Ocugen, Inc. (OCGN) Earnings Call Transcript & Summary

Hello, and a warm welcome to this edition of the Scrip podcast. I am Vibha Ravi, sub-editor with Scrip and Pink Sheet. And I'm having a conversation today with Mr. Shankar Musunuri, who is Co-founder, Chairman and CEO of Ocugen, Inc. Most of our listeners will likely know Ocugen on account of its tie-up with Bharat Biotech for the COVID vaccine [ COVAXIN ], but Mr. Musunuri's company is working on a gene therapy platform that we will talk about more during the course of this podcast. First, some bit about the man himself. Mr. Musunuri, who has a PhD and a business management degree, is a seasoned biotech veteran with over 25 years of experience advancing and commercializing a diverse portfolio of products. Prior to cofounding Ocugen in 2013, he held leadership roles at several companies ranging from big pharma to start-ups. Dr. Musunuri spent nearly 15 years at Pfizer, where he gained extensive product launch and life cycle management experience playing a key role as global operations team leader for the vastly successful launch of Prevnar 13. Prior to the Pfizer stint, he was a group leader focused on clinical development at Amylin Pharmaceuticals. He is a recipient of the distinguished alumnus award from the University of Connecticut School of Pharmacy and serves on the advisory board of Fuqua's Center for Entrepreneurship and Innovation at Duke University. Apart from Ocugen, he has also founded Nuron Biotech, where he serves as President and CEO. So Dr. Musunuri, welcome to this podcast.

Thank you, Vibha. Thank you for having me today.

So being an entrepreneur, what will be the top 3 pieces of advice you would give to an aspiring start-up?

One needs to have a clear vision where they want to go. That's extremely important. The second thing is don't reinvent the wheel. You really need to have a sounding board off other entrepreneurs and so that you don't repeat the mistakes. At least minimize the mistakes. As an entrepreneur, you will make mistakes. You will fall. You need to move on. That's the second advice. Third one I would say is never give up. Entrepreneurial journey is very hard, very complex. At times, when I left a nice job at a large pharma, became an entrepreneur -- people around you -- I mean it's tough. They see you are doing extremely well. Then you are struggling sometimes, moving up and down. And so that's something you have to learn. You need to be very resilient. You can never give up, and you will be successful. As long as you have a clear vision, you focus on it. And so that means you have a vision. You have a goal [ of ] targeting that. That's what entrepreneurs do. They never give up. That's very important. So those are the 3 things I would say: clear vision. Never give up, and don't reinvent the wheel. Always have a sounding board off other entrepreneurs. They have been there, done that. Learn from them.

That's really interesting because this is the same thing that Mr. Robert Langer, who we all know is a great entrepreneur and innovator, said to us recently at a conference. So yes, that's a good one. Now what is the most memorable and forgettable part of your entrepreneurship journey?

I mean, the forgettable part, I'll come to that part. I mean it's not easy to forget things, but I would say the new company goes through ups and downs. When you are on the downside -- we ended up, a few years ago, laying off some employees at Ocugen. We had a result in the clinical trial we didn't anticipate, and so as a company to survive, we had to lay off some employees. That was very hard for me. That's first time in my life I ever did that as an entrepreneur, and it's hard. I mean people only see, when the companies are growing and the growth mode, how many people they hire. That's tough and it stays with you. And how do you go from there to a journey, most exciting journey? The last year, the company, we were in gene therapies and we [ adopted ] into getting into vaccines because our leadership has a deep history in vaccine development. And we could not sit on the wayside, jumping into this war of COVID and fighting it. How do we contribute? We have prepared our soldiers, right? We all have a lot of experience, [ network ]. And we thought we cannot sit aside -- and jumping into it and are really trying to contribute to that. I think that whole dynamic -- I think, if it -- and in fact, we had a year-end get-together, all the employees. And every one of those employees in my company, I could probably state they really care about patients. That's the moment I can never forget. I mean, every management team member in my company, when you look at Ocugen, they all focus on patients. Just like Steve Jobs -- they focus on the customer. We focus on our customers' patients. This is a byproduct. I think that's the culture we're instilling and we all believe in it. We -- and every employee who comes into the company, they are so passionate about the therapies we are working on, either vaccines to -- for the public health initiative to save lives; or gene therapies we can have significant impact on many, many, millions of lives globally. We focus on the patients. That's very important. That's the memorable moment. Yesterday, that get-together, when I talked to people around -- going around and talking to employees -- and the sense of pride that they have that they work for Ocugen is something you don't earn in a day. It takes years of effort, and when you see that, I mean, it's just a matter of time. It's not about commercial success. It's about really instilling and hiring good people. And also they have the same culture, same accountability, same -- the positive attitude. We really have to do something for the patients. That's important. That can take us a long way. And that's a memorable moment for me, yesterday, when I had a great get-together with all our employees before holidays. They have been working extremely hard. During COVID times, when some of the people were sitting in places -- they don't even want to come out when the COVID started, and we could not stop. Our employees continued to work throughout the COVID. Even today, they continue to work. Omicron is not going to stop our work, or any other variants. That's a memorable moment for me as an entrepreneur.

Great. So I agree that people do make a company and its culture, but as you said, every entrepreneur has its worries and motivations, so what keeps you up at night? And what energizes you in the morning?

I mean every -- I mean, this whole COVID -- COVAXIN, for example, it's not easy, right? I mean we started the journey. We have great intentions and we continue to do that. Our goal is to contribute to this public health initiative and save lives, and we're going to get there. There are bumps on the road. We are not the first ones, all right? We have 3 other vaccines which are -- had emergency use authorization. 1 already has approval in the U.S. So that's a tougher road, but that's what keeps you up. How do you navigate? How do you make this, from a public health perspective, working with the government, working with agencies; and take this to the next level so that this can be available as an option? That's what keeps me up. How do we get there? How fast can we get there? When you see the -- Omicron, some other variants -- tomorrow, it could be some other variant coming up. We do know how valuable this vaccine could be in saving lives. And however, what energizes me is my employees. When you go there -- everybody has such a positive energy. And I look forward. Every day, I get up. It's a new day, [ number one ]. And they -- my employees and my colleagues, they energize me. And what we are doing together, that's what energizes me.

Right. So obviously this partnership with Bharat Biotech counts for a lot. So given that there was a recent hold on the clinical trials for BBV152 or better known as COVAXIN, what is the likelihood and anticipated time line for FDA approval? You applied for emergency use in children also, so what would -- what do you see as the likelihood and anticipated time line for use in adults and children?

For the adults, as we stated before, we wanted to follow for the biological licensing application because this pandemic is going to stay for, we believe, several years. It's not going to end, so therefore we wanted to make sure we have a longer outlook. How do we have a product approved, not just emergency use, biological license application? And what are the studies we need to do in the U.S. demographic so that we meet those requirements since most of the data is coming from abroad? So one of the requirements is doing a bridging trial which does immunobridging to large safety and efficacy trials our partners have done in India, which is about 26,000 patients. So this immunobridge will allow you to state this is [ equivalent ]. And if -- any other additional studies we need to do for safety in the U.S. demographic, we'll be happy to establish that. We did provide a significant amount of data coming from our partners. And also, safety surveillance data after 35 million doses administered from the government of India system, just like our CDC system here, all that data was provided. So that's the clinical trial we are trying to do in order to meet the requirements with the BLA sometime next year, and that trial is the one which is on hold. Typically when FDA has the clinical hold, they have up to 30 days to provide questions, any clarifications they need on our filing; and so we are anticipating, hopefully, they'll respond to us this month. And as soon as they respond -- our team is -- standby. And we're going to work as soon as possible and provide those responses so that we can move on the clinical trial. We do have a large CRO and -- lined up. We got everything ready to go. We have the product. We have everything ready to go, the clinical sites. Everything is established. As soon as we get a green light, we are going to initiate those immunobridging trials in the U.S. population. The second one, question you asked, is about emergency use authorization in pediatric population. Emergency use authorization is based on unmet medical need. And FDA has a lot of leverage based on the unmet medical need from the public health perspective, leverage that and see when they can issue U.S. emergency use authorizations. So in case of pediatric population, when we received data from our partner from India, they already filed for emergency use in India. [ The equivalent ] of advisory committee, SEC, in India has already approved it. They're everything -- [ the equivalent ] of a [indiscernible] approval for 2 to 18 pediatric population. And the data, when we looked at it, it's compelling. First of all, it showed [ equivalent bridge ] to large adult trial they did, immunobridging. And the clinical trial also has compelling safety data, including 100% prevention of hospitalization. None of these kids in the trial got hospitalized [indiscernible]. And also the trial showed solid safety data, especially related to severe adverse events such as myocarditis or thrombosis or known [ as blood clots ]. Many of the parents in the U.S., they are looking for choices. If you provide choices to people, it will automatically improve the vaccination rates. And so when it comes to children, there are other outside surveys people have done, independent surveys. And they do support having choices for kids in this country. So one is unmet medical need, which is 2-to-5 age group in the U.S. Today, there are no approved or authorized vaccines. From 5 to 18, we do have Pfizer vaccine which covers. So if you take that entire range of 2 to 18, obviously there is a need. There is a need, unmet medical need, from 2 to 5. We are the only ones, first one, to file for that age group. And 2 to 18 group also provide -- having a vaccine such as ours which has a broad immune response beyond spike because we have -- it's a whole virus-based vaccine and gets support from other antibodies, which could be beneficial for current variants or future emerging variants. And a solid safety data with no severe adverse events such as myocarditis or blood clots could be beneficial option for parents. So that's the reason we applied for EUA. Currently it's undergoing review with FDA.

Right. So staying on the topic: As you said, Pfizer is already approved for the 5-to-18 age group. And given the nature of the trials, pediatric trials, it's a step down to the various age groups, so do you think, by the time you get a go-ahead for the clinical trial, Pfizer would have also got approval for that age group which is the unmet need you're talking about right now, which is the 2- to 5-year old?

Pfizer has not filed 2 to 5 yet. They may file in the future. Currently -- so the clinical trial is for adult. That's a trial which we're going to bridge for biological licensing application in the future. But emergency use authorization is already under review with FDA. So again, if Pfizer files it tomorrow, we are hoping FDA is going to treat us fairly as other companies and give us an opportunity to get a fair review, including advisory commentary review, so that they can make a decision.

Okay. And so when this clinical whole was -- I mean, hold, was placed, did FDA communicate at the time anything to you about the reason why a hold was placed?

At the time of clinical hold, we didn't get any specific questions. I mean they have up to 30 days to give us questions, and that's what we're waiting.

Okay, fair enough. So now does Omicron complicate the equation for you in terms of the BLA license? Would you be -- would you have to conduct additional tests to see if it works against the variant? And what kind of a number of volunteers are you looking at to conduct this trial for the BLA?

So the first question is Omicron. As other companies are doing, we will have the data on Omicron too, yes. And again this is not a clinical trial. Everybody is doing a -- neutralization assays. And it's important to do whole-virus neutralization assays in the labs, not with a [ pseudovirus ], to get meaningful results, so we're going to wait for that. However, what we have seen, the other variants, we have shown with COVAXIN a good neutralization effect. I mean I think even with the Delta we were the only company which showed through our partner's data in controlled clinical trials -- when they were doing clinical trials in India, about 90% were variants. So when the vaccine trials were done, for others which are here in the U.S. right now in the market, at that time, the variants were not emerging, right? It's original strains. So compared to that, in India, 90% were variants when they were doing the Phase III clinical trial. Within that, they showed prevention of hospitalization is 93.4%. That's significant. That's what people are looking at today with Omicron. People are stating, "Oh, if you can prevent hospitalization even 70%, that's good." And so people are looking at how do you prevent hospitalizations, right? And that's very important. And in the middle of all those variants, they showed overall efficacy of 78%. And they specifically did the map to find out which of those subjects had specifically Delta variant. And majority of them had Delta variant, and they showed 65% efficacy from a controlled clinical trial. That's real data. So the data you are getting from surveillance studies and all, they don't have control. So when you do a clinical trial with -- a controlled clinical trial, that data is solid. And this is the only vaccine which showed solid data from a controlled clinical trial on Delta variant; and also showed prevention of hospitalization, a very high percentage, when they had 90% of the cases were variants. That's important to note. So that's the first part, of Omicron. So as other companies [ have stated ], when we get the data, it'll be known to the market how it does, but however, when they also did neutralization using other variants -- they had Delta prior to that, but there were other variants. They always showed very high neutralization with this vaccine because the -- how this vaccine is made. The problem with this variant and current spread and emerging variants is the virus is mutating in the spike part. And all 3 vaccines which are here in the U.S. today, they're all based on the spike-based protein. They target the spike, and that's the part which is mutating. So if you made a vaccine based on the original structure -- and now even in the RBD, they have about 10 mutations, so scientifically you tend to believe your efficacy is supposed to go down. That's exactly what they're observing. However, our vaccine is built similar to polio vaccine, right, which eradicated polio with a whole inactivated virus, with an adjuvant that boosts the immune response which actually was co-developed by [ MIH ] in the U.S. and [ which was ] licensed back to our partners Bharat Biotech. Because of those key elements, this inactivated [ virus ] vaccine is different than a couple of inactivated vaccines in China. This has that additional adjuvant which boosts the immune response. In fact, this week, we gave a press release based on the memory data, the B cells and T cells, which are extremely important for the protection. First of all, that publication [ pre print showed ], up to 6 months when they measure, they still have antibodies which are broad antibodies, not just spike. They also have antibodies for the [ conserved ] portion nucleoprotein. And that's the part which potentially doesn't mutate. So when some part is mutating and you have a vaccine which will -- just on that, scientifically you are going to reduce the efficacy. Eventually, you will create a viral escape. However, if you have a broad-based vaccine such as ours, you are producing antibodies again as like a nucleic protein and other parts. And scientifically it has to neutralize it. And that's exactly what the studies actually observed in the past when they did neutralization assays. They fared much better compared to spike-based vaccines, so we are hoping, based on how it's built, this vaccine should show good -- potentially it should work on Omicron and future variants. So that's [ the crux ] of that. And as soon as we get the data, it will be known to the market.

Right...

And so that's the -- I think you are talking that part. And you are also asking about clinical trial, right, the second question. Could you repeat?

Yes. I was asking, what are the numbers you are looking at for the bridging study?

Yes. The bridging study, typically, Vibha, you don't need a large trial. You're talking about a few hundred patients. And the good thing about this study is it's very difficult to get [ new ] patients, as you know, at this stage in the U.S. I mean even though there are a significant portion of Americans who are not vaccinated. However, our study also allows people who got vaccinated with mRNA vaccines. However, they have to be vaccinated 6 months or earlier. That means those subjects will also be enrolled into the study. So hopefully, what this study will give us in this -- in a few-hundred-patient study, will not only bridge the immunobridge with large efficacy and safety trial we did our partners [ have connected ] in India successfully. It will also allow us to learn about some of the subjects with the other vaccines in the U.S. And this is almost like giving them a booster. I mean how -- this booster and broadening the response in these patients, not just giving you [ another spike ] booster, right? You are giving an inactivated virus vaccine, which should broaden [ your ] response. That means you will have antibodies beyond spike, like nucleoprotein, and other antigens [ against them ]. So that should really help and protect because you always want to think about vaccinating and giving boosters to subjects in the middle of the pandemic. Number one, the durability, how long it's going to work. Did you create a memory? I just mentioned about that memory paper. And at 6 months, they showed good T cells and B cell. That means it's -- the memory is going to last a lot longer. It should be durable. Number three, it should also have a broad immune response. That means not only getting worried about the current variant or the future variants. If that -- one part is changing, you have other parts of the virus you are developing antibodies. You have ability to neutralize them. That's important. That's -- so you really have to have multiple vaccines in the tool kits to do different things, and then you need to come up with a solution. How do you control the pandemic? That's very important. People have to really take a step back. It's not about having billions of doses of one type of vaccine. Unfortunately -- if it is that simple, we could have controlled the pandemic by now. It's not that simple. Number two, you need to vaccinate majority of the folks globally. It's not like a few countries. Just vaccinating in a few countries and giving boosters to them and -- this is never going to stop. This pandemic is going to continue to spread. So all the developed countries -- U.S. is pitching a lot. And I'm hoping they'll continue to increase their global vaccine diplomacy and get the vaccines and give it to the global population to control this pandemic. So there are multiple ways we can help out in order to get a -- inroads into the U.S. We are happy to work with our government, not only for U.S. vaccination in this country. We want to contribute to the global vaccinations.

Right. So on that note, could you tell us, what's the status of the approval in Canada?

The approval in Canada. This is -- when we filed for approval in Canada, they had something called interim order, which is [ equivalent ] of our emergency use authorization. That interim order was -- expired. I mean, when we filed it, we were just on the tail end of it. They accepted our application. They did tell us, "Hey. This order is going to end potentially. [ Then we'll roll it ] into new drug submission," so this is almost like a BLA in the U.S. So that's going through rigorous review process. And as -- in between the review, we do get questions, sometimes deficiencies, and we are working with Health Canada to respond to them, yes. So that process is ongoing, so I mean at this stage I cannot comment on specific time line when the approval is going to come. I mean our goal is -- when any regulators review our file, when they have questions, our goal is to respond to them as soon as possible in a very efficient manner so that we can continue to move the clock and make sure they get all their questions addressed so that we're putting the company and the product in the best position to get approval.

Right. So if I got this right: It will go through a full regulatory process in Canada as well now.

Yes, that's right because the -- that interim order [ equivalent of ] EUA got expired...

Okay, when did this expire?

I believe it expired in September.

Okay, okay, yes, but the process is on now for the full regulatory review and...

Yes, yes, yes. That's -- yes. It's called new drug submission process. That's [ equivalent ] of a biological licensing application in the U.S.

Right, right, okay. So moving on to a slightly unfortunate development that had happened in the U.S., which was the class action suit. So has there been any favorable negotiation there? And if not, what is your plan to mitigate the impact on the company?

First of all, the company believes the lawsuits are without merit and intends to vigorously defend against them. At this time, no assessment has been made as to the likely outcome or whether the outcome will be material to the company. So I think at this stage, because it's in the process, it's difficult for me to comment any more.

Okay. So then we move on to more -- a brighter topic, which is your gene therapy platform. So what commercial promise does this modified (sic) [ modifier ] gene therapy platform hold for Ocugen? And could you share the plan for the clinical trials of OCU400? Is this -- is it expected to begin by the end of this month, or is this some kind of a delay?

With this -- our gene therapy platform, it's very exciting. I'll briefly mention about what this does, this platform means, what it means to patients globally. And then I'll talk about the clinical trial. There's no delays with the clinical trial. We got very exciting news last week. This is a new platform technology with a lot of potential promise -- patients across the globe. And we did get our IND approved within a month, stipulated time, and we are ready to go to the clinic. And I'll give you more details about it. Coming to the -- this gene therapy platform, which is known as modifier gene therapy. This is based on -- this is typical of like -- these are like master genes in the retina. Unlike traditional gene therapy, you have a mutation or a defective gene. And you have a functioning gene you inject through typically AAV vector. And you -- I mean typically one dose for the lifetime -- potential lifetime treatment, right? In the -- in our case, if there's another mutation, then you have to start the product development, start from scratch. It will take you 8 to 10 years. So the differentiation of our technology is this is based on -- these nuclear hormone receptor genes, they like master genes in the retina. I mean, if you take like OCU400, [ our first ] product which is about to go into the clinic with the IND approval very shortly, this has potential to treat many mutations with a single product. If you take a disease such as retinitis pigmentosa, there's about 150 mutations under that. There is another disease, Leber congenital amaurosis, that has about 25 mutations; total together, 175 mutations. If you take the traditional gene therapy platform, it's almost impossible to develop products for that many mutations, right? And some of them could be thousands. Some of them [ will be ] 10,000 patients. And globally, about 22 million people struggle with these diseases. And many of them are desperate for [ rescue ] because many of them could become legally blind by the time they're in mid-40s. And there's only one product approved in the U.S., which covers very small population for one mutation. Therefore, using this technology -- these are like [ master genes in the retina ]. And they control the functional [ network ] from cell development to survival in all associated gene expressions. They bring the homeostasis to the retina and the [ rescue ]. Therefore, based on the data we have, we have 4 orphan drug designations for the same product from FDA. We also have a -- broad orphan drug designations from EMA, 1 for RP, retinitis pigmentosa; another 1 for LCA, Leber congenital amaurosis, covering all this. So this clinical trial we're embarking on is going to start with 2 mutations, NR2E3 and rhodopsin. And potentially we may start another mutation in clinical trial [ 2 ]. So this is Phase I/II, small clinical trials, as orphan diseases, less than 10 patients per mutation. And then we move on to Phase III clinical trial after 1 year of Phase I/II. The Phase I/II, the primary objective is safety. And along with that, we'll be looking at observational end points for efficacy and multiple ones we are going to monitor during the clinical trial. And depending on the mutation, the end points for primary analysis for the Phase III may change. So we're going to pick one based on this 1-year-duration clinical trial then we move on to Phase III. That Phase III will have a primary end point based on the data we generate from Page I/II. However, all the patients are going to be monitored for safety every 3 months. So we do have all the clinical centers, everything lined up [ in the ] U.S.; very excited to get it going with the patients as early as in the first quarter of next year. So that's what is going on with that product. The second product -- so once again sum it up: We've got one product from this master gene technology platform targeting potentially many, many inherited retinal diseases which are orphan diseases which are under the umbrella of RP and LCA; about to embark on that. And so at least there is a potential hope of the -- for those 2 million patients globally waiting for it. And the second part is we have OCU410, [ another ] gene therapy program, targeting dry age-related macular degeneration. This is big. About 150 million people, patients, globally struggle with this disease. Currently there are no approved therapies -- significant unmet medical need. It's a very complex disease. So we have a program based on RORA gene which is OCU410, and we do have promising in vivo and in vitro data. And currently, based on the agreement we have, a road map from the FDA, we're executing those studies; doing the GMP, manufacturing; doing the preclinical toxicology studies. Once they are complete, we'll be taking this program into the clinic. Can you imagine giving a single injection and for a potential cure for this large population globally, the impact we can have in this gene therapy? That's the reason we get so excited with our gene therapy programs. Developing gene therapy, mutation by mutation, with the first program is almost impossible for any pharma company when you have 175 mutations and to treat those major orphan diseases and underlying mutations. You have mutation-agnostic gene therapy program like we have, its potential to treat all those, such an immense impact that the patients can have in the coming years and then another program targeting a large population. People don't think about it, millions and millions globally, 10 million patients in U.S. alone. That's going to be a huge impact from health care perspective. That's why we're excited about it. And that's the differentiator, a game-changing technology platform in the eye space and specific to retina. That's what we have in Ocugen. And we are working extremely hard [ to move them ]. The first program will be in the clinic, as I mentioned, very shortly with an approval of U.S. FDA. We are really thankful to them. They have been extremely collaborative on this program.

Very exciting. And I -- for the patients' sake, I do hope that they do come to market. On the same products, you have an agreement with CanSino biologicals -- CanSino Biologics, sorry, for CMC development. And you also have the option to support commercial manufacturing. Would you be able to share some commercial terms regarding royalties and/or milestone payments with them?

Yes. CanSino has manufacturing collaboration on both OCU400 and OCU410 programs, and they have option to commercialize. As you know, gene therapy manufacturing is very complex. It takes several years to get to the finish line and come up with a process; and make it clean, GMP process; and scale this process up. We already outlined and also notified the markets: With the help of our partners, we scaled up this process to 200 liters. In gene therapy terms, it's pretty good scale, kind of a commercial scale, so this is good. And we have a solid process going in. And we also can use them for commercialization while we look for other options. We will have several years before we get to commercialization of this product, so we will -- as any other biotech or pharma company, we will start looking into other potential commercial options or commercialization for manufacturing options in U.S. or the European Union, while we can count on our partners CanSino for first commercialization because they do have large facilities. They have excellent expertise. And with our gene therapy expertise and their expertise -- and we have a great team together. So the -- as far as the licensing terms are concerned. They do have Greater China rights for these products, where when they sell the products, Ocugen gets mid- to high single-digit royalties. Similarly, CanSino is taking a lot of risk on these programs. They do entire chemistry, manufacturing and controls; all the clinical trial manufacturing; all the development support, everything -- taking risk on it because we don't pay them for any of this. They absorb all the costs. And on the back end, in our markets, [ ex bridge ], everything belongs to Ocugen. And in all markets, they get single-digit, low to mid-single-digit, royalties. So that's the arrangement we have. There are no milestones. That's why this partnership is very strong. They have significant skin in the game for our success. And they're a very large company, biotech company, very well established on significant expertise and great facilities. That's really good.

Right. So gene therapy in general, it's a bit of an area [ fought with ] risks, so what has been your experience in this regard?

Gene therapy, yes, just as any cutting-edge technology, Vibha, you always have risks in science, right? And so we just have to overcome that for the benefit of the patients. And we have to understand and to use the science to slowly solve the problems. What are the risks? So I just wanted to differentiate for the listeners' sake: In case of gene therapy, there is a systemic gene therapy and then what we are studying in [ almost like a pocket like eye ]. And then in the eye space, typically it's targeted, right? I mean we are giving subretinal injections; and it goes into where the target is, where the gene is supposed to function. And in case of systemic gene therapy, unfortunately, our technology is not there for targeted delivery to specific places where you want to take it, specific target cells or organs. We don't have that. And so lack of that -- I mean obviously, I mean, I can give you -- typically, in the eye space, we target [ 10^11 ] gene copies per dose or below. And in systemic delivery, you are talking about [ 10^14 ] and up, so you are talking about thousand-fold minimum increase in systemic gene delivery. So that's where probably you're seeing, when you look at the data coming out of other companies, there are significant side effects or safety issues. So the technology and science still has to evolve and get there. I mean ideally, if you have targeted delivery, you will be able to control the dose and reduce it to be more effective. I mean, luckily, there is a product in the marketplace in the eye space. It's safe. And it's a kind of a protected -- almost like a barrier. It's an organ they're targeting in the eye space, and you really go into the target. So that -- those are the benefits we have with our gene therapy. And also there are manufacturing issues some of the companies have, the purity of the product and how they make it. And as I mentioned, we have great expertise in gene therapy, very solid expertise. And our partners have solid expertise, so as a combined team, they did a phenomenal job. Kudos to them. I'm very proud of them, what they did with a new technology platform. We have been working with FDA ever since we had the pre-IND meeting. They have been very collaborative addressing our questions. And when we filed the IND, also we had a very collaborative back-and-forth. And within the 30-day clock, 30th [ date ], we got the approval. So that's, I think -- I'm very proud of our team, what they did. It's phenomenal. Getting that first IND approved for our new technology platform is phenomenal, so we'll continue to take steps very cautiously. And safety of our patients is at the top of our list. And that's why, this Phase I/II clinical trial, the primary end point is safety. And we will be continuously monitoring these patients. And along with that safety, we are going to get -- monitor for some signal on the efficacy side so we can be better prepared to go into Phase III.

I wish you the best. And it was really nice talking to you, Mr. Shankar. Thank you for your time. With that, we come to an end of this podcast. And I hope our listeners found it interesting. If you're not already subscribed to pharma intelligence products, you can begin with a free trial by registering on our sites. This and all our other podcasts are available on the pharma intelligence channel on Apple Podcasts, Google Podcasts, SoundCloud, TuneIn and Spotify. Bye for now. And I hope you'll be listening in the next time around as well.

Thank you, Vibha. Thank you. Thank you for having me today.