I get them off these nitrogen scavengers, which are quite burdensome? And that's what Ultragenyx is doing as well. And I think these are all in the category of full approvals because they are clinically meaningful.
On the SYNGAP1 program, can you just, obviously, proceeding towards the selection of a developing candidate, I guess, what can we anticipate to that end in terms of additional sort of preclinical updates related to that program as it kind of transitions from early to nearing clinical development?
Yep. So we have a few different oligos that we're really excited about. I shared some of the data on a prior slide in terms of the humanized mouse data. And we're very close to nominating a clinical candidate. We're just finishing up some non-human primate studies. And then we intend to start our GLP tox studies. So thus far, we're feeling very good. But as I mentioned, we tend to take an approach where we want to have as much good data as we can to give us confidence going into the clinic. So at present, we have a few different oligos that we're quite excited about. And I anticipate in the coming months we'll be nominating a DC and formally going into GLP tox.
Compared to urea cycle disorders or other metabolic disorders where the liver is the primary delivery target, here you're going to be administering to a more privileged CNS region via intrathecal administration, at least I presume so. I guess, are there unique formulation or chemical considerations when you're sort of designing an appropriate candidate that you need to work through here?
Yeah. It's a good question. The answer in some cases, no. We are taking. They are modified. And typically what you do, and it's not specific to CAMP4, is you take a few different candidates into primate studies, for example, because sometimes there's idiosyncratic tox. Thus far, our drugs look pretty good. And you just use the intrathecal delivery to get there. Through the work we've done, though, we find that the distribution matches with what you'd expect for other oligos that have been administered intrathecally. And to go back to how we choose diseases, we also, for the CNS, choose diseases where, despite the CSF gradient, we know that intrathecally we can still get enough of the drug there at concentrations that will be efficacious.
So in this case, there's nothing, I think, beyond what other people would do for the state-of-the-art chemistry to essentially get the distribution and safe properties you want. So thus far, we feel pretty good about it.
Just in the neurology field, neurological clinical practices, I guess, what, if any, sort of measures or programs are underway that might sort of help to streamline the patient identification process of patients that might be suspected SYNGAP1 or have SYNGAP1-related disorders, given sort of the question about overall population sizing?
We've got a lot going in our favor for this disease. First of all, it's high unmet need. And as such, the patient organization, to give them credit, is completely organized, proactive. There is a registry. They are doing a natural history study that we're going to plan to participate in. It was a very high-profile discussion at the AES conference. So they're doing all the right things. These patients are now getting diagnosed more and more frequently with genetic screening. And we ourselves have been spending time with the KOLs that are both in the U.S. but also Australia and other places. And we've found the physicians to be extremely proactive. So one benefit of there not being a lot of competition in the clinic right now is that these patients are in desperate need of getting a therapeutic.
So I think we're going to have a lot of opportunity to identify the right patients to select for. So, for example, not all patients have epilepsy. However, we might optimize for getting patients to start with that have a high amount of epilepsies. Therefore, we can use that as a biomarker. Places like CHOP, for example, and Penn are also doing a lot of innovative work here to come up with EEGs and other measures of being able to look for efficacy instead of months and years to weeks and months. And so I think across the board, there's a lot of innovation happening here to the endeavor of making sure these drugs that are coming into the clinic have the best opportunity to succeed in a patient population that's never really had anything.
Maybe just one last question, kind of more to the platform and the potential for additional business development. I guess, how much effort, I guess, or time is dedicated to additional BD activity at the organization? And as part of those considerations, are there certain realms that are more perhaps partnerable versus those that you might reserve for your own proprietary pipeline?
Yeah. So we are right now, for our own purposes, it's a bit of an Ionis- Alnylam approach. We are intending to move forward rare genetic diseases that we think have biomarkers, that we think we can get quick readouts out, and that, of course, have a high unmet need. So we are intending to maintain and hold on to those because we want to be a commercial-stage company. On the other hand, we are starting to work on more prevalent diseases, sometimes taking them a bit further, like the GBA1 Parkinson's program, which we expect at some point we would need to partner. That's a larger disease. At the same time, we're incredibly efficient on the front end with our platform. As I mentioned, once we apply our platform to a cell, we call it mapping. It's a once and done.
It creates an entire database and catalog of regulatory RNAs. So anytime a partner wants to come to us and has a desired gene they want to go after, in the course of months, we can identify ASO candidates to upregulate it. So what we've begun to also do with our resources is generate small data sets as well for some of these bigger diseases because we found that's helpful for partners as well. It's always useful to have a little bit of data. And so that's starting to, one, create benefits. I also think that every pharma company and company alike recognizes that there are so many diseases where you just need to put some healthy protein back into the system. And there's just not a lot of therapies there. CNS has over 30 different haploinsufficiencies.
And so we're starting to get a lot of questions and conversations about opportunities to do work there. So business development is going to be very important for a company like ours, both because we don't want to leave value on the table, but also because we want to get this moving, as many programs moving as possible. So we're going to devote a lot of effort to that over the coming year.
Yeah. All right. Great. Well, I think we might leave it there for time. Thanks, everybody, for tuning into the session. And thanks, Josh, for the presentation.
Thank you.