In this episode of Raising Biotech, Surani explores the fascinating world of vision restoration and optogenetics with Ray Therapeutics. The company raised an oversubscribed $100m Series A in May 2023, which was particularly noteworthy during a extremely grim year for fundraising. Surani speaks with CEO and Co-Founder Paul Bresge about how his daughter's retinitis pigmentosa diagnosis drove him to enter the biotech world. He discusses meeting Co-founder Sean Ainsworth and seeing groundbreaking experiments in blind mice from inventor Dr Zhuo-Hua Pan that lead to the formation of Ray. He also talks about what drew investors to Ray's mission, its clear regulatory path forward (first in retinitis pigmentosa patients followed by Stargardt disease and geographic atrophy) and visions for the company's future. Surani is also joined by renowned optogenetics leader Dr José-Alain Sahel, Distinguished Professor, Department of Ophthalmology, University of Pittsburgh School of Medicine, to talk about Ray's scientific foundations, early data, theoretical safety/efficacy profile and potential to make a meaningful impact in late-stage retinal disorder patients with close to no vision.
Timestamps:
00:35 - Partner segment: Mindgram.ai
01:05 - Background of Ray Therapeutics and focus on late-stage retinal diseases
04:30 - CEO Paul Bresge's backstory, personal motivations to enter the biotech world
07:30 - Formation of Ray Therapeutics in 2021 with Co-founder and Chairman Sean Ainsworth
09:50 - Leveraging breakthrough science from inventor and optogenetics pioneer Dr Zhuo-Hua Pain
11:00 - How Ray was able to attract investors and secure an oversubscribed $100m Series A
16:40 - Dr José-Alain Sahel gives his take on Ray's early data and scientific potential
19:45 - Ray's plans to get the drug into Retinitis Pigmentosa patients and regulatory pathway
26:50 - Safety benchmarks and meaningful efficacy outcome measures
38:20 - Optogenetics competitive landscape and Ray's potential edge
29:00 - Future financing goals and the company's longer term vision
This episode is partnered with Mindgram.ai, a research tool powered by AI to better serve the biopharma community. Listeners of the Raising Biotech podcast are able to get an exclusive free trial of Mindgram via this link, using the code: raisingbiotech24
For any comments, questions, feedback or suggestions you can connect directly with Surani Fernando on LinkedIn or email: raisingbiotech@gmail.com
Music composed by: Yrii Semchyshyn (Coma Media)
[00:00:03] Welcome to the Raising Biotech podcast, I'm your host, Surani Fernando and thanks for tuning in.
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[00:01:08] Today we're exploring the fascinating world of vision restoration as we look at the journey
[00:01:13] of California-based optogenetics company, Ray Therapeutics. The company is trying to tackle a bunch of
[00:01:20] late-stage inherited retinal disorders and return vision to thousands of close-to-blind patients
[00:01:27] with an effective gene therapy. The company raised $100 million series A in May 2023,
[00:01:34] and is making good tracks to get its lead asset into abbreviated clinical trials
[00:01:40] first in Retinitis Pikmentosa. Joining us to talk about Ray's journey is CEO and founder Paul
[00:01:46] Brzewky. He talks about how his daughters diagnosis motivated him to enter the biotech world to
[00:01:52] find a cure. His journey found in Ray Therapeutics and Raising funds in a tough market,
[00:01:58] and the company's players to enter clinical trials and beyond. Also joining us is Professor Jozé
[00:02:04] Sahel, one of the leading pioneers of the optogenetics world coming from the University of Pittsburgh.
[00:02:11] He discusses the general optogenetics landscape which is now a very active space
[00:02:16] and how raised program differentiates from the rest. I hope you enjoy the episode.
[00:02:22] Hi Paul, thanks for joining the podcast. Thank you so much for inviting me. Happy to be here.
[00:02:27] Just to start us off, can you give us a brief, high-level overview of the company's technology
[00:02:34] and offering and the problem you're trying to address? Sure, very happy too. So,
[00:02:39] at Ray Therapeutics we are an optogenetic gene therapy company, targeting patients with
[00:02:46] retinal degeneration. So, our first program is targeting patients with retinitis pigmentosa
[00:02:55] and that we have a follow on program for patients with starguards disease
[00:03:00] and then we're very excited about moving into geographic astrophysic because starguards
[00:03:05] diseases essentially are juvenile form of geographic astrophysic. So, we're very patient centric.
[00:03:12] Optogenetics is a technique that enables the control of neurons or other cell types with light.
[00:03:18] In the field of ophthalmology, it's a technique being used to restore vision.
[00:03:23] Retinitis pigmentosa starguards disease and geographic atrophy
[00:03:27] are three distinct original disorders each characterized by genetic factors and progressive
[00:03:33] vision loss. Retinitis pigmentosa affects peripheral vision, starguards disease impact
[00:03:39] central vision and geographic atrophy involves a macular function deterioration. And while Ray will
[00:03:46] look to target all three of these diseases, the major and first focus is on retinitis pigmentosa.
[00:03:52] And our patient population is really late to end stage disease patients. So there's a lot of
[00:04:01] really exciting work that's going on in the field, particularly in, you know,
[00:04:06] cell engineering therapy for patients with blinding diseases. But unfortunately for
[00:04:12] MCH patients, most of those programs are focused on slowing down the progression of disease.
[00:04:17] So we're very excited to bring hope and hopefully a new therapy to patients who have already
[00:04:24] lost low or even all of their vision.
[00:04:28] So going back to where it all began, the story behind Ray's founding is especially unique
[00:04:34] given that Paul isn't your typical biotech founder. My dad was the founder of what became a large
[00:04:42] generic pharmaceutical company in Canada and then I started off my career in contract manufacturing
[00:04:49] with the POSA format and cosmetic industries. I left the industry and went on a
[00:04:56] number of different things including I've got very heavily involved in real estate and
[00:05:01] operating businesses. The majority of his professional career was spent working for a Canadian
[00:05:07] logistics company where he climbed his way to CEO. But it was some personal circumstances that
[00:05:13] drove him to enter the world of biotech around 14 years ago after his middle daughter Tamar
[00:05:19] was diagnosed with a retinitis pigmentosa just a couple of weeks after she turned 15.
[00:05:26] We were told by the chief of the model just to Toronto's sick kids hospital that she was going
[00:05:31] blind and that there was nothing that could be done to save her vision. Essentially we were
[00:05:35] sent off with a booklet for the Canadian National Institute for the Blind and that was it. And
[00:05:41] that day I spoke to her and made her a promise that I would do everything that I could to find
[00:05:53] her agreement or a cure and in return her promise to me was that she should live her life as
[00:06:00] fully as she wanted to and could and we both kept our ends of the bargain. At that particular
[00:06:10] appointment where she was told that she was losing her vision the doctor told her,
[00:06:15] you need to think about what you're going to study. You need to think about your career choices
[00:06:20] which was actually so inappropriate in hindsight and here we are 13 years later, 14 years later
[00:06:28] and she has a mass difference degree in visual arts from tough university. She's an incredible young
[00:06:35] woman and incredible artist and my inspiration to do what I do every day. So that led me to find
[00:06:44] some really exciting science out of university California Irvine brilliant researchers,
[00:06:50] a husband and wife team Henry class and then Jing Yang. They were working on a written
[00:06:56] oprogenator self therapy program that had incredible proof of concept in animals and I got
[00:07:05] involved with them that led to the formation of Jay site and I had the good fortune of taking
[00:07:13] that program all the way to what is now the initiation of a space three study with Santa and
[00:07:20] pharmaceuticals. Jay site signed a significant partnership with Santa and pharmaceuticals in
[00:07:26] which was also the year that Paul decided to leave Jay site to start his new and current
[00:07:31] venture co-founding Ray the Reputix in February 2021. The difference between what Jay site is doing
[00:07:39] and what Ray their appearance is doing is really addressing different patient populations. So
[00:07:45] what we learned from the Jay site data is that the therapy works best in patients who are at the
[00:07:51] earlier stage of disease to stop the progression of the loss and also to provide
[00:07:57] perfect factors to dormant voter receptors to get them working again. But for patients at the
[00:08:03] end stage of disease who no longer have voter receptors that the therapy is less likely to be effectors.
[00:08:11] So Ray is addressing those more advanced patients where the Jay site therapy is no longer an option.
[00:08:17] So while these patients no longer have voter receptors, those cells responsible for converting
[00:08:22] light into brain signals. They still do have either rational ganglion cells or by polar cells
[00:08:29] basically neurons involved in vision signaling. So we're targeting ready the remaining cells
[00:08:35] in the retina and then using an AA lead gene therapy to get those cells to function like
[00:08:43] voter receptors. So for the retinaces pigmentosa program we're targeting the retinal ganglion
[00:08:49] cells and then for our Starguys disease and geographic astrophied programs we're targeting the bite
[00:08:55] polar cells. So knowing that Paul isn't the scientist in the equation, where do the science come from?
[00:09:03] Well that tracks back to Ray's now co-founder Sean Ainsworth. Sean was a former CEO of another
[00:09:10] optogenetics company retro sense that got acquired by Alagan in 2016. So Sean actually was the one
[00:09:19] who brought this to my attention. I saw the data that had come out of the really the same lab
[00:09:26] that formed retina sense and you can imagine over the last 13, 14 years I have looked at so
[00:09:33] much animal data that it's quite remarkable but I had never seen anything like I had seen in the
[00:09:41] data which really formed right there a few days. So just to give you an idea of what excited me so
[00:09:47] much about this and what differentiated it from reading everything else I had seen before. Our inventor
[00:09:53] had used that inventors name is Dr. Pan and esteemed ophthalmology professor and one of the pioneers
[00:10:00] of the optogenetics field coming out of Wayne State University. He had previously developed a drug
[00:10:06] program with retrosense based on a light sensitive protein called channel rovedopsin and was working
[00:10:12] on some new experiments with that protein in a bunch of blind mice. So these mice are
[00:10:20] on blind they don't even have the developed cortical pathways for vision and yet we are able to
[00:10:28] I was going to say restore but it's not really restoring we're able to give them 50%
[00:10:35] of the vision of purity, of wildlife and new normal contrast sensitivity. So when I saw that data
[00:10:44] you know and again given that the bar is so high with the animal model it was an absolute no brainer
[00:10:50] that this was where I was going to hang my hat and this was a program that could translate into
[00:10:56] reading meaning for therapy for patients. So Paul and Sean decided they were going to pursue
[00:11:01] this endeavor and went out to raise funds. The company raised a seed round of $6 million in 2021
[00:11:09] led by four biocapital and they also received a grant of $4 million from the California Institute
[00:11:16] of Regenerative Medicine. Then two years later in May 2023 they closed an over subscribed
[00:11:22] $100 million series A led by nover holdings with participation from de-filled management,
[00:11:29] nor West partners amongst other investors. I'm so grateful that the investors saw the same
[00:11:36] opportunity for this therapy that I did that we were able to do such a successful route particularly
[00:11:42] in 2023. This was one of the biggest financing announced in 2023 and Paul and I spoke about
[00:11:48] what the investor saw in raise mission given that this field is not new,
[00:11:53] has been around for close to 10 years with a number of programs in the clinic. We made sure that we
[00:11:59] could articulate and demonstrate why our program was so highly differentiated from all other
[00:12:08] programs out there in the space. The you know exciting part for me is that I think it's an
[00:12:15] question of all that proof of concept is there. We seen in other programs, you know,
[00:12:21] particularly the genocide program that was published in Nature. Genocide biologics is a
[00:12:27] French biotech that Paul says was the first to publish clinical data and successfully confirmed
[00:12:33] that octogenetics really works to restore vision in patients and in fact the founder of that company
[00:12:38] is going to join us later in the podcast. The problem today has been that the proteins that have been
[00:12:46] used are not light sensitive enough so in the genocide program for example, patients have to
[00:12:54] wear light enhancing goggles in order to activate the protein to be able to see and we know that
[00:13:02] patients will do anything to get some vision back. The problem is that it's really difficult
[00:13:09] when you think about combining a therapeutic with a device through a regulatory and commercial
[00:13:16] program. Obviously, that makes it much more challenging. So the fact that our protein is activated
[00:13:22] in ambient light and shouldn't have the need for any additional light enhancing, I wear gives us
[00:13:29] you a much clearer regulatory and commercial path. The other thing that we've done this
[00:13:34] differentiated from everybody else in the space is that we've highly bio-engineered this protein
[00:13:40] very specifically for human vision restoration. So it's not just you're increasing the light sensitivity,
[00:13:46] it's also taking into account all of the different functions of vision that are required to have
[00:13:53] meaningful change. Paul said that another thing that was attractive to investors was that Ray was
[00:13:58] able to replicate the studies multiple times and show the same results which obviously gives investors
[00:14:04] a lot of confidence in a field with a huge amount of need. The data really spoke for itself,
[00:14:10] and then of course the management team as well. So I have an actually brilliant chief scientist,
[00:14:19] chief medical officer by the name of Peter Francis who I consider really the most knowledgeable
[00:14:25] person on the planet for after genetics and then a chief development officer, your name is Jenny Holt
[00:14:33] you know who's been involved in the marketer space for for many years. So it was really I think
[00:14:39] a combination of the technology and then the team that gave the investors confidence in our program.
[00:14:45] Ray was also able to demonstrate to investors that their data should translate pretty well
[00:14:50] to approval and points. So we don't have to rely on you know on end points that are maybe more
[00:14:58] in discovery, we believe that patients should be able to gain vision acuity and or you know high
[00:15:06] functioning navigation. And those are already you know really well known to the SNA.
[00:15:13] So the team at Ray were able to successfully articulate their differentiation value
[00:15:18] and clear part to market to investors, but something that did cross my mind especially hearing
[00:15:24] Paul speak about his daughter and motivations for entering the field is that this mission is
[00:15:29] very personal and emotional for Paul. And while that might be incredibly compelling for most
[00:15:35] people for investors that could do a bit of a risk. Yeah and that's actually a great question
[00:15:42] because there were investors who challenged me on you know obviously the investors
[00:15:51] they want to return on their investment and you know they they care about patients for sure
[00:15:56] but they also care about you know their investments. So there was a lot of pressure testing
[00:16:01] you know are you going to make the right decisions as a CEO that are good for the program
[00:16:08] as well as you know be good for the patients and they converge there's no question you know
[00:16:14] if we can develop a successful therapy which you know I have a very high confidence level
[00:16:19] that we will the investors will do even enormously well. And I think that I'm you know I'm able to
[00:16:25] have you know a very balanced approach. So I wear my senior hat you know and I wear my parent hat
[00:16:33] but I think they they work very much hand in hand. So let's get a different expert perspective on
[00:16:39] this. I was super privileged to talk to a bit of an optogenetics legend and that's Professor
[00:16:45] Jose Sahel. He's the distinguished professor and chairman of the Department of Ophthalmology
[00:16:51] at the University of Pittsburgh and he's the founding director of the Vision Institute in Paris
[00:16:57] but he's also an inventor with more than 40 patents. I has co-founded a number of Vision
[00:17:03] Restoration focus by a text including the optogenetics company mentioned earlier. Genside
[00:17:09] publishing some groundbreaking clinical proof of concept data that has really elevated the field.
[00:17:15] I heard of the writer of the text first because I knew of the one of the scientific
[00:17:20] sources of the panel and he's a landmark worker years ago in optogenetics. And then I
[00:17:27] had the opportunity to see very early actually be up a pair with using a new protein that seems
[00:17:33] more responsive to light and we've forgotten the tips of responses. So I was interested in that
[00:17:39] and then over time I got to know the team. This is a unique situation as Professor Sahel knows
[00:17:46] Paul and the team at Raytherapeutics but he's not connected to the company in any adviseary role
[00:17:51] and he's technically a competitive being one of the co-founders of Genside. But he's happy to speak
[00:17:57] objectively on Ray's potential and also cheer it on from the sidelines if it happens to be a success.
[00:18:04] Because my key focus is on patients I'm happy to support any of the fault that could
[00:18:09] provide good outcomes to patients. Dr. Sahel said that the drivers for investor interest in
[00:18:15] this company will likely a combination of a solid scientific and management team along with
[00:18:21] the unique and novel protein that originated out of Dr. Pan's lab which is responding to levels
[00:18:27] of lighting that is below what is currently used in the field. One of the key issues with stogeyetic
[00:18:33] is that we are trying to transform cells but not responding to light or not only responding to
[00:18:39] light, interferatory sectors and so that we have to express proteins that are responding to
[00:18:44] light but because we are not benefiting from all the transition gascane that exists in the normal
[00:18:50] of the receptors you have to amplify your signal. So the biggest barrier to efficacy for every company
[00:18:56] is amplifying that signal and as Paul mentioned earlier Dr. Sahel's team at Genside is using
[00:19:03] amplifying goggles that can codes that signal but with raised therapeutic not using any device
[00:19:10] their special protein is able to respond in a normal range of lighting that we're living in daily.
[00:19:17] While Dr. Sahel said that if you don't use a device this can be at the extensive speed of response
[00:19:23] and it can be slow to optimize the ability to respond to light but in raised case
[00:19:28] the kinetics so far are looking pretty good. Based on the experimental data of XNV12 and in
[00:19:35] people you know small animal models, we seem to have a good answer to provide the good response to
[00:19:41] light in patient. Obviously we won't know until we get to patient. So speaking about patients,
[00:19:46] Paul said the ray plants are getting lead compound RTX 015 into the clinic sometime this year 2024
[00:19:54] and the first trial will be in retinitis pigmentosa patients. Now just for some context there are about
[00:19:59] 100,000 patients with retinitis pigmentosa in the US but rays are dressing later stage patients
[00:20:05] which gives them a target population of around 20,000. But a huge benefit of this therapy is that
[00:20:12] it's independent of the cause of genes so in retinitis pigmentosa there are 70, 80 genes
[00:20:21] you know already identified probably many more because so many patients are unable to even find
[00:20:27] an identifiable gene that it's really important that we can address patients independently of what
[00:20:34] their cause of their gene is. The sparklexerna program which was the first gene therapy that was
[00:20:43] ever approved that was in retinitis pigmentosa. Just jumping in here for a bit of a biotech refresher
[00:20:49] sparklexerna was a huge deal back in 2017 for its major gene therapy approval milestone.
[00:20:56] Distrog made a number of big headlines at the time not only for its dramatic vision restoration
[00:21:02] efficacy but also it's price at a one-time treatment of $850,000 and this drug sparked
[00:21:10] pun intended the company's eventual acquisition to rush in 2019 for $4.8 billion. A classic
[00:21:18] biotech unicorn success story. Gene Bennett is an absolute hero of mine as you can imagine
[00:21:24] she's you know behind the lectern of program it's been incredibly meaningful for patients
[00:21:31] it's your absolutely life changing and we've seen that are sustainable over so many years
[00:21:38] the problem is that it only addresses one mutation of around $4,400 patients around the world.
[00:21:46] So the fact that we are targeting patients independent on whatever the specific gene they have
[00:21:53] really you know broad and supropulation greatly. Paul and I discussed the regulatory part for the
[00:21:58] drug in retinitis pigmentosa perhaps abbreviated timelines and benchmarks for an approval from the
[00:22:05] FDA and Paul said that he's seen the FDA change their position over the years and that's giving
[00:22:10] him some optimism. So for example you know at Jay site we were one of the first companies actually
[00:22:17] with the first in off the modulator in the third ever to receive a designation called RMAT and that's
[00:22:24] a real gift from the agency because it allows you to have constant interaction with them and they
[00:22:31] really work with you as their partner. As you need to establish that there is proof of concept
[00:22:37] so we hope to apply for RMAT you know really quickly after we go into clinic but even just given
[00:22:44] the fact that this is a rare disease it allows for a limited number of patients in an
[00:22:50] clinical study you know phase one nine to twelve patients and then a quick pivot based on efficacy
[00:22:58] into you know a phase two three pivotal study design. So there there is really an expedited
[00:23:06] path to approval here. In addition to that expedited pathway to approval with a low number of
[00:23:11] patients recruited onto clinical trials another advantage of studying late stage patients is the time
[00:23:17] it takes to reach the meaningful data. We expect to see efficacy within a few weeks of dosing the
[00:23:25] patients so within a couple of months we should be able to see how the therapy is working in the
[00:23:31] patients and that will give us data obviously very quickly. When you think about these other
[00:23:38] programs that are you know and again there's so important for patients but generally because they're
[00:23:44] looking to slow down the progression of disease by virtue that these are slowly progressing to
[00:23:49] these so just by virtue that there are longer you know timelines and that makes it a long
[00:23:57] development program obviously we're going to show durability to the agency that at least you
[00:24:03] know the patients will hopefully see a fit quickly and will be able to measure how quickly we
[00:24:10] can move based on that efficacy reader. And Professor Siles can testify to that with his own
[00:24:15] experience in the clinic. The advantage of vision restoration approaches is that you're not comparing
[00:24:22] to a natural history of a disease with demonstrating a change in the stroke of vision loss
[00:24:27] in that case you want to show vision restoration so this kind of curve you know experience for
[00:24:32] example in the genitality tend to grow months after the therapy so we are talking about years
[00:24:37] you're able to meet a lot of shorter pathway to demonstrate vision benefits. Even with these
[00:24:43] obvious advantages we know how biotech works and we will likely have some curve balls ahead to navigate.
[00:24:50] It won't be all smooth sailing so Paul and I talked a little bit about the major hurdles ahead.
[00:24:56] No I'm smiling because I'm hoping it will be smooth sailing. We're doing everything that we can
[00:25:04] to make it smooth sailing and we're doing things differently. For example you know when
[00:25:12] he said that in order to prime the study for success I guess in anticipation of some potential hurdles
[00:25:18] instead of using existing retina clinics that are not the most experienced with testing patients
[00:25:24] who are blind or nearly blind raised developing its own testing facility in the form of a vision
[00:25:30] research and assessment institute in California. We've brought on some of the best low vision
[00:25:37] experts in the country and we will actually be using the facility to do this end for interesting so
[00:25:46] it's very generous on the patients as you can imagine a lot of these patients haven't been tested
[00:25:52] and we're going to be asking them to even get a baseline on neurovision security and to see
[00:25:57] whether or not they're able to navigate a maze you know without the guides that they use
[00:26:02] like their dogs or their family members and we're going to ask them to see if there's any
[00:26:08] your peripheral patient. Paul said that all of those things can be very stressful and time
[00:26:14] consuming for patients so a common controlled environment with low vision experts that are able to
[00:26:19] evaluate baselines and changes from baselines gives ray a better shot at success.
[00:26:26] So while we're relying on vision security and the navigation maze you know we we're also incorporating
[00:26:33] you know every end point that we believe could be meaningful. Professor Sael said that
[00:26:39] he's aware that Ray has had quite productive conversations with the FDA to accelerate the program
[00:26:45] but he cautioned that one can accelerate too much as to not compromise on safety assurances.
[00:26:51] Obviously like every opportunity trial because we are expressing a protein that is
[00:26:55] far into your mind you have to check on the even response you have to check on the
[00:26:59] tolerance of the vector. We thought trial we showed that this was working well with puttory
[00:27:05] runs and of course we need to make sure that we don't compromise the chances of patient
[00:27:09] to retain vision they have and more importantly to be involved in our future therapies.
[00:27:14] It would be promising for them. There are a number of factors that support the safety profile
[00:27:18] of Ray's lead therapeutic firstly it's using an adenovial vector to deliver the protein and this
[00:27:25] vector has been used in humans in several trials then there's the special protein.
[00:27:30] This protein has already proven to be efficient and safe in small animals I'm I'm
[00:27:36] pretty sure about currently doing the primary work to show that the immunogenicity and the
[00:27:41] biogistribution are relevant and then the that is pretty straightforward to you know designing the
[00:27:48] trial and we are looking at really above the safety but it's also beyond common measures especially
[00:27:52] the demonstration of benefiting real life what we have vehicles functional vision which is
[00:27:58] the demonstration but tasks people are doing in daily life made possible using this technology
[00:28:03] which obviously would be the best possible demonstration of the efficiency so I think we are
[00:28:08] touching this you buy all possible angles in a very relevant way because the team again is
[00:28:14] very experienced and very knowledgeable. As Ray and Barks on its optogenetics journey Dr. Siles
[00:28:21] talked about the landscape in general and there were quite a few players in the space including
[00:28:26] sparing vision nanoscripped their pudexpionic vision and a few others around the globe.
[00:28:32] The good news for patient is that the lot is happening. Fortunately there will be competition
[00:28:36] but I think Ray have the net also we have a resource season we have a team to get it to
[00:28:41] we have a multitude to the broad for the vision to. In terms of future out the for the company
[00:28:49] where does that money take you in terms of timeline like when would you look to raise more money and
[00:28:54] need more money like a series B. Yeah let me send another great question this was session
[00:28:59] RJP Morgan for me because I can't remember your JP Morgan then I was not out raising money
[00:29:07] in some form or another you know I mean that's just the world that we live in in biotech
[00:29:12] we're so fortunate that we have enough funding to take us really all the way through to BLA
[00:29:19] if we need to in our lead program. So obviously you know depends on what we do you know strategic
[00:29:25] me and how we think about this in terms of a program versus a platform and the other products
[00:29:31] and the other indications that that we want to address but we have enough funding you know to take
[00:29:37] us to major infection points and that it's an absolute gift because it allows us again to put
[00:29:44] all of our resources into just getting this here a few patients. If all goes well poll did say that
[00:29:50] in the next five years they could actually have a drug on the market given their abbreviated timelines
[00:29:56] and this might sound premature but my immediate thought went to pricing given that Luxeterna was
[00:30:01] price at 850,000 and I wanted to know if Cole and his team had given that any thought.
[00:30:06] I'm so looking forward to having those discussions.
[00:30:12] That means that we're you know when you're talking and debating about pricing
[00:30:18] that's a really exciting time and I hope that we're having those conversations.
[00:30:22] Obviously there are a comparable products like the Luxeterna products on the market
[00:30:27] so at least you know we do have certain data points as we think about this you know from a
[00:30:33] commercial standpoint but right now we are just we're so focused on getting into clinic and having
[00:30:41] patients see and you know getting this to a commercial program really as quickly as possible.
[00:30:48] Given Cole's unique journey I was curious to know where he saw the company be on that
[00:30:53] and whether the company was looking to build itself into a big up to genetics powerhouse potentially
[00:30:58] going public or whether it's so itself safer in the hands of a big farmer once he got its approval.
[00:31:05] I would say both were very focused again on getting to clinic and I'm getting if there are
[00:31:11] future patients with blinding diseases but there is enormous potential for oxygen addicts in
[00:31:17] other indications particularly in the neurospace and we're very interested in that as well.
[00:31:25] So I see the potential for rate therapeutics to bring be the optogenetic platform company
[00:31:32] in the future. We're excited to get the therapy to patients in regular diseases,
[00:31:38] particularly our P first followed by starguars and then as I mentioned GA but the potential for
[00:31:45] optogenetics as a therapy is absolutely enormous. Is that potential for you to also do partnership
[00:31:51] deals on the side with the platform? I have a very open mind I mean we are again in a really great
[00:31:59] place and there's a lot of interest from the statistics in our program there was even before we did
[00:32:05] the series A-rays and I think again Matt Gaze validation to the investors that there is opportunities
[00:32:12] for partnerships, for licensing, for exits everything else but because we have the funding
[00:32:19] and because we're just so focused on clinic that metric we're doing at this time but I'm
[00:32:23] always open as one has to be as a CEO in this space. Moving forward a key challenge for not only
[00:32:31] Ray but everyone in the field will be improving resolution and activating individual cells within
[00:32:38] the retina to replicate the natural vision signal. Current methods just lack that necessary
[00:32:45] specificity. One of these questions is a light adaptation, how do we cope with different levels
[00:32:50] of lighting? How do you avoid foot-to-for-bears or you want to could responsiveness which no
[00:32:55] one foot-to-for-bear means something that needs to be explored and probably addressed?
[00:33:00] The ultimate goal in the field would be to bring back color vision which professor Siles says is still
[00:33:07] a long way off in the up to genetics field but some researchers are working hard on it so it could be
[00:33:13] a future possibility. So that's it for another episode of raising biotech, a truly
[00:33:20] groundbreaking area of science that will note out have a lot to show for itself in the coming decade.
[00:33:27] Thanks to an Iguest called Brezgi and Professor Siles for sharing their insights on this episode
[00:33:32] and thanks to you for tuning in. Remember if you like the episode please share it around
[00:33:37] and follow and subscribe for more episodes. For now I'm Serani Fernando and I'll catch you next time
[00:33:44] on Raising Biotech.