Ever wondered how far we've truly come in the realm of gene and cell therapies, and where we're headed next? This captivating conversation with Oscar Segurado, the CMO of ASC Therapeutics, will satisfy your curiosity. Oscar, a veteran in the biopharmaceutical industry, shares fascinating insights on ASC Therapeutics' groundbreaking innovation in personalized medicine and synthetic biology as they set out to redefine the future of therapies for rare diseases.
First In Human is a biotech-focused podcast that interviews industry leaders and investors to learn about their journey to in-human clinical trials. Presented by Vial, a tech-enabled CRO, hosted by Simon Burns, CEO & Co-Founder. Episodes launch weekly on Tuesdays. To view the full transcript of this episode, click here.
Interested in being featured as a guest on First In Human? Please reach out to catie@vial.com.
🎧 Stay in the Loop!
For the latest news and updates, visit our website: https://vial.com
Follow us on social media for real-time insights:
Twitter: https://twitter.com/VialTrials
LinkedIn: https://www.linkedin.com/company/vialtrials
Ever wondered how far we've truly come in the realm of gene and cell therapies, and where we're headed next? This captivating conversation with Oscar Segurado, the CMO of ASC Therapeutics, will satisfy your curiosity. Oscar, a veteran in the biopharmaceutical industry, shares fascinating insights on ASC Therapeutics' groundbreaking innovation in personalized medicine and synthetic biology as they set out to redefine the future of therapies for rare diseases.
First In Human is a biotech-focused podcast that interviews industry leaders and investors to learn about their journey to in-human clinical trials. Presented by Vial, a tech-enabled CRO, hosted by Simon Burns, CEO & Co-Founder. Episodes launch weekly on Tuesdays. To view the full transcript of this episode, click here.
Interested in being featured as a guest on First In Human? Please reach out to catie@vial.com.
🎧 Stay in the Loop!
For the latest news and updates, visit our website: https://vial.com
Follow us on social media for real-time insights:
Twitter: https://twitter.com/VialTrials
LinkedIn: https://www.linkedin.com/company/vialtrials
You are listening to First In Human, where we interview industry leaders and investors to learn about their journey to in-human clinical trials presented by Vile, a tech-enabled CRO hosted by Simon Burns, ceo and co-founder. Featuring special guest host Rich McCormick, evp of Clinical Strategy and Head of Oncology. In this episode we sit down with Oscar Segurado, cmo at ACS Therapeutics. Join us as we explore ACS Therapeutics' revolutionary gene and cell therapies, personalized medicine and the transformative landscape of synthetic biology, offering a brief glimpse of the future of rare disease treatment.
Speaker 2:Hi, I'm Rich McCormick, Executive Vice President of Clinical Strategy, here at Vile. Today I have the pleasure of welcoming Oscar Segurado, CMO at ACS Therapeutics, to our First In Human podcast. Oscar, thanks for joining us. Would you mind letting us know a little bit about yourself?
Speaker 3:So, first of all, glad to be here and especially, it's a pleasure for me. I'm Chief Medical Officer at ACS Therapeutics. This is a biotech based in the San Francisco Bay Area and we are focused on cell therapies, especially allogeneic cell therapies in hematologic malignancies, and gene therapies, together with gene editing approaches to Hemophilia A, which is a rare disease, and we have three platforms that work together very nicely. My background is as a professor of immunology. When you have this kind of expertise, you have been working, like I have, in molecular biology, cell biology and protein biology and over 30 years experience in the biopharmaceutical industry with companies like Avibecta, dickinson and several other big pharmas and mid-sized pharmas and biotechs.
Speaker 2:Great Thanks for being all with us today. So, as you mentioned, ASC Therapeutics is on a mission to advance gene and cell therapies of patients with mostly rare diseases. What would you say sets your company apart from others in the space? I think?
Speaker 3:that the most important aspect of what we do is the fact that we are running concomitantly three platforms, and all three platforms are next-generation platforms.
Speaker 3:What I mean by that is that we have a gene therapy for Hemophilia A, which is a second-generation gene therapy, and that means that it overrides what is already available even in the market, with genics for Hemophilia B, with roctavians for Hemophilia A, for bimarine, and also work being done right now with giants as Pfizer and Roche in the same space.
Speaker 3:What we are bringing also to the table is the fact that we are working on gene editing, and that means that we can go beyond the standard requirement for all these gene therapies to be delivered to patients 18 years old at least. That means that we can incorporate this gene into the genome of the patient, and this requires a technology that is well-established for Cas9. Additionally, we are working on in the cell therapy space on analogenexal therapy, with cells called deciduous thromal cells with immuno-regulatory properties that are derived from the placenta, which is discarded after a woman delivers, and they are used for patients with hematologic malignancies. Additionally, just to give you a sense about what differentiates us, is the fact that, based on my experience over many years with Abbott Laboratories and Bechton, dickinson and other biotechs. It is our focus on personalized medicine and the use of biomarkers.
Speaker 2:That's great. So you mentioned some of the technologies like CRISPR, Cas9, also familiar with your target technology as well. Do you think you could maybe just dig into these a little bit and share how these are really helping advance the gene editing and gene replacement?
Speaker 3:Yeah, I think that CRISPR Cas9 is now a revolution in medicine. A number of companies are already working on that and some of them very recently in teleatherapeutics called an IND to start treating patients. We are working on that too, and the advantage again is that we can incorporate the gene that we want to be replaced into the chromosomes, and this is something that you do not do with gene therapy. With gene therapy, you use an episomal approach. That means that the gene is lurking around the chromosome, in the nucleus of the cell and delivers the protein. But when you incorporate that, you also need something which is a promoter and the way we have identified and we work on the exact intron or place in the genome where we're going to benefit from the promoter of albumin. Albumin is the main driver of proteins in general by the liver.
Speaker 3:We also are developing collaboration with Stanford University. We are very close to them in terms of our location, target and target is a technology that, just to try to explain that in late terms, is like having a pickup truck that we also position in the same place where we would insert our gene using CRISPR-Cas9, which is a cat and paste type of approach for gene editing. But what this pickup truck does is that it's able to hold a trailer, and that trailer in some way means that we can incorporate a larger gene than is usually available when using the standard CRISPR-Cas9 technology, and we're working on that at this moment in terms of preclinical work, but it looks very promising.
Speaker 2:None of what you're describing sounds simple. So I'm sure there's been challenges along and maybe just talking us through some of the challenges that ASC therapeutics has faced trying to develop these treatments for such niche patient populations.
Speaker 3:So when we talk about niche patient population and I think you're absolutely right that's where you have to start before broadening up all this work. But the main issue, let's say, or aspect that we need to tackle and this has been the main focus also of the regulatory agencies, and we mostly interact at this point with the FDA is in terms of off-target effects. When you do all this kind of gene editing, with whatever technology CRISPR-Cas9 off-target we need to make sure that when we do this cut and paste, we do that exactly in the place where we want to do that. We don't want to have any type of insertion in a place where it could have any kind of deleterious effect or we could have a gene that is not required being activated.
Speaker 3:So this is what we do, and we do that through different ways. We use synthetic medicine in some way because we approach that with large data sets. We are well-defined and they tell us if we are doing the right thing. So it's all kind of an artificial approach. But then we need to go into preclinical models and at the end of the day, we will obtain approval from the regulatory agencies. Again, as you said, it's not easy, but we need to focus in first and foremost on the safety of the patients.
Speaker 2:That's great. So maybe shifting to a broader topic, so you worked across different continents. So how does the landscape of gene cell therapies differ across the regions and maybe what does this really impact in terms of development and accessibility for these therapies to patients worldwide?
Speaker 3:So let me focus on accessibility, which I think is the sticking point here. Science and technology across continents are the same. Obviously, what we are doing here in the United States can be applied in Europe or Asia Pacific, which are the three continents we are focusing on, and the regulatory agencies understand that. But they are not involved in reimbursement. And this is accessibility.
Speaker 3:That's what changes the way the private health insurance I would say almost fully mostly private, except obviously Medicare, medigap and Medicaid in the United States is very different to the way it works in Europe.
Speaker 3:In Europe, the majority of the people are under any type of public insurance, and the same also for most countries in Asia Pacific. So that means that when I see what is happening to companies that are already more advanced than us in terms of clinical development, that they already either are in pivotal trials or they already got marketing authorization Everyone everywhere is struggling across different continents, but it's different in the way it happens in the United States. By the way, what happens in the United States, because everyone is with a different health insurer, is that you can't change anytime. So what company is going to be able to shell out $3 million, which is the average of these therapies or gene therapies, when available, versus this patient potentially moving to another health insurer, while in Europe, although it seems easy, there is also a lot of reluctance because there are public funds that need to be used for supporting these patients. And very similar the situation mixed in some ways in Asia Pacific.
Speaker 2:And saying personalized medicine and genomic biomarkers are key areas of your expertise. Can you shed light on how ASC Therapeutics is incorporating personalized approaches into its therapies and how this customization is enhancing the effectiveness of treatments for patients with a disease?
Speaker 3:Personalized medicine, precision biomedicine, biomarkers are my passion. This is the way I started my career over 30 years ago, when I was hired by Abbott laboratories to work on tumor biomarkers and I was working on a prostate specific antigen carcinumbleronic antigen which were at that time, at the top of the approach to looking to cancer biomarkers. After 30 years, just a few weeks ago, I read an incredible article showing how we can revolutionize everything that we're doing in cancer through the so-called CT DNA, or circulating tumor DNA. What is that? When a tumor is developing, even in the initial stages, they shed small fragments of DNA and these fragments can be assessed by sequencing an incredible computing power.
Speaker 3:And this article but this is just one of many showed for the first time that this CT DNA, this type of biomarkers, cannot, could can replace radiology. So the way you assess the way a therapy in oncology works is because you see that the tumor shrinks or stays or grows. So now you can do all that with a simple block test, and this is a real revolution. We are using this technology to approach our cell therapy. In our case it's analogous cell therapy. I mentioned before the seizure of normal cells and we want to see how these cells are performing and how they are circulating in the body and how they do their immunoregulatory effect. And we do that through DNA, through these fragments of DNA that identify these cells and tell us how they are doing. This is the way we do the so-called pharmacokinetics of our technology.
Speaker 2:Thank you. So, considering your academic background and industry experience, how do you see the role of regulatory agencies evolving related to gene and cell therapies?
Speaker 3:So, first of all, we are interacting, as I mentioned a moment ago, with the regulatory agencies in North America MOTREFDA, european Agency, ema and also in Asia, especially with NNPA in China. So all these agencies interact with each other and talk to each other continuously, and one of the ways to do that is that they don't even need to actually go and ask or send an email or please tell me what did you do. These regulatory agencies are externally transparent and they provide open forums and, by the way, there is one that started this morning by the FDA which is going to be several hours open to the public about their decision or not to send muscular dystrophy therapy by vertex, and they are going to spend several hours and this is again available in the FDA space. I can tell you that all regulatory agencies around the world are looking and are able to see that they are going to be watching. That I will also in a moment.
Speaker 3:So this means that there is a cross-pollination between agencies and also the fact that we are asked to provide a number of details about clinical trials. In clinical trialsgov. The information is really widely available. There is also something that the regulatory agencies do they provide guidance, and these guidances have two steps. One is when they have a draft that they put out for everyone to provide feedback, and then they publish the final draft and all these agencies do that, and so that in some way, what we're seeing is that the focus and the fact that gene and cell therapies are relatively new type of therapeutic modalities. That means that the effort and all these public open forums are even more and more relevant.
Speaker 2:Great Thank you. As a renowned author and speaker, can you provide insights into how public awareness and understanding of cell and gene therapies have evolved over the years?
Speaker 3:First of all, thanks for saying that they do my best. I really try to contribute to the field and I try to go beyond my role and therapeutic focus, and I do that through participation in panels and speaking engagements, participation in podcasts like these, and also through publications which are more focused into more lay people or the medical profession in general, without being too scientific. So I like to combine my scientific work and I recently had several peer review publications with other activities that I think are relevant for our field and for other clinical developers and CROs, and I want to make sure that these articles, these medical articles, provide also a direct communication to the patients, and I have developed. If you go to my website, we have a specific tab for patients. I recently completed a video with a number of opinion leaders which are talking directly to patients about why they should enroll our clinical trials. It's going to be uploaded as we speak and I also want to make sure that something as complex as they inform consent, which means that the patients need to read up to 80 pages Very complex, very detailed. We have also condensed that in a couple of pages where we tell the patient this is all, what is all about, and we also provide several other tools. We completed an animation video. All of that is already uploaded in our website. So we think that communication to patients, to physicians that are going to be involved in our clinical trials, is absolutely critical.
Speaker 3:And, by the way, there is also a trend towards what we call patient centricity.
Speaker 3:That means that we, as developers, want to make sure that we forget a little bit the fact that we are so focused on again what the regulatory agencies are telling us, how we interact with principal investigators, pis, with key opinion leaders.
Speaker 3:We want also to make sure that patients really understand what's in it for them and for the entire medical community and patient community. We interact directly with the patient advocacy groups. In our case in the US is the National Hemp Affiliate Foundation. There is also the World Federation of Hemophilia. We are involved with them continuously. And we also want to make sure that there is another concept that I recently wrote an article called patient centricity and decentralization, and by that I mean that we need to increase the diversity of patients that are included in our clinical trials. What is happening until now is that the majority of the patients that enter in clinical trials across the board are patients that live in big cities and close to reference centers. We need to decentralize that, and I think that there is a big effort about that. It's one of the guidance that is going to be published by FDA.
Speaker 2:So our last question has us looking into the future. What excites you the most about the potential of gene and cell therapies, and maybe are there any specific diseases or conditions that you see these therapies having a profound impact on in the coming years?
Speaker 3:So, as we talked a moment ago, we are focused on rare diseases. The reason we do that is because these are diseases where we have a precise knowledge of the biological function of genes and cells. It's relatively easy for us to focus on what we already know. We know the activity of those genes, we know what happens when we replace them, we know that there is something missing in these patients. But where we want to go and there are already several companies working on that is broadening up our sites, and one way to do that is going to some genes that are involved in susceptibility to large types of other therapeutic areas, such as cardiovascular and autoimmunity chronic inflammation. So there are already a number of companies working on that. We have this project on cell therapy, focused on patients with hematologic malignancies, but our cells, these deciduous stromal cells derived from the placenta, have an immunoregulatory property that could be used for many of those diseases that I mentioned autoimmune diseases, rheumatoid arthritis, crohn's disease, psoriasis and also several other types of chronic inflammation. So we think that that's the point.
Speaker 3:There is another very important aspect that is going on, and I'm very keen to talk about that, and this is an article that I published in Forbes in November last year. The title is about synthetic medicine, and by that I mean something that is very relevant to CROs like yours, and I don't know that Viall is already working on. That is how you take all these big data. You not only collect the data, you interpret the data, you analyze it. You also gain knowledge about that. But there is also something very important, and this is to obtain wisdom.
Speaker 3:I call it data knowledge, but the most important is the wisdom for management knowledge to wisdom, and with wisdom I mean that we're going to take all this information, all this data sometimes in many clinical trials, over millions of data and we use, obviously, the buzzword artificial intelligence so that we combine all this information and we use that to create this personalized approach to medicine. One aspect that we have been talking about today, which is related to synthetic medicine, is synthetic biology. All these genes that I've been talking about and that we use in gene therapies are synthesized in a lab, so we know the sequence, but we are able to just synthesize exactly what we want, even with all the aspects that are relevant to ensure that this gene works in the right way. So I think that what really excites me is how the future is already written in the world and I recommend everyone to take a look into that article about synthetic medicine.
Speaker 2:Great thank you. So, oscar, it's been a pleasure meeting with you today. Thank you for being a guest on the First in Human podcast. The team here at Bio wishes you and your team at ASC Therapeutics nothing but future success.
Speaker 3:Thank you so much. It's the same for you. I think that what you are doing and all my conversation with you and your team is that you are really at the forefront of what we expect from ASI ARO. Thank you.
Speaker 1:Thanks for listening. Be sure to follow us on Spotify, Apple, YouTube and Google.