Bobby Gaspar 0:04
"It just crystallizes to just one thing for me, trying to improve the lives of individuals with genetic diseases through this gene therapy."
John Simboli 0:14
That's the voice of Bobby Gaspar, founder and CEO of Orchard Therapeutics, headquartered in London, with U.S. headquarters in Boston and offices in California. Listen in now to hear Bobby's thoughts about leadership, and how Orchard Therapeutics is pursuing the opportunity to change medicine and change the lives of people with severe genetic diseases. I'm John Simboli, you're listening to BioBoss.
John Simboli 0:43
Today I'm speaking with Bobby Caspar, founder and CEO of orchard therapeutics, headquartered in London, with U.S. headquarters in Boston and offices in California. Bobby, welcome to BioBoss.
Bobby Gaspar 0:56
Thank you, John, thank you very much for inviting me on and it's a real pleasure to have the opportunity to talk to you.
John Simboli 1:04
Bobby, how did you find yourself at Orchard Therapeutics?
Bobby Gaspar 1:07
I'm a physician by training. So I'm trained as a pediatrician, I was very interested, even in my medical school days, about genetics. Even at a very early stage in my medical training, I did a science module on genetic engineering. So this was back in 1986, I think it was, so even then I kind of had a kind of kernel of interest in genetics and the power of genetic manipulation.
Bobby Gaspar 1:44
But the story really starts when I came to a specialist children's hospital in London, Great Ormond Street Hospital, which looked after some very rare diseases. And when I was there, I was looking after children who had very severe immune deficiencies. So children who were born without immune cells, and who essentially would die in the first year, or so, of life without any treatment. And the problem arose from a single gene that was missing in their bone marrow. I can't tell you really the clinical impact upon me was extraordinary, because you had children in cubicles, essentially ravaged by infection, and they would either die from their infection, or they would die from the complications of the treatments that we were giving them, and only about 50% of the children would survive. So the treatment at that time was trying to take immune cells or bone marrow from a normal donor and give it to the child, so a bone marrow transplant. And that had complications because you're giving these children somebody else's cells, those cells see the children qs different, they cause something called graft versus host disease. And that causes all kinds of complications. And they were, you know, six months, nine months, a year old, ravaged by infection, being given a treatment that could cause even more complications and a high death rate. It was just kind of heartbreaking to see that kind of disease.
Bobby Gaspar 3:28
I can remember a ward round where we went around with the professor at the time we passed one of these children. And the professor said, this child's going to have gene therapy, and this was in 1992. And I thought it was a joke that was played on all residents at the time. In case they fell for this because I just thought this is science fiction medicine. But that was the first child that was going to receive gene therapy in the UK and one of the first children worldwide. And the idea was not to use somebody else's cells, but to use their own bone marrow cells. And now to introduce a working copy of the gene into those cells. So having gone over that, I just thought, this is amazing. This is extraordinary that this is actually going to happen. After that, first that six months job, I actually didn't have a job to go to but I said I would stay around and work as a research fellow and they needed somebody to look after this child and to help with some of the kind of ethical applications and things like that. So I stayed on for six months initially to do that. And I became the child's own kind of personal physician, as it were. When we took bone marrow from the child, I actually had the cells, I took them to the laboratory in the Netherlands, they brought the gene-corrected cells back to London, I picked up the scientists from the airport, came back to the hospital, and then administered the cells myself to the child. So this is the first child of the UK to receive gene therapy. And it didn't work for that child, this was back in 1993. It didn't work for that child, but we learned a lot from doing it. And I was hooked at that stage. So I then went into the research lab, I did a Ph.D. learning about this form. This was a nascent era of what is hematopoietic blood stem cell gene therapy.
Bobby Gaspar 5:46
And I worked with a very, very close colleague and friend, who, we worked together, on then developing this for other conditions. And we took it from the laboratory to doing the relevant studies before then being able to put it into patients. And we treated our first patient in 2001. And again, it was another form of this very severe mutation the kind of bubble babies that you've heard about where children are confined to their, their bottles. And that child, again, incredibly unwell on intensive care came out of that, no bone marrow donor available, they entered the gene therapy study, and he was treated in July 2001. And now still comes back to the clinic every year with a functional immune system, having received his gene-modified cells back at that time. And it was just such an extraordinary, and it was rewarding on so many different aspects. The kind of scientific curiosity and the ability to take something that was scientifically so interesting. But also seeing the impact on that child and the family. And to see that you could actually correct a condition using this kind of approach and the difference that it made to the child and his family. And so we then developed that approach, this HSC gene therapy, not just for that disease, but for three other diseases and built up an expertise and experience in the laboratory that was doing this. And doing for multiple different diseases. There was a kind of fundamental view that this was the best way to treat these diseases, this was the safest way. And it allowed us to potentially correct or cure the disease.
Bobby Gaspar 7:46
And we then, in 2012, we had a new trial for another one of these bubble baby diseases. And we were doing this in conjunction with a very close colleague at UCLA. And as this trial evolved, the patients were being referred to us from, essentially, all over the world. Because, you know, it is a very rare disease, that patients come to us from all over Europe. At UCLA, they were coming from all over the US, I had a child come in from Australia to access therapy. And the bottom line was all these children survived. And virtually all of them recovered their immune systems as a result of the one-off administration of the gene-modified cells. And at that point, I mean, I suppose we felt, normally what you do is you do one trial, and it kind of works. And then you do another trial and you kind of work. Here was a trial that was working, and it was making such an impact that we felt we had to now make this available as a medicine for patients wherever they might be. And it had to move from one center in London, one center at UCLA, to being made available much more extensively. And that's when I think we felt Well, this now has to go to a commercial entity that brings into place all the manufacturing infrastructure, the clinical infrastructure, the regulatory infrastructure, to make this into medicine, a new genetic medicine. And at the same time, the kind of commercial investment, were also starting to think about gene therapy as well. And so we had investors who wanted to do something like that, not just for one disease, but a whole series of diseases that were using the same approach. And that's when the idea of Orchard came into being and that's through the investment, through the licensing of the work that we had been doing. We then created Orchard as a company. And I kept my academic clinical role for quite some time, as well as being CSO with Orchard. But it got quite busy after a while, trying to do all of those things. And so that's why at that stage, I stepped over a CSO and then became CEO of Orchard. And so, it was never an intention to be CEO of a company, it was always the intention to bring what I feel is the best way to treat these diseases through this genetic therapy to patients worldwide. And Orchard, and being CEO of Orchard, is part of that as part.
John Simboli 10:42
Did you go through any tug, between wanting to remain a practitioner, a professor, and also realizing that if you didn't do this thing to create this company to investigate this and launch this it might not happen?
Bobby Gaspar 10:58
You've described it very well, actually, that was exactly what I went through. Whatever I do, nothing will be as rewarding as what we did with the patients that we treated. I mean, I loved it. I absolutely loved it. I would see the families when they first came in, counsel them about their disease, talk about what options were available, say that here was an alternative that we could use, it hadn't been trialed, it was in the process of being trialed. We couldn't give them guarantees, but we felt that this could offer a safer outcome, etc. And then, when you see the patients come back after treatment, and they recover their immune systems, and you can actually say to the families, you know, it's the same for parents, we think your child's fixed now, you know, and you don't have to worry anymore. I mean, that's the most rewarding thing as a physician that you can do. So to leave, that was very, very difficult for me, I have to say, but in the end, there was a limitation as to what we could do clinically and academically. It would mean that we were constantly only being able to do a limited amount. And here was this opportunity to take this and use it for patients on a global basis. And do it not just for one disease, but a whole series of other diseases, as well. And although I still miss that kind of personal contact, and I really, really do miss that, I do feel to myself, were successful at what we do that we're going to make a bigger difference,
John Simboli 12:31
is it the case that at the same time that you're the CEO and moving Orchard forward, that you retain your contacts with your academic connections and physicians around the world? You're focused on your own work, but you're no doubt still very closely involved with practitioners, right?
Bobby Gaspar 12:50
I was very lucky, as a physician and a scientist, to be working with some of the leading academics in this field. It was such a small field that we were all connected. We would see each other at multiple meetings during the year and we would exchange ideas, we would collaborate, we wrote scientific papers together. So that was a very, very close network. We were all working in slightly different but connected areas, and of course, I was championing this gene therapy approach. And so, to a certain extent, I am very connected with them still. So, a number of those colleagues are on the Scientific Advisory Board of Orchard, we are conducting trials with our collaborators from the start, and some of their programs were licensed into Orchard as well. So it is still a very connected community.
John Simboli 13:51
And that makes me think of conversations, a couple of times, where I've talked to founders and CEOs. I've heard several people describe what a CEO is, as, they say things like, you know, really, I'm a Chief Education Officer, which I think is an interesting idea. I know, executive means you make decisions, but in particular, I would think in gene therapy, that a good deal of your work, over and above scientific work, has to be to help people understand what the need is and how it could be filled. Is education a large part of how you spend your time?
Bobby Gaspar 14:26
Yes, I mean, I think education in many different ways. And I've always loved teaching. As a clinician, as an academic, giving lectures, etc, workshops. And so, I love it and I do think there is a need, because I think, in the role that I have within Orchard, everyone needs to be connected with what we're trying to do, what kind of difference that we are making, to patients. And that difference is being made through a science, a technology. And the more our staff, everyone at Orchard, understands what that is the potential of hematopoietic stem cells, the way we introduce genes into those cells, why giving the cells once could potentially correct an individual for their lifetime, how that is different from the way other gene therapies are done, for example. The more they're connected with that, the more they feel, I hope, motivated by what we're trying to do, understand what we're trying to do, talk about what we're trying to do, to others. So that education I try and get across as much as possible, they're probably sick to death of me talking about hemopoietic stem cells and everything else. But you know, but to a certain extent, I do feel that we're a small company, and in a small company, you want everyone to be connected as much as possible, to what the company is doing. And knowing as much as you can, understanding as much as you can, helps connect people to what we're trying to do.
John Simboli 16:19
When you describe what you're doing, to people, how do you pull together all those strands?, Because they're obviously all connected—professor, physician, leader.
Bobby Gaspar 16:30
It's about one thing, John, and it just crystallizes into doing just one thing, for me, and that is trying to improve the lives of individuals with genetic diseases, through this gene therapy. And that's what I think my whole professional career has come down to, right from the very start. Back, when I saw that child at Great Ormond Street, and thinking about how we can best do this, trying to make that science fiction, that I thought at the moment, into a reality, not just for that disease, but a whole series of diseases. So it has been a pretty singular path to try and achieve that then. On the way, I have had the most extraordinary opportunities of interacting with some incredibly bright, smart people. Just the reward of being able to talk and be with families and be involved in some of the most important decisions than they'll ever make about the life of that child. And, you know, being able to travel and communicate what I believe in so passionately, to many other people, and hopefully, tell them that there is a way now of treating these diseases, there is a technology, there is a science that is evolving, that maybe has the potential to treat many other diseases as well. So, to have been involved in this for such a long period of time, it's just given me opportunities that I never thought I would have.
John Simboli 18:19
Bobby, can you remember way back when you were eight or nine or 10, or something like that, what you wanted to be? I think for most of us, we were trying to be something we thought our parents wanted us to be.
Bobby Gaspar 18:29
So my father was a doctor, and my mother was a doctor. My father was a psychiatrist, actually, my mother was a GP. And I was the oldest of five. My mother once sat us all down and she said, I don't care what you do, as long as one of you is a doctor. Four out of the five of us became doctors. And we were just surrounded by doctors. You know, there was no other career. I do you remember this, at the age of seven, being asked, you know, we were all at school and being asked, what is what do you want to do when you grow up? And of course, that's what I wrote. I wanted to be a doctor and I wanted to save children. That was what I wrote when I was seven years of age. I remember wavering, at some point, and being called to the headmaster's office and you had your chat about what you want to do. And he said, Gaspar, you're very good at Latin and Greek and I think naturally, a career in classics and law would be the right thing for you. But I know you're going to do medicine. So there's no point in me saying anything. Why am I talking to you anyway?
John Simboli 20:01
What do you see when people say who is, as opposed to what is, who is Orchard Therapeutics?
Bobby Gaspar 20:07
Orchard Therapeutics is a family of people who are trying to do the same thing and that's what I want people to feel about it. It is a company, a group of people who are connected to achieving the same thing. That's what I've talked about before, it's about bringing these genetic therapies to patients. And I just want to kind of instill that into everyone who comes to work for us at Orchard.
John Simboli 20:35
What's new at Orchard Therapeutics?
Bobby Gaspar 20:37
The thing that defines us, really in respect to that question, is that we're one of the first companies that are bringing these genetic therapies to the world. And so I feel we're right at the forefront of making this happen. And we're making this happen for a whole series of diseases. And, you know, I feel very much that's part and parcel almost of what I've been doing the whole way along. You know, we were developing this in the lab, for the first time, we were putting it into patients, for the first time, we received the results, the first time, going through the ethical questions, for the first time, doing the manufacturing in the academic setting. That was a kind of, you know, a first, as well. So I think Orchard is there in the same position, we're going to be getting these approved for the first time. These will be some of the very first blood stem cell or hematopoietic stem cell gene therapies that will be approved. They will be the first that will be commercialized, they will be the first that will be reimbursed. So we're at the forefront of making this happen. So, you know, what's new, almost everything is new, you know, because nobody's done this before. Nobody's really done this before. So it's challenging, it's invigorating, it is stimulating, it's difficult. But you know, that's where we're at is, as I say, getting these as approved genetic medicines, and making them available worldwide. That new. All of that is new.
John Simboli 22:27
Many of the founders and CEOs I speak with when they tell me that, and I've observed when they present to people about what the company is doing, and what your vision for the company is, a certain percentage will come back and there'll be a meeting of the minds, and they'll talk about going forward. A certain percentage will come back and say, I'm just not interested. And then a third group will come back and say, oh, now that I understand it's x, let's talk about that. And you'll be thinking to yourself, but that's not what I said. I said, Y, not X. So when people do misperceive the story, what did they get wrong? And then how do you help them to figure it out?
Bobby Gaspar 23:04
That's really interesting. I think there's two things. One is the perception that all gene therapy is the same. There's gene therapy using the two main areas, there's many areas, but you know, one area, for example, in vivo, AAV gene therapy. So there's a lot of interest in that, there's a lot of programs and a lot of companies. And sometimes, all gene therapy could be seen as AAV gene therapy. But in fact, what we have is something very, very different. We're using, and I like to talk of it as HSC gene therapy or hematopoietic poetic stem cell gene therapy, where we're essentially taking the cells out, we're modifying them, and we use a lentiviral vector to modify the gene corrected cells. And we're returning those cells back to the patient.
Bobby Gaspar 24:10
So there are a number of different aspects and one is that we're taking the cells out. So it's ex vivo, that's when we're manipulating cells when it's manipulated outside the body. We're using a lentiviral vector to carry the genes into the cells and then we're returning the cells. So that's very, very different to AAV gene therapy. And so I think one important thing that I try and get across is gene therapy comes in many different forms. And you can almost think of it as a toolbox, as it were, with multiple different tools. And when you're trying to correct a disease or do a job, you will use the right tool. And so you use the AAV for certain jobs, for certain diseases, and HSC gene therapy is better suited for other diseases. And you pick the right tool for the right disease. And I would like that concept, really, to get across, that HSC gene therapy is best suited for certain diseases and these are the advantages.
Bobby Gaspar 25:17
The other thing, I think with the Orchard is people think, Oh, well, it will treat this disease and it can treat this disease and this disease. What they don't fully get, and this is something again, I'm trying to impress more, is that it is an approach or a platform that could be used in many different areas. And once you understand, or you get the concept of the platform, the concept of using hematopoietic stem cells or gene-modified hematopoietic stem cells, then you see that the potential is enormous, really. You've got so many different diseases. So rather than thinking of Orchard as a collection of this disease, this disease, this disease, I'd like people to think of Orchard as a company that's using HSC gene therapy. And the potential is enormous to treat whole series of different diseases.
John Simboli 26:09
You just talked with me about the idea of a toolkit and how different approaches can work for different needs. For the HSC platform, can you describe how it is that that is a useful approach?
Bobby Gaspar 26:25
These cells, the cells that we're using, they're the kind of early cells of your bone marrow. And they're the cells that will give rise to all the cells in your bloodstream. So they give rise to your immune cells, they give rise to your red blood cells, they give rise to cells called platelets that make your blood clot, they also give rise to some specialist cells that can actually migrate into your brain so they can actually cross the blood-brain barrier into your brain. So the way I explain it is that if you take these blood stem cells, and introduce a gene into these blood stem cells, you've got a route to deliver a gene to all of these different cell types. So it means you can correct immune cells and, so, correct immune disorders. It means you can correct red blood cells and so correct red blood cell disorders like polycythemia. And it means you can correct these specialist cells that get into the brain, naturally, get into the brain, and therefore gives you the opportunity to correct certain neurodegenerative conditions. And we've been able to do that within our programs at Orchard.
Bobby Gaspar 27:37
And the other really important aspect is that these blood stem cells have the ability to self-renew, they renew themselves, as well as giving rise to the cells in the bloodstream. And the way we introduce a gene into these blood stem cells means that the vector that we use integrates the gene into the blood stem cell. So every time that blood stem cell divides, whether it's renewing, or whether it's becoming a cell in the bloodstream, that genetic information is being passed on. With every cell division, the genetic information is being passed on. And that's why when you give these gene-modified cells back to the patient, they are renewing, they're passing on their genetic information. And that's why a single administration can have such a long-lasting effect.
Bobby Gaspar 28:33
And so we've seen now that from the patients that we treated, and some of the programs at Orchard, the first patients we treated were treated over 20 years ago. And as a result that once-off administration of the gene-modified cells, and the self-renewal, the passing on of the genetic information, they've been corrected for that period of time. So this is why we think it's potentially a curative approach. We have seen that when we give gene-modified stem cells, hematopoietic stem cells, back to patients, we don't see any diminution of effect. So they continue their self-renewing, they're giving rise to gene-corrected cells in the bloodstream. And that doesn't go away. And we see the level of correction being sustained for many, many years. And in the world of bone marrow transplantation where, for example, you're getting somebody else's blood stem cells, that effect has been seen for 50 years now. That's when the first bone marrow transplants were conducted, 50 years ago. That experience tells you that if you give normal hematopoietic stem cells to an individual, and in our case, it's gene-corrected hematopoietic stem cells, you can have that duration of effect, and so that's why it is potentially curative.
John Simboli 29:52
I would think that there are people who would hear that and say, well, that sounds magical, sounds wonderful, I hope you achieve it, Bobby, I wonder if that's just for a narrow, narrow set of genetic subpopulations. But it seems to me based on what you've told me that the platform could conceivably address, not just rare, or ultra-rare diseases, but other diseases, too, Do I understand that?
Bobby Gaspar 30:17
I've worked with rare diseases all my professional life. And they can teach you a lot. The fact that in these ultra-rare diseases that we're currently working on, there's a single gene that is missing, you know, that if you replace that gene or the ability to replace that gene, you could correct that disease. So it's very tractable from that perspective. And we've learned a huge amount in using this HSC gene therapy for these rare conditions. The first thing to say is, there are many, many of these ultra-rare conditions that could benefit from HSC gene therapy, and we already have quite an extensive portfolio at Orchard, but we could add to that portfolio with a whole series of other ultra-rare conditions. So it could benefit many other diseases as well.
Bobby Gaspar 31:05
But the learnings and the kind of the understanding of the mechanism, I give you one example of this, is a very rare neurodegenerative condition called metachromatic leukodystrophy. So, MLD, for short. So there's a single gene that is missing, the lack of that gene means that it doesn't make an enzyme. And the lack of the enzyme means that it builds up certain toxic substances that predominantly affect the brain. So they affect neurons. And so what happens is these unfortunate children are born, normally, they develop, and then after the first year of life, or so, they start to lose their function. So when they were sitting, they stopped sitting. They were walking independently, they can't walk anymore. They lose the ability to communicate, to talk, to interact with their families. And that's because their neurons, their brain cells are degenerating because they've built up these substrates. And it's a devastating condition in the most severe form. Nearly all the children will die by 10 years of age. So that's how difficult it is. What we have at Orchard is a therapy, or we have been developing a therapy, for MLD. And what we're seeing is that if you take the child's hematopoietic stem cells introduce a working copy of that gene into those cells, you're actually overexpressing that enzyme through this, and you give it back to the children intravenously, that a population of cells, those hematopoietic stem cells, are able to cross into the brain, they're able to engraft in the brain, they secrete the enzyme in the brain, it's taken up by neurons. And now it prevents those neurons from degenerating. And it what it does is it allows, and we've been able to show this, that it preserves their cognitive function. These children now, some of them are learning in the same way as children of their own age, they're able to play with their friends, they're preserving most function. And it's just a remarkable difference between untreated children and children who have now received this therapy. It's a huge understanding that you get from, one of the clinical impacts, understanding the biology of what's happening there, and the fact that these HSCs are able to cross into the brain and secrete the gene in the brain.
Bobby Gaspar 33:45
And we have some other earlier-stage programs of this nature, again, ultra-rare, that we are taking forward at Orchard. But it also tells you that you can express other genes in the brain. And so can you now do this for other much larger conditions as well? And so you now have the potential of introducing a gene into HSCs, giving it back to patients, allowing it now to migrate into the CNS and then targeting specific genes, proteins, and therefore preventing neurodegeneration in much larger indications. And so we're now working with a brilliant scientist who I've known for a very long time in Italy, in trying to address some much more common disorders of neurodegeneration. The first one is going to be a certain subset of Frontotemporal Dementia, which after Alzheimer's is the most common form of dementia, but it's that understanding in rare diseases and ultra-rare disease, understanding the biology of this HSC gene therapy that now allows us to move forward to try and address more common conditions and that's just one example. But there are other parallels in other areas as well.
Bobby Gaspar 35:02
But really, we have at a late stage in the development of MLD. And that has been filed with the European Medicines Agency. And we filed in November last year, we've been working through, as per their procedure, working through the questions they sent back to us. So, hopefully, we will get a potential approval in the fall this year, which will be in a very, very exciting moment for us as a company. And most importantly, we'll be able to bring this medicine to patients who at the moment don't have anything available for what is a very devastating condition. So that will be a very exciting moment, I think. And we also have late-stage programs in another condition called Wiskott Aldrich syndrome, which is an immune deficiency, and also causes platelet problems, as well. And again, we're working through dialogues with the agencies, and hopefully, we'll be able to file that next year in the US and in Europe. We have, as a company, some very exciting times ahead of actually taking what has been done over many, many years to being approved and then being commercialized, while at the same time building a pipeline using this HSC template for other rare diseases, but also, in the longer term for larger indications as well.
John Simboli 36:28
Your life must be filled with a lot of precision around the details of getting to the next step of all the things that you're working on. But I'm wondering if, at this point in your career, in your life, do you still find yourself on a weekend, a time when you're with your family, some time away from the immediate need, when you're thinking, boy, if this works the way I hope it will, I can really help some people?
Bobby Gaspar 36:51
You have to have a bigger viewpoint, obviously, but also, it only gets done by the detail. You have to make sure the detail is right, as you said. And whether it's on the scientific level, it's on manufacturing, whether it's on all kinds of different areas, you have to get the detail right in order to progress forward. But at the same time, the bigger view is that you want to change the way these diseases are treated, you want to change the landscape. And I do you think that if we can go in the direction that we're heading, we will change what these diseases are like. Because it's not just about treatment is when we're also working on diagnosis and early diagnosis as well. And so you can get to a stage where you can identify these patients extremely early, even after birth, through newborn screening, you can prevent them from getting the disease and you can give them a therapy, that means that they'll never get the disease. So you've completely almost eliminated the disease for that individual. And that will happen, that will happen through these advances. And I just think that's an extraordinary opportunity that, I, as a physician, have an opportunity to be part of. You're changing the landscape of a disease.
John Simboli 38:20
Bobby,. thanks for speaking with me today.
Bobby Gaspar 38:22
So thank you very much. It's been a real pleasure to be able to talk to you and I really welcome the opportunity.
John Simboli 38:28
Every now and then we meet someone whose life has followed a singular path, a straight line from first falling in love with an idea to realizing a vision through a lifetime of work. Bobby Gasper told me, for him, it all comes down to one thing, trying to improve the lives of people with genetic diseases through gene therapy.
John Simboli 38:50
As I spoke with Bobby, I could hear the passion in his voice as he talked about his first experience as a young physician in London, caring for children who had a very severe immune deficiency, children born without immune cells who would die in the first year or so of life, without any treatment. At that moment, Bobby spoke of envisioning a future, "When you see patients come back after treatment, and they recover their immune systems. And you can actually say to the child's parents, we think your child's fixed now, you don't have to worry anymore."When Bobby said to me, "That's the most rewarding thing, as a physician, that you can do. It was clear to me this was a man with a calling.
John Simboli 39:32
And now as CEO at Orchard Therapeutics, Bobby continues his lifelong pursuit. As he says, "It's not just about treatment, it's also working on early diagnosis, to get to a stage where it's possible to identify patients extremely early, even after birth through newborn screening, and prevent them from getting the disease by giving them a therapy. That means they'd never get the disease." To me, this sounds like it would be a dream come true.
John Simboli 40:01
I'm John Simboli. You're listening to BioBoss.