Nanoscope Therapeutics is working on the potential first-ever optogenetic therapy for retinal diseases
In this episode of The Ophthalmic Project, host Mark Dlugoss and guest Sam Barone, MD, discuss Nanoscope Therapeutics’ new optogenetic therapy to help restore vision in patients with retinal diseases like retinitis pigmentosa and Stargardt disease. Dr. Barone explains how the MCO-010 platform uses light-sensitive proteins to help surviving retinal cells respond to light again. The discussion also covers how this treatment differs from traditional gene therapies, along with early clinical trial results showing improvements in vision and long-term safety.
Mark Dlugoss:
Optogenetics for vision restoration is an alternative gene therapy that utilizes light-sensitive proteins, commonly known as opsins, to restore sight by making surviving retinal cells light-responsive. While optogenetics has been researched in ophthalmology for roughly 15 years, there is 1 platform emerging that is showing promise ahead of phase 3 trials. Hello, this is Mark Dlugoss, senior contributing editor for Ophthalmology 360, and welcome to The Ophthalmic Project, powered by Ophthalmology 360.
In today’s edition of The Ophthalmic Project, we will learn about a company that is developing an optogenetic platform to treat retinitis pigmentosa and Stargardt disease. Joining The Ophthalmic Project to learn about optogenetic technology and its role to addressing retina diseases is Sam Barone, MD, Chief Medical Officer of Nanoscope Therapeutics. Dr. Barone, welcome to The Ophthalmic Project.
Sam Barone, MD:
Thank you, Mark. It’s a privilege to be here. It’s an exciting time for us at Nanoscope. I really appreciate the interest in our work and the opportunity to share it today.
Mark Dlugoss:
It sounds like incredible technology. Before we get to our discussion about the science, let’s talk about the overall view of Nanoscope Therapeutics. Can you provide a history of Nanoscope, when and how the company has started, the individual principles behind the company, and any key business milestones that the company has experienced coming to this point?
Sam Barone, MD:
Nanoscope is a registrational phase biotechnology company, and we’re committed to developing and commercializing novel disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degenerations. We really see 2026 as a breakout year for Nanoscope on multiple fronts. Our lead program, MCO-010 in retinitis pigmentosa. We are on track to complete a rolling BLA submission with the US FDA in June and a marketing authorization application to the Japanese PMDA to follow shortly thereafter.
With very limited treatment opsins of diagnosis of retinitis pigmentosa, as you know, is also known as RP, it’s a life sentence of progressive vision loss to blindness. If approved, MCO-010 would represent the first-ever optogenetic therapy that’s been approved and it’s representing a category-defining milestone and really an opportunity to impact the lives of many patients.
Approximately tens of thousands are addressable at launch in these 2 geographies alone. We are therefore additionally actively preparing to stand up the infrastructure to support a launch, so that’s a second major milestone. Then additionally, we’re expanding the platform with 2 new trials this year, a phase 3 trial in Stargardt disease and a phase 2 randomized, controlled trial in geographic atrophy.
Together, these indications represent over hundreds of thousands of additional patients that could benefit from our technology. You’ve heard me talking here, ability to impact patients’ lives keeps coming up because this is these patients who are our north star. They’re the ones who are driving us at Nanoscope to do what we do. This originates from our CEO, Sulagna Bhattacharya, and she comes from a family history of RP.
She’s actually witnessed firsthand that tragic impact of the progressive vision loss to blindness on not only the patient, but on the patient’s loved ones and the caregivers as well. She’s leveraged her background in financial services and management consulting into pursuit of a treatment for such vision robbing degenerations. She actually characterizes efforts as her personal mission.
She met Samarendra Mohanty, PhD, who’s our president and chief scientific officer, and she recognized his breakthrough work in optogenetics as the vehicle that could complete her mission. They started working together in 2013 and they founded Nanoscope in 2017. It’s their tireless dedication and commitment that have fueled our progress over the last 10 plus years and placing us on the verge of these important milestones that I talked about and something very big in 2026.
Mark Dlugoss:
As you’re talking about your executive and leadership teams at Nanoscope, they appear to be committed to developing therapies for patients obviously blinded by complex retina diseases. Can you highlight some of the key members of Nanoscope Therapeutics’ team and what their experience bring to the company?
Sam Barone, MD:
I mentioned Samara and Sulagna. Beyond that, they have officially built expertise within the organization that’s rooted on our executive team. I’m the chief medical officer, and I’ve been in this role for almost 3 years. I’ve actually been involved with the company since 2017. I was an advisor who helped design the first-in-human phase 1/2a study.
I’m a board-certified ophthalmologist and retina specialist; and I’m really motivated by our cutting-edge science and, again, the impact we’re making on patients to give them back vision. I also spent almost 5 years at the FDA reviewing cell and gene therapy technologies at the Center for Biologics in the very office where our Nanoscope submissions are residing. This experience is particularly applicable with the BLA review that’s upcoming.
The rest of our executive team is well positioned for success and execution of our upcoming milestones. We’re particularly looking forward toward commercialization and launch. Our executive vice president of value and access, Ramesh Arunji, he’s had similar roles with other successful launches in gene therapy outside the eye, namely Zolgensma for spinal muscular atrophy and Vyjuvek for dystrophic epidermolysis bullosa.
We’re also very happy to announce, a couple of weeks ago, that industry veteran Paul Hallen, who spent almost 30 years at Alcon and most recently as the global head of retina. He has joined as our chief operating officer to lead global operations, including commercial readiness, supply chain, and cross-functional execution. Paul’s really great addition and really rounds out the collective expertise and our ability to address the high unmet need of vision loss from retinal degenerations.
Mark Dlugoss:
Before we divulge into Nanoscope Therapeutics’ lead technology, which is the multi-characteristic opsin platform, or commonly called MCO, we should probably explain with optogenetic technology, what’s the science behind it, the technology? How is it different from traditional gene therapies and basically its role in the development of MCO?
Sam Barone, MD:
It’s really a great technology. Light response in nature exists at the cellular level, essentially through light-sensitive things like ion channels, pumps, or enzymes. You can have everything from phototaxis in a single-celled organism that moves away or toward a light signal. To the mammalian retina, where the vision, the exposure to the light—it triggers a biochemical signaling pathway, and it’s usually through a conformational change of a specialized protein that’s sensitive to light.
Optogenetics specifically is the biological technique that harnesses that ability, allowing for the characterization and manipulation of the activity of neurons or other cell types with light. The applications for optogenetics are broad. While we’re just using it to the potential to restore vision, there’s a lot of research ongoing for its use in things like Parkinson’s disease or other neurologic and psychiatric disorders like autism, schizophrenia, drug abuse, anxiety, and depression.
In 2010, optogenetics was chosen as the method of the year across all fields of science and engineering by the interdisciplinary research journal Nature Methods, and it’s also highlighted in the journal Science as one of the breakthroughs of the decade. When we talk about optogenetics and what we’re doing, we are employing it as an intravitreal delivery of an adeno-associated viral vector, carrying a transgene for the expression of an opsin protein.
In retinal degenerations where the vision is lost because of photoreceptor damage and death, opsin expression in the inner retina cells that remain intact even late in the disease allows these cells to function as defacto photoreceptors and allow for the restoration of vision. Now, there’s 2 main advantages of optogenetics in comparison with other gene therapies like you’d asked about traditional gene therapy, and 1 is the ability to restore vision despite photoreceptor loss.
Traditional gene therapy or other therapies offering neuroprotection, they’re best suited for the patients who may have had a diagnosis but still have photoreceptors in place and very usable vision. These therapies can only prevent further degeneration and vision loss, but are going to be of minimal impact in advanced disease. This is in contrast to optogenetics, which can actually improve vision by replacing the function of the photoreceptors.
A second feature that distinguishes optogenetic treatment from traditional gene replacement therapy is the fact that optogenetics is mutation-agnostic. This is important in a disease like RP where there are over 100 genes that have been implicated as causal. Spark’s LUXTURNA, for example, is only effective with 1 of these mutations, individuals with mutations in the RPE65 gene. With optogenetics, it does not matter what the cause of mutation is.
We are bypassing the retinal damage by targeting the initiation of the visual signals downstream. In fact, because of this mechanism of action, it doesn’t even have to be an inherited retinal degeneration caused by a genetic mutation. Optogenetics can truly be disease agnostic as a potential treatment in any setting of decreased vision from photoreceptor loss. It really has the potential to help a lot of patients.
Mark Dlugoss:
Nanoscope Therapeutics’ core technology is obviously the multi-characteristic opsin or MCO. Let’s discuss MCO’s technology platform, specifically as MCO-010. Can you elaborate on this platform? How does optogenetics interact with what you’re trying to develop and the kinetics of the opsin used, how they reprogram cells, just to get a better understanding of the science behind it?
Sam Barone, MD:
The key in optogenetics is that light-sensitive opsin protein. Our opsin protein is a bioengineered fusion protein, it has 3 different light-sensitive elements, and we call it a multi-characteristic opsin or MCO. It comprises primarily a transmembrane ion channel, and that undergoes a conformational change when exposed to light that opens up the ion channel, dipole rises the bipolar cell in our case, and causes the initiation of the action potential there in the bipolar cell instead of the degenerated photoreceptor.
This action potential then is transmitted through normal visual pathways to the brain for the perception of vision. It’s important, not all opsins are the same. The different characteristics of the various proteins are really what differentiates all program in optogenetics, including those that have been tried before and failed. Now, there’s 3 different elements of our MCO, as I mentioned, and they synergistically worked together for better function than any single element or naturally occurring opsin could provide by itself.
In this way, MCO is differentiated and truly designed to optimize vision in the optogenetics setting. For example, opsin proteins are usually only maximal responsive to a single wavelength band of light, and they respond less or maybe not at all as the wavelength is further away on the electromagnetic spectrum. For example, an opsin sensitive to blue light would have difficulty with red light.
Not just the recognition of the color, but the opsin might not even be turned on by a red light. However, each of the 3 elements in the MCO is sensitive to a different wavelength band of light and works in unison to give sensitivity across the visible light spectrum. A second important characteristic that differentiates opsin protein is the strength of the light that’s required to stimulate the protein.
Many opsin are not sensitive to normal ambient light and require a goggle or some kind of external device to amplify the brightness of the light getting to the back of the eye. Not only would a device like this be cumbersome for patients and activities of daily living, but the amount of light required in these settings can also be toxic to the retina. In contrast, MCO is sensitive to ambient light levels without the need for additional amplification.
Interestingly, light sensitivity in low light, it usually goes with the slow kinetics or vice versa. Opsins with rapid kinetics require higher light intensities. However, the coordination across the 3 different elements of MCO allows for quick, both on and off response time, like less than 2 milliseconds, in the setting of low light sensitivity levels.
Functionally, this minimizes the blur that an individual might experience with fast moving objects going past them. Then the final characteristic of the MCO program that allows it to provide optimal vision and optogenetics is through the targeting of bipolar cells, in contrast to retinal ganglion cells with a lot of programs dupe target. This is because our proprietary vector and the inclusion of a promoter enhancer sequence that is specific for on bipolar cells.
In fact, we’ve demonstrated that we’re able to achieve greater than 70% transduction of the on bipolar cells in multiple animal models, and this leads to better vision potential than when targeting the retina ganglion cells really for 2 main reasons. One is that there’s approximately 10 times the number of bipolar cells than retinal ganglion cells in the retina. There’s 10 million versus 1 million.
There are simply more pixels in a picture that results from bipolar cell stimulation. Two, the bipolar cells are the cells that are right next to their photoreceptors and are upstream of the retinal ganglion cells. Stimulation at the level of the bipolar cells allows for retained retinal circuitry and signaling across, for example, like the amacrine and the horizontal cells. It’s more similar to when the photoreceptors initiate the visual signal.
Again, our MCO platform was designed for optimized vision with maximal functionality across these 4 characteristics: stimulation across the visible light spectrum, sensitivity at ambient light levels without the need for external amplification, rapid kinetics, and the targeting of bipolar cells. These truly differentiated from the other programs, again, allows to be set up from optimal vision in optogenetics.
Mark Dlugoss:
Let’s move on to the clinical application of MCO-010. I know it’s targeted toward RP and the Stargardt disease, but is there a specific patient profile you’re looking to target with MCO-010?
Sam Barone, MD:
As I mentioned before, there’s other strategies in development for retinal degenerations, and they target patients who still have usable vision because again, they can only prevent further degeneration and loss, but optogenetics can address that unmet need of severe vision loss in these patients with advanced retinal degenerations. We want to make a difference where other strategies are unable to help and are looking toward a patient population with legal blindness, 20/200 or worse.
In our RESTORE randomized, controlled phase 2b/3 study in RP, more than half of the patients had even more advanced disease with vision that was hand motion or worse, and some were barely able to perceive light. While all of these patients did not have measurable improvements specifically in visual acuity, they showed other improvements in functional assays that we utilize, and it’s still indicating a benefit from MCO-010. It really seems feasible that MCO-010 can be considered in advanced degenerations as long as the bipolar cells and inner retina are intact.
Mark Dlugoss:
Is there any specific restrictions to the kind of patients that can be treated? What I mean by this, maybe patients who have undergone prior gene therapies or maybe severe cases of ophthalmic diseases that would prevent them from being treated with MCO-010.
Sam Barone, MD:
Of course, in a clinical trial, there’s a lot of restrictions that we implement in an effort to minimize confounding variables. We look forward to the labeling discussions with regulators, but I don’t envision that the label is going to be nearly as restrictive. When you talk about other diseases, other ophthalmic diseases, it’s going to be important that the inner retina is intact.
Unfortunately, patients who have vision loss because of optic nerve disease like glaucoma or an optic nerve degeneration, those patients are not going to be able to benefit from the current iterations of optogenetics and MCO-010. Prior gene therapies, they’re an interesting consideration. Generally, repeat administration or dosing with another gene therapy, it’s not occurred because of the immune response to the vector.
Essentially, the patient is being administered a vaccine against that vector. What’s interesting is that most people have had prior natural exposure to the adeno-associated virus, and that’s the vector that we use and it’s commonly used in ocular gene therapies. To get a sense of baseline immunity and its potential impact, we actually looked at our patients’ baseline neutralizing antibody levels, but we did not make that part of inclusion and exclusion criteria.
We did not see any correlation between baseline neutralizing antibody level and safety or efficacy outcomes. This suggests that repeat dosing or dosing with another gene therapy may be an option. I think more work needs to be done in the field, but having received a prior gene therapy, it may not be an absolute contraindication.
Mark Dlugoss:
Does MCO-010, from the patient’s perspective, does it require genetic testing? Let’s talk about other things from the patient’s perspective. How is it administered? What’s the duration of the treatment effect, and what is the sustainability of the visual improvements that will occur?
Sam Barone, MD:
As you can imagine, we’re on the verge of completing our BLA submission. We’ve done market analysis and preparation for launch, and we’re really excited about the feedback. Greater than 90% of the healthcare providers indicate an intent to utilize MCO-010. A lot of this has to do with the patient journey that you’re referring to. It’s because it’s much more similar to a biologic like an anti-VEGF rather than other gene therapies like Spark inside the eye or even ones outside the eye.
MCO-010 is delivered by an intravitreal injection. It’s the most common procedure in all of medicine, and it’s done right in the office. There’s no surgery that’s required or time-consuming infusion. Because of the disease agnostic mechanism of action, genetic testing is not necessary. A clinical diagnosis should be sufficient. This is actually supported by our clinical trial data where we looked at genotype, but again, we did not make it part of the inclusion and exclusion criteria.
We saw no correlation with gene mutation and outcomes, including those in whom a cause of mutation was not even identified, which is about 33% of patients with retinitis pigmentosa. In terms of durability, we are transducing the bipolar cells and they’re terminally differentiated and no longer dividing. We expect the protein to continue to be expressed for a long time. When we translate our mouse studies, we can see a sustained benefit for 20 years in humans to be possible.
We continue to follow the patient in long-term follow-up and are very happy that we see vision improvements from baseline that are equivalent of 3 ETDRS lines. We’re seeing this to 3 years and counting. In contrast to the natural history of continued vision loss and RP where you expect to see at least another line of vision loss at 3 years, this level of gain is really impactful to patients.
They’ve reported that stopping or even just slowing the progression of the disease is meaningful to them, let alone actually improving the vision. It’s really set up for a balance to be in the benefit for patients and the providers too to be able to deliver it easily in their office.
Mark Dlugoss:
Is this a 1-time dosage?
Sam Barone, MD:
Yup. It’s a single intravitreal administration.
Mark Dlugoss:
What’s the safety and tolerability profiles associated with MCO-010?
Sam Barone, MD:
Safety continues to be a pillar. We’ve had no ocular SAEs in well over 60 patient years of follow-up. Anterior chamber inflammation has been the most common adverse event, but it’s been generally mild or moderate and controlled with topical steroids. It’s interesting, there’s a lot that’s been learned as the field of ocular gene therapy continues to develop. There’s a lot that’s been learned about control and prophylaxis for gene therapy associated inflammation following intravitreal administration.
There’s large programs like ones by Adverum and 4DMT and wet AMD who’ve dosed hundreds of patients. Our studies actually had been dosed before there was this sort of better understanding. Our patients were actually underdosed in terms of the duration in comparison to what now seems to be considered the standard of care for steroid prophylaxis regimen. Even though the benefit risk profile was very much in favor for our patients because there is such a high unmet need, I think there’s even the potential for a cleaner safety sheet moving forward.
We’ve additionally not seen any evidence of retinal toxicity clinically or in preclinical studies. One thing that’s important when we consider things like our Stargardt program where there are peripheral areas of intact photoreceptors, but the atrophy is limited to the central macula, there’s been no reports or any visual disturbance or permanent photopsias related to the bipolar cell transduction in these areas of more intact retina. One other thing that comes up too in terms of safety is kind of the immunogenicity.
I commented on the baseline neutralizing antibodies. Antibody levels that we evaluated, they were not correlated with safety or outcomes. Of course, we’ve continued to follow those antibody levels over the course of the study too, and we saw increases in those neutralizing antibody levels following dosing, but they decreased over time. There were also no correlations with level of increased safety or efficacy outcomes. Safety continues to be a pillar of the program and considering the risk benefit in these patients, again, very much in their favor.
Mark Dlugoss:
Let’s talk about the clinical trials that have been conducted so far for MCO-010. Let’s start with the RESTORE study, which was a phase 2 for RP, and also the STARLIGHT study, which also was a phase 2 trial for Stargardt disease. Can you provide the clinical results and the primary efficacy endpoints of these 2 trials and what insights did these results confirm to the company moving forward?
Sam Barone, MD:
RESTORE was our randomized, controlled phase 2b. We called it phase 2b/3 because it’s what’s being used to support registration. This trial randomized 27 individuals to 1 of 2 MCO-010 dose groups or to a sham control. Both MCO-010 dose groups had best-corrected visual acuity gains greater than 0.3 LogMAR, which is the equivalent of 3 lines or 15 letters on a standard ETDRSI chart, at the 52-week primary endpoint. This was statistically significant compared to the sham control.
Importantly, greater than 40% of the dosed patients reached this 3-line improvement threshold. As you know, that’s the threshold that’s been used to support approval in multiple indications in ophthalmology like in wet AMD and diabetic retinopathy. These improvements have been durable, not only to the week 76 key secondary endpoint, but also in long-term follow-up to 3 years and counting, as I mentioned, and where we continue to see that approximately 3-line improvement.
These data in RESTORE, they’re the basis for our marketing applications, first in the US FDA and with the Japanese PMDA. It allow us to be full throttle moving forward towards approval and commercialization in terms of an insight from RESTORE.
STARLIGHT was a phase 2b, open-label study of MCO-010 in patients with Stargardt disease. In STARLIGHT, MCO-010 continued to be safe. We saw vision improvement across multiple parameters, particularly in patients whose disease was limited to the macula.
The results from STARLIGHT support continued development, and we have alignment with the FDA on a single randomized, controlled phase 3 trial design, which we’ll be initiating this year. In terms of insights, STARLIGHT also provide proof of concept and read through when we think about vision improvement and macular atrophy. This really supports moving directly into a phase 3 randomized, controlled trial in geographic atrophy with an actual vision endpoint, not just looking to slow the progression of geographic atrophy, but actually looking to improve visual acuity.
Mark Dlugoss:
The success of both RESTORE and STARLIGHT studies have allowed Nanoscope Therapeutics to begin follow-up trials. The follow-up trials were REMAIN study for RP and the SUSTAIN study is for Stargardt disease. What’s the status of these studies and when does Nanoscope expect to have data to report?
Sam Barone, MD:
Yes, you’re right. REMAIN and SUSTAIN, they’re the long-term follow-up studies of the patients who are dosed and RESTORE and STARLIGHT respectively. They continue ongoing. Fortunately, there have been no surprises in data, and we look forward to providing updates at upcoming scientific conferences as soon as this fall.
Mark Dlugoss:
Phase 3 has started for Stargardt disease is planned for 2026, as you mentioned. Can you shine some light on Nanoscope’s plans for the trial? Can you talk about it?
Sam Barone, MD:
Again, the STARLIGHT results, they support advancing to phase 3, and we have alignment with the FDA on a single registrational trial design. We plan to randomize 60 patients 1-to-1 to MCO-010 versus sham control with a week 52 BCVA primary endpoint. In contrast to other programs currently in development in Stargardt disease, which are looking to slow the progression, the rate of advancement of the atrophy.
One important thing to note is in this study, we’ll actually be using the commercial dose and the drug in the phase 3 Stargardt study. That’s important because that means that no bridging work will be needed when we get the results at the conclusion of the study, and it will facilitate potential approval in market entry in Stargardt disease as well.
Mark Dlugoss:
Nanoscope Therapeutics’ active pipeline is currently focusing on MCO-010. However, you are developing another platform called MCO-020, which also uses optogenetics to address geographic atrophy. Can you discuss details of MCO-020 platform and where it stands in the clinical process?
Sam Barone, MD:
MCO-020 is really a cool technology. It’s actually our next-generation platform, and it uses non-viral vector delivery. Instead of using the viral capsid shell, the plasmids are injected directly into the vitreous. There are some advantages with using non-viral vector delivery like manufacturing, supply chain handling, and a lower immunogenicity. The challenge when you step away from the vector is: how do you get that genetic material into the cells?
The pathway or the means that we are employing is a use of a low power near infrared laser, and that allows us to stimulate the cells and allow the uptake of the plasmid. This laser, it’s OCT guided, so that really offers the potential for very exquisite localization, and it makes it particularly conducive for geographic atrophy. This is a pipeline program. It’s undergoing some continued IND-enabling work, particularly while we focus on the commercialization of the vector programs for inherited retinal degenerations.
Mark Dlugoss:
Nanoscope Therapeutics is obviously committed to the optogenetic therapy for restoring vision. What future applications is the company possibly exploring to advance that platform in ophthalmology?
Sam Barone, MD:
As I mentioned before, it’s disease-agnostic. There are almost endless possibilities where you have photoreceptor degeneration and loss and intact inner retina where it could be applicable. We’ve done some exploratory work with Leber congenital amaurosis, and that one actually supports direct to phase 2 considerations as well. When we talk about some of these other pipeline or potential therapies, we’re a bit bandwidth constrained right now as we maintain our focus on commercial execution in RP and beyond.
A trial in RP may not even be necessary because we continue to get traction with regulatory authorities for a broad retinal degeneration claims consistent with the disease-agnostic mechanism of action. Some of them, and some of the way the discussions are going, it might not require another specific study for RP, another study for Stargardt disease, another study for Leber congenital amaurosis.
With the data and the body of data that we are collecting, it’s gaining traction where regulatory authorities are starting to get their heads around, this could be a potential therapy for any inherited retinal degeneration where you have photoreceptor and RPE loss, and really to be able to get access into and be able to help many of those patients with broad spectrum disease.
Mark Dlugoss:
Genetic cell therapy can open a door to so many, not only in ophthalmology, but everywhere, but there’s so many diseases in ophthalmology. You could do wonders once you get a handle of everything that cell therapy can offer.
Sam Barone, MD:
RP is really like final common pathway phenotypically. There’s a lot of different…As I said, over 100 different mutations. They ultimately lead to photoreceptor loss, but there’s different ways that the photoreceptors die off. With a technology like ours, it may be able to be broadly applicable regardless of the underlying mechanism of photoreceptor loss.
Mark Dlugoss:
What we’ve discussed today is amazing. It sounds like the folks at Nanoscope Therapeutics are doing incredible research and developing optogenetic therapies and the MCO-010. It looks like Nanoscope appears to be on track with this technology to revolutionize retinal disease management. Are there any other points of discussion we may have overlooked today, or is there something you would like to add about Nanoscope Therapeutics or MCO-010 before we close?
Sam Barone, MD:
Thanks, Mark. With years of experience and the programs that you’ve seen come and go, it’s truly an honor for you to use the term revolutionized to describe what we’re doing. That’s how we see it too. Patients are getting meaningful restoration of vision from previously irreversible photoreceptor loss as has never been done before. This is with a single intravitreal injection that can be done right in the office setting.
We have patients telling us that it has been 15 or 20 years since they’ve seen the way they are seeing after MCO-010. They’re again able to do things like navigate an unfamiliar environment with confidence and see loved one’s faces. These are very important activities for patients with severe vision loss who have in many ways given up hope. The science is fascinating, but it’s not without nuance.
I really appreciate your interest in what we’re doing and allowing me the opportunity to describe it and try to educate your audience. It’s a very exciting program to work on. We look forward to some very meaningful milestones this year toward getting MCO-010 into the hands of providers and the eyes of patients. Thank you again for the opportunity.
Mark Dlugoss:
Can’t wait to see when MCO-010 is approved by the FDA and see what happens then. Like I said before, gene therapy is going to revolutionize everything. It’ll be really fascinating to see where the evolution of this technology goes.
Sam Barone, MD:
Appreciate your support.
Mark Dlugoss:
That concludes today’s edition of The Ophthalmic Project. I want to thank Dr. Barone for outlining Nanoscope Therapeutics’ work with optogenetic technology and its development of the MCO-010 platform. I also want to thank you, the listeners, for tuning in, and I hope you’ll join us for the next edition of The Ophthalmic Project, powered by Ophthalmology 360. Until next time, have a great day.
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