Leveraging a Gene Therapy Approach to Maximize Speed and Minimize Costs

November 11, 2021

While gene therapies hold great promise for patients with rare genetic diseases, one obstacle for ultra-rare conditions is that drug developer may view patient populations as being too small to make the development of a gene therapy economically viable. Taysha Gene Therapies, through its partnership with UT Southwestern, is rapidly developing a robust pipeline of gene therapies that leverage the same vector, manufacturing, and course of administration to enable the company to pursue indication that might not otherwise be feasible. The approach shows how rare disease patient advocates, academic researchers, and biotechnology companies can collaborate to enable treatments that would not otherwise be developed. We spoke to R.A. Session II, founder and CEO of Taysha Gene Therapies, about the origins of the company, its unique relationship with gene therapy innovator Steve Gray and UT Southwestern, and the critical role patient organization have played in the process.



Daniel Levine: RA. Thanks for joining us.

RA Session II: No, I appreciate you guys having me on today. It’s a lot going on, but always excited to talk about what we’re doing.

Daniel Levine: We’re going to talk about Taysha Gene Therapies, its unique partnership with the University of Texas and how this provides a translational pathway for gene therapies for rare neurologic conditions that might otherwise not get on the radar of drug developers. I’d like you to begin with the founding of Taysha and how it was created through a partnership with Steve Gray at UT Southwestern and how it all came together.

RA Session II: Yeah, it’s a great story. I’ve been fortunate to be in rare diseases the majority of my career, gene therapy, probably for the last four or five years starting with our time over at AveXis, which was really the company that helped validate the modality of gene therapy with the great clinical data generated by Zolgensma and then eventually the approval. Now, just announced yesterday, it’s reached blockbuster status, with now having over a billion dollars in sales in a calendar year. Being part of that first management team really gave us some insights on how to take this technology that was so groundbreaking at the time and really one where you wait your whole career to work on, curative therapies, and to really help scale it.

So, when we sold AveXis, I took a little bit of time off. I was kind of looking for my next opportunity. I’m born and raised in Dallas. I just so happened to live about five minutes away from the university and have been a part of a number of spinouts at the university either as a member of the management team, as a board member, and now as a founder. And I’m really kind of looking to what I was going to do next. And I knew that I didn’t want to go back into conventional therapeutics and really wanted to work on curative therapies. At the same time, the university showed a lot of foresight, recruited this young, up and coming translational scientist in the gene therapy space—up and coming meaning the guy’s just been prolific over the last 10 years—but [UT] showed a lot of foresight into recruiting him away from UNC to UT Southwestern and basically gave him the opportunity to build from the ground up a gene therapy program that was fully integrated meaning, had translational capabilities, GMP, manufacturing, clinical care, all under a single umbrella and really allowed him to partner with another, phenomenal, clinical scientist on the clinical care and clinical trial side of things, Berge Minassian.

Ultimately I came on campus and met with Steve, and this was in the summer of 2019, and I’ve known Steve through my time at AveXis, but had the opportunity to reconnect with him. It really started off with a simple conversation of just, “Hey, what are you planning to do now that you have this new role at UT Southwestern?” And Steve puts up a slide of about 30 programs, and says, “Hey, this is what we’re doing. We’re going after these.” And I just looked at him and I looked at the slide and I said, Steve, this is a company. This is not an academic research lab. How are you planning to take on all of these? And, he basically said, “Look, we’re using the same capsid. We’re taking what we’ve done before with AAV9 intrathecal delivery HEK 293 suspension and trying to apply that technology at scale, and basically using some of the learnings of what you guys had pioneered at AveXis and really trying to spearhead, really trying to accelerate and move these programs along into the clinic quickly for patients.” And, I basically told him, “Steve, this is a company, how are you funding this?” And he said, “Well, we’re bootstrapping, I’m writing grants, I’m getting funding from patient advocacy groups, and ultimately, I’m funding a lot of this work out of my startup funds for my academic lab.” And I told him, I said, look, gene therapy was extremely hot at the time. Big pharma, big biotech were looking to place bets, VCs were looking to place bets. I basically said, “If you spun this out into a company, you would never have to fundraise again. You would have all the funding that you needed.” And he kind of looked at me and said, “Well, that’s a great idea RA, but would that be something you’d be interested on helping to spin this out and, and coming to run it for us?” And I said, ‘Look, I’d love to help you spin this out, but I wouldn’t be interested in coming to run this for you, because I just really never saw myself as a CEO. It’s not where I wanted to go. I was a maker, I liked doing deals. I was fairly successful at it and it gave me a sense of autonomy, right? I could do a bunch of things at the same amount of time, or in parallel. And I told him, “Look, I’d help you put together the framework for this, I’d help you, kind of think about it, I’d help you kind of build it out.” But I really didn’t see myself as the CEO for it.

Ultimately as I got closer and closer to the technology, closer to the indications that we were going after, closer to the patients of the lead indications that were going to be first into the clinic, I got even more excited about what we were trying to do and decided to come on and help him full-time as CEO. At the same time, a lot of my old AveXis colleagues—AveXis had just gotten approved [to be bought out by Novartis] a few months ahead—and a lot of them had made the transition into Novartis but were looking to get into something that was going to be a little bit more entrepreneurial, kind of getting the band back together, so to speak, and were looking for the next opportunity. So, I made a couple of phone calls, the first being to Sean Nolan, who was the former CEO at AveXis, who now had transitioned to a number of board roles and to investing from his own family office to kind of just get his advice on what this could potentially look like. And he loved the idea. The second call we made was to Paul Manning who runs PBM Capital and PBM Capital was the first investor in AveXis. They held a board seat on AveXis up until the time we sold the company and subsequently between PBM and Nolan Capital, they were the first seed investors in Taysha. And ultimately that gave us the credibility to where a number of our old AveXis colleagues joined us on the ride. So we had a management team that was highly experienced, and we were able to couple that with an academic research institution and a gene therapy group that was second to none in the world.

And many people asked me about Steve Gray. In my opinion, he’s the best translational scientist from a gene therapy perspective in the world. And the guy is literally prolific; he’s credited with having five clinical programs in the clinic. Currently, the hope is that we’ll quickly get to six by the end of the year, five of those being Taysha programs. And then, into next year, we get to double digits, but really without that academic collaboration, which is very unique in the way that our structure works, we would not have been able to accelerate the development of what Taysha has become in the amount of time that we’ve done it. Not only have we transitioned from a preclinical company to a clinical company, within call it a year, but we’ve also transitioned from a clinical company to a pivotal stage company about to embark on our first regulatory discussions around approval pathway, as well as having four programs currently in the clinic with our fifth entering the clinic for Rett syndrome by the end of the year. So, what they’ve been able to do was really help accelerate the development of these programs in a way that we would not have been able to do without them.

Daniel Levine: I take it a lot of these conversations took place with you at your kitchen table in the midst of a pandemic to boot.

RA Session II: Yeah, it was a pretty unique situation last year. So, we closed the seed round in April, the capital markets were falling apart right around us, but we were fortunate to have some investors that were convinced, understood the modality, understood the unique nature of what gene therapy could bring. And then the capital markets totally flipped for biotech. They were probably some of the best capital markets you could have been in, post that meltdown in late Q1, early Q2. At the same time, my wife was stuck outside of the country. See my wife’s from Ethiopia. She had gone to visit her family in Ethiopia in late January and subsequently got stuck in Ethiopia for a year and a half due to this pandemic and the ability to get back into the country. My daughter was with me, so not only was I raising a nine-year-old throughout this pandemic by myself, but we’re also starting a company, raising capital, putting together the foundation of what a management team would look like, and then trying to shepherd some programs through the translational process into the clinic. Fortunately for us, we were able to get it done. And what I would say is, it was the unique combination of an experienced management team coupled with an experienced translational group at UT Southwestern, coupled with just a really good financing environment from a biotech perspective. We raised $307 million from my kitchen table. It’s crazy. We took the company public in six months from my kitchen table. Gone were the days of those road shows where you’re flying around on a plane for two weeks, visiting five different cities trying to raise capital. We did this all literally in four days at my kitchen counter. So, it was definitely a weird experience.

Daniel Levine: I think of some of the relationships that have been forged by Jim Wilson and the Gene Therapy program at the University of Pennsylvania. How unique is the partnership with Steve Gray and UT Southwestern? And how does it differ from typical agreements we’ve seen between academia and [companies].

RA Session II: Yeah, it’s a really good question. What I would say is the way that we initially started off the collaboration is certainly more, the way that we initially structured the way Taysha and the UT Southwestern Gene Therapy Group were going to work together is that they would focus on translational science, early GMP manufacturing, tox material manufacturing. As soon as something reached IND enabling, they would initiate the IND enabling studies and then will throw it over the goal line for us to take the lead on regulatory discussions, clinical development, GMP, late stage GMP manufacturing. and then, obviously, commercialization. The way that it’s morphed into is—it’s almost a single organization to be quite honest. UT Southwestern really acts as our discovery research arm. We talk to these guys daily. Our corporate offices are literally across the street from UT Southwestern, and UT Southwestern’s admin functions are literally all moving downstairs from our office. So, we’ll be in the same building here in the next month or so and it’s really morphed into this organic collaboration in a sense that in some cases they’ll take things from translational research, lead things all the way through the translation development cycle, including IND enabling studies, including regulatory interactions and lead the clinical development program. A good example of this is our SURF1 program where once we initiate BLA discussions, we’ll take the program on, take the lead there, and then we’ll do all the commercial distributions, versus other programs where they initially led the development and we decided to take it on and lead the late-stage clinical development programs.

A good example of that is CLN1, which we just recently announced we’ve taken an option to, but really, we co-collaborate on multiple areas. What still holds true is that they typically take on all the translational research, early discovery, science construct, design, and whatnot. We’ve taken more of a leadership role in GMP manufacturing for all clinical trials. We’ve taken more of a leadership role on the IND enabling studies, and obviously we work closely together on protocol development and clinical development, but we’ve always said UT Southwestern is going to be a gene therapy center of excellence for us on the clinical side. And, on the translational side, they really operate as our discovery research arm. So, we’re constantly taking on new programs, assessing new programs, new targets with them, and discussing ways that the portfolio will either grow or augment based on new research that they’re doing. So, it’s very dynamic in a sense versus other academic collaborations where there’s a very finite set of experiments that the academic research institution is leading and then once those are done, the collaboration is over. That’s not the way that our collaboration works. We’re constantly looking at new targets together, constantly assessing new targets, running new experiments, and hopefully, continue replenish the portfolio as we get things into the clinic, into the commercial setting. Obviously, we’re retooling the early research programs and making sure we always have this dynamic pipeline.

Daniel Levine: Earlier this year, you expanded the relationship with UT Southwestern with the launch of an innovation fund. What is this fund? How does it work and what will it do?

RA Session II: What this innovation fund was really built for was to cut down on a lot of bureaucracy that was happening based off being able to see new ideas coming out of the cross-collaboration between UT Southwestern and Taysha. Essentially what would happen if you came up with a new target—you’d have to go write a new SRA, negotiate a new option agreement, and then, you’re now feeling clear to execute on those set of experiments. From start to finish that could take a few months. And ultimately what we want it [the fund] to do is, if collectively the cross-functional leadership team decided to go after a new target, we wanted to have a pool of money that was already approved by Taysha and the University to be able to just pull from—to quickly go and get and initiate new research. So, basically this is a cross-functional fund that is managed both by Taysha’s R&D leadership and R&D leadership at UT Southwestern’s Gene Therapy Group. They collectively approve new ideas, new targets, new assays or experiments to go after. And once that’s been approved by that cross-functional group or that cross-institutional group, then that funding is released and we don’t have to necessarily wait to go and run new SRAs. So, it was a way to basically cut down on the time that it would take to get new experiments and new targets funded, and that work could get underway. It’s worked beautifully. Now, since those funds are in place, all we have to do is go amend that resource plan to add to those funds. We’ll be adding to those funds every year, and that’ll be a pool of money that both institutions will be able to go pull from in order to keep moving science forward.

Daniel Levine: You touched on the large set of programs you can choose from here to move into development. Given those choices, how do you prioritize the indications you’re pursuing and is that done through discussions with Steve Gray and his team?

RA Session II: It’s a really good question. We were fortunate not to have to take on too many of those type of discussions because of where the portfolio was. When we in-licensed it, it was already appropriately staged. You had some programs that were approaching the clinic, you had a program that was right at the clinic. We were just doing GMP manufacturing. You had some programs that were approaching the clinic, and then you had some programs that were just at the animal proof of concept, but you needed to do some further pharmacology studies to tease out what the dose response would look like. And then you had some just ideas, right? So now, as we see we’ve made significant progress in the portfolio, you’re just now starting to have this natural movement of the pipeline where programs that were in the clinic or programs that were approaching the clinic are now in the clinic, programs that were in late-stage animal proof of concept are now in IND enabling studies, and programs that are now starting to approach regulatory discussions around an approval pathway. So, as you have programs that are now moving over to the commercial setting, you need to be reinvesting in those early-stage targets. And you’ll constantly see this, and prioritization happens very collaboratively with the team over at UT Southwestern. Again, we go after targets together. We decide what are the best targets, and we kind of move at the speed of science. So, what we never want to do is slow down, slow down something that works. What we’ll do is decide who is the best partner to take on that program or to lead the development of that program. We’ve seen a good example of that with UT Southwestern taking back the SURF1 program. We basically just did a swap with us taking on the CLN1 program and leading that development.

We’re fortunate now to be a company of about 180 employees, but if you couple that with the infrastructure that’s been built over at UT Southwestern and the Gene Therapy Group, they have another 70 employees that work cross-functionally in that group. So collectively we’re about 250 FTEs working to move this pipeline forward. So it’s a pretty robust group. What I’ll also say is, just through the natural design of our portfolio, we’re able to take advantage of a number of economies of scale. Keep in mind, all our programs are AAV9. They’re all using the same manufacturing platform and HEK293 triple platinum transfection and suspension culture. They’re all using the same route of administration, which is intrathecal delivery. So, there’s a lot of commonalities that you get just out of controlling for what we consider validated gene therapy technology. At the same time, we feel very strongly that this improves program probability success and reduces overall program risk. Those are just commonalities from the scientific perspective. Keep also in mind, all these programs are the same call point. The vast majority of these—about 90 percent—are all geared towards pediatric CNS indications. The remaining 10 percent address adult CNS indications. Still, there’s a lot of synergies that you gain by holding a lot of things consistent between one program to the next, which allows us to gain the advantage of a lot of economies of scale that other companies aren’t really able to tease out. If you’re using a different capsid for every program, that means you have to use a different manufacturing process for every program, which probably means you’ve got to use a different route of administration for every program. And you’re not able to really take advantage of significant economies of scale. So fortunately for us, we control for a lot of things. And by controlling for these things, not only are we able to gain significant economies of scale, but we feel strongly that we improve the probability of success and reduce the portfolio risk.

Daniel Levine: I’ve had a series of conversation with patient advocates where there was just an amazing streak where they’ve told me they went to Steve Gray and all of a sudden, they’ve got a program in development. What role have patient advocates played in driving these programs and getting them on the radar and into development?

RA Session II: Yeah, it’s a great question. This is what is so nice about being able to partner with a translational scientist like Steve Gray, because he does it for the right reasons. He knows the right balance between perfection and good enough and good enough to make sure that we have an impact for patients. And that’s a very tough nuance for a scientist, an academic scientist, because they’re inquisitive by nature, and they’re always trying to solve the next problem, but sometimes the next problem really doesn’t matter and today is what matters. What we say all the time is time is neurons, right? Particularly in these neurodegenerative diseases, once a neuron is gone, you’re not getting it back. So, for Steve his motivation starts with the patient. All of his funding, a lot of his early funding came from patients. I look at the giant axonal neuropathy program. This is the first intrathecally dosed gene therapy in history. The clinical trial started in 2015 and Steve is credited with dosing the first patients intrathecally. That’s six years ago and that funding came from Hannah’s hope and really, they drove the early development of gene therapy as a modality and really helped to educate the field. When you start to think about the CLN1 program, that funding and that motivation came from Taylor’s Tale, which was the advocacy group leading the way in CLN1. When you think about GM2, you think about advocacy groups like the NTSAD. When you think about Rett syndrome, you think about advocacy groups, like the RETT Research Foundation. It’s just a number of these groups have just been instrumental—Angelman, it’s the FAST advocacy group, right? A number of these advocacy groups have literally been the reason why these programs have moved forward, particularly some of the local research groups that were really instrumental as Taysha was getting started. They were working right along with Steve Gray on a number of programs, SOC6A1 Connect, Cure SURF1, a number of these programs that I think Steve Gray and the great science wouldn’t be there without these advocacy groups that too many to name.

Daniel Levine: What role do you see them playing in the development of therapies if any and in the commercialization of them?

RA Session II: Ultimately from a development perspective, we always run patient focus groups, particularly in our clinical development as we’re thinking about endpoints—understanding what’s important to patients, understanding functional areas that would be meaningful to them, and understand the natural history of these diseases. These patient advocacy groups have been extremely instrumental in just allowing us to have an insight to what’s important for them and the day in the life of a patient, the diagnostic odyssey that they go through to get diagnosed and honestly, places where that can be approved. These are always areas I think are going to be important. If you’re in the development cycle of any one program, once you’re commercial, or at least embarking on those regulatory discussions, making sure these regulatory agencies know what’s meaningful to them, what ‘good’ looks like, right? And making sure that advocacy is playing a hands-on role and making sure that these therapies get over the goal line. I think we’ve seen this play out in Duchenne muscular dystrophy. We’ve seen this play out and in spinal muscular atrophy. I think we’ve recently seen it play out in Alzheimer’s research, of advocacy playing a real role in helping these programs get registered.

Daniel Levine: I’m wondering if there’s an example of how an advocacy organization may have helped make some decisions about the development of a therapy for you.

RA Session II: Yeah, I think helping us understand what endpoints are meaningful. I’ll give you an example. For our lead program, TSHA-120, which is giant axonal neuropathy. People always think about this as a disease of the central nervous system or a neuromuscular disease. But really one of the biggest functional areas that patients find debilitating is loss of vision because when a patient typically loses their vision, they lose the ability to communicate. They lose that connection with the world. And that’s something to be quite honest that would have necessarily just stuck out to you. It’s not something that necessarily is thought to be a big issue, but as you go into the natural history, a lot of these patients have debilitating loss of blindness. And it’s something that, second to loss of motor function, this is something that really worries patients and caregivers—the loss of vision. And so, as we started to think about endpoints for our clinical trial, this was something that was smartly built into the natural history, studied by the partners, which has been led by our partners at the NIH and Carson Bonderman, but also the interventional trial, which is also being led by Carson Bonderman at the NIH. This was something that we’re certainly going to be talking to regulators about because it’s not something that would naturally stand out to a person when talking about a neuromuscular disease.

Daniel Levine: As you talk about TSHA-120, your lead program, where is it the clinic? And what’s known about its safety and efficacy to date?

RA Session II: This is the first intrathecally dosed gene therapy program in history. We’re fortunate to have clinical efficacy data on two doses where we see clear arrest of disease progression. The first patients were dosed in 2015. We see clear arrest of disease progression at our two middle doses, which is 1.2 E to the 14 total VG, and 1.8 E to the 14 total VG. We’ll be reporting data on our high dose cohort, which is 3.5 E to the 14 total VG later this year. A win for us is basically recapitulated the data that we’ve already seen, which is essentially clinically meaningful slowing of disease and clear arrest of disease progression. We now, with some patients, have more than four years-worth of data so, not only do we have a clear separation and clear arrest of disease progression as measured by the MFM 32 and across a whole host of other functional endpoints, including visual acuity at multiple doses, but we have long-term safety, long-term durability, and dose response data. So, we feel strongly that with the data that we have in hand today that this program meets all the requirements for approval. So that’s going to be our going in position as we embark on scientific advice, and in the phase meetings with the regulators, both in the U.S. and in Europe.

We’ve characterized this is into a couple of scenarios. We look at ex-U.S. and U.S., in two separate buckets. So, if I took ex-U.S. first, this program, as it stands today, meets all the requirements for what we consider conditional approval to meet and that’s [also] a clear pathway in Europe. So, that’s going to be our going in position that this program hits all those boxes and it’s a clear pathway. I’ve been fortunate to be able to get programs through that pathway and so has our head of R&D and chief medical officer, Suyash Prasad. So, as we embark on those discussions, we think that that will enable an MAA in late 2022 and a commercial launch in 2023. Keep in mind that a conditional approval in Europe is a full approval. You’ll be able to reference that approval to get reimbursed early access for the named patient programs over the goal line in multiple markets around the world, which are known to reference Europe and U.S. approvals. So that could be Turkey, Israel, the GCC region of the Middle East, parts of Latin America that have good reimbursement for rare diseases like, Brazil and Columbia, parts of Asia as well. So again, this is the tried and true pathway.

The U.S., as we see it, breaks down into a couple of s high probability of scenarios and one low probability. The two high probability scenarios essentially are that the FDA looks at the dataset that we have in hand and basically says, good safety, good long-term durability, good dose response, clear arrest of disease progression, good natural history. We now have eight years of natural history data and basically [FDA] says this study checks all the boxes. If you look at the guidance that the FDA issued around the development of gene therapy for neuro development and neurodegenerative diseases that was issued in January of this year, this study meets all those requirements. Essentially, the argument is really going to be around your commercial grade material.

The goal is to basically manufacture our product using that same process. We’re using the same cell line, the same media, the same bioreactors, and we’re using the same CDMO that manufactured the clinical grade material—a ‘like for like’ process. So that’s what we’re going to be going in with. The only difference is we’re moving from the clinical manufacturing facility to the commercial manufacturing facility. That’s literally it. And so we’re going to be making the argument around doing analytical comparability. If the FDA goes for that argument, that would enable a late 2023 BLA. The FDA could come back and say, look, we’d like for you guys to treat a few patients using the commercial grade material. The patients are already identified because we have a number of patients that are currently in the natural history study that we’d be able to roll into the interventional trial. So they may say, look, treat two to three more patients for six months, prove clinical comparability, then use that as the basis for the BLA. That would enable probably a mid-20-23 BLA, and then hopefully a late-20-23 approval, or an early 2024 approval.

Then a low probability of what we would see would be the FDA coming back and saying, potentially do a confirmatory study. Honestly, we just wouldn’t know what else to do. You just basically do this study over again. So we see that as a low profitability and the FDA’s guidance basically says when it’s appropriate to use natural history as a comparator, and this study checks all the boxes. So, we think scenario one and scenario two would be high probability. It would either be a worldwide launch in 2023 or ex-U.S. launch in 2023 with an early 2024 U.S. launch. That’s how we’re framing it.

Daniel Levine: I think of the NIH’s platform vector gene therapy program. It’s bespoke gene therapy consortium, which you’re a part of, I should note. You seem to be doing what they’re trying to work out. You’ve got this single vector, you’re just swapping out the genetic material essentially, and delivering it in the same way. How much time and cost savings are you able to realize doing this? And is it enabling the development of gene therapies that otherwise wouldn’t be commercially viable?

RA Session II: You’re absolutely right. It allows us to be able to go after smaller indications that from an economic perspective wouldn’t necessarily get the return on investment. But for us, if the biology is clear and we have the technology to address it, it really allows us to go after those Ns of hundreds versus having some arbitrary cutoff around NPV. Not only is it allowing us to go after smaller, smaller indications. You’re absolutely right around time and cost of tests. Keep in mind, we’re not changing the manufacturing processes, the same manufacturing process. In most cases, a lot of these diseases have the same endpoints. And, when you start to think about operational aspects from a clinical trial perspective, we’re using the same pharmacy manual, because again, it’s the same capsid and the same intrathecal route of delivery. So, you’re using the same drug to device interaction studies.

We’ve even had in the last three months, seven interactions with regulatory authorities around the world. And we’ve had regulators basically tell us, look, when it comes to certain aspects of your filing, you don’t have to do these studies over again, just reference your last filing, think about this more from a platform perspective versus individual programs. That was actually reassuring to us, from some of the regulators, as they started to think about this as a true platform—a plug and play. And so for us, it’s another reason why we’re able to do so much and actually do it at scale and the speed in which we we’re able to do it. And keep in mind, we’ll have five clinical programs. We started the year with one clinical program. We ended the year, last year, with our first just being approved to be in the clinic. And we’ll finish this year with five and hopefully, somewhere between eight to 10 next year.

Daniel Levine: You’re well-funded at this point, you’ve got a rich pipeline, you’ve got a BLA in your sights. What do you think the challenge of proving this model is going to be?

RA Session II: I think at some point, it’s going to be you can’t do everything, right? You’re going to have to make some trade-offs. There are going to be some really interesting opportunities that you want to go after, but that you’re not going to be able to go after. I’ll give you a perfect example in this. We have a program in tauopathies that we’re extremely excited about, particularly where Alzheimer’s development is and the excitement around some of the new advances in Alzheimer’s development. That program itself is a pipeline, right? MAPT associated tauopathies include not only Alzheimer’s, but also frontal temporal dementia. It also includes progressive supranuclear palsy, CTE, just name a few. As a company, our size and what we’re doing, we don’t have the infrastructure that’s going to be able to support an Alzheimer’s indication, right? So eventually you’re going to have to go out and seek scale and partnerships with Big Pharma and other places to where you’re able to utilize some of that natural breadth and scope that they have that they’ve been building up over the last hundred years or so since they’ve been in existence. But ultimately, I think we’re going to always be a source of innovation. Our goal is to be fully integrated. We’ve brought on the best and the brightest from a gene therapy perspective all the way from the development, from gene therapy manufacturing, clinical care, all the way through commercialization, and have started to build out that commercial team. That commercial team is the same commercial team that launched Zolgensma.

We started to build and put in the framework in order to be leaders in rare diseases. But also, we have aspirations of being able to go after larger markets and helping to drive that innovation cycle in larger markets. So, I think eventually you’ll see us go after larger indications, whether it’s indications that are not monogenic and where gene therapy could really play a role, whether it’s vectorized antibodies or knocked down approaches, or areas where you’re able to go after novel mechanisms to address a large number of diseases with a single construct. We have the first of these, our micro RNA program for MAPT associated tauopathies. We have another for GYS1 knockdown where you’re able to take a single construct and go after a number of indications, glycogen synthase disorders like Lafora disease or adult polyglucosan body disorder, or even Pompei. You’ll start to see more of these platforms within a single program and learn more about these next year. And we’ll start to get away from these monogenic diseases as we solve more of these. Certainly, I think the use of partnerships and collaborations with big players that have more scale and scope is going to be needed.

Daniel Levine: And did your wife get home from Ethopia?

RA Session II: She eventually did about six months ago. It wasn’t that long ago but we were certainly excited to see her when she got back. So, all is, well.

Daniel Levine: RA Session II, second founder, president, and CEO of Taysha Gene Therapies. Thanks so much for your time today.

RA Session II: Thank you for having us. We really enjoyed the conversation.

This transcript has been edited for clarity and readability.



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