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At Davos’ Open Forum “Where Biology Meets Choice,” leaders from academia, biotech and global youth networks explored how rapidly advancing biology could reshape health—and society—by 2050. Oxford Vice-Chancellor Irene Tracey framed the opportunity and the risk: science is accelerating, but “there’s sadly always a dual use,” demanding earlier, better ethical training and public dialogue.
ProQR CEO Daniel de Boer offered a plain-language primer: DNA is transcribed into RNA, which becomes protein; disease-causing “mistakes” can be targeted via gene editing (DNA) or RNA editing (RNA). Nobel laureate Victor Ambros positioned microRNAs as part of an “ancient suite of mechanisms” enabling cells to regulate hundreds of genes, with therapeutic spinouts like siRNA that can “knock down a toxic protein.” Natalie Edwards described synthetic biology as building gene circuits “analogous to the game Legos,” urging democratized science literacy, especially in Latin America.
Panelists repeatedly returned to access and affordability. Ambros warned high-precision therapies risk widening inequity unless designed for “accessibility and low cost.” De Boer argued AI and platform approaches could lower failure rates and, over time, reduce costs. On ethics, the panel rejected non-therapeutic embryo editing; de Boer likened misuse to using “a brick to hit someone.” Looking to 2050, hopes ranged from women’s health becoming obsolete as a category to truly predictive personalized medicine.
Well, a very good evening to you all, and welcome to this open forum where we're going to think about biology of the future and what the world is going to look like in 2050. It's wonderful to see a packed auditorium. So thank you for your interest in this subject. And let me at the start, encourage you to be thinking throughout the discussion that we will be having questions that you might have for our expert panel. So start thinking now about the questions that you would like answered or things explained. Even in this really exciting space of biology, DNA, RNA editing, synthetic biology, what it means to be human and what the world and what Davos will look like in 2050. My name is Professor Irene Tracey, and I'm vice chancellor or president at the University of Oxford. I'm actually a scientist by background. I've spent 30 years of my career developing neuroimaging technologies to understand the human brain, and then specifically to understand them in the challenging problem of acute and chronic pain. So I've spent my career as a neuroscientist, but now I don't run the lab, I'm running the university. But of course, we have lots of exciting biology going on there. So I shall be drawing on my background as a scientist and the career I have had in some of the probing and the questions I have of this magnificent panel before us. What I'm going to do is just briefly introduce the panelists, and then I'm going to invite them each to speak up to five minutes about their background and how they see the future for biology, particularly drawing from the careers they've had and what they're currently working on. And then we'll get into it with some questions and between ourselves, and then we'll open it up at the end for about 20 minutes of questions and answer from yourself. So immediately to my left here, I'm delighted to introduce Natalie Edwards. Natalie is a global shaper in the Santiago hub and a molecular biotechnology engineer. She is also the iGEM ambassador for Latin America for 2025, and in that role, she promotes the advancement of synthetic biology across the region and continues to support these efforts throughout the year. Currently, she coordinates the School of Engineering undergraduate research program at Pontificia Universidad Catolica de Chile. Please excuse my terrible pronunciation. To her left is Victor Ambros and Victor is a professor of molecular medicine at Massachusetts Medical School. Throughout his career, he worked with David Baltimore, a co-recipient of the 1975 Nobel Prize in Physiology or Medicine, and with Robert Horvitz, who shared the 2002 Nobel Prize in Physiology or Medicine. He has received numerous honors for his scientific achievements, including the 2024 Nobel Prize for Physiology or Medicine for his co-discovery of micro RNA, short single stranded RNA molecules that are now understood to play critical roles in post-transcriptional gene regulation. To his left is Ava McClellan, and Ava is a global healthcare executive at Roche and the co-founder of the foundation unlocking Eve. She is a passionate advocate for reimagining the new health economy and ensuring that innovation serves humanity, especially women and people who have historically been left behind in health and opportunity. She is also the recipient of Canada's Life Sciences Top 20 under 40 award. And last, but by no means least, we have Danielle de Boer. Daniel is founder and chief executive officer and board member of Proqr Therapeutics, a biopharma company dedicated to developing RNA editing medicines for genetic diseases. He is a passionate advocate for rare disease patients. He also has founded several technology companies, and acts as a strategic advisor for a range of pharmaceutical companies. He was named Emerging Entrepreneur of the Year by Ernst Young, and is a member of the Young Global Leader program at the World Economic Forum. Can we please give our panelists a round of applause? So, Natalie, may I start with you to kick us off and just give us a brief overview.
Perfect. Oh, hello, everybody. I'm so thankful for being here. This is my first Davos. And, besides being a global shaper like Irene said, I work at the Universidad Catolica de Chile and where I promote, you know, students going into research, especially in different engineering fields. But my background is actually in biotechnology. And I was, presented to the world of synthetic biology, which I was so interested in and just realized that it's not developed, that much in my country, which is Chile. And that later took me to work in a different direction, more on applied science and developing a treatment for psoriasis based on, products of stem cells. You know, to just improve, the skin disease of other people that are affected by it.
Great. Thank you so much, Victor.
Did you want us to speculate on the life sciences in 50 years or something.
Like that. Well we'll get to that. We'll get to that. But maybe just give a little bit of background about yourself and your thoughts just, just, just very briefly. And then we can get into the questions.
Yeah. So I'm a basic scientist, meaning I have a PhD and I do research into animal development. And in fact, I've been I've had a lab since 19. When was it 1984 or something. And I've been funded by the US National Institutes of Health for over 40 years to study the development of a little nematode, Caenorhabditis elegans, C elegans. So it's a worm that only gets about a millimeter long and only takes two and a half days for its life cycle. But you can see every cell in the animal and watch every cell as it divides, as the animal develops. And, this has been an organism that folks all around the world, hundreds of labs study. And it's been the source of four Nobel Prizes from work in C elegans. And so one of the things I'm here, I like to, sort of see myself as a cheerleader for basic science and for, the idea that there is an enormous amount still to be learned about living things. And I like to think of it this way, that probably there is more to be learned than we know about living things. There's probably more to be learned than we can imagine. And the reason is because in the life sciences, everything seems to be a surprise. Right? And so daily in the labs, up and down the corridors of our university and your institutes and your university, people are encountering living systems with experiments and getting data that surprised them and caused them to challenge their presuppositions, presuppositions. And the dialogue in these labs and in these institutes is so exciting. And so because so we know in this moment, basic science research is unlocking new perspectives and completely new ways of looking at organisms. So, I think that's what I want. You know, I think it's very important to try to remind ourselves that we're not in a place where we've learned everything we need to know, and now we just need to apply it.
Great. Well, I'm going to get into the whole sort of how do we keep curiosity alive and the challenges to basic science a little bit. Victor with you later. But, Ava, please, over to you.
Yeah. Thank you. And it's a pleasure to be here with such amazing panelists and I'm sure many scientists in the room as well. So my name is Ava McClellan. I love this quote. I have no special skills except I'm insanely curious. And I think that is what drew me to biotechnology. I still remember being in grade school when we had two paper plates, and with spaghetti and pasta, we had to basically recreate mitosis and meiosis, you know, the spindles and the fibers. And I thought, are these people for real? Like my teachers? I thought, are they for real? I was created from a single cell that multiplied into multiple cells that that became a tube, and then that tube differentiated into every single thing I have right now. I still remember this moment, my seven year old self, and you're like, wow, that's what I'm very curious about. And fast forward into university, I studied life sciences at the University of Toronto, where a lot of research has been done. You know, Banting and Best invented insulin, for example. And there I got very curious about biotechnology. And and I still remember sitting fast forward from seven year old to 17 year old sitting in my bio 150 class at the Convocation Hall at the University of Toronto. There's like 2000 people all wanting to be scientists. And the, the, the news that year, which is more than 20 years ago, was that the human genome sequence was complete. And that's extraordinary. And I knew it there, because what that meant is we had the sequence, the genetic sequence to the human genome. And I knew then, as a 17 year old student, that that would change the trajectory of human health 20 years hence. And I wanted to be part of that as well. So 20 years later, here we are today. I then decided to go and work for, an industry that was really pioneering at that time, recombinant technology, because it really interested me is the fact that until then, only chemists could make medicines. But now biologists were able to make medicines too. And so I continue to be curious, and I'm very excited to hear from all of us and have this conversation of what the next 20 years is going to look like.
Great. Thank you. Ava. Daniel, over to you.
Yes, thank you for the organizers for inviting me and happy to be part of this amazing panel. Yeah. So I'm a serial entrepreneur. I love creating things that weren't there before, but first and foremost, I'm a father. One of my children was born with a rare genetic disease now 16 years ago. And that made me decide to pivot from being a tech entrepreneur to exploring drug development, biotech and biopharmaceuticals. That led to me starting this company that I run today as the CEO, Pro-cure Therapeutics, which is a company that's focused on developing RNA editing medicines. So we made an invention that allows us to change the human genetic code at the level of RNA, which allows us to modify how certain proteins function, take away genetic defects that lead to genetic diseases, like the one that my son has. And it allows us to also think about how we can prevent disease and how we can help people live a longer and healthier life. I think it's a really exciting time to be in biotech. I think there's a tremendous amount of opportunity. I think in in the current time that's being amplified by artificial intelligence, that allows us to see through some of the unknown unknowns that we have in biology and can help us to in a in a much more predictable, predictable way, finds molecules and medicines that can help us to, I think, in the end, develop better, more and more, productive, more efficient medicines for patients. So I'm happy to be part of this panel and look forward to the discussion.
All right. Well, thank you, Daniel. And, I'm sure I speak on behalf of everybody that we wish you and your son all the very best going forward. Maybe what we'll do is start more on the sort of obvious benefits of this future biology, which is, of course, the application to disease and what we call the translational side of the basic science. But I would like to then go back to the basic science and explore a little bit some of the challenges there and the opportunities and the excitement and the curiosity, but the ethical challenges that that brings. Maybe we should just get some basics right, though, because I'm mindful that this is an open and public forum, and we're using these words gene editing and RNA editing and scripting. And maybe, out there, you're not quite sure. So, Daniel, I'm going to ask you, you gave a little description, but could you just sort of, in a nutshell, describe again, just the basic building blocks of DNA, makes RNA, makes protein, and what we mean by gene editing. And maybe I'll ask you to weigh in as well, from an industry perspective, how that field has just evolved so rapidly that we can now code our whole human genome, you know, very cheaply and very quickly, whereas that was the major goal for many, many laboratories only, just what, 20 years ago. But Daniel, can you just get us all on the same page in terms of our biological understanding of gene editing?
As the only non biologist on the panel to explain the basic biology.
Here, you'll be as she often that's better for explaining. Exactly.
So as I just alluded to about 20 years ago, the scientific community figured out a code for human life, which is the human genome. And in the human genome, it's essentially, inscribes how all of our genes, how all of our cells function. So our bodies are made up out of a whole bunch of cells. Every cell has a has a nucleus. And in the nucleus there we have DNA that provides a specific code for your cells. The DNA is continuously copied into RNA, and RNA is used as a blueprint to make proteins and proteins make you make your cells function and make us living human beings. Essentially, in genetics, we can find in that DNA, all the features that we have, the way we look, the way we, grow, etc., but also diseases often captured in that DNA. So certain mistakes in the DNA, can create disease or can create a predisposition for certain disease manifestations. Those can be targeted with gene editing. By modifying the DNA, they can be targeted with RNA editing by modifying the RNA. So the blueprint or they can be treated with other medicines that target a protein. Ultimately. And I think today we will focus on the first two gene editing and RNA editing.
Yeah. Thank you. And maybe you can just tell the journey that's been on between the public and private sectors. So industry as well as academia, how we've developed then at scale the technologies to do this at ease as opposed to something that's just in our private research labs, where we're tinkering around with trying to achieve this goal.
Yeah. No, absolutely. And I think, this is one of my favorite topics, you know, the partnerships with academia and industry. Academia has this thing called tech transfer offices. I would say, you know, the the scientists there, you know, scientists in academia, my perspective is scientists, academia, they're genuinely curious people, and they want to find answers to unsolvable questions. And so they get to do that for as long as they are doing their work. They get to play around and find out really cool things. And, and many of those cool things have real application to human life. One example of a cool thing that had an application to cold to life. Before we get into gene editing, my colleague Daniel talked about DNA going to to becoming a protein one protein that we, some of us have, but some of us are missing is insulin, for example. And you know, those those people who are missing insulin, they don't have the DNA code to make that insulin that all of us enjoy when we have some food. And that helps us control our glycemic control. And so scientists somewhere in the lab have have to give University of Toronto a little bit of a shout out here. Banting and best discovered how to make, you know, synthetic. You know, how to reproduce, use recombinant technology to make, protein that insulin that humans can then take. And today that is taken for granted. Diabetics all around the world live a very normal life. And that's a very good example of how a university discovered something that was needed and then worked with industry to think about, well, how do we create, this at scale? So recombinant technology is essentially taking that DNA, putting it into a non-human kind of Chinese hamster ovary or something, and then producing it at scale and democratizing it to everyone. And so that's one example of how universities really then work with industry and through tech transfer offices or partnerships. And I just think it's wonderful. I think we need to be doing way more of it. I think we need to find models where we can do it faster and more collaborative. And one of my other dreams is that universities collaborate with each other even more. So that would be wonderful for human progress.
Yeah, well, we can get into that, maybe. And I'm sure Victor will have things to say about the the desire to have more collaboration from a basic science perspective and sharing of data and other aspects. So when we're using the word recombinant, it basically just means recombinant technologies recombining different combinations in order to be able to produce things. And again, in the first instance, as this discussion, we're thinking very much about some of those benefits that are brought to patients with diseases. And and again, particularly some of those maybe rare diseases, the public private partnership is so essential. And I think we are seeing more examples of public, public, public private. If I think about our university, we produce the vaccine for Covid, for the pandemic. And again, that was a wonderful example of a public private partnership. There are things that we just can't do at scale in the private in the public sector. But we can be nimble and we can develop things quite quickly, often drawn from decades of investment of basic research. As I often say to my government, you've got to have something to translate, and that means you've got to invest in the basic research. Otherwise the pipeline runs out. So we've got a few definitions sorted. I hope those are really clear for you all. Victor won the Nobel Prize, and I'm going to come back to the question I know all of you will want to know is how does that work when you get the phone call? But maybe we'll leave that towards the end, because I know that every Nobel laureate gets that question. So we're all dying to know. But let's let's keep with the science for now. MicroRNAs, could you please just explain to the audience what they are and why are they important?
Yeah, microRNAs are part of a, an ancient suite of mechanisms that operate inside the cells of almost every cell of every kingdom of life. And the focus of it is a protein called argonaute. And that ancient protein has evolved, a way of living where it associates with a short RNA, sometimes a short piece of DNA, depending on whether it's a bacterium and so forth in the systems that we study, which are animals. And as I mentioned, I studied C elegans, the Argonaute associates with a small RNA that's produced from another part of the genome of the, of the animal. And that part of the genome makes a microRNA and then that argonaute microRNA complex can now go around in the cell and regulate the production of protein from sometimes hundreds, hundreds of different messenger RNAs. So this is a vast network of communication that happens, between genes in our genome and other genes in the genome. So it's part of a regulatory apparatus that enables cells to really function minute by minute, second by second, day by day, and how cells can stay the way they are if they need to or change as, as would happen, for example, during development or wound healing or something. So microRNAs are part of all that apparatus of gene regulatory mechanisms. It's also closely related to another kind of, of basically a defense system that cells have. And in this case, the argonaute associates with a different class of small RNAs, and that small RNA argonaute complex surveils the cell and detects foreign genomes. And there's several different kinds of small RNAs that do that. And that's important because these so-called small interfering RNAs, or siRNAs, are a very prominent and exciting mode of therapy where, the, the the aim is not to edit the RNA, but to sort of to kill it. So if you have a toxic gene product of some kind, a protein that's not desired in the, in, you know, in a disease context or indication context, it can be knocked down by treating the individual with a completely synthetic small RNA that's been synthesized, you know, on a machine. This is very analogous to what you know about the, messenger RNA therapy, vaccines, where the messenger RNA, which is a long RNA, it's going to make a protein that you want to express, that's capsulated in a, in a little, microcapsule. And that is delivered into the, into the person. And that makes the protein that you want, the small RNA would be encapsulated in an analogous little capsule, and it's administered to the patient with the aim of producing the small RNA in the cells to knock down or eliminate a toxic protein. So it's the opposite of messenger RNA therapy. Messenger RNA therapy is where you want to produce something that's desired, be it a vaccine or a missing protein of some kind. And the small RNA therapy is where the objective is to knock down a toxic protein.
Right. That's fantastic. It's a really clear explanation. And just maybe one more explanation to go. Natalie, in your bio, you again have been part of what we call synthetic biology. Do you want to just explain to the audience what that means?
Sure. Well, so iGEM stands for International Genetically Engineered Machine, which is a foundation that for the last 22 years, has promoted the advancement, advancement of synthetic biology. And the definition of synthetic biology is, creating new circuits, involving genes to produce a new tools and systems that are not available in nature by itself. So that's why it is called synthetic. And by that you construct the networks using different pieces. So genes, promoters and creating a full system, which is very buildable. And it's actually, analogous to the game Legos. So it also goes by modularity, which each component can be, mixed with another to create different, a different system. And all of these parts are registered. So that's the main component of synthetic biology. And it comprises biology engineering and computing sciences to achieve this.
Great. That's a very clear explanation. Why do we want it? I can sense from the audience it's like really you're all creating new things that we've never experienced on this planet before. And goodness knows, as we go off to other planets and we develop and maybe discover completely new elements, will have completely novel, maybe, you know, types of structures that go beyond proteins and just completely new chemistry. So, so this is scary. And and whilst I'm excited by what synthetic biology can do from an intellectual level and just the sheer, you know, you know, sort of brilliance of it really. Can you each tell us why we think this is a good thing to pursue? Because more and more universities and public private are developing big synthetic biology efforts. And and I think this gets into some of the ethical things. Often as scientists, we are compelled because it's exciting. And you want to get the answer. And it's just something that you're very curious to do. And then we're often not trained to think about the dual use of what is often commonly a problem with every discovery we make, whether that's a chemical discovery or biological discovery or a technical discovery, is that there's sadly always a dual use for these things. We tend to focus on the positives. It's going to cure diseases, it's going to enhance life. It's going to give better quality of life, whatever that might be. There's always bad actors, and there's often a way to then abuse that discovery for, again, this negative outcome. And that's why we always refer to things as dual use. My own view is that we've got to get much better in universities in training. And I'll come back to this with what you're doing. Students, early career researchers, scientists to in parallel, think about what the consequences are of some of their discoveries. And I think we're seeing this writ large in AI and brain computer interfaces, lots of different areas where often it's out there. And then we think, oh, I didn't anticipate that. And then we're trying to sort of constrain it, hold it back, it's too late. So we've got to get much better at this. And it's hard to think, frankly, in the life sciences of an area where we've actually done this. Well, I'd love to know whether the panelists think there is a good example where we thought about the ethical implications, the dual use. We developed the technology, but we put guardrails around it and we controlled it. I struggle to think of one, but maybe to you. So can we make the case for why we want synthetic biology and some of the sort of challenges that maybe some of these things bring? And how can we get better at sort of making sure we don't ruin the world historically?
I mean, there is the Asilomar Convention, which was in 1975, or whatever it was, where recombinant DNA had just been, invented, let's say. And, and scientists were concerned. And so right at the outset, there was an effort, an organized effort to think about consequences. And that was the, you know, that it gave rise to the NIH guidelines on the use of recombinant DNA. And nowadays, I suppose there's, you know, in the Crispr gene editing field, I can't really speak to this directly, but, I mean, it feels like there has been a community reaction to, you know, the one case public case of editing children before they were born. And I feel like, like the community is thinking about it anyway and developing and trying to develop a consensus. If I could just briefly comment on synthetic biology, I'm one of the things, I don't know if it qualifies as synthetic biology, but we're really excited by protein design. I'm sure you guys are too. And so, you know, David Baker, won the Nobel Prize for protein design, which was an incredible breakthrough. If you want a protein of a given shape, they can design the protein sequence, and then the DNA sequence, and the organism will make it and the protein will fold into the shape that you want. And so this enables us for the first time to really design new contacts inside cells. Right. Which is kind of the basis for these circuits you're talking about. Right. And in biopharma there's a lot, a lot of excitement, I'm sure as well.
There is. Yeah, we call it lab in a loop. You know, how to design. And then I think it's exciting for many reasons, maybe to take that question around, you know, what is it serve versus the risk? You know, it's it's hard, right? Because self-driving cars, what's the risk? You know, and and then and what's the benefit? You know, maybe people who are, you know, have disability could now transport in a self-driving car before they couldn't, you know, you have to really look at the cost, cost of the benefit of something versus the cost. So my point of view is, you know, focusing on purpose and medical necessity. And I and I think that's really the essence. I mean, in healthcare or physicians, they have an oath which is do no harm. And I think all of us think about that as human beings, hopefully do no harm. But how can you make progress from medical necessity and not to, pivot to Daniel? You know, you can choose to, you know, share your perspective. But this is very personal. When I talk to parents of children, I don't I'm not a mother, but I talk to a lot of parents and children. And when they have a medical necessity, whether it's a diabetic or a hemophiliac or what, something else, they're not thinking like, oh, you know, is this they're thinking like I, you know, do I share my data or do I not share my data? And I've talked to a lot of parents who said it's irrelevant. I want a better life for my child. I think that hit me home many times when I've had conversations and kind of struggled myself. I think any tool could be used for good or for for for not for good. But when it comes to what's currently available, 20 years ago we had a watershed moment because 25 years ago, recombinant, we we discovered the sequence of a genome. Now we have another watershed moment, I believe, which is AI. And being able to make proteins on a computer. So remember chemists used to make medicines. Then biologists were able to make medicines. Now computer scientists can make medicines. And I think we need to be really focusing on human, on leadership, what it means to do no harm on on all of that kind of, I would call it it's not even soft stuff, hard stuff. So that we are developing and progressing humanity and science in a, in a moral, ethical way, but in a way that helps us all move forward, from a, from a medical necessity perspective. So, yeah.
No, to build on that, I would say there's a huge need for more and better medicines. And today there is out of the 7000 rare diseases, there's only 4 or 5% of those. There's a drug that's approved that treats patients that are born with that disease. And if you're a patient with a disease or a parent of a patient with that disease, you feel very vulnerable and very left out and left alone because there is, you know, so limited progress for these, vastness of diseases. I think the progress that we are making is really accelerating in the last 20 years through inventions like microRNA and siRNA, through gene editing, RNA editing, and a really good understanding of the of the genetics, but also through new chemistries that help us to engineer molecules, proteins, RNA molecules that are much more effective in treating disease. And as we now enter the age where AI can help us to design those medicines and can help us to iterate on those medicines to continue to make them better, to better understand what they do so we can hopefully reverse engineer how we got there and get better medicines for other diseases quicker. I think those new chemistries are really helping us to make a step change in how impactful these medicines can be. So I think it's actually very exciting time. And, yeah, to Eva's point, any product, any invention can be used for male intents. I think, we are fairly well regulated around, doing that unintentionally. I think the intentional part is difficult to regulate. We always have to keep that in mind when we put things together. And one of the things we spend a lot of time of thinking about is what are genetic modifications that are that are ethical to make and which ones are enabling people to maybe adopt lifestyles that you don't necessarily want to be healthier. So, for example, through human population research, we have found that people in the Asian population cannot absorb alcohol as well. And it's known as the Asian flush. This leads to people that when they drink alcohol, they get a flush in their face. And they also can't digest it as much. So it gets drunk very quick. The genetic mutation is known and we could reverse that. It's very questionable if we should. Yeah, yeah.
Well, that's a great segue. So. So what? We've just to conclude a little bit this section, as Eva and others have pointed out, you know, we've got a great rich heritage, actually very based in Europe, you know, for pharma, many of the big pharmaceutical companies of the world were born from Europe and from European countries built on chemistry from the 18th, 19th century. And, and of course, that's transitioned now. There's still obviously pharma. There's still a lot of need for medicinal chemistry. But biology has become this very targetable thing. And doing biology and creating biology, if you like, is now a fantastic. That's the sort of biotech. If you like bioengineering industry and biologics, you'll have heard of all these words. So we are in this new era and as you say, very targeted, you know, for health benefits, which is again, nobody's going to argue with that. There are some challenges that maybe we don't need to get into the weeds as to how you localize it. You spoke about microRNAs in a particular area. So there's some systemic problems. But this is the world that we're sort of in now. And I think we can probably all agree that that's a great thing. We've got more tools at our disposal to tackle some of these diseases that affect people. But of course, it can also enhance just life and the quality of life. And as we all are in countries where our populations are aging, most people don't get ill or don't necessarily get chronic diseases. They will eventually, but there's a great emphasis on having a much longer, better quality of life. And that's where one gets into not necessarily that example you've given, but where maybe the synthetic biology or some of the other editing might be able to help facilitate a better quality of life, whatever that means for that particular culture or that particular country. Could you all say sort of whether you're going into that side of things or transiting into sort of healthy living for longer as part of the industry, or maybe some of the research that you're doing and what you see as some of the ethical implications that we should be thinking about now that we put in place. Because I take your point, Victor. You know, that's a good example of the recombinant and stem cell biology might be another one, but we're not very good at doing it globally. So whilst a country might do it, Britain took all the people who left America to come and do stem cell research in Britain because they could, because we had a different approach to the ethics of it. So as a globe, we tend to not sort of we tend to don't go and sink. So there's still some challenges, I think, for us there. But who would like to start? Daniel, please.
So I'll give an example of the other side of the human population research. So in the human genetics research. So essentially comparing people's features at scale to their genetics, we have found that certain genetic mutations lead to genetic disease. We've also found we, the scientific community at large, that certain genetic variants actually give a health benefit. So there's populations found, for example, in the US that have a 36% lower chance of cardiovascular disease, which is obviously very interesting because if we could all have that, we would suffer less from cardiovascular disease, which still is the number one cause of death in the world. They were able to pinpoint that finding to one specific genetic mutation. So one building block in the DNA that was different in these people from everybody else. And this particular mutation is one that we could potentially introduce. And when we introduce that variant in animals that have a cardiovascular disease phenotype, we actually see that we reverse that phenotype and that these animals now restore normal cardiovascular system. So I think that's a really good use case of where you can introduce an edit to not therapeutically but prophylactically prevented people develop life threatening disease.
Yeah. That's great. And Victor, I'm just mindful of the build up of protein toxins in the prelude to maybe dementia. Can you see the microRNA technologies being in focusing on very decades ahead of something becoming clinical or symptomatic, that one could correct what one can see as maybe a trajectory that is going to lead to something down the road, and we should be intervening. And are you comfortable with that?
Well, I'm oh, oh, in principle, would I be comfortable to for intervening, where it would increase the quality of life? Not not by germline editing, though. We're talking about somatic editing or something like that, or using an RNAi to knock down. You know, the thing is about these, we whenever we talk about, let's say, these high technology, you know, precision medicine modalities, whether it's editing of, of the genome or not, RNAi therapeutics, these are expensive. And so we one of the things we inevitably have to come up against is the is the, the issue that we can invent all these wonderful technologies, but if we can't, if they're not available to most people on the planet, then, that's a problem, right? It's not like a negative side. Right? It's not like a oh, this is a negative thing. It's almost like the thing we've invented is positive. But the problem before us now has become something that's societal. It's economic, it's humanistic, and it's all that, you know, so but I but but technologically though, it need not be, impossible. Right. Because we can think about ways right to, to approach engineering these new therapeutics in a way where we're trying to anticipate accessibility and low cost and so forth. You know, so I think that, I'm sure you all do that to some degree, but I wonder to what extent we may need, let's say in education, let's say in a program, we in our med school, we have a program in translational medicine. I don't think that we take up really formally the issue of affordability engineering for affordability, engineering for.
That we talk about how, well, if you get the technology to work, then maybe the market will drive down, you know? But, the idea is and that, oh, the modularity you mentioned as well. And now you've got to stop me. Yeah. Okay.
So you're on a roll. A lot of these things.
Are these these modalities are modular. Right. And so you have a you have an informational part of it which is going to edit or it's going to knock down. And you just design that on the computer. Or it maybe it's a synthetic protein. You design that. That's one thing. That's the thing that's interact with the living system to make the change. Then you have the, the, the sort of the formulation or the little, little thing conjugated to it, the chemistry that takes it to a particular organ. And so those two engineering challenges are, are the main sort of components. And the biggest challenge in my understanding now is the delivery component. The the informational part is much more straightforward. So there's the opportunity for, you know, the engineering bioengineering community, to develop these so-called platform. You all could talk more about platform technology, you know, where it becomes more straightforward to just sort of mix, as you mentioned, right, this with that and you have a new thing. And if this is is approved and this is approved, this is going to be it's approved, it's going to be approved very quickly and cheaply. And so my colleagues that do this sort of thing at UMass Chan are talk a lot about this.
No, it's a.
Very optimistic I think view of the future. Yeah. But but but pressing on the need for the community, whether it's, you know, you know, funding agencies and so forth, to deliberately try to promote, these kinds of effort to, really, you know, develop platforms efficiently. So they're broadly available.
Yeah.
Shared data.
Yeah. Yeah. Well, I'll come to that momentarily, but you're absolutely right. I mean, maybe Natalie, you can comment on what you're doing because obviously, you know, you're at the coalface teaching the students. I'd like to know a little bit about what you're doing about ethical training as well. But to design right from the get go, the capacity to scale and disseminate at pace and affordably, as Victor was saying, are you doing that? Are we training the next generation of scientists and engineers to think in that way, noting that different health systems are very different. So again, you know, I think some of the challenges we've got is that, you know, I'm from a public health system. Obviously, in America, things tend to be designed knowing that there's a middleman to take a profit. That tends to change the cost base. So there are challenges, but tell us what you're doing. Give us some hope about how we're taking this on.
Okay. So, effectively Latin America, specifically Chile situation is very different. In this sense, I think, that going for education and students and promoting, you know, new interests in them, specifically democratic, democratizing science, making it accessible, easy to understand, because how will you be able to take part of a conversation if you don't understand the meaning of it and the impact it can have, in a long time. So I think that's the first, way to to approach this situation. And I do also believe that at this moment, Latin America is behind. And that's the reason why with some other ambassadors, we're trying to create different initiatives in each country and also collaborate to spark that interest, show that it's possible and, you know, encourage young, scientists and maybe high school students that are interested in biology and engineering and computing sciences to delve into this world. I think that the, you know, the regulations of ethics and, and the decision making in the next few years is not entirely, on the hands of scientists. It's very important to understand policies, governance, how to, you know, have different stakeholders being part of that conversation. So in this meaning, Universidad Catolica de Chile, we have an undergraduate research program, which, is mainly from the School of Engineering, but of course offers opportunities in different faculties. So in that sense, at this moment where I'm working on, working in each student, goes after their interest, contacting a professor and getting to know more about a certain topic. It's just that not everybody is interested in in politics and regulations and how it's going to change. But that's where I think the conversation is going. To know a little bit of everything, collaborate, through academia and more experienced people with younger scientists, just like in this moment, to have the conversations, you know, we have more technologies nowadays, but the expertise and different perspectives, can create something that's, stronger and can have a better societal impact in the next years.
Fantastic. Well, that gives us a tremendous amount of hope. And you're absolutely right about the need for people to learn the language of interdisciplinarity. You know, I'm a big advocate that people have to be trained very deeply in specialist, but they do have to learn the language of others and the courtesy and respect for other disciplines. And I think one of the mistakes often scientists make is you think if you've discovered something and it's obvious and it's going to work well, the world will just adopt it, and then you realize, oh, humans get in the way and society and, and there are anti-vaxxers and there are people that just don't want to adopt, you know, energy solutions for the climate problem. And there will be many examples of that. And that's why we need our scientists to be comfortable to talk to people from policy, from social sciences, from humanities. And that's why meeting like this is good to, to get people comfortable with that. But the earlier I think we can teach people from undergraduates the better. So I really applaud what you're doing there. Ava, have you got anything to say around some of these issues that we've just been talking around? Yeah.
Maybe just that I'm actually quite bullish on technology democratizing access to to to medicines. And just two examples. Every tech biotechnology is just technology as well. So every technology gets cheaper as as it goes. I mean how much did it cost to buy a computer in the home 20 years ago? 30 years ago? How much is it now? And and, and so I think the human genome sequence, I don't know how much it costs to, to get a sequence now, is it or is.
It $300, $300?
How much was it when it first came out?
Oh, it was billions. What?
Yeah.
I wanted you to say that because, see the order of magnitude. So I'm really, really optimistic about technology actually improving the democratization. And just a little side note before insulin, back to my insulin story. Before insulin was, the protein for insulin was discovered and we could democratize it. You know, only very, very few wealthy people could get access to it. You'd have to stimulate, I believe, the pancreas duct of a dog to produce a little bit of insulin. And only, you know, the kings and queens of, of, of the state would be able to support if they had a diabetic kid. So I only share that here just to kind of give it this some, some weight. It's not just, you know, it's not just empty words. Technology does allow us to do things in a way that it's then available to everyone, and then there'll be a new technology that will come out again, will take a couple of years or a decade to democratize. But I always say, you know, everything we need is available. It's just not equally distributed. But over time it will be equally distributed, and then something else will come that's not equally available. But I'd rather live in that world where where we're moving that forward. In, So that's all I'm gonna say about that. I was going to go a bit rambling into a story of, but I'm not going to do that. I'm going. I'm going to reel myself in.
Well, we might come back. We're all intrigued what that story is. And I want to.
Say it out loud just to kind of reel.
It back. Stop yourself.
And everyone. I am done.
We'll definitely pursue that one. But but you know, the point that I think the victor is making is, of course, we're getting quicker at democratizing the technology. Yes, the speed, but meanwhile there is a gap. And and that's an opportunity loss for either treating people or enhancing life. So how can we, right at the design stage, build it more cheaply so that it can, right out the gate, be something that can be widely disseminated? Is that mindset yet in your industries, or is that something we've got to just train a completely different generation to come forward to, then hopefully join your companies and just do it differently?
I think access to health care, access to new therapeutics is front and center for every company that works in our industry. I think we're all very focused on, the interest of patients and how we make sure that we actually help patients with the work that we do. It's difficult enough to it be super frustrating that at the end of the at the end of the day, when you succeed in developing a product that it doesn't reach patients all over the world. So I think companies are really focused on it. The reality is that drug development is very difficult. It's extremely expensive, it's highly regulated. And as a result of the failure rate of new therapeutic development is really high. And that feeds into significant cost for every product that reaches the market. I think as we progress and as we look more into platform technologies that actually help to become predictive for every next drug on the same platform that gets developed, but also by implementing AI to help us fill early and really invest deeply and with high conviction behind the ones that have high probability of success. I think we, over time, can drive down the cost of development. And with that make, make, make access more equitable across the globe. So that's certainly an objective in my company and I'm sure that's across the industry.
Yeah. No it's fantastic. Well listen the time is going by very quickly. And I want to make sure there's time for the audience to ask questions. But at the end, just a heads up for my panelists, I would like you to just conclude each with a thought about the world you see in 2050, in terms of what excites you from your curiosity. And let's assume that we're still really supporting basic science and its translation. What is it that you're excited about that you would like to see be there in 2050, in terms of, of biology? And that can be anything. You know, it can be sort of, you know, changing things on we're all living on another planet maybe at that point and what it means to to do that. So you can be thinking about that whilst we open up for the audience for questions. I believe that there's a microphone. So if you could just say who you are, if you're willing to say that and we'll we'll get started. Okay. So it's hard with my blinding lights here, but there's a gentleman here. Thank you.
Can we have some lights? Oh, great.
Thank you. Yeah.
Hi. Oh. Sorry. I'll stand out. Hi. My name is Wilson. I'm a law student from Hong Kong, I came. Thank you for delivering the, talk. I just want to ask the panelists. There's been a lot of, conversation about accessibility based on, you know, affordability. But I think we can all agree that increasing affordability for gene editing and all that technology is a good thing. It prevents, you know, rich people from hogging all that technology. But I wonder, I wonder, I want to pick your brains on the ethics. If we do include everyone, what if a person chooses to edit genes of, let's say, their embryos to make it have a certain condition? For example? Currently there are like deaf people prefer that their child be born deaf. And there's been cases where they explicitly request that their child be born deaf. So, I wonder what your takes are on that. For example, like, they might this might be a case of being deaf. Maybe in the future it might be something else where they want the kid to be, you know, something that's less therapeutic. I just wonder. Yeah.
No, it's a great it's a great question. And I'm only cutting you off so that we have time again. Just everybody keep their questions really short. So I guess the question is it gets into the sort of, you know, determinism, free will choice. Who has that choice? You know, again, particularly on dependence. Who would like to take this on Danielle, please. Yeah.
Yeah, I think that's a that's a real concern. I think you can use gene editing in particular for, malicious, editors as well. But I think, like, that's the case with everything. You know, you can use a brick to build a house, or you can use a brick to hit someone to the head and kill them. And it's clear that that's illegal. And I think everybody would agree that deliberately making human being build a be born with a defect would, would not be what we as a society would accept. I think there should be significant regulation around that. And I know that when we, for the first time saw someone experiment with editing in embryos, there was a significant, countermovement in, in, across the scientific fields against that. So I think it's really important to be aware of the risks that it imposes. And I think it's important to have sufficient guardrails around that from a regulatory perspective.
Yeah. Thank you. Any other comments in the panelists.
Position on that is quite clear. Just that's personal. Eva MacLellan personal position is you focus on medical necessity and prioritize things that you can treat and prevent, medical illness or serious disability. And on the other one, I, I yeah. I don't have another comment.
Yeah. Thank you know. Yeah. Let's take a question from the from the back of the audience. So let's see if we can go to maybe the, the woman in the middle there with a black sweater on. I'm just going to distribute around spatially as I can see it in the lights. Thank you. Please do stand up and say who you are if you're willing.
So my name is Ayana Kapler. I'm a high school student from Zurich, and I was wondering if gene editing regulations should be decided nationally, or do we need global rules to avoid ethical gene editing tourism, as we earlier had in discussion.
Right? Right, right. Gosh, gene editing tourism. I haven't heard that expression again. Okay. Who would like to take this on? Natalie, do you want to have a go? It's a great these are tough questions. Good.
I guess, regarding what was brought up before, I, I came into the biotech world because I really had a feeling that I wanted to do something that was able to create a good experience for people to, create something, a prototype, a system, whatever, something to impact people's lives. I had the experience of working with psoriasis, and, you know, it's such a common disease with that. People only think it's, like a skin condition, but it's a multifactorial origin and it's multisystemic. It affects all of your organs and can have detrimental results if not treated accordingly. So in that sense, I do believe we have to use gene editing and creating, you know, using synthetic biology to produce good for the people. I don't believe in, you know, editing for obtaining a certain trait and, and trying to control things that that I don't know everything all our traits make us unique. So I would encourage diversity, which is also how nature is. So, I do stand with that, being able to, to use these technologies for doing good.
I guess it begs the question about what the social norms are of what we define, which might vary on countries as to what's in that grouping. That is a required need, required disease for want of a better expression that needs a therapy. Right. And, and I think there'll be different social norms for what a medical need is. And I think as we evolve and our expectations and our longevity improves, I suspect that that bell curve of the norms of what's in that will change and it will it will skew, it will grow. I Victor. Ava. Yeah.
Your question was more like, where would the decision be made also. Right. National. And you know, so one thing that just popped in my mind, I thought, well, do we have an analogy? You know, do we have an analogy where. So one thing that just came to my mind was medically assisted death. Right. And then I was just quickly trying to go through my Rolodex of countries, not without mentioning them here. I was like, okay, some have it, some don't. And then, I had a little bit note around, you know, how do we make the how did they make that decision? And then, you know, we use independent multi-stakeholder oversight, to do that. And those multi-stakeholder oversight bodies, you know, the best decisions use all perspectives. They usually are guided by ethics boards that include clinicians that include scientists that include patients that include the community and back to the norms and values of that country. And I have not said in one of those boards on such a consequential decision, but I imagine it's a long process of finally deciding what's the thing. And I would I would also imagine that the that the public dialogue, similar to what we're having here, is critical in that conversation. If I was feeling super edgy today, I'm not going to do it. I would actually just say show of hands. Who would be for or against genetic tourism? I'm not going to do it. But if I felt edgy, maybe another time when I come back, I will, because public dialogue, you know, would starts to also, you know, citizens need to vote. In, in in realms of law, legality and ethics that's been established in other parallel systems that are really hard decisions to make. And now, as I'm talking, I can think of 3 or 4 more, but maybe that's helpful.
It's such a good point you make, and I think the assisted dying is a great live example that we're going through in the UK at the moment, with much discussion and debate, and that it emphasizes that need to have, as you say, that dialogue, which is what of course, is all about on this particular meeting, and about the training and sort of this desire that how can we get better as scientists talking to the public and making sure we serve them fundamentally right? And we need to know what how we need to serve you best and what it is you want us to do, as opposed to just leaving us free reign to come up with this stuff. Victor, it looked like you wanted to say something on this topic.
Comment that the question is so good because what it points out is these questions. They seem so simple, but they're so hard that the chances of getting an international convention on something like this is, is is so remote that it feels like each country is is needs to go through these conversations, do these processes, and each of us learns from the other, as, as they go through their process, you know, we're all trying to figure out how to be humans living on a planet, right? And we don't know how to do it. And so none of us can sit here and say, this is the way it should be done worldwide. And so that's the messy business is that each each, each community, each country needs to wrestle and and invent the process and teach, teach and teach each other.
Yeah, absolutely. Yeah. And your generation will be really facing you know, AI will be coming up with creative new biology independent of any of us. So we won't be using it just as a tool. It's going to come up with something new. And that brings enormously interesting yet challenging questions. Okay, let's get another question. I'm going to okay, I'm going to come right to the front as we haven't had a question. This gentleman here and let's take what we'll do is we'll take three questions. We'll try and remember them and we'll answer them please.
So hi, how are you? Congratulations. Was very amazing speech from your side. So my name is Aramis, and, I am I do political analyst, from the and I work between governments doing lobby. So one of my questions is, how can we make medicine more affordable for the countries that are also developed and rich?
Great. That's a great question. Okay. Thank you. So how do we make medicines more affordable for countries? Let's take a question from this gentleman here. And then we'll go to the very back. And the lady at the back with the black sweater please.
Hi, I'm Herman, thank you very much for the awesome panel. I'm a PhD student in Zurich. We spoke about ethics, education and understanding that each technology is dual use. And so I was wondering, practically speaking, what needs to be done in the education system to include ethics education for budding scientists. And and to what extent do scientists hold responsibility for their own discoveries? Thank you.
All right. That's a great question. Thank you. And the lady right at the back of the black sweater. Thank you.
Hello, I'm Leah, I'm from Romania and I'm a youth advocate. I'm working with teenagers around the world. And, I see, a lot of, let's say a lot of fatigue in students, in teenagers, overwhelm or lack of motivation, and a lot of lack of interest in, in health. So what would be the, activities or the work or the steps that we will need to do as leaders in order to help the educational system to introduce this kind of, activity or systems in the school in order for them to get the information. Because if if a parent gives the information home to the child, it's okay. But the majority of the parents, they don't take care, they don't take care of themselves. So they don't bring or give this information to a teenager. So in order to, to have a sustainable performance on a long term, you need as well your health, your body to start with your body. So how we can do that?
Yeah. Okay. Great. So we've got a couple of things around sort of the broad education thing. So one is around the sort of ethics and whether actually, you know, the Pi or indeed the algorithm, you know, if an algorithm comes up with a new drug and it kills people, who's to blame? The company, the scientist or the computer or the mathematician that came up with the algorithm? These are real ethical questions that philosophers and ethicists are dealing with in the context of AI. So ethics, education, who's responsible if things go wrong? How do we get health education better in the school system so that people learn about health? Natalie, I'm going to turn to you first because you've spoken a little bit about this. But then if the other panelists would like to comment and then we've got the more affordable medicines, which I'm looking to Ava and Danielle to answer. Natalie, do you want to start?
Sure. So, you know, regarding the lack of interest in students in health and biology or even Stem, I think it starts with communication. Sometimes it is so hard to understand, you know, each word, yeah. Being in a in a niche and so technical. How do you translate that into something that you can understand, that you can keep in your mind and you, you can do something about it? And regarding that, you know, actually because of iGEM, which is funny, I really wanted to, be really involved in the lab work, but, since our group was. So it wasn't. Heterogenic. Let's say, between 12 to 13 people over the team were only biotech. So that is also a huge bias on how you want everything to work. It's important to have diverse, backgrounds and information. And I encountered, the communication coordination of our team. So doing that science communication, which at the time I didn't know it was called that way, just made me be able to connect, transfer these ideas to communities. You know, even speaking with kids, maybe 4 or 5 years old until, you know, grownups from rural areas in, around Santiago. That's my city. So I do think it starts changing the content, making it accessible and understandable. And also I want to delve hopefully next year. And how to create content that is also entertaining. That is fun, that you don't associate it to being boring. But sparks curiosity. So I think it starts there. And for that, I've known that there are new, more formats. So there are video games. They are magazines. And I actually discovered a couple of years ago, frontiers, which is also promoting open science tests and initiative that, they conduct articles that are written by experts but edited by kids. So I think it has that input of youth, and it makes it more fun and closer for them to understand.
Yeah, it's a great answer about the dissemination. And I think, you know, my sense is I've just recently finished being president of the Federation of European Neuroscience Societies. And neuroethics is a is an area of concern because of just the manipulating your mind as something that people find very, threatening. And, you know, we brought in neuroethical training and it was it's so popular. So my sense to the gentleman that asked the question is, the people want to be trained students, postdocs, early career researchers alongside their work. They actually are welcoming the opportunity. So I think academic societies that your members of should be laying on as part of the conferences, as part of the educational training, they should be laying on ethical training as well. Can I just ask a very briefly, because then we need to conclude, how do we make medicines more affordable?
I think AI will really help us to accomplish that. I think I mentioned it earlier. If we are able to drive down the failure rate, increase our probability of success of the molecules that we invest behind, that will lower the cost of drug development and ultimately will lower the price of the products. I think it's important to realize, though, that currently medicines, when they are approved, have a significant price tag, but only for a short period of time. When the patent expires, they become what's called generics, and they become typically readily available at a very low cost to society. So the majority of our medicines actually are very affordable. I think AI will help us to get new innovative medicines to be more affordable at lunch.
Yeah, fantastic. Great. Well, you've been great as an audience. If you can indulge us, maybe an extra minute over time. So thank you for your questions. And then we'll appropriately give a round of applause. But before we do that, one more task for our panelists. And that's just to go along. Maybe start with Natalie as to what do you see as the future for biology in 2050 and what is it? What is it you'd like to see? What are you going to be particularly excited about hoping will be the case?
Well, I'd like to see more youth in Stem, especially girls in Tula is maybe in Stem careers. Around 30% of girls that are participating. So first of all, there's that. And then, you know, being able to encourage youth, giving them tools and the space also to have hands on experience and also get to know what other stakeholders think and how they can engage and and create collaborations. So for that, just, you know, my university does this by a program inviting people from different sectors and trying to explain what what they do to students. So I think that's the start. And just for 2050, I just want to see a better world.
A better world. Yeah. Victor.
Also biology in 2050. I mean, what jumps to my mind is by then I hope we will have detected habitable worlds in other star systems and detected the signature of life there by then.
Yeah, yeah. No, I said I was hoping somebody would give that sort of answer, so. Thank you. Victor. Eva.
2050 65 I hope that we don't use the word women's health anymore. I hope that half of the world's population is living not only longer, women are living longer, but they're living less well in the last 20 years of their lives. So my wish for half of the human population would be that, that women would be continue to live as long as they live, but the lives that they have would be with better well-being and health and that we would understand, the genetic differences, sex differences as it relates to response to environment and medicine. We've solved that. And women's health is no longer a topic.
Fantastic. That's a good one. Great. And, Daniel.
I think my hope is that by then we understand our therapeutic platform so well that through personal medicine becomes a reality, and we can be so predictive that we can treat people with new medicines without lengthy validation processes and trial and error. That goes straight to a solution. I think that's potentially within reach.
Fantastic. Brilliant. Well, can I ask you please to thank our panelists and Natalie, to Victor, to Eva and to Danielle. It's been absolutely wonderful. We wish them well in their work going forward. So keep up the great work. And and thank you for the contributions you've made to science and to to the betterment of society. And thank you all for coming. And to the organizers. Thank you. Thank you so much.
Very much. Oh, my pleasure, my pleasure. No thank you. Excuse me.
Could you ask if I could have a quick word with.