Talking Biotech Podcast #384
Gene Therapy to Cure Rare Disease - Dr. Gaurav Shah, Rocket Pharmaceuticals
===

[00:00:00] Kevin Folta: Hey everybody, and welcome to this Week's Talking Biotech podcast by Collabora. One of the sad chapters of my life was watching a friend succumb to a rare cancer. It was a type of cancer that nobody really worked on. Few people researched and an affected vanishingly. Small number of very unfortunate individual.

Sadly, she had to look for any kind of trials or generic therapies that they may allow her into because nobody knew much about her disease. The number of orphan diseases affects a large number of people, yet there's been less research and fewer dollars invested in their therapies. However, we're at an interesting time where technology has reached a place where it can be applied to some of these rare problem.

This is where Rocket Pharmaceuticals comes in, and Rocket Pharma is working on using gene therapy to approach correction of rare genetic diseases. Today we're speaking with Dr. Gav Shaw. He's the CEO of Rocket Pharmaceuticals or Rocket Pharma. Welcome to the podcast, Dr. Shawah. 

[00:01:11] Gaurav Shah: Thanks for having me today, Kevin.

[00:01:13] Kevin Folta: Yeah, this is really cool because I love to focus on innovative ways of correcting novel problems, and your company is really focusing on rare genetic diseases and using gene therapy as, as a way to resolve them. But at the same time, there's some unique problems with dealing with orphan disease. So what is an advantage of operating an orphan disease space?

[00:01:35] Gaurav Shah: That's a great question. So, I would say there's a few answers. First of all, truly devastating, rare monogenic diseases don't really spread widely in the population because these are patients who sometimes pass away early in life, or aren't able to live normal lives. So the mutations themselves tend to be somewhat restricted in the nu terms of the number of patients they affect.

So, deal and try to cure potentially rare genetic diseases. It's going to be in the orphan disease space. So another point to make on why the orphan space also is that big pharma companies often don't want to. Go into that space for whatever reason sometimes to do with profitability, sometimes to do with the expertise needed to develop rare disease drugs.

So it's a place where small companies like Rocket can truly be first, best and only in class. I would say that. The development of complex therapies such as gene therapies is difficult and we have to keep the patient and drug development in mind. And it's not good to be trying to compete with somebody or another company.

So it's just great to be first, only in best in classes. All of the programs with 

[00:02:56] Kevin Folta: rocket art, No, that's, that really makes a lot of sense and, and it's really important to somebody if you're the person who's affected with that disease and unable to find a cure out of the major pipelines. So what are some of the current disorders that are being targeted and maybe how common are they?

Yeah, 

[00:03:14] Gaurav Shah: so we. Are looking at two therapeutic areas. One is the bone marrow. The best way to approach the bone marrow with gene therapy is an ex vivo, lentiviral based approach. What this is, is there is a lentiviral vector, a lent antivirus, which is a modification actually of the H I V virus, which has been inactivated, but at the same time is highly prolific in its ability to deliver DNA material to millions of cells or billions of.

and this lentiviral therapy is transduced means introduced into cells outside the body. Hence it's called ex vivo. So ex vivo, lentiviral based therapy. We take patients blood and bone marrow into a lab. We introduce the gene using a lentiviral vector, and then we reinfuse that blood. Into the patient's body, and it ultimately defines its way to the bone marrow and corrects the disorder.

So that's X vivo lti. We have three x vivo LTI programs that address bone marrow diseases, and those are fcon anemia, L A D one, leukocyte ion deficiency one, and PY RVA kinase deficiency. We also have an in vivo, a AAV program. This is where we use another virus called AV to introduce correct genetic material directly into the.

and in this case it's the heart. So instead of taking it outside and culturing these cells outside, we directly infuse the AAV V into the bloodstream iv. It goes to the heart. And it adds this gene to cardiomyocytes. Dana disease is a heart disease, so we have two therapeutic areas, bone, marrow, and heart, and we've also now added two more heart diseases that can be potentially treated with AAV V in vivo therapies as well.

[00:05:09] Kevin Folta: This is really cool. So let's start with this lentiviral thing and drill into this. I'll touch more is when you're doing the lentiviral approach, this is done in in vivo, as you say, you do the genetic change in vivo, then do you have to oblate the patient's bone marrow and then you just give this transfusion back in and then bone marrow cells figure out how to get back in the bones, which is really cool.

Is that how this works? 

[00:05:34] Gaurav Shah: That's exactly how it works. That says elegant as an explanation as I heard Kevin. That's exactly how it works. And the advantage here is that you're not really bathing the, the bone marrow directly with these lentiviral viruses, right? These lentiviral vectors. That's one thing.

And also we're able to control the conditions of transduction means introduction of genes into these cells outside the body so we can really tweak that and develop it over time. So there's a lot of advanced advantages to that style. 

[00:06:06] Kevin Folta: and are those lentiviral approaches kind of one and done that once you deliver the DNA n in vitro and then those cells no longer can spread the virus?

Like, is, is it kind of disa inactivated or unlikely to spread after that? 

[00:06:21] Gaurav Shah: Yeah, it is one and done. The idea is that a single infusion is potentially curative for the rest of the patient's life, and we have evidence. That lentiviral based therapies can last at least 10 years in some cases a few years, more than 10 years based on some of the early work using lentiviral therapies.

Not in these programs, but in other programs. So there's good reason to believe that this is a durable, potentially one and done treatment. 

[00:06:50] Kevin Folta: Gosh, I love this stuff. So the, the length of viral stuff that makes sense to me. Now when you talk about the in Viv. Approaches with aav, which has been used for a long time now in many different places, but how does the AAV know to deliver the target to the heart?

[00:07:08] Gaurav Shah: There are many types of AAVs and many of them are naturally occurring. The one we use is AAV nine. Nine represents the capsid. The capsid is the way. These AAV vectors find the cells and bind to cells and insert themselves into the cells. AAV nine loves the heart. That's been proven in cell-based experiments.

It's been proven in animal models, and now it's been proven in humans. AAV V nine loves the heart. It also likes skeletal muscle and c n s. For example, a AAV nine was. Vector, the capsid that was used for zolgensma, which was currently marketed by Novartis a therapy that addresses spinal muscular atrophy, type one, and it's, it, it's an effective therapy.

AB nine loves c n s as well as the heart, but it loves the heart more than any of the other other organs in terms of what we call trophism. Trophism is. Ability to hone in on a particular cell. So the trophism of AAV nine for heart is very high. . That's how we, that's how we select for specificity.

[00:08:14] Kevin Folta: Well, I think that a lot of people who would learn about this may be concerned about off-target then. So, you know, you have something that you want in the heart that maybe is showing up, is going to other places by nature of that of that virus. So do you use regulatory elements in the gene, in the, in the gene that's being added therapeutically to ensure specificity of the therapeutic.

[00:08:37] Gaurav Shah: So the capsid determines t tropism, right? It's the number of cells that the vector is going to transduce and insert DNA into promoters and other regulatory elements. They determine protein expression, which is the next step after DNA insertion, right? So you can, you can insert a lot of DNA with the right capsid and tweak the protein express.

Using promoters that might be cell specific or tissue specific, you know, organ specific. So the whole spectrum of how to influence a particular target, a p particular disease target using an AV vector has such a wide range of inputs across the, the full spec, across the full rainbow here, ranging from the type of caps that you select like you said, the promot.

Enhancers, other regulatory elements, other initiation sites that can tweak up or down particular protein expression. And all of that is it's an art as well as a science. And it's also sometimes serendipitous to discover the right Goldilocks type gene therapy that works. And I think for Dana disease, that's exactly what happened.

We had a therapy and if we tweaked up some of the. , it became too toxic if we tweaked some of them down. It didn't get in into enough cells. So the one that we developed and moved forward with was exactly the right one. I think it, it's a beautiful question because people just probably don't realize that gene therapies can go wrong really quickly if you have the wrong tweak on there.

But they can also be curative in the right way, you know, with the right variations and marrying the arc, the. . 

[00:10:28] Kevin Folta: Well, it's just like anything, right? The dose makes the poison. And what I think has been so exciting about the genomics era is that we've been able to discover so many little, not just on off switches, but volume knobs that can help us right target the, the, the right expression in the right tissue at the right time, right place, right?

Developmental state. So many things that we can do that we couldn't do 10 years ago. So super cool that you're doing this. And, but you know, I think about this from the science side, that's great, but from the business. Yeah, you know, you're talking about rare diseases with a, like you say, a handful of patients that maybe get one treatment , and if it works, then you don't, you lose your use, your lose your customer.

So how does a publicly traded company expect to be highly profitable from treating obscure disease? Yeah, 

[00:11:14] Gaurav Shah: so first of all, I would say that each rare disease is somewhat rare, but rare disease is not rare, right? Because there's a lot of rare diseases and you add them all up. They become more common sometimes than what we call common diseases, right?

So I think the ability to address several diseases that might be considered rare, each one put them together using a platform approach, like, like I said earlier, ex vivo lte or in vivo a v to quickly be able to address the science behind these diseases, get them into the clinic, and then get them commercial.

Can certainly lead to a pretty profitable business case. And the precedent here, there, there are precedents that that might be well known of companies that started as rare disease companies that continue to be rare disease companies, but they've really been successful in addressing a large number of rare disease patients.

There's Vertex there's Alnylam, there's Alexion and these, these have very strong business cases and, and now positive revenue. . 

[00:12:16] Kevin Folta: That's a really good point. And it's also the idea that once you develop the toolbox and a great set of tools, you may find more screws to turn that are a little bit more mainstream.

So it's, it's exciting not just from a innovation standpoint, but for future collaboration. That's pretty good stuff. 

[00:12:31] Gaurav Shah: Just to add to, to that last point you made, we're addressing monogenic diseases. These are diseases in which one gene has gone awry via mutation. once we crack this monogenic code, which has already been cracked.

But once we're able to, to address a lot of these you could call them low hanging fruit in some ways because it's a simple fix in, in many ways a gene replacement for the gene that's wrong, we can then address bge, tri multigenic diseases and ultimately the multigenic diseases. Even many diseases like Alzheimer's are considered to be multigenic, so over time.

I think gene therapy has the potential to have an impact on a large swath of human diseases right now. We just opened up the door a crack and we're, we're letting light in. 

[00:13:23] Kevin Folta: That's awesome. So we're talking with Dr. Shaw, he's a c e O of Rocket Pharma and their cracking open doors, a new technologies that are allowing realistic application of gene therapy.

And this is the Talking Biotech podcast by Collabora. And we'll be back in just a. So now we're back on the Talking Biotech Podcast by Col Collabora, and we're speaking with Dr. Shaw, and he's the c e O of Rocket Pharma, and we're talking about gene therapy solutions to monogenic problems. So where there's a.

Underlying genetic condition that's causing a problem. These are therapies that are designed to fix it, and we're speaking about some specific examples in their pipeline on the diseases that they chose to pursue. And it's it's really interesting because these really sit in two different places.

We talked about bone marrow and cardiac, but can you give us an idea of the pipeline overall and how does this work? Do you start with animal models first and then kind of move towards a human focus? So even 

[00:14:22] Gaurav Shah: before animal models, we have cell models. So we can actually, for example, in heart diseases, look at cardiomyocytes, and there are ways to derive cardiomyocytes that serve as a proxy of human cardiomyocytes.

And we can even test these vectors first in the lab without animals. And then we. Move into animal studies, we typically start with either small animals, either mouse or, or rat models. We may at some point move into larger models such as pig or in some cases non-human primates. And this whole package comes together.

The programs are highly de-risked by the time we get into humans or at least de-risked as much as possible by the time we start a clinical trial in, in. . So that's, that's the whole process. It takes a while. One of the things that we pride ourselves at Rocket about is the fact that we've been able to take programs from discovery to clinic in about 30 months, so about two and a half years.

So over time, as we develop more programs, our capacity to bring multiple rare disease gene therapies to market is, is is quite. . 

[00:15:35] Kevin Folta: Well, that's it's really impressive and it's exciting to think about that this pipeline is relatively short for these diseases that are rare. And let's start with a couple of them.

That's the fun part for me about this is it's great to learn about new technology, but it's also interesting to learn about new diseases. And let's start with fcon anemia. I've never heard of this before. So what is it and what is the current line of treatment that before your therapies would. 

[00:16:03] Gaurav Shah: Yeah, FCON anemia is a bone marrow derived disease and the disease is caused by defects in DNA n a repair.

So usually when most of us are exposed to the environment, there's radiation around, there's random mutations that happen in our body. There are other environmental toxins that lead to DNA damage. And we have an intact dna, n a repair mechanism. Fcon anemia, patients have a defective d n a repair mechanism, so when they have injury to cells, they can't self-repair the organ that's affected first.

And most. In a devastating way in these patients is the bone marrow because the stem cells and the bone marrow are especially prone to DNA damage. And then if they can't repair themselves, the stem cells eventually die out in the bone marrow and the bone marrow fails. Over the course of the first 10 years anemia patients, 80% of these patients go into bone marrow failure, and later in life also develop.

The current therapy, the current way to get around this is an allogeneic bone marrow transplant from somebody else's bone marrow into the patient's marrow. But as you can imagine, this can be toxic, can be deadly, and it leaves the patient even if successful for many years of frail health status. The gene therapy approach actually is different for Fanconemia versus almost any other gene therapy program in.

We're not requiring conditioning, we're not requiring chemotherapy to kill out the bad native stem cells before introducing the gene corrected cells. Why? For the same reasons that the disease exists, those stem cells fizzle out on their own, so we don't need to use chemotherapy to eradicate them and make room for the new gene therapy cells.

So just by infusing gene therapy cells with. chemotherapy we're able to, over time to repopulate the bone marrow with normal corrected stem cells that have an intact d n a repair mechanism. So that's sort of one of those situations where pcon gene therapy was not just pushing the pipeline for Rocket Forward, it actually pushed the gene therapy field forward in a very, in a very big way in terms of the science.

[00:18:35] Kevin Folta: That's super exciting because I, I've watched gene therapy for a long time. I did speeches on this in 1980. Wow. Three or something. I mean, I, I used to talk about it back then and all the companies that had these glowing ideas that we thought, okay, this will all be figured out by 1990. And it took a lot longer to get to where we're getting, but I think that it's so realistic now because of focuses like this part.

Approach. And what about leukocyte adhesion deficiency? This is another one on your website, but I'd never heard of this one either. What is it and what is the current treatment? 

[00:19:11] Gaurav Shah: L a d one is perhaps one of the most devastating diseases on earth. It is a disorder of a type of blood cell called the neutrophil.

We all need neutrophils to fight infections as sort of the first line of. and the issue with patients with L A D Lide Adhesion deficiency in this case, subtype one is that the patient's neutrophils lack a certain protein on a surface called CD 18, or they have very low levels of it. And without that protein, the neutrophils can't get outta the bloodstream and get into areas of infection.

So these are children who have recurrent and often fatal illness. Early in childhood. In fact, two thirds of patients in the world with l a d one, severe l a d one pass away by the age of two. And even those first few years of life are marked by re frequent hospitalizations and infections that are devastating for patients and families in the community.

So really a devastating disorder. We're using a lentiviral ex vivo approach here. To address l a d one patients, nine patients have been treated. We revealed the results last year. All nine patients are l a d one related, infection free. They're coming off their antibiotics, they're, they're blood levels of other biomarkers that might suggest L e D one have all normalized.

And while these patients expected only to. 2, 3, 4, 5 years. It's possible now that they live normal lives, 82, 83, 84, 85 years or more. So, you know, obviously I don't wanna point toward results that haven't happened yet. That's, that particular statement was actually made by the principal investigator.

But these diseases do have the potential to find curative solutions through gene therapy. When gene therapy works, it really. , 

[00:21:10] Kevin Folta: and I think that's what people don't appreciate. This isn't a molecular bandage here, this is a molecular solution. This is replacing what the problem is, isn't just treating the symptom, this is solving the causal defect.

And I, I think that's just such a important part of the approach. 

[00:21:26] Gaurav Shah: Yeah, and, and to your point in the history of our species, there's been very, very only a few instances where, We can actually address the root cause. One example is many types of infectious diseases where we can eradicate the bacteria, and I think after infectious diseases, the next big break breakthrough in terms of curative potential is gene therapy.

you have defective DNA and you replace it with correct dna n which is as fundamental a cure as, as we could get for anything. 

[00:21:55] Kevin Folta: And let's and maybe we can, should approach this question now while we're at this point, is what are the limits? Because you're using bone marrow where you can do this in vivo, or you're using targeted AAV V, which can find a specific delivery place with all the regulatory elements, but how many mono.

Is there a way to even guess what, how many of these defects are monogenic and can be approached with these kinds of therapies versus others, which, you know, if it's in all your muscle cells, you'll never get to it, that kind of thing. 

[00:22:29] Gaurav Shah: Yeah. So there are more than 7,000 known rare diseases. Many of them are monogenic and amongst the monogenic diseases, you know, the, the, the.

The question you're asking is the exercise that we performed when starting Rocket Pharma several years ago, we can whittle that number of 7,000 down to a few hundred where there's a clear monogenic cause associated with a protein in a cell that causes the disease. I would say that number is several hundred.

And I think out of those several hundred, not all of them are common. to sort of warrant the large number of investment or the large amount of investment that's needed to develop these therapies. So we whittled those few hundred numbers down into several dozen, and I do think that there are several dozen diseases that are relatively common, even as rare diseases are still relatively common, where again, all these other philosophical tenants of asset selection come into play and where we.

Really make a, a big impact in a large number of patients' lives and also have a business case to move forward. We've started, as I mentioned, with six of these four in the clinic, two others. We have about the same number in what we call wave two, a pipeline right behind, right behind. And over time we do anticipate being able to, to address a few dozen diseases.

And at that point, it's no longer rare diseases, like I said earlier. , you add all the rare diseases together and it's no longer rare. So we put, could be getting into several hundred thousand patients that we could affect over time as we expand our pipeline. 

[00:24:03] Kevin Folta: and, and maybe I'm putting a cart ahead of the horse, so just correct me if you don't want to answer this question, I'll edit it out.

Yeah. I, I'm expecting a daughter in May, and one of the things that we went through was because both my wife and I are advanced age parents, and one of the things they did was a significant panel for monogenic diseases in. Her to ensure that we didn't have a a child coming with some special needs or or special issues to address towards delivery.

And is there a possibility that these types of screenings could happen early on and that corrections could be made even in a early embryonic stage to create, to solve the problem before you ever, before the baby's even born, or before a human even is, you know, boots on the. , 

[00:24:46] Gaurav Shah: I think early in life is certainly a possibility.

In fact, the youngest patient is treated in some of these programs. Both Lent D and AV are less than one years old and in one case in our trial, three months old. So certainly identifying the disease early in life is only gonna be beneficial for everybody because not only can you treat the disease, but you can prevent many of the manifestations of disease if you catch it early in life.

Hmm. in terms of embryonic gene transfer. I think that's probably a ways away. I would say that the field would have to advance a little bit further where we can get more and more comfortable with the overall benefit roast profile before we go there. But yeah, why not someday? 

[00:25:29] Kevin Folta: Absolutely. Yeah. Yeah.

There's a guy in China who could tell us all about the fallout when you jumped the gun on that one. . Yeah, right, 

[00:25:34] Gaurav Shah: exactly. , 

[00:25:35] Kevin Folta: we're not there yet. Let's go back to your pipeline. And it's really, cuz this is really the good stuff. What's Danin disease? It was totally new to me. And how does gene therapy help to.

[00:25:47] Gaurav Shah: Yeah. Danin disease is actually one of the most life-threatening and devastating cardiomyopathies on the planet. In fact, the largest heart on record in in human history in terms of weight is a danin disease patient's heart. And this is a disease that's X-linked. It affects boys earlier than girls, but eventually affects.

It's a disease where there's something called autophagy that keeps all of our cells clean. It's like the vacuum cleaner or the recycling center of ourselves, of, of our cells. Without autophagy, you get garbage building up and rearranging these cells function. It happens mostly in the heart, but it can also happen in the skeletal muscle and the.

Dana and disease patients have basically a mechanism, sorry, a faulty mechanism whereby their heart malfunctions over time again affects boys earlier than girls and it affects 15 to 30,000 patients in the US and Europe. That's our our best estimate. Boys, unfortunately pass away in their late teenage early.

Unless they get a heart transplant, which is only available in rare cases, even when patients get a transplant, though the 10 year, what we call survival, meaning the chance that patients can survive for 10 years is only about 50%. So even with the cardiac transplant that the, the odds are not great. . Our gene therapy for Dana disease is an an IV AAV nine approach like we discussed earlier, where we directly inject, inject the corrected gene into the heart using an AV vector, and once it gets into the heart, it turns on autophagy because the defect in den is caused by lack of a protein that we then.

Institute into cells and turn that vacuum cleaner back on. And once that vacuum cleaner is back on, we've seen in the patients that we've treated based on biopsy, that the vacuoles actually start going away. We can look at those and we can even quantify them. And there's a massive drop in VAs over time.

And that massive drop in Vacuoles is associated with, we can see the protein being express. , it's associated with labs like b n p that start improving. These are measures of heart failure. It's been also associated with improvements on cardiac imaging. You can actually see the heart shrinking in size on, on echocardiography, and it's also introdu.

It's also manifested by patients actually feeling better. And in some cases, patients who thought they were gonna be on a transplant list or passed away by now who are in the early twenties, who got our gene. Are now going back to work and going to college and starting to live normal lives. So this was for us, a really big breakthrough in the field because while gene therapy has been effective to date in bone marrow diseases in the liver and in the c n s, this is the first time we've shown the impact of gene therapy on the heart and knowing that the heart is the biggest killer.

of, of people in the us it could really open the door to something 

[00:29:14] Kevin Folta: very large. So all of these are really exciting and they seem to be very linear and straightforward approaches to really important problems if you're in that subset who are affected. So what is the regulatory climate like on these?

How much evidence do you have to produce and, and especially with a few number of people, are the clinical trials really challenging to, to perform? Or what, what's the bar like? 

[00:29:36] Gaurav Shah: Regulatory landscape for gene therapy has actually evolved and recently has become highly supportive. So I would say early on in the field, there was a lot of positivity and harmony between drug developers, of gene therapy and regulators.

There was a middle zone, I would say it's between one and three years ago where some of the toxicity of gene therapy came out. Into the public arena and just required a little bit of a pause. I think a lot of gene therapy trials went on clinical holds while the FDA and the industry figured things out together in terms of how to de-risk these programs.

And now I would say that the, the faucet is, is open again. In terms of FDA guidance and support for gene therapies, for example, for Dana disease, we just got our MAP designation, which is like the old breakthrough design. Last week. And this allows us to have frequent dialogues with the F D A, including with some of their senior management and a commitment to help us develop this, this therapy for patients as efficiently as possible.

So the regulatory landscape has evolved. It's wax and waning, but I think we're in a really good place at the moment. , 

[00:30:47] Kevin Folta: and that's one side of the coin. The other thing that always concerns me and others in this area is access. So as you develop a novel solution that maybe affects a small number of people, the price tags may be excessively high, and is that really going to be an issue, or do you imagine that there actually will be value in gene therapy rather than lifetime treatment of someone with a severe disease?

[00:31:13] Gaurav Shah: Yeah, so I, I think a lot of. Gene therapies that have been commercialized have come with large price tax, but that has to be compared with the cost of not getting gene therapy. For example, in danin disease, the cost of a cardiac transplant is often about 1.5 million, and even then it doesn't work in 50% of cases.

So that's the comparison versus the gene therapy price target and also the socio socioeconomic cost. A person losing life early, you know, what, what's the number on that? And if a patient can be cured with gene therapy, you know, some of these, these higher costs start making sense. So I, I think in this case, in the case of devastating genetic disorders you know, high price tags are not just , problematic for us and, and for patients, but they're also not, don't seem to be problematic for payers who have approved these price tags based on the socioeconomic models.

[00:32:12] Kevin Folta: Mm-hmm. . And I guess the, maybe the last question I would ask you is, you know, the c e o of the company and looking out into the future, is there really a, is there a disease out there that you find particularly intriguing, maybe a little bit tough to tackle at this moment, but maybe something that you would like to see?

Especially taken care of? 

[00:32:32] Gaurav Shah: Ultimately, we want to tackle something as complex as Parkinson's. Or even Alzheimer's, if there is a gene therapy approach to that. I think that's when we're there, that means we've solved a huge portion of humanity's health problems and I think we're decades, if not a century or two from there, in my opinion.

But we'll get there because the gene therapy revolution that's going on right now has cracked open the door already. I would say that however, even just address. The multiple rare disease monogenic conditions that exist today would make a major dent in, in the world's health. And I think that's our immediate, medium term and long term aim at at Rocket Farm.

Yeah. 

[00:33:16] Kevin Folta: I absolutely love your optimism on it and I, I love the fact that I disagree with your timeline. I think , this is gonna go a lot faster cuz we got a lot of great people working on it and amazing companies and lot of really great innovation. I mean, look how far we've come in 10 years I guess.

But if people wanted to learn more about about your company, where would they look online or follow you on social media? 

[00:33:37] Gaurav Shah: Yeah, so by the way as c e o, as you. My job is to under promise and overdeliver. So hopefully we overdeliver deliver on that, that timeline. 

[00:33:48] Kevin Folta: I'm sorry. Yeah. I'm sorry for kind of throwing you under the bus there a little bit, but I Hello?

I, I, I hear you. I hear you loud and clear. Yeah, you have to underpromise and overdeliver. I absolutely appreciate that. So, , so where can we find out more? 

[00:34:00] Gaurav Shah: Rocket pharma.com. There's also you can follow us on LinkedIn as well. 

[00:34:05] Kevin Folta: Excellent. So thank you very much Dr. Garrahan. And if when the next big innovation happens, I really, really hope you come back and talk to us about it.

Whether it's how the existing therapies move forward and are changing the lives of people, or as new things show up on your radar that you wanna chase please reach out because I just love this topic and I really appreciate you being with me here. 

[00:34:26] Gaurav Shah: It was great talking to you, Kevin. Really enjoyed this conversation.

[00:34:30] Kevin Folta: And as always, thank you for listening to the Talking Biotech podcast and I think we can look forward to a much more sunny future where these are the tip of the spear of these therapies really hitting home. In conjunction with things like the innovations around sickle cell disease and other approaches with rare diseases of the eye that we've seen cured with gene therapy.

There's many new things on the horizon that will eventually grow. And to the more common illnesses. So keep the orphan diseases on your radar because they are a harbinger of good things to come. Thank you so much for listening to The Talking Biotech podcast, and we'll talk to you again next week.