Gene Therapy

Discover the transformative potential of gene therapy in our latest Deep Dive video as we explore the groundbreaking technologies and innovative companies leading the way in treating inheritable diseases and many others. Learn about the revolutionary techniques that make cures once thought impossible a reality and how regulations and payment pathways must adapt to accommodate these advancements.

In this comprehensive Deep Dive, we discuss gene therapy techniques that have the potential to cure genetic disorders and other illnesses by repairing, replacing, or introducing new genes into cells. Explore the challenges and opportunities that come with these novel therapies as we examine the changing landscape of regulations and payment pathways.

Don’t miss the chance to enhance your understanding of gene therapy and its transformative potential in medicine. Watch our Deep Dive video now to stay informed on the latest developments and breakthroughs in this exciting field.


Good morning everyone. Thanks for joining us. For another I Select deep dive. My name is David Yocum. I’m an analyst on the I Select Ventures team. Today, I hope to give you an overview on the state of gene therapy technologies and investment opportunities.

Gene therapy is the modification replacement inactivation or introduction of a gene into a patient’s body with the goal of durably treating and preventing or curing diseases.

There are a number of technologies that have evolved to allow us to edit specific cells or entire genomes to achieve specific disease, care and benefits.

I will not pretend to be an in-depth expert as the nuance of each of these technologies hope to provide a useful overview of what is being used today and for what purposes. I further hope to give an overview of what the market opportunity looks like, the regulatory path forward, what has been approved, who is investing, what starts in the space are doing, who is innovating in academic institutions, and the general guidance for investment strategy.

Without further ado, let’s jump in.

Further, please feel free to comment or ask questions using the go to webinar side panel at the end of the presentation. So let’s start with an overview of gene therapy what it is and some of the technologies that are available today.

The gene therapy can be summarized by the modification replacement inactivation or introduction of genes into a patient’s body with the goal of derrably treating, preventing, or curing diseases.

Can essentially break this down in sort of two bodies of thought, somatic cell gene therapy, which essentially transfer of a section of DNA into its cell of the body that doesn’t produce from her eggs. Essentially, these are the functional cells of your body that don’t go into reproduction. And there’s germline gene therapy. Now this is something that hasn’t quite been as actively pursued so far due to controversy, essentially, to transfer of section of DNA to sell the produced eggs or sperm, So, therefore, these types of genetic changes would be heritable into the next generation.

Now when you think about gene therapy and and genetic changes in sort of three major categories, gene augmentation therapy in that we essentially go in and we change exchange a gene that we want to have present in a cell.

We essentially want to add genetic information into the construction manual of the cell. Gene inhibition therapy, the idea is that there are certain genes in our in our DNA.

That are causing various proteins to be produced that are essentially bringing unwanted results. They’re either causing disease or they’re creating malfunctions in the body. And so we can go in, we can we can edit, essentially, genes in our DNA, to inhibit these types of genes.

Further, we can also use gene therapy, the editing of cells to target and kill specific cells and this really plays into CAR T cell therapy, which is sort of one of the branches of gene therapy today.

Shaver screen.

In this, this. Apologies to the attendees and believe I and now sharing my screen. One moment, please. Thanks, everyone.

Thanks everyone for the brief technical difficulty there.

We’re we’re jumping back in and hope you we can see what we’re what we’re looking to talk about here. So I again, where I was before, the killing of specific cells, essentially our technologies that allow us and specifically CAR T cell therapy to go in, edit T cells in our in our immune system and enable them to target cancer specifically.

There are sort of three major technologies that we’ll dive into a little bit further here, viral vectors, potentially using viruses to deliver genetic material into a cell. There are non viral vectors, which is actually direct insertion of DNA into cells. One example, that would be naked DNA or synthetic oligonucleotides.

And we have gene editing, which is the use of of CRISPR cas nine or zinc finger nuclease, which basically harness the power of enabling enzymes to cut and edit and A in certain locations.

So here we can we can jump into an illustration of sort of our the the three main categories we talked about earlier and just sort of help see what we’re talking about here. So really, we’re taking that functioning gene adding it into a cell where the gene either does not exist or it needs a gene in order to perform a certain function.

Gene inhibition, here we can see it’s the adding of a certain gene that blocks the functioning of another gene to enable the cell to perform optimally or normally as other cells would. I know we have the killing of specific cells, where we can either target a cell by adding a certain gene into the cell that essentially causes the cell to die.

Or we can cause the gene to present a certain type of marker that enables the immune system to find that cell and specifically kill it.

These these are some of the technologies that are available.

We put it on the slideshow rather than presenter mode.


Okay. Now so let’s sort of jump into some of the technologies they’re available today.

Viral vectors biologists are a means of using viruses with specific genetic information to cause cells to present certain characteristics. In the case of CAR T therapy, viral vectors are used to cause t cells to express antigens that allow them to identify and destroy cancer cells. We can sort of see an illustration here on the right sort of procedure we’d be looking at. And again, CAR T for anybody who is unfamiliar stands for chimeric antigen receptor T cell therapy.

Essentially, we pull out some T cells from the body. We present them with a virus that causes them to be able to identify a specific antigen present on a certain cancer cell, identify that cell and destroy it.

Some of the other — some of the viral vectors that are available today include retrovirals, adenovirals, herpes simplex, and adeno associated viruses all have various – all sort of have various advantages.

To other types of advantages compared to other means of presenting genetic information.

But the main advantage from what I’ve been able to glean has been that these cells that are presented with viruses have a high sort of DNA transformation rate, which essentially means they present the DNA quickly and efficiently compared to other forms of gene transfer. At one second, please.

Just one moment. We’re having a little bit of technical difficulty here.

Hey, Chad.

You are unmuted now. Is that screen working better for you. Sorry to be a problem. No. It’s right now, it’s just it’s it’s black. Like, there’s nothing.


Got it.

Not sure why that’s happening.

If if I if I’m the only one then don’t worry about it. But I think you’re not the only one.

Susan Williams is saying the same thing.


Normally, it works just fine.

How you on mute? How is that?

Still black? Yeah. It hasn’t changed at all. Yeah.

Are you sharing the screen again?


Hold on a second.

Share go share your second screen. I just got the folks what I had.

Is that You guys still see my notes? I don’t I don’t see anything.

It’s just black.

Yeah. Are you running two screens? Or it says it says I’m viewing I select fun screen, but there’s nothing there. Okay.

Maybe try the main screen again and share that. Of course. Five one. Okay.

Now, we’re back to the title slide.

Okay. Now, I see that now that’s presenter view.

Now that’s yeah. It shows notes. And can you click that screen icon in the bottom left corner?

Let’s see. Okay.

Otherwise, we may just can you guys see this?

Can you see this?


I can see that.

Yeah. It’s the slide. We’re gonna roll.

We’re gonna roll from here.


I think Sorry, everybody. I’ll just blow up the screen so I can read it.

Yep. Okay.

So apologies again for that.

Okay. So we’re going to hop into non viral vectors here.

Essentially non viral vectors is a means of sort of injecting DNA directly into the cell.

And so in sort of the naked DNA here, we can see that sort of direct insertion In antisense therapy, it serves synthesizing genetic material that will bind to messenger RNA produced by that gene and inactivated. So you can think about naked DNA as direct presentation. And you’d think about antisense as more direct presentation of genetic information to inhibit genetic information.

And lipoplexes and polyplexes are means of coding these coding the insertion of DNA into these types of in this type of technology.

Now as we jump into gene editing, which is sort of the hot topic these days. And there’s sort of three prevalent technologies.

The most prevalent and the newest and sort of the most advantageous at this point is CRISPR cas9. CRISPR stands for clustered regularly interspaced palindromic repeats and essentially utilizes the CAS9 enzyme and guide RNA to sniff to basically unzip both ends of the DNA strand and insert a new insert a new genetic material or remove genetic material that’s undesired.

Now Talin, which stands for transcriptor activator, like effector nucleases, and zinc finger nucleates, both use the FAKL enzyme to cut DNA for specific nucleotides.

The zinc finger nuclease interact with DNA triplets while tail end interacts with individual nucleotides.

There’s sort of some of the technical differences between the two of them, at least at least to my understanding. Now, CRISPR has a certain number of advantages in that it’s essentially easier to edit multiple parts a genome at once using CRISPR, it’s a more efficient means to do it.

And it’s it’s it’s more accurate and it and it’s actually cheaper to be able to do so.

So for that reason is a large part why it becomes so popular technology and why a number of of companies and and research organizations are building off of the CRISPR platform in order to to study genetic editing.

And in terms of the market opportunity, the market size gene therapy is expected to reach between four point three billion dollars and ten billion dollars by twenty twenty five. According to some research with a with a compound annual growth rate, forty eight – forty point eight percent which is quite substantial.

Currently to date, There are over a thousand clinical trials for gene therapy products. And sort of the top areas for investment and use cases include cancer in oncology, genetic diseases, genetic cardiological conditions, and genetic neurological conditions.

And here we can see basically a grouping of where the trials are focused on today and sort of reiterate some of the information from the last slide and that we’re seeing a lot of focus on cancer.

And when we’re talking about cancer, what we’re really talking about here is essentially investment and research in CAR T stem cell therapy. So if you go back to that viral vector slide, that’s really what we’re focusing on.

We’re talking about that. And essentially, it’s a means of of presenting a viral vector into T cells in order to target specific cancer cells. And so that’s really what the a lot of the trials are focused on today. And and a lot of that is because there’s a lot of value created that industry, cancer is incredibly prevalent.

Rare diseases, when it comes to bringing a new therapy to the market, sometimes you might be working with a very small population of individuals.

And so while there has been a lot of investment and a lot of research in that area, it’s not as it’s not as prevalent to to attempts to bring gene therapies for genetic conditions to the market, a little bit wide. It’s a little bit that it’s unknown.

And there’s not necessarily the patient populations that are in your support. The trials or or sort of receiving the the payback for the investment in the therapy.

And here we can see sort of another another look at sort of the breakdown of where the clinical trials are coming from.

This is coming from the Journal of Gene Medicine. And again, we’re seeing the cancer diseases and genetic diseases involved with a with a genetic mutation in someone’s cells are sort of the focus of a lot of the a lot of the trials today.

So from here, we’re gonna start jumping to the regulatory and insurance landscape.

One thing to sort of premise this There’s only been a number of gene therapies that have been brought to market to date, a couple of those being CAR T stem cell therapies.

CRC cell therapies.

And it’s important sort of premise here that, you know, because of the curative nature of gene therapy.

There are some issues in both bringing products to market from a regulatory standpoint, and there’s some challenges in terms payment process because the way the – essentially the way, you know, healthcare payments are set up today is you support recurring treatment over a long period of time.

As opposed to a one time cure for a disease that was one thought incurable.

So something that’s going to be, you know, a topic of conversation for years to come as gene therapies more prevalent.

But here we can see an approval, an overview of the gene therapies are approved today.

The most recent was Luxterna, and Luxterna is sort of the – is the only gene therapy treatment that is available in the United States today.

It’s treatment for inherited retinal dystrophy and helps cure sort of partial blindness.

And we I sort of highlight here that it’s gonna be it’s it’s priced right now at approximately eight hundred and fifty thousand dollars per treatment.

And we can sort of see that as we run the gamut down the gene therapies here and the CAR T therapies here is that glovera, which was the first gene therapy brought to market for lipoprotein lipase deficiency with an approximately one million dollars for treatment and only one treatment was ever administered. And so the cost for bringing these products to market and then pricing them accordingly to the value that they bring is a challenge for for both patients drug developers and payers because it’s it’s hard to to find a way to pay for a one million dollar drug in a way that is that is fair to all hearts involved. When we first see that down here, strimbeles, seven hundred and fifty thousand dollars for treatment administered, Kim Rea, five hundred thousand dollars treatment, Yescarta, three hundred and seventy two thousand dollars per treatment.

An interesting trend, we can see in this next slide that as the therapy prices are strongly correlated with a number of eligible patients per year.

So if there’s a large pool of patients who can ask to a certain gene therapy, then there’s a strong correlation with the fact that the price might be a lot lower.

And so in terms of of where the investment is today and in terms of where the trials are focused on today, they’ve got a sense that it’s focused in cancer and certain inheritable diseases because, you know, the likelihood that someone actually paying for one of these treatments increases as number of patients and cruises.

Now to to sort of progress with with some regulatory landscape, the twenty first century recruit cares Act was passed in December twenty sixteen, and was able to accelerate the approval of REGenerys and Product, including Jared, Gene Therapy, HealthU Therapies, for otherwise critical conditions to market more quickly than having a sacrifice patient safety. Essentially, this act allocates four point eight billion dollars than an age budget. At the end of twenty sixteen, the FDA five hundred million dollars implemented strategy over nine years.

And as the implemented strategy that came up with definition, for regenerative medicine and advanced therapies. So within advanced therapies, this includes gene therapies.

And so they created a definition And when we look at what that means, it means that within that, this accelerated path forward for these types of these potential.

For an accelerated path forward. Under the twenty percent of this act, FDA would have the authority to grant credit approval for regenerative medicine giving straight from animal models and stage trials over testing in humans to post market review.

Now with that, obviously there’s some benefits, you want to be able to bring these markets, these therapies to market because they have a huge potential benefit of large people in a way that is unprecedented.


Chad is wondering, is the pricing value based or is it more based on cost of the R and D to get it to to the point where is that.

So the pricing is definitely value based, and that’s something that I found fairly prevalent and at least some of the articles I’ve read.

And I think it is some degree being a cost to market. Obviously, bringing one in terms of market a new type of drug.

But if you look back at, like, a luxe turn up, it’s the first one in the in the March of the US. And obviously, it was resource intensive and took a lot time to bring to market. I I don’t know exactly the cost to bring that drug to market or the time frame, but it’s safe to it’s safe to assume that that it was extensive.

But there’s a lot of evidence to show that basically these types of trees are being brought to market with the the thought that they’re bringing a huge amount of value and they’re being priced accordingly. But there there’s an issue with pricing by value on the pest treatments because when you price a drug, it’s a million dollars, it it’s hard for anybody to be able to to keep paying for it. They’re in a really, really high, you know, income packet.

So some of the concerns about bringing accelerated drugs to market, Obviously, if you legitimize, you know, various gene therapies gene two therapies too early, you you lose the ability to successfully recruit individuals for you know, phase three trials.

And you miss kind of the perception if if you if you bring one to market too early, and there’s, you know, something something was wrong or there’s, you know, something was unforeseen and you and you in bringing that drug to market seemed a little brash in terms of timeline, it kind of sets back the other gene therapies and other medicine therapies that might come to market.

So you have some of the concerns that come out of this exciting path forward.

One area that identified is for a big area of issue for gene therapies was in clinical trials and the FDA guidance outlined this accordingly.

And sort of they they outlined it of trial, the way it features that it influenced trial design and that there could be potentially uncontrolled gene expression the activation of unintended neighboring genes and for both viral and effectors potential for transmission of those viral effectual vectors vectors to others.

One area, and I was surprised. And I know it wasn’t really too prior to this presentation was that there’s some manufacturing considerations going into these trials. And so there are some difficulties in terms of producing gene therapy technology at large scale because they’re so personal and sensitive.

And so this is something that has to be taken into account when when trying to design a a drug trial in the If you don’t have the manufacturing for the people that you’re that you’re hoping to to treat, then you’re gonna have some some problems in validating that trial. And then there’s also some clinical considerations in that animal models are quite important in choosing an animal model that really reflects how the new genetic information presented into the cells is gonna react in humans really affect that trial design and its validity.

This next slide is something that I would encourage anybody who’s curious to look into in-depth once this presentation is posted online.

Essentially, sort of talks about some of the mechanisms to handle affordability, because you know, if we’re if we’re gonna pay if we’re gonna pay for value for for a period of disease, for for curative periods, and that value was very, very high, There’s going to be some challenge in terms of getting the therapies paid for by insurance companies and those paid for by the part paid for by patients and making sure that those who develop the drugs are receiving fair value what they’re about to market And so I won’t go into each of these in-depth for the sake of time. Basically, the outline, you know, this chart outlines, you know, based agreements, insurance, loans to consumers, third party financing, manufacturer, managed financing, and then government information financing.

As means to address this.

Now the the what what works best will will change over time, and there may not be a perfect solution. When prices are at this time, it may just be to a certain extent price will have to come down to be able to to pay for these kinds of treatments. But these are some of the various means of of which they can be paid for. I would encourage you to to read through them if you have time.

So here we’re gonna start with some of the key players in financing activity.

The next slide I’m going to show here sort of outline some of the some of the groups that they’re sort of more advanced in gene therapy development and some of the startups that are they’re innovating.

So here we can see the far therapeutic fiber in bluebird bio, gen type biologic feature also have been later stages of of it of developing various kinds of gene therapies.

And at least it seems to say that in hemophilia a gene therapy treatments are being investigated by by multiple large organizations, a smart therapeutic is responsible for in coughsterna, the first gene therapy approved in the US.

Now below that, we can sort of see some of the, you know, startups per se. Now a number of these have raised us a substantial amount of money.

And I want to highlight a trend among these groups that are listed below in that a lot of, you know, the gene therapy products in early stages are attracting really large rounds of financing in a series a stage. So at least from from five from the databases that I pull from I’ve found that, you know, if it’s a gene therapy and it’s in a high profile in any way, it’s it’s likely seeing, you know, a a straight series a, forty million dollars or more, which is which is unlike most of the industry that we see and that most other VCs are focusing on.

So an interesting trend that I’ve seen. And I I think some of that is is in anticipation that there’s going to be a high value received for these products in the future, but there’s also some athlete that people aren’t quite sure how much it’s going to cost.

Because only only one gene therapy to improve in the US are to say how other gene therapies are gonna are gonna go through you know, regulations and and and essentially a benefit to be up to market.

And so there’s unforeseen challenges. I think that’s part of the root of why for seeing such financing sizes.

There’s there’s known there’s known value to be created, but there’s unknown cost in bringing drugs to market.

So here, we can sort of see the VC investment in gene therapy has grown in the last three or four years substantially.

From approximately three hundred million dollars in twenty fourteen to, you know, two point two billion in twenty seventeen. Now this does include CAR T therapy, and so that would can I would sort of, you know, manage for a large amount of of that investment?

But we’re seeing, you know, forty four deals in twenty fourteen, a hundred and two in twenty seventeen more than double what was going on four years ago.

And so there’s obviously increased investment, increased awareness in the in the field, and I expect that to grow over time.

This is another sort of good reference slide for anybody who’s interested in getting engaged with people who are engaged with the VCs in the groups who are you know, really engaged in gene therapy and CAR T therapy.

And so we can sort of see some names that we’re familiar with. You know, we think about early stage, invest in in in gene therapies and CARK and other types of innovative therapies, you know, we see Orbiomed atlas atlas ventures at prime, you know, there’s a lot of a lot of these groups are are getting engaged. We see someone like with OrbiMed with, you know, seventeen investments in gene therapy or CAR T space in the last at least in the last six or seven years and with three in the last in the last year. And on the later stage, investment side, we’re seeing groups like RA capital management, foresight capital management at Prime in there again, or from that as well. With, you know, anywhere between four and eleven investments over the last six or seven years and most with, you know, one to three investments in the last year. So so, obviously, the early stage is is a little more common.

But I think we’ll, again, tend to see more later stage deals at a number of deals begin to climb.

So here’s here we can sort of see the exits that have occurred in in gene therapy products that that have that have been acquired anywhere sort of between know, we’ve got to we’ve sort of run the gamut here. We’ve been thirty three million and twelve billion at at up at the the Kite pharma size.

And and they again, this is this is a good reference for sort of what’s been available.

I mean, what’s what’s been what’s been active in the last you know, six years or so, there are solutions available in in the appendix for anybody like to investigate for which which of these companies do specific things. Just sort of gives a a an overview of what’s been what’s been what’s been acquired in the last number of years.

See some of the leading universities in gene therapy and CAR T therapy. So, you know, you can obviously with with Carl Jun Lab, who’s the father of CAR T Therapy, Michigan, essentially is working on various viral vector technologies, the Baylor School of Medicine, which is which is in clinical research in stem cell transplantation, gene therapy, cellular therapy, Mount Sinai, which is focused on research in various assets of gene therapy such as understanding basic virology, efficient gene delivery into the nucleus cells, various other applications.

So if anybody who’s who’s intrigued trying to to find where where some of the newest research is coming from, you know, the gene therapy initiative and comprehensive, human genetics, University of Minnesota, University of Iowa, These are some of the the universities that are leading the way in terms of bringing new technologies and new new knowledge. So here we’ll sort of look into a couple highlight a couple startups and support that they’re working on.

Oxallis Pharmacy, raised thirty thousand dollars grant back in twenty sixteen, is working on drug discovery for therapies for the treatment of hunting news and Parkinson disease.

Indeed LAS, is working on essentially hardware to enable the essentially to hide the large scale manufacturing of gene modified cell therapies. That kind of technology is something that’s really intriguing. That’s a sort of a major platform and enabling technology in terms of bringing the cost down and enabling more efficient clinical trials. And so that’s that’s the kind of technology that I would I would keep my eyes out for.

Forty molecular therapeutics, is working on gene therapy products for customized AAV vectors in their clinical focus on cystic fibrosis.

Synpromix essentially develop type promoters that are designed to regulate gene expression to desired levels, which will require specificity. Essentially more more accurate means of of gene control and and gene editing.

And sort of to jump to the next slide, a few others that we’re looking at Antorgin, which is working on rheumatoid arthritis gene therapy treatments. Brainvectus out of France, working on gene therapy for Alzheimer’s and huntington’s disease, Drive bio, which is a technology platform for developing viral vector technologies for the treatment of rare diseases. So there’s a number of interesting scenarios at the early and late stage, sort of earlier stage we’re seeing here, later stage, from one of my earlier slides.

And so there’s there’s a good opportunity to see, you know, what’s what’s been invested heavily now, like, you know, the recent past and where the new players are coming in the market.

So from here, we’ll jump into some of the risk factors and investment strategy in terms of sort of summarizing some of what we’ve learned in sort of guiding the the pathway forward. So some of the main risk factors in the in a gene therapy investment trying to bring a gene therapy market sort of product functionality and evidence.

So essentially being able to prove without without a doubt that this gene therapy works that there aren’t a ton of unwanted side effects and that it performs fairly, you know, uniformly within the patients that it’s that it’s been assigned is difficult to it’s difficult to achieve that kind of certainty.

Stacey sort of plays into that, you know, are there gonna be unintended consequences down the road from these types of therapy is gonna be life altering in a negative way to somebody. Clinical trial design, shooting the right test animal models, making sure you have manufacturing lined up in a way that that works out for everyone.

In reimbursement.

Again, that’s gonna be a major issue of this gene therapy revolution.

And being able to find the solutions that’ll enable enable people to receive treatments in a way that doesn’t force them to pay a one million dollar treatment.

The perceived value of that treatment is gonna be an area which which needs to be solved in order for these to be to be adopted in a way that that makes sense.

Again, the capabilities manufacturing supply chain, being able to produce these types of therapies in a way that is efficient, but still personalized as they are inherently personalized medicines.

Regular hurdles, you know, there there is a an accelerated path pathway forward as we discussed.

But there’s still regulatory hurdles, and we still have only seen one gene therapy that’s been brought to market Only one gene therapy brought to the market in the US to date, and so there will still be some regulatory hurdles to be overcome.

And then again, pharmaceutical company strategy and exit potential, certainly understanding how these these companies can exit and how much money it’s going to take to bring them to that to that stage. And so in terms of areas in which, you know, we’d like to invest you know, platform technology that advanced the speed efficiency and affordability of gene therapy development and regulatory approval.

You know, any any way in which that the cost can be lowered, speed can be can be brought up or models that can be developed in order to understand better how gene therapies interact with various types of people.

Our technologies that are gonna help bring the body of gene therapy into the light.

Innovative solutions to the payment crisis for expensive new therapies that create synergies between stakeholders involved in the payment process.

We need to you know, if if there’s a startup out there that’s working on bringing together those type of stakeholders in a way that payments are done in a fair way. That’s gonna be something that’s gonna be very valuable.

Therapies with larger patient populations and a higher number of eligible patients per year. We saw that slide earlier that showed number of patients eligible for trials per year or number of patients eligible for treatment per year strongly influences cost. And so if you can bring those types of therapies to market, there’s a higher chance that more people are gonna be willing to pay for them.

Therapies are the right partnerships that enable early exits to be far you know, if if you can invest in a company in a technology that you think is inherently possible, but you don’t have to be of the process of actually bringing it to market, which could be given a very capital extensive process.

That’s that’s an advisable strategy to take. If you can, you know, enable that technology to to be sort of brought to market by someone with more the ability to provide that kind of capital.

And then supply chain manufacturing solutions to gene therapy. It’s a really interesting place where that can be where the manufacturing can be optimized and where the supply chain can be optimized for these types of technologies is going to make easier to bring them to market.

Some areas in which We would not advise investment at this time.

You know, therapies that are catering to extremely small patient populations, not these therapies aren’t worth bringing to market.

But in terms of the type of capital that we’re providing here, it doesn’t necessarily make sense to provide that kind of capital because inherently produces a therapy with a high cost and and a difficulty to to model that and and and bring it bring it through trials and and bring it to market.

And that sort of plays into, you know, therapies that will require a sense of capital investment in the venture stage, you know, prior to an acquisition.

For for us, it doesn’t make sense to get involved in a in a therapy that this that’s gonna force us to engage more capital than we’re able to provide.

Germ line cell therapy is a social area.

Again, you know, germ line speaking to the fact, you know, to to essentially any any therapies that are gonna be inherited from, you know, mother, father to to children is something that’s uncharted territory at this time and couldn’t see that being an area of comfortable investment for quite quite a while.

And that any therapies that indicate extremely high cost per patient, we saw GLvera only ended up GLvera in Europe, the first gene therapy bought to market a million dollars per patient only ended up serving one patient before the essentially, they they stopped administering that treatment. And so that’s those are the areas in which we, you know, wouldn’t at least from our our standpoint and our strategy and our ability to provide capital don’t make sense. And some of the areas above, do you make sense of enabling technologies or early acquisitions?

So without further ado, that’s the sort of gene therapy as as I’ve come to understand it over the last last few weeks and happy to answer any questions at this time.

Should anybody have any?

No questions yet.

David, I’ll start off with one. In your research, did you find anything involving gene therapy for livestock or or animals?

And and is that roughly on pace with human developments in gene therapy, is it behind?

Is it is ahead?

Or did that’s made in government. So I I didn’t so I studied mostly specific to human interaction.

So I speak to one anecdote that I’ve been aware of.

So I know that the teal bile labs started working out of the area because they were initially working on sort of genetic testing and in tech stock management. And so, one of the the advantage they saw about going after livestock testing initially was that there’s sort of a lower threshold to approval for the types of segments.

So in a sense, I think there’s a huge opportunity for that in animals and plants.

And we’re already seeing some of that know, there’s there’s a little bit of the anti GMO movement, but there’s other means of of genetically altering these organisms.

Doing that can selective breeding for a very long time. In terms of gene therapy as a medicine as a therapeutic, I couldn’t see that reaching, you know, animal populations, but I could see genetic optimization being an area for increased investigation.

Great. Thank you.

Any other questions from the attendees? Looks like just a chance of a question.

Oh, so just a comment. It’s nice. Yeah. I’m dropping it down to investment criteria.

Thank you guys.

Great. Well, if if there’s no further questions here, first want to thank everyone for joining today, bearing with us some of the technical difficulties.

Hope it was a valuable presentation to all I know it was it was really interesting for me to get to learn about this.

This this was a sub segment, an original presentation done on regenerative medicine.

Believe will be will be published online soon. So if you’d like more context to where where this initial thought on gene therapy came from and my initial research came from, it was within sort of the sub segment of regenerative medicine.

But thanks everyone for joining today. We will be continuing these Wednesday mornings at nine AM.

And look forward to seeing you next week for a briefing on the soil microbiome.


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