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Edited Transcript of EXAI.OQ earnings conference call or presentation 18-Aug-22 12:30pm GMT – Yahoo Finance

Posted: August 22, 2022 at 2:25 am

Q2 2022 Exscientia PLC Earnings Call Aug 19, 2022 (Thomson StreetEvents) -- Edited Transcript of Exscientia PLC earnings conference call or presentation Thursday, August 18, 2022 at 12:30:00pm GMT TEXT version of Transcript ================================================================================ Corporate Participants ================================================================================ * Andrew L. Hopkins Exscientia plc - Founder, CEO & Executive Director * Ben R. Taylor Exscientia plc - CFO, Chief Strategy Officer & Executive Director * David Hallett Exscientia plc - COO * Richard Law Exscientia plc - Chief Business Officer * Sara Sherman Exscientia plc - VP of IR ================================================================================ Conference Call Participants ================================================================================ * Chris Shibutani Goldman Sachs Group, Inc., Research Division - Research Analyst * Michael Leonidovich Ryskin BofA Securities, Research Division - Director in Equity Research & Research Analyst * Peter Richard Lawson Barclays Bank PLC, Research Division - Research Analyst * Vikram Purohit Morgan Stanley, Research Division - Equity Analyst ================================================================================ Presentation -------------------------------------------------------------------------------- Operator [1] -------------------------------------------------------------------------------- Hello, everyone. My name is Chris, and I'll be your conference operator today. At this time, I'd like to welcome everyone to Exscientia's business update call for the second quarter of 2022. (Operator Instructions) At this time, I would like to introduce Sara Sherman, Vice President of Investor Relations. Sara, you may begin. -------------------------------------------------------------------------------- Sara Sherman, Exscientia plc - VP of IR [2] -------------------------------------------------------------------------------- Thank you, operator. A press release and 6-K were issued yesterday after U.S. market closed with our first half and second quarter 2022 financial results and business updates. These documents can be found on our website at http://www.investor.exscientia.ai, along with the presentation for today's webcast. Before we begin, I'd like to remind you that we may make forward-looking statements on our call. These may include statements about our projected growth, revenue, business models, preclinical and clinical results and business performance. Actual results may differ materially from those indicated by these statements. Unless required by law, Exscientia does not undertake any obligation to update these statements regarding the future or to confirm these statements in relation to actual results. On today's call, I'm joined by Andrew Hopkins, Chief Executive Officer; Rich Law, Chief Business Officer; and Ben Taylor, CFO and Chief Strategy Officer. Dave Hallett, Chief Operating Officer; and Garry Pairaudeau, Chief Technology Officer, will also be available for the Q&A session. And with that, I will now turn the call over to Andrew. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [3] -------------------------------------------------------------------------------- Thank you, Sara. In the first half of 2022, we made significant advances across the business to scale our team and operations. We have continued to deliver progress across our collaborations and pipeline, positioning us for long-term growth. A detailed overview of our pipeline progress is included in our press release. We believe the decisive actions we've taken to continuously refine and focus our strategy have put us in a strong position to deliver across our portfolio of drug projects, whilst remaining agile and entrepreneurial in our approach. We are well capitalized with $730 million in cash at the end of the quarter, providing several years runway, which is highly pertinent in today's market. On the operational front, our unique, nearly equal balance of talent between drug discovery scientists and technologists continues to be an area of strength. It's what underlines our ability to combine expert drug and the knowledge with tech scalability. In the first half of this year, we continue to add new talent to our team in key areas of growth from companies, including Dr. Mike Krams as our Chief Quantitative Medicine Officer, whose expertise is invaluable as we begin to plan for clinical development in the coming years. And Professor Charlotte Deane, who joined to lead Biologics AI in January. Charlotte is a renowned leader in the field and was recently awarded an appointment to the most excellent order of a British Empire to lead in the U.K. Research and Innovation's rapid response call for projects addressing issues arising from the pandemic. And also Eileen Jennings-Brown has joined us as our Chief Information Officer from her previous position as Head of Technology at the Wellcome Trust. We've also gone through a period of transformative growth. At the time of our IPO in October 2021, our team was around 200 employees. By the end of June in 2022, we had grown to more than 400 employees at locations across the world, building new areas of expertise and growth for a company, such as our clinical and precision medicine infrastructure. During this time, we've also made considerable progress announcing our historic deal with Sanofi to develop a portfolio of AI, precision entry and medicines. Together, with Sanofi, we have formally accepted 2 programs into the collaboration, 1 in oncology, and the other in immunology and information. We are also exploring the potential role of dozens of additional targets identified using a central biologist AI platform in these therapeutic areas, and we look forward to providing additional updates this year. Beyond small molecules, you may recall that our collaboration with Sanofi includes leveraging our translational platform for biologics. We're pleased to say that we have initiated our first project to help Sanofi select patients for one of the important antibody discovery programs. By leveraging our translational platform for our partners, we aim to potentially increase the probability of success for the patients we are seeking to help. Our journey to achieve automation in drug discovery and development is well underway. We're working towards automating not just design, but also some of the biggest bottlenecks in the design-make-test cycles, such as compound synthesis and molecular profiling. Our state-of-the-art automation suite will become operational next year, and we're excited about the potential for ushering a new era of what's possible in accelerating productivity. And with the announcement of top line data in our A2a program, EXS-21546, one of our first-ever AI design molecules entering the clinical trials, we demonstrated the ability of our AI platform to create novel molecules based upon defined design objectives. We're engaged in ongoing translational work to establish a predictive biomarker to enable targeting patients most likely to benefit from our molecule. This has the potential to unlock the key challenge in effectively treating patients with high adenosine signature cancers. We look forward to showing more details on our progress. We're using simulation guided clinical trial design to plan our clinical trial for EXS-21546, which we're calling [Ignite AI]. Identifying the right patients remains core to our approach. We're also using our translational platform and innovative approach to trial design, as we plan our CDK7 CTA submission by the end of the year. We expect to start a trial in patients in the first half of 2023. Overall, we look forward to nominating additional drug candidates and reporting further pipeline progress in the second half of the year. And now, I want to take a deeper dive into the interplay between our business models and our technology strategy and how we're able to achieve productivity and why we are so confident in our ability to achieve success in the future. In short, the more we do, the more we learn. Exscientia's overarching mission is to create better drugs for patients by applying AI and novel technologies, which allows us to be faster and more scalable from traditional methods. We apply the same tech-enabled strategy to all our programs, wherever we are developing new medicines for our own pipeline or for a partner. A fundamental aspect of what we do is based on the scalability of our technology platform. In previous earnings calls, we described how different aspects of our filing approach to AI-first drug discovery in precision medicine and why we differentiate it. It is not simply how differentiated technology platform for this key to success, but also the opportunity tech scaling gives to a strategic interplay across our drug discovery pipeline between our partnerships and our own internal pipeline. Scalability and systems of a technology company means we can create a different way to balance risks compared to a traditional biotech. In our internal pipeline, our focus hones in on areas where we haven't established expertise and where we believe our precision medicine platform can provide the greatest probabilty of success. Our CFO, Ben Taylor, will provide more details on this in a few minutes. Our partnerships not only generate substantial standalone value, but also have a practical application of allowing us to scale. The more we do, the more platform learns and improves. By applying our tech to do challenges, we're expanding our knowledge base and, more than that, increasing the impact that we can bring to the world. So how does translate into the actual pipeline? Because of our technology capabilities, today, we are able to progress a multitude of discovery programs in our pipeline balanced between internal and partner programs. We have a Phase I asset about to enter Phase Ib/II, 3 programs in IND enabling studies, news that which we expect to announce in the coming months, and the rich pipeline of early drug discovery programs that we've highlighted here. Furthermore, beyond what we have presented here, we have an active program of early stage portfolio of target indication and validation projects. We continue to generate targets on our partners, including Sanofi, as mentioned earlier, and have 2 approved programs moving into design. Focus is critical for us. We have prioritized the results at both areas where we can have the greatest impact on better drug development, translation, and patient selection. This has resulted and decided to de-prioritize a few early stage programs so that both resources can be moved to our core areas where we believe outpatient-based translational platform can be applied to increase the overall probability of success in selecting the right patients. Our message today is one of focus and how we can leverage all our platforms and truly impact the probability of success in the clinic going forward. In previous earnings calls, we have talked about Exscientia's AI-first end-to-end process to drug creation. We originally pioneered AI-driven drug design. But as the business go, we have grown downstream into clinical development and upstream into targeted indication. We have a number of parallel and complementary approaches to generate new projects to our pipeline and our partnerships. New projects can come via our center (inaudible), which applies deep learning to genome scale knowledge grasp to identify connections and predict target disease associations. This technology has been actively used to prioritize targets for validation as part of our collaboration with Sanofi. We can also use our patient tissue translational platform to identify new drug targets. You may recall at the AACR earlier this year, we presented an example of how we discovered ALK as a potential new target for ovarian cancer used in our human tissue precision medicine platform. We are also sourcing new assays, models and targets from outside Exscientia, with programs, such as Xcellomics, where we're seeking to accelerate early-stage research with phenotypic screening of novel disease models with academic labs. I'd like to highlight an example where using our capabilities in just 1 quarter, we were able to identify around 20 novel target hypothesis with high confidence from the initial genome-wide search in a collaboration with one of our partners for an inflammation and immunology indication. This is a nice example of the power of our operations, technology and platform, and we can continue to improve them. We plan to present the deep dive of our experimental and AI driven approaches to targeted implication and validation at a future earnings call. I'm now happy to hand over to Richard Law, our Chief Business Officer, who will walk us through how we think about our platform and business development strategies tied together as evidenced by our news flow. Richard and the business development team are quite prolific and created exceptional value for the company. Richard? -------------------------------------------------------------------------------- Richard Law, Exscientia plc - Chief Business Officer [4] -------------------------------------------------------------------------------- Thank you, Andrew. I'll now take a few minutes to walk through our approach to business development at Exscientia and how this has evolved over time. Since the founding of the company, we've continued to expand our capabilities. And in turn, this has increased the value of partnering with Exscientia over time. Using the combination of AI tech platform and the human tissue platform Andrew mentioned earlier, our approach can validate targets in ways that others can't. Rather than relying on the limitations of the traditional industry-dominant human expertise-led approach to selecting targets for new drug discovery projects, one which is a long and sequential process, but then often relying on a single key academic publication. We are using AI to bring to bear a new level of scale and connectivity to turn publication overload and biological complexity into a data-driven confidence builder on which to base the start of new drug discovery projects. Another area to highlight is how we have integrated advanced molecular dynamics and quantum mechanics into our approach to help us fully utilize and understand the potential for a protein structure, whether it is determined or AI predicted to guide molecular design. Molecular dynamics allows the quality of a structure to be assessed, but also provides a movie-like insight into the target's flexibility instead of just a snapshot. This is just one example. As we continue to build and scale these enhancements then builds our value add, not just for us as we can improve on our efficiency, quality and probability of success, but also for our partners. And so the deal terms, milestones and royalties have reflected this build-out as well. We've seen a shift over the past few years. The more end-to-end, fully externalized drug discovery programs with all of our pharma partners, such as BMS and Sanofi. As we expand our capabilities, so the breadth and value of what we can bring to partners will grow. Going forward, within our partnered programs, our aim is not just to do more. It is to have increasing ownership of the partnered projects within our pipeline, principally by enabling a match of drug to patients in the clinic. As an example, our collaboration with Sanofi includes an option for clinical co-investment with a royalty rate up to 21% on net sales of co-funded projects, without any reduction in milestones for the delivery of the asset by the AI discovery engine. There are several potential ways in which we can deliver value for partners, leveraging our end-to-end process. Our partnerships with pharma are often focused on the ability to design a better molecule from an idea, whether that's for a known or relatively novel target. Normal targets are often the least well understood or studied, but the value may be the highest here and where we can provide differentiated expertise since well-known targets will already be well known by others in the industry. It's a balance, but with a combination of biology AI and patient tissue data, we believe we can break the dogma of novel targets being riskier. We can predict who the patients are going to be, even before we start the discovery project. This is key to altering the probability of success equation in the industry in an end-to-end fashion. For known targets and/or molecules, the platform can assess molecules to select the patients and indications for which they are best suited in a way that others can't, as we are testing against actual patient tissue. Thirdly, we have the potential to match the right drug candidates or combinations to the right subsets of patients with our translational work. This is something Andrew mentioned earlier that we are doing for Sanofi. Consider the number of molecules that may have been pushed to the side, that may have unrealized potential for patients. For instance, certain drugs that successfully treated patients in our EXALT-1 trial that had previously failed in clinical trials for that same indication. As we work with partners, we're often generating a number of high-value potential targets, not all of which will be selected to advance. As the internal pipeline advances, some assets will need to be monetized to manage cash flow, and this will mean finding partners for those molecules that will best shepherd them in the clinic with our help, of course. And while there are many opportunities to add value for our partners, our partners are increasingly tapping the full extent of our capabilities from early target discovery all the way through to eventual patient selection. Put simply, the real potential lies in a reengineered process of drug discovery and development, which is the vision and track record our business development team aims to articulate. That is, how when implemented, alongside the reengineered process, AI can drive a step change in the probability of advancing new precise medicines to exactly the right patients. I'll now turn the call over to Ben to walk us through how we think about scale and impact business development has on our platform. -------------------------------------------------------------------------------- Ben R. Taylor, Exscientia plc - CFO, Chief Strategy Officer & Executive Director [5] -------------------------------------------------------------------------------- Thank you, Rich. We are often asked 2 important questions. First, how do we determine if a new target should be developed internally or through partnership? Second, how do we think about the value of a partnership program? In the following section, we'll address those questions from a strategic and economic perspective. Before we talk about our business strategy, it's important to spend a moment on the operational value of scale. Economies of scale certainly can benefit us. But much more importantly, increasing our scale can help us design better medicines. The more pipeline programs we work on, the more data we generate and the more design problems we solve. This data and learning can then feed back into the platform to design better drugs more efficiently in the future. Traditionally, in biotech, developing 2 pipeline programs in parallel requires approximately twice the number of people where CRO services is developing one. However, by using a combination of AI-based technologies and novel processes, we have repeatedly demonstrated exceptional productivity gains on both the time and cost compared to traditional benchmarks, while maintaining a relatively small infrastructure. So for us, scaling is not just about developing a bigger pipeline. It is actually helping us develop a better pipeline. This is exactly why we continue to expand our closed loop operating model from target ID through the clinic. The more aspects of development and data we control, the more robust our system can become. Not only do we already have a broad pipeline, but our capacity allows us to initiate another 5 to 10 programs per year. This means that we can continue to expand both our internal pipeline as well as our partnerships into the foreseeable future. We would expect the majority of new programs to be initiated in the partnership model going forward, with a few focused candidates chosen every year for internal development. Here, we can see the valuation impact of our partnership terms by comparing the net present value received in a partnered program versus the same drug as a wholly owned internal program. The chart shows risk adjusted discounted cash flows over the life of a drug candidate, in this case, at a 10% overall probability of success to reflect a slightly better rate than the industry average of 4%. For the orange wholly owned line, we have used publicly available data to create an example of the average cash flow profile for a drug from discovery through commercialization. The cash flow potential is very high, but requires substantial investment, time and risk. In other words, this is a typical biotech model. And you may recall, we shared the assumptions behind this model last November as well. For the teal partnership line, we have used the general terms of our 15 target Sanofi agreement from earlier this year. That partnership uses a broad range of our capabilities, including our target identification, AI-based design and precision medicine platforms, resulting in per project economics of $343 million in potential milestones as well as royalty rates from the high single digits up to 21%. In this example, we are using a low teens royalty that would be appropriate for the average product described. To sum the wholly owned scenario, it results in about twice the discounted cash flows as a partnership, but that only comes after several years of large investment, with substantial binary risk. By comparison, the partnership is always cash flow positive from a direct cost perspective, which strategically makes up for some of the lower NPV. To be clear, we would always prefer twice the value. However, our capacity allows us the flexibility to do both. We can select a focused group of wholly owned targets where our internal capabilities may improve the probability of success, while still capturing a large portion of the additional pipeline value with lower risk through partnerships. In addition, our partnerships are providing us with an operational benefit by building out and improving the technology platform. Now let's take a step back and look at what our current partnership business means in terms of value. If you run all of our BMS and Sanofi programs through the same NPV analysis, it would equal just under half the NPV as if we were to internally develop those candidates as wholly owned programs. Hopefully, this gives a sense of the importance our partnership business contributes to our long-term business model. On this slide, you can see how the model's net present value changes based on our assumption of overall probability of success. The greater the probability of success in the model, the greater the magnitude of value we may achieve from a wholly owned program as compared to a partnered asset. Importantly, even at 100% probability of success, the wholly owned program is only about 3x the partnered NPV, highlighting the importance of having a combined strategy. What this shows is that if we believe our internal capabilities can significantly increase our probability of success, pursuing a wholly owned strategy becomes more valuable. On the other hand, if we think we can increase the probability of success with the partner's capabilities, the partnership model will become closer to or even surpass the wholly owned value. And so we are thoughtful in evaluating each target on our internal ability to improve probability of success. It needs to not only be a target where our AI design will create a differentiated drug, but we also need to know that our precision medicine platform will guide our clinical development, and we will have the internal expertise to execute and potentially commercialize the program. You can also see why we would invest in better translational systems that enable patient enrichment and improved data-driven clinical trials. Both these investments are directly tied to increasing the probability of success at critical points of development. Our approach also provides us benefits and risk diversification. As I just mentioned, we focus our operations on improving probability of success by targeting the most common points of failure in discovery and development. However, on a very basic level, we target binary valuation risk by having a broad portfolio. We believe that having dozens of pipeline candidates increases our chances of having commercial products. As you've seen from our financial results, our partnership business has brought in over $200 million over the last 18 months. This allows us to continue rapidly expanding our platform and pipeline with substantially less dilutive financing required. Finally, we also believe it is underestimated that many biotech companies have a pipeline based on a single scientific hypothesis. If that hypothesis fails, it may undercut all of the products in the pipeline. At Exscientia, we execute projects across therapeutic areas, target classes and types of small molecules, which provides a high level of scientific risk diversification. Andrew earlier spoke about our strategy in the 2 different pipelines. However, it's important to note that, operationally, these 2 models look very similar until the point of entering the clinic. This has actually provided us substantial flexibility in adjusting our pipeline to fit changes in our long-term goals. We have moved programs from our internal pipeline into partnerships as well as converted partnerships into joint ventures. Our core focus is to successfully launch new medicines, so we will continue to be nimble and rational on how we achieve that. Now we'll take a minute to close with highlights from our financial results. Full results are detailed in our press release and Form 6-K. We ended the quarter with $732 million in cash equivalents and bank deposits. We believe this gives us several years of cash runway and the resources to continue investing in our business expansion and differentiated pipeline. Our cash inflows from collaborations for the 6 months ended June 30, 2022, were $117 million, which included $100 million upfront payment from Sanofi. Importantly, our strategy, which we have just reviewed, creates a potential stream of nondilutive capital inflows, helping maintain a strong balance sheet to fund our business. As an example, our operating cash flow in the first half of 2022 was positive $63 million. It is important to note that we do not expect to be operating cash flow positive for the full year, as we continue to invest in our pipeline and platform to best position ourselves for the future. With that, I'll turn the call back over to Andrew. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [6] -------------------------------------------------------------------------------- Thank you, Ben. 10 years ago, Exscientia was founded by a small team in Dundee, Scotland, nearly all of whom are still with us today. Since that time, we forged our way as a leader using AI-driven approaches to modernize how new medicines are created. We've pushed the field by bringing the first AI-designed molecules to clinical trials, and we were the first to improve clinical outcomes in oncology using AI. We are truly transitioning from rapid growth and scaling to now entering a period focused on stability and execution against our long-term vision. And with that, we'll open up a call for questions and answers. ================================================================================ Questions and Answers -------------------------------------------------------------------------------- Operator [1] -------------------------------------------------------------------------------- (Operator Instructions) Our first question is from Michael Ryskin with Bank of America. -------------------------------------------------------------------------------- Michael Leonidovich Ryskin, BofA Securities, Research Division - Director in Equity Research & Research Analyst [2] -------------------------------------------------------------------------------- Great. And appreciate the overview. I was just wondering on that -- some of the last points you kind of touched on the ability to execute on the (inaudible) platform across multiple programs. Obviously, with the $700 million cash balance, you're really well positioned for a number of year. So could you give us a little bit more color on your hiring plans, your investment plans, both in R&D and the rest of the organization over the next 6 to 12 months. How should we think about deploying some of that capital? -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [3] -------------------------------------------------------------------------------- Excellent. Mike, good to hear from you. Thanks for the question. So Ben and I will actually give an answer to this question. So we're well positioned, actually. We've been through rapid scaling over the past few years. We doubled in size year-on-year for the past 4 years now. And at the time of the IPO, we're around 200 people, and we're just over 400 people at the moment. But we do expect to see -- now we can start to see how now the business starts to sort of stabilize around the capacity, which Ben started to describe in his slides, and the ambition we have then for the partnership business. But at the same time, we do continue to invest in technology areas where, we believe, it actually give us a continuous advantage, both for developing our own molecules and our own programs, particularly focusing now in the areas of precision oncology and also areas where we see that we have the next generation of productivity enhancer is going to come from, where that's from, investments in automation, investments in biologics, designed by AI as well. So we do see that the investments we have put in place are actually allowing us then to grow a company, which we believe actually would create a portfolio of agents, which will be designed with translation biology and precision medicine in mind right from the start, but also in a platform, which we believe actually will continue to be one of the most productive drug discovery engines in the industry. So that's a core sort of areas where our strategic plans for the next years are leading to. But to give you a bit more color on that, I want to introduce Ben actually, who has been -- whose team has been doing a lot of work actually in preparing our plans behind this. -------------------------------------------------------------------------------- Ben R. Taylor, Exscientia plc - CFO, Chief Strategy Officer & Executive Director [4] -------------------------------------------------------------------------------- Mike, always great hearing from you. So -- well, I think, what hopefully you picked up from the presentation is both of the business models have a great value proposition. So I think what this really highlights is, no question, we've got the balance sheet capital to make investments. However, what we want to continue doing is make smart investments. So where we start everything is how is this either going to benefit our long-term platform or how can we improve the probability of success on a drug that we're designing. And so that's really where we start. It's not about, hey, we're going to set out and invest x number of dollars this year. It's more of a question of how can we make investments that have a lot of impact to them. If we can come up with a great translational strategy and do a patient enrichment scheme around the clinical trials, where we're really impacting the potential to get to market and get to patients, that's something that we're much more likely to do internally. However, we've got a lot of great partners who come in with their own technologies and viewpoints and capabilities. And sometimes, they have a way to do it, and that should be partnered programs. So I think when we take a step back, what we want to do is make sure that we are always in control of the business. And so we have the flexibility to continue investing, but also to make good business decisions. We never want to be held to outside influences. And so I would expect us to have a continuing balanced business model. And to be honest, I kind of like having a low burn rate. So I don't think we're going to flip that the other way around. -------------------------------------------------------------------------------- Michael Leonidovich Ryskin, BofA Securities, Research Division - Director in Equity Research & Research Analyst [5] -------------------------------------------------------------------------------- Okay. Great. That's helpful. And if I could ask a follow-up on the 21546 Phase Ib to start, could you remind us of any details you provided on sort of the format of that trial? Sort of how many patients are you looking, the duration? When should we expect the next data point there? Just -- and if there are any other -- any other things we should be looking for in the clinic in the next 6 to 12 months? -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [6] -------------------------------------------------------------------------------- Excellent. Mike. So yes, we've given further details of the 21546 trial design in the next few months. We're really excited by what Mike Krams, our new Chief Quantitative Medicine Officer has been bringing to the organization and particularly thinking about how we're using simulation driven clinical trial designing that and how that has been integrated then with our work on using a precision medicine platform for thinking about sort of translational biomarkers with that. So we'd be really excited to bring that forward to you in the next few months within it. Just to sort of recap on the timings and how we're thinking about the practicalities of it in the trial, I just want to hand this over to Dave Hallett, our COO. -------------------------------------------------------------------------------- David Hallett, Exscientia plc - COO [7] -------------------------------------------------------------------------------- Yes. Thanks, Andrew. Just to reiterate what Andrew said is that the sort of precise details about kind of study designs for both 21546 and the CDK7 asset will be forthcoming in the second half of the year, so watch out for that news flow. But as Andrew said, I think one of the beauties of kind of Mike Krams joining us who is a world expert in kind of, let's call it, creative kind of clinical trial design, reminding that he was the first person ever to kind of lead an effort to use kind of a Bayesin statistics approach to running clinical trials. So what he's brought to the team, I think, not just for the A2a asset, but other ones is that we're running a lot of simulations, kind of hundreds of simulations, in the last few weeks and months just looking at how best to kind of optimize that forthcoming study, which obviously will be in patients, looking at kind of interactions with kind of standard of care. So I can't give you precise details today about patient numbers, about kind of -- about cancer types, but be patient. That will be forthcoming in the second half of the year. In addition to that, clearly, a key part of that is the work that we're doing with our translational platform at the moment in terms of both kind of drug response biomarkers, but also kind of patient response markets. So yes, that will all be forthcoming and a lot of details during the second half of this year. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [8] -------------------------------------------------------------------------------- And Mike, just to the second part of your question, and I hear the word sort of further catalyst coming through as well, and I just want to sort of open it up to Ben, actually, who's been looking at actually the range of potential events we got flowing down actually over the next few months. -------------------------------------------------------------------------------- Ben R. Taylor, Exscientia plc - CFO, Chief Strategy Officer & Executive Director [9] -------------------------------------------------------------------------------- Yes. And I would think about it as 3 different areas where you might see things. One is pipeline, two is operations and three is business development. So on the pipeline front, obviously, we've got a lot of candidates that could be moving forward over the course of the next 6 and 12 months. Certainly, our CDK7 and A2a drugs are looking to start the next phase of clinical trials soon for both of those, and more updates coming on that, as you noted. But we have a number of other pipeline programs that are moving forward both internally, and that have been in-licensed by some of our partners. Obviously, we don't have control over the timing of events for our partners, but we will let you know when that happens. And then in addition, I think, operationally, we're looking at a lot of new platform developments coming on. We actually have a lot of technology development going on internally. I think we've probably got one of the biggest and best technology teams in the industry, and they're being incredibly productive right now. So we'll continue to keep you updated as those things come forward. And then finally, Richard has obviously been busy on business development, and we hope he's not going to coast for the next 12 months, and we'll be very busy as well. -------------------------------------------------------------------------------- Operator [10] -------------------------------------------------------------------------------- The next question is from Vikram Purohit with Morgan Stanley. -------------------------------------------------------------------------------- Vikram Purohit, Morgan Stanley, Research Division - Equity Analyst [11] -------------------------------------------------------------------------------- So my first one is on the A2a program. So I know that we're waiting to see more details about the design of that study over the next couple of months here. But on the simulation guided design principles that you mentioned in the release and throughout the call, is there any more color available on particularly what kinds of design features you may be thinking about adding, how these design principles might impact time lines to initial data and the parameters of data we might initially see? And then lastly, how transferable are some of these design features from the A2a program to other pipeline compounds that you're going to be looking at and that are going to be moving forward in the next 1 to 2 years? -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [12] -------------------------------------------------------------------------------- Vikram, good to hear from you, and thanks for the question. That's a great question, actually, and it touches upon one of the reasons why it was so important for us to bring someone with Mike Krams' skill set into the organization because it's fundamentally for us about we want to be as innovative in the clinic as we have been as innovative in developing new technologies in discovery. So the way we're thinking then about sort of computational driven, patient-driven clinical trial design in all these approaches is, as you hinted at, this isn't actually just a technology and approach for the A2a molecule. This is actually a philosophy, which we're looking to apply to all our clinical trial design approaches as we go forward. And we've got a number of other projects right now, which we're also actively working on in thinking about sort of trial design. So what we're looking at then is fundamentally how we cut the goal then of how to identify the correct dose and the correct treatment regime as efficiently as possible in the trial design to do that. So that would be potentially -- so obviously, you could think some of the key parameters, and we'll be trying to do that. So as Dave has just hinted that, these simulations allow us to go not just hundreds, but actually thousands of variations then on trial design to be run. And the simulations then allowed us then to think where we can find sort of a maximum tolerated dose and specific on oncology indications. And also really think about how then we design that trial. So we test in A2a then with standard of care and what are the right sort of indications where all sort of -- if there's a combination of standard of care is best applied. As I said, there'll be more details to come on that trial design, particularly how our trial design sits alongside, how we're thinking about developing and validating translational efficacy biomarkers, which is also key, and that's what we use in our human tissue patient based platform for as well, and actually then bringing those 2 fields together. I actually think it creates an almost unique approach for Exscientia on how we start to think about clinical innovation, and that actually is something we really look forward to telling you about in future earnings calls and talking to you through that. This is about how would we bring out innovation, apply some of the really greatest thinking we've seen in this field starting to emerge. So you don't expect sort of a traditional clinical trial approach from Exscientia. We do want to think about how we bring these 2 leading approaches to adaptive trial design and precision medicine together in how we're thinking about our trials, not just for A2a, but actually for all our programs. -------------------------------------------------------------------------------- Vikram Purohit, Morgan Stanley, Research Division - Equity Analyst [13] -------------------------------------------------------------------------------- Got it. That's helpful. And then a quick follow-up on -- going back to capital deployment and business development. So how does the unwinding of the Bayer partnership and the thought process that went into that mutual decision and then also the lessons learned that you've accumulated through the rest of your partnership that you have ongoing? How does all of that impact your perspective of what future partnerships could and should look like, looking out over the next couple of years? -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [14] -------------------------------------------------------------------------------- Good question, Vikram, and it's a good opportunity now to introduce you for the first time on our earnings call to Richard Law, our Chief Business Officer. Richard? -------------------------------------------------------------------------------- Richard Law, Exscientia plc - Chief Business Officer [15] -------------------------------------------------------------------------------- Thanks, Andrew. Thanks, Vikram. Yes, as you heard in the press release, we've sunsetted the collaboration with Bayer. And I think what you've seen is an evolution of the company over the last few years. We're much more end-to-end in what we're doing. But most importantly, we've created a system and a process that is now in place with every other partner of ours, whereby we drive everything. This is really vital to both making the best use of how AI works, but also in how we create value from the platform. So we ended on a high with Bayer. We delivered the lead candidate to the design criteria of the collaboration. And we wish them luck with that in the future. -------------------------------------------------------------------------------- Ben R. Taylor, Exscientia plc - CFO, Chief Strategy Officer & Executive Director [16] -------------------------------------------------------------------------------- I'll just add in one thing on that, Vikram. So as far as -- I would expect more in the vein of the BMS and Sanofi type of deals on the partnership side going forward. And obviously, that has a great economic return to us. But what we really think about is we want to be a partner on everything that we do across, at least, through the end of discovery. And our partners often may pick it up in clinical, but it's really got to be something strategic in a partnership before that. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [17] -------------------------------------------------------------------------------- Absolute. And look, in many ways, the reason why we sort of ended on a high with Bayer after getting the first milestone with them was actually to do with real development of how would you apply AI, and that's exactly how we've been building our end-to-end platform. And what we find sort of these older type of deals, which are sort of AI only sort of design deals are not the optimal way, which is why we built out Exscientia now, so we can really control the whole process of how we generate experimental data, how we add on upstream and downstream capabilities of target ID and precision medicine, and integrate all these into a new process. And that's fundamentally the advantage we see, and that's why now all our deal structures, all our partnerships are built that way. -------------------------------------------------------------------------------- Operator [18] -------------------------------------------------------------------------------- The next question is from Chris Shibutani with Goldman Sachs. -------------------------------------------------------------------------------- Chris Shibutani, Goldman Sachs Group, Inc., Research Division - Research Analyst [19] -------------------------------------------------------------------------------- With really a foundational tenet being the company's ability to continuously learn and weave into your efforts that what you're learning, I think when you think about your internal pipeline, I always think about your focus as being on small molecules and in oncology. And yet you made some comments about leveraging this towards biologics with some of your partnerships, et cetera. Can you talk about how you were thinking about this sort of therapeutic and modality focus as far as circling what you think might be priorities from an internal pipeline standpoint? And then I have a follow-up. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [20] -------------------------------------------------------------------------------- Excellent. Thank you, Chris. So yes, we're still developing these new areas. And you can see from some announcement we've made where you might think the direction of travel is going. I mean, in January, we hired Professor Charlotte Deane from Oxford to head up and build our new biologics AI design group. That's obviously a strong signal, and that has been going incredibly well. I'm very pleased to see Charlotte join the company, really apply her talents and also win her MBE actually earlier this year. The other thing as well, of course, is there's some important elements around our precision medicine platform, which we think actually opens up a really interesting possibility in this whole biologics space, which is we've already validated our precision medicine platform using human patient tissue for use in both small molecules and antibodies. And in fact, it's already built into the Sanofi deal. And in fact, we've already started our first precision medicine projects with Sanofi, where, actually, we're bringing in their antibodies discovered by Sanofi into our sort of precision medicine sort of pipeline and is a whole set then of milestone-based success fees then based on how we can help Sanofi select patients. So you'll be hearing more of us in the future, I think, Chris, once we're actually sort of ready for prime time to talk about these new developments. But I think you can start to see things taking shape where we are, it's public knowledge, actively interested in how we can design biologics using AI and, importantly, have already validated how we can use precision medicine downstream to test biologics. And we think those 2 things coming together create something very interesting. -------------------------------------------------------------------------------- Ben R. Taylor, Exscientia plc - CFO, Chief Strategy Officer & Executive Director [21] -------------------------------------------------------------------------------- Yes. I think the other really important aspect is we're actually building a very modular system. The way that we do it and how we remodel the process actually allows us to slot things in and out. And so if you think about our 5 areas of capabilities, so we do target ID, we can do the drug design using our AI systems, we do a lot of experimental biology, we do our translational technologies, and we're expanding into the clinic. So if we put another modality into design, for example, that would really just be sliding out the small molecule design and putting in a different modality. But the rest of those modules that I talked about would still function in the exact same way. And so we are designing from the foundation to be able to build and grow our business over time and be very flexible on how we advance medicines to patients. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [22] -------------------------------------------------------------------------------- That's a great point, Ben. It's about engineering that process and how we can bring in new technologies and really get synergies of that new technology across the rest of the platform. Chris, it was another part to your question I just wanted to follow up actually. So today -- sorry, sorry, which was oncology focus, yes. So certainly, we do see that for the internal pipeline. As of today, it is small molecule focused and more and more so now focusing on where we can see we can gain an advantage of increasing our confidence and probability of success in the clinic, where we believe we can build validated and high-confidence translational models. And right now, that's in the area of precision oncology. That's where we believe our precision medicine platform, which we already got the first sort of clinical validation of, is giving us confidence. And our goal then is to align our sort of target validation, therapeutic area strategy towards areas which really align then to the clinic and areas where we believe we have higher confidence and probability of success based on these platforms. -------------------------------------------------------------------------------- Chris Shibutani, Goldman Sachs Group, Inc., Research Division - Research Analyst [23] -------------------------------------------------------------------------------- Right, and that leads exactly to my second question. I guess, there was a discussion earlier about 546. Your other near to approaching the clinic asset 617, the CDK7 inhibitor, I believe, with Apeiron. So I think that is that challenge, doing the preclinical into the clinic transition with confidence and enhancing the probability of success. I suppose a standard question will be what types -- what remains to be seen in order for you to feel that you can transition to the clinics just from a housekeeping standpoint. But what might be the first signs that you can demonstrate clinically that 617 is differentiated? And what do you need to learn in humans to inform the next stages? Or can you already set the clinical path based on your preclinical work alone? -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [24] -------------------------------------------------------------------------------- Thanks, Chris. I'm going to open this one up to Dave Hallett, who's been leading the 617 program. -------------------------------------------------------------------------------- David Hallett, Exscientia plc - COO [25] -------------------------------------------------------------------------------- Again, Chris, good to meet you virtually. Really good question. The housekeeping piece, let's deal with that one first. So the regulatory talks, kind of clinical formulation piece, all the kind of the boring but necessary pieces we have to do to get it compound to is the patients. They're nearing kind of successful completion, so that kind of regulated the IMPD piece and the tox package that kind of goes with that molecule is there. So that's why we remain confident about giving guidance about opening the CTA later this year. In terms of differentiation where we go next, the things that we'll update you on kind of during the second half of this year and probably a medical meeting into next, the things we're looking at the moment was the -- particularly for CDK7, what's the kind of optimal dosing regimen to kind of maximize the therapeutic index. So -- and preclinically, we're doing work at the moment looking at different dosing schedules to maximize that. In addition to that, we've released some data, and we'll release some more in the next 6 to 9 months, most likely around -- so I refer back to the AACR poster where we started to kind of get an understanding of both the phenotypic level using our platform, but now looking at more molecular levels. What is kind of -- I'm sorry, referring to the ovarian cancer work that we did. We're now getting an understanding, and we look forward to releasing that data as to why certain subgroups of patients in the ovarian cancer setting, what were the molecular markers that underpinned the differential response to the drug. So I think -- yes, so some of the things we're looking for will be to show differentiation and progress is really -- the early part of the clinical study will really be kind of looking not so much for efficacy, but maximizing the therapeutic index, making sure that we find the right dosing regimen in patients, so that we properly test the mechanism. And then we're just narrowing down at the moment, and we'll release that information in due course, about the specific kind of counter subtypes that we're going to look at. So I can't give you any more precision on that at the moment. But like 21546, Chris, we're pretty close and in very advanced discussions about the kind of clinical trial design. So look forward to sharing that with you and the rest of the world in the second half of the year. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [26] -------------------------------------------------------------------------------- Sorry. Ben is on the line. Wonderful, but you go first. -------------------------------------------------------------------------------- Ben R. Taylor, Exscientia plc - CFO, Chief Strategy Officer & Executive Director [27] -------------------------------------------------------------------------------- Okay. I was just going to say, Chris, this goes back to something that is very close to our heart and how we think about operations when you were talking about what can we do before we start the trial. So throughout our company, starting from target ID, all the way through the clinic, we always try and use a data-driven modeling, tech-forward approach to doing things. However, you have to balance that with experimental validation. And so the clinical trials will be no different. We intend to start our clinical trials with a tech-driven thesis that has been backed up by the best experimental data that we can get. However, if you think about the heart of an adaptive trial, for example, and that's just one way to do it, there's different ways to do it. That is actually validating that hypothesis in the clinic. You're using Bayesin statistics to do it much more efficiently on much smaller patient populations, but you're basically validating that hypothesis and then designing that, so that the trial can continue once that's been validated. And so we'll continue to use methods like that, that are familiar to the FDA and that the FDA actually really appreciates. But you have to go through and validate these things in the early-stage trials. I think one of the differences is we can do it a lot earlier than other people would. Most of the time, you see people running Phase I and Phase II trials, doing a post hoc analysis and trying to figure out what the right strategy is. We're able to do it from the beginning with the early design and then factoring that into even the Phase I trials. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [28] -------------------------------------------------------------------------------- And Chris, just one final point I just wanted to add to that, particularly around Ben's point on validation, which is, as well as obviously running trials on our drug assets, we continue to look to clinically validate our precision medicine platform, all those things. So as Ben and Dave said, we get our validation as well alongside the innovative trial design that we've run in into leasing the biomarker that we're developing from our platform. But also we're looking for further clinical validation of our precision medicine platform beyond blood cancers, beyond the EXALT-1 trial. And we are looking to be initiating shortly further sort of studies where we start to think about how to validate our precision medicine platform in its own right in other solid cancers as well. -------------------------------------------------------------------------------- Operator [29] -------------------------------------------------------------------------------- The next question is from Peter Lawson with Barclays. -------------------------------------------------------------------------------- Peter Richard Lawson, Barclays Bank PLC, Research Division - Research Analyst [30] -------------------------------------------------------------------------------- Great. Just kind of dovetailing into Chris' question just on CDK7, just a number of [peer] molecules and whether you've kind of synthesize those and whether -- where you think you could potentially have differentiation based upon whether the structure of those molecules or the way they're kind of targeting. Any details around the CDK7 differentiation would be great. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [31] -------------------------------------------------------------------------------- I think probably just to remind you, refer back to information that was kind of generated and disclosed at the time of the IPO. So in the case of CDK7, differentiation against kind of competition, so because most of the molecules that we're aware of that are in the clinic at the moment, and that we have synthesized the compound itself or patent the examples from those companies. Potency selectivity is something that kind of stands out with our molecule. There are molecules that are a little bit ahead of it in the clinic. But in our hands, those molecules are the less potent against CDK7 or are less selective against the wider [kinase]. And we expect that to have kind of manifested several kind of negatives against our approach in that CDK7 itself is a promising mechanism, but it does come with mechanism-based toxicity. If you then add on kind of other kinase activity, you're going to erode that therapeutic index even further. So we're optimistic that we've designed a molecule that can push the therapeutic index as far as it will go in human studies. I mean, the other balanced properties of the molecules as well, so we kind of factored that over to the design. One of the key aspects is that one of the competitive compounds we looked at, we know for a fact actually that the molecule is -- experimentally is a substrate for a number of key transporters in humans. That itself is likely to kind of cause problems in terms of variation and exposure in humans. And just as importantly, that one of the transporters is actually present in tumors that are overexpressed and polarized, such that it is trying to push drugs out of a tumor rather than in. So I guess, in summary, differentiation from potency, selectivity and balanced properties, allowing us to kind of dose a molecule or really test the CDK mechanism, yes, we are not -- as we go into the clinic, I think doing simulations around dosing regimens to maximize that therapeutic index, again, using data and simulations, as we alluded to, kind of looking -- using kind of simulations as well actually on CDK7 to look at kind of likely effects that will happen together with standard of care. So a very -- a lot of data-driven methodology taking place at this moment, which we look forward to share with you in the next 6 months. -------------------------------------------------------------------------------- Peter Richard Lawson, Barclays Bank PLC, Research Division - Research Analyst [32] -------------------------------------------------------------------------------- And then just around the Bayer collaboration or the termination of the program, just any further details around that? And also I think you mentioned the potential to develop one of those targets. Any details around the target and then potential implications? -------------------------------------------------------------------------------- Richard Law, Exscientia plc - Chief Business Officer [33] -------------------------------------------------------------------------------- Yes, I can speak to that a little bit. So yes, so we successfully delivered the lead on the first target that we worked on with Bayer. We're really happy about that. We looked at several other targets. So we use the central biology AI platform and other elements of our target evaluation. And we decided on one other target to start work on. We also pushed back on several other targets that we felt like the target validation wasn't there for them. So we did start on another target. But essentially, at the point of deciding to exit, we agreed with Bayer that we could get full freedom to operate back on that target. So we'll potentially look to start to work on that, either ourselves or maybe even with another partner. -------------------------------------------------------------------------------- Ben R. Taylor, Exscientia plc - CFO, Chief Strategy Officer & Executive Director [34] -------------------------------------------------------------------------------- And one other thing to add on there, Peter. Just to be clear, this was, in many ways, an economically driven decision. Even though we've got wonderful scalability, it's not infinite. And so we need to be really disciplined managers of how we use our resources. So we showed the NPV analysis during our call for the Sanofi deal in comparison to wholly owned. I won't go into the details on Bayer, but it didn't look as good. And so if we're thinking about where we are going to have our team spending time, we would rather have them spend time on programs where we have better economics. And so you've heard it from several of us already. We had a very nice relationship with Bayer, and we'll do business with them again in the future, certainly. But that deal itself wasn't the right deal for the way that we do business now. -------------------------------------------------------------------------------- Peter Richard Lawson, Barclays Bank PLC, Research Division - Research Analyst [35] -------------------------------------------------------------------------------- Any details around the target that you would develop out of that relationship? -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [36] -------------------------------------------------------------------------------- I'll leave this, a very interesting kind of I&I target, but we -- again, just to kind of -- to link back to kind of one of the key themes of today's presentation is really around what we're looking at the moment is again being very accretive of, okay, what is the validation around that target, how does it fit with our precision medicine, is it a target that we feel, either ourselves or a partner, may have a different kind of pharma partner may have kind of technology that changes a view on a public success. So it's scientifically very interesting, but scientifically interesting only gets you so far. So we're spending a lot of time looking at what kind of what are the right patients for that target, kind of what's the landscape and then figuring out is this one for us to take on internally or one that's actually best within a partnership. -------------------------------------------------------------------------------- Peter Richard Lawson, Barclays Bank PLC, Research Division - Research Analyst [37] -------------------------------------------------------------------------------- And just a final question, I guess, for Ben or Andrew just on the ideas of any bolt-on acquisitions. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [38] -------------------------------------------------------------------------------- Good question, Peter. And Exscientia actually, I thin, has been very successful on how we've carefully acquired and integrated companies to grow from our early days, acquiring connected discovery would help us build our experimental biology capabilities, which then actually allowed us to really truly run in our own projects. That allowed us then to really enable the Celgene deal that we signed because we could also have first new deals of the new type we're running, where we could actually then manage the whole thing, running the experiments to doing a project management. And then, of course, last year when we integrated precision medicine into our platform with the acquisition of Allcyte, which has been an incredible integration into the company. And the team has been absolutely fantastic. It is as if we formed Exscientia together actually, I think, the feeling of team is. And so where we think about it now is how do we then position any sort of new acquisitions into our core strategy. I think, hopefully, what came across today, you can see there's a focused strategy emerging now. One that is -- if you think about the platform, how do we increase productivity continuously and how do we increase our probability of success. And that's why for us, building in precision medicine, using sort of the high content approach was so important. But what was more important was actually the network of biobank samples collected from dozens and dozens of hospitals, and that network is expanding as we speak. So what's important for us now would be to look at any acquisition, sort of think about how does it increase our probability of success, moving forward to the clinic, does it potentially allow us to maybe expand into new indications with a greater degree of probability of success, as we've seen with oncology with our current platform, those are the ways we think about something, actually. Those are ways we try to sort of position it, et cetera. And so that's the way we -- that's the way to think about it, Peter, as we go forward. Ben, anything to add to that? -------------------------------------------------------------------------------- Ben R. Taylor, Exscientia plc - CFO, Chief Strategy Officer & Executive Director [39] -------------------------------------------------------------------------------- Yes. I'd say we're always going to be data driven. We're not a dogmatic team. So we're just looking at it and saying, how -- if we're going to invest anything in an external technology, it's got to exceed our internal ROI capability in that area. And we've got a very productive operating team, so that's a pretty high bar. Typically, that's not going to be something that is a tech standalone. We are more interested in things that can integrate with experimental operations, like how are you making experimental data better, the focus of what would probably be interesting. But again, we've got plenty to do internally. We don't worry about doing M&A as a growth driver. But if we find something that makes a lot of sense, we're not opposed to it either. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [40] -------------------------------------------------------------------------------- Absolutely. So Peter, just to reemphasize what Ben just said. Coming back to our concept of modularity, as we build out new technologies, whether we build them ourselves or whether it's an acquisition, are we getting synergy? Is it truly something that we can see could be plug and play that would also synergize with the rest of the platform, whether it's target identification, drug design, different type sort of modalities, design engines or downstream into sort of precision medicine and into clinical sort of innovation, and that's the way to think about it actually. We don't standalone technologies. We want things that actually really give us synergy. So as we plug something in, we can really see that new modality concept gains from actually the rest of the platform. And I would say, our precision medicine, actually, precision medicine has gained so much more by being part of an end-to-end AI-driven drug discovery engine as well. -------------------------------------------------------------------------------- Operator [41] -------------------------------------------------------------------------------- We have further questions. At this time, I'll turn it over to Andrew Hopkins for any closing remarks. -------------------------------------------------------------------------------- Andrew L. Hopkins, Exscientia plc - Founder, CEO & Executive Director [42] -------------------------------------------------------------------------------- Thank you, Chris. We now have a growing pipeline that is progressing towards and actually into the clinic now. And we aim to be as innovative in the clinic as we've been put into our efforts into developing drug discovery and design, and then start to see that come to fruition and come to life as we advance our pipeline. We believe there are many more firsts and achievements that lie ahead of us. Thank you all for joining us today. And operator, you may now disconnect. -------------------------------------------------------------------------------- Operator [43] -------------------------------------------------------------------------------- Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.

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Edited Transcript of EXAI.OQ earnings conference call or presentation 18-Aug-22 12:30pm GMT - Yahoo Finance

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Roche launches COVID-19 test that detects and differentiates the emerging variant of interest BA.2.75 – PR Newswire

Posted: August 22, 2022 at 2:25 am

PLEASANTON, Calif., Aug. 22, 2022 /PRNewswire/ --Roche (SIX: RO, ROG;OTCQX: RHHBY) and its subsidiary TIB Molbiol have developed a COVID-19 test for researchers that detects and differentiates the latest subvariant of interest BA.2.75. The test specifically targets two of the known unique mutations in BA.2.75, which allows clear differentiation against other notable subvariants.

"Roche is pleased to have developed a test for researchers that provides insights into the epidemiology of BA.2.75, helping to understand its impact on public health," said Cindy Perettie, Head of Molecular Labs at Roche Diagnostics Solutions. "Being able to differentiate emerging variants and understanding their similarities and mutations, can provide a basis for experts to make predictions of their spread and response to treatment strategies."

Since it was first detected in India in May 2022, cases of BA.2.75 have been reported in more than a dozen countries. The BA.2.75 is a subvariant related to the Omicron variant. The subvariant is being monitored by both the World Health Organization as well as the Centers for Disease Control and Prevention.It has not reached the level of a Variant of Concern, but differentiating it from BA.4 and BA.5 allows for improved tracking to understand how it is spreading.

The available research use only test, VirSNiP SARS-CoV-2 Spike 147E 152R, adds to the broad suite of COVID-19 test kits developed by Roche and TIB Molbiol, for instance for the detection of the recent BA.4 and BA.5 subvariants, as well as other mutations present in the Omicron SARS-CoV-2 variant.

Roche and TIB Molbiol's researchers continue to work in collaboration with partners globally to continually screen for new variants and emerging diseases.

About the VirSNiP SARS-CoV-2 Spike 147E 152R

The test specifically targets two of the known unique mutations, K147E and W152R, in BA.2.75, which allows clear differentiation against other notable subvariants. This test is for research use only and is available globally. The VirSNiP SARS-CoV-2 Spike 147E 152R for use on Lightcycler(R) and cobas z480 analyser.

About Roche's response to the COVID-19 pandemic

As a leading healthcare company, we are doing all we can to support countries in their fight against COVID-19 and minimising its impact. That is why we are working with governments, policy makers, healthcare professionals and others to help contain the COVID-19 pandemic and make sure patients continue to receive the tests, treatment and care they need.

The pandemic has profoundly raised awareness of the role diagnostics play in COVID-19 diagnosis, treatment development and disease management. Roche has developed and launched more than 20 COVID-19 diagnostics solutions, including polymerase chain reaction (PCR) and rapid antigen and antibody tests. Our solutions serve the entire diagnostic continuum, from high-throughput laboratories to point-of-care and home self-testing, and cover all currently known variants. To help meet global demand, we have supplied more than 1.5 billion tests for COVID-19 since March 2020.

Roche continues to evaluate its existing therapeutic portfolio and is researching future options to help benefit patients with Covid-19. Our IL-6 inhibitor Actemra/RoActemra (tocilizumab)has been approved forpatients hospitalised with severe COVID-19 in more than 30 countries including the European Union and is authorised for emergency use in the United States. The World Health Organization has prequalified Actemra for use in patients with severe COVID-19, facilitating its availability in low- and middle-income countries. In addition, we have been improving access to Actemra by introducing an international differentiated pricing strategy, providing the medicine at cost for use in low- and middle-income countries and non asserting patents in these regions during the pandemic.

We have also been partnering with Regeneron to jointly develop the antibody combination Ronapreve (casirivimab and imdevimab, known as REGEN-COV in the US). It has been approved in multiple territories including the European Union, Japan, and Switzerland and authorised for emergency or temporary pandemic use in many countries including the US. The antibody combination has been made available to patients in more than 60 countries, across many geographies including low and middle income countries. As the virus continues to evolve, we are constantly monitoring Ronapreve's activity against emerging variants of concern, and will share results with health authorities as soon as possible.

Our utmost goal remains to be a trusted partner who acts with urgency to save and improve the lives of patients with COVID-19 and to reduce its burden on society. For more information please visit our COVID-19 response page.

About Roche

Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.

In recognising our endeavor to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the thirteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.

Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

For more information, please visit http://www.roche.com.

All trademarks used or mentioned in this release are protected by law.

For further information please contact:Elizabeth BaxterHead of Communications, Molecular Labs & Roche Diagnostics Solutions Portfolio Centre of Excellence[emailprotected]+1.925.523.8812

Nicole ClarkCommunications Manager, Roche Diagnostics Solution Portfolio Centre of Excellence[emailprotected]+1.317.361.9512

SOURCE Roche

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Will Omicron BA.5 Be the Last of the COVID Variants? – The Epoch Times

Posted: August 22, 2022 at 2:25 am

On January 20, 2020, the first case of COVID-19 emerged in the United States. More than 30 months on, the pandemic is still considered ongoing.

COVID-19, whose official name is coronavirus disease 2019, is caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2 virus). The COVID-19 pandemics continuation and severity are mainly due to the viruss fast mutations and its many variants. As a type of RNA virus, SARS-CoV-2 has a very high mutation rate. This is because the RNA polymerase that replicates the viruss genes doesnt have excellent proofreading skills. However, this actually helps the virus by allowing its variants to find new hosts and escape immunity induced by vaccination or previous in

fection. As a result, during the spreading of the virus, different geographic areas have generated genetically distinct variants.

In June 2021, the World Health Organization (WHO) started using the Greek alphabet to describe variants of interest, which are SARS-CoV-2 virus strains that are considered more dangerous than earlier forms of the virus. These variants are typically considered important due to their increased transmissibility, increased virulence, or the COVID-19 vaccines reduced effectiveness against them.

Prior to the WHO rolling out these rules, researchers around the globe came up with their own lineage naming rules to facilitate their genome sequencing of the virus. As SARS-CoV-2 is a rapidly evolving virus with a high rate of lineage turnover, some Western scientists used the letter A to denote the ancestral type, which is the Wuhan original strain, and the letter B to denote a derived lineage. Many variants of interest, such as alpha, beta, delta, and omicron, belong to lineage B.

The current variants of concern, or more precisely, subvariants (i.e. subsidiary variants) of concern, are BA.4 and BA.5. As of August 13, 2022, BA.5 cases accounted for 88.8 percent of all COVID-19 cases in the United States, followed by BA.4 (5.3 percent) and the latters own newer version BA4.6 (5.1 percent). From the statistics, we can see that BA.5 is more transmissible than BA.4, but so far, scientists havent gained an understanding as to why this is the case, as both subvariants are very similar.

As BA.4 and BA.5 are derived from BA.2, theyre very similar to the latter. According to a preprint study on MedRxiv, the spike proteins of BA.4 and BA.5 are identical, and comparable to BA.2. However, each of these subvariants has its own different mutations from BA.2 in other areas of the virus.

BA.4 and BA.5 are more contagious than any other subvariants of the SARS-CoV-

2 virus. According to a Japanese study, the Re value of BA.4 and BA.5 is 1.2 times higher than that of BA.2. The Re value, also known as the Rt value, is a viruss effective reproduction number, which represents the number of people in a population that can be infected by an individual at any specific time. This number represents a viruss transmissibility. BA.4 and BA.5 are 18.3 times more infectious than BA.2, and are currently the subvariants with the fastest increasing transmission rate.

BA.2 used to be the dominant subvariant around the globe, and BA.4 and BA.5 have rapidly replaced BA.2 in many countries, including the United States, since April 2022.

Furthermore, BA.4 and BA.5 appear to have higher pathogenicity than BA.2.

In a preprinted study of animal models, some Japanese researchers infected some wild-type Syrian hamsters with 105plaque-forming units of BA.4, BA.5, or BA.2 subvariants and performed histopathological analysis. They discovered that inflammation, hemorrhage, congestion, and alveolar damage were significantly higher in the lungs of BA.4 and BA.5-infected hamsters than in BA.2-infected ones. Therefore, BA.4 and BA.5 are more pathogenic than BA.2.

So far, the symptoms of BA.4 and BA.5 appear to be mostly upper-respiratory, including sore throat, running nose, incessant cough, headache, and fatigue, which are similar to the typical Omicron symptoms.

In comparison with the other subvariants, BA.4 and BA.5 are masters at bypassing immunity from a previous infection or vaccination. So people who were formerly infected by other Omicron subvariants can also get re-infected.

As the existing COVID-19 vaccines have been developed based on the original strain. Their protection against Omicron has been significantly reduced, and their protection against BA.4 and BA.5 may be even lower.

In the preprinted Japanese study, by analyzing peripheral blood sera from some convalescents, the researchers discovered that the serum neutralizing antibody titers with BA.4 and BA.5 in those patients who had had two to three doses of COVID-19 vaccines and later had a breakthrough infection by BA.1, were significantly lower than those with BA.2. That is, the combination of full-course vaccination and breakthrough infection cannot protect people from BA.4 or BA.5 infection.

Back on June 30, 2022, the Food and Drug Administration (FDA) warned that the effectiveness of existing COVID-19 vaccines had started to wane against Omicron variants, including BA.4/5. According to the FDAs statement, post-authorization observational studies have shown that effectiveness of primary vaccination wanes over time against certain variants, including Omicron.

Is the immunity gained from a previous COVID-19 natural infection effective against BA.4/5? The answer is: it depends. Although BA.4 and BA.5 subvariants are highly immune-evasive, the immunity acquired from an Omicron infection can still be helpful in protecting people against them. However, if the infection was by a different strain, then the protection offered by the infection-induced immunity may not be great.

The following studies shed some light on this issue.

According to the latest data from the UK Health Security Agency, similar to the pandemic situation in the United States, BA.5 has become the dominant subvariant in the UK, accounting for almost 80 percent of all COVID-19 cases.

The UK Health Security Agency has been carrying out a systematic cohort study (pdf) called the SARS-CoV-2 Immunity and Reinfection EvaluatioN (SIREN).

The participants of this study are more than 44,000 National Health Service healthcare workers from 135 hospitals across the UK. These participants are under active follow-up and undergo asymptomatic SARS-CoV-2 PCR testing every 2 weeks. The cohort had a high seropositivity rate of 30 percent before the second wave hit and is now over 95 percent vaccinated.

The incidence of new infections and reinfections is evaluated in this cohort. Reinfection is defined as a new infection (i.e. PCR positive) 90 days after a prior one.

The graph shows the fortnightly trends of PCR positivity in the SIREN cohort study, and they appear to be consistent with the trends of the alpha, delta, BA.1, BA.2, and other COVID variants. Starting from mid-May 2022, due to the prevalence of BA.4/BA.5, the trend has been going upwards again.

According to the graph, there has been an increase in both primary infection and reinfection rates since May 2022. The increase in primary infection rates indicates that Omicron and its subvariants have significantly enhanced transmission rates, resulting in a large number of infections. The increase in reinfection rates indicates that people who were previously infected can still get infected right now. Therefore, the immunity from natural infection doesnt necessarily offer protection against BA.4 and BA.5. Infection-induced immunitys effectiveness in preventing BA.4/5 infection depends on the strain of the previous infection.

Another preprint study was conducted by a team of researchers from Weill Cornell Medicine-Qatar in Doha. According to the study, young and middle-aged adults who were previously infected with earlier subvariants of Omicron may have substantial protection against BA.4 and BA.5. However, for the people that were infected with a variant that appeared before Omicron, they would not have similar protection against reinfection with BA.4 or BA.5.

This preprint study used the S-gene target failure (SGTF) infections to estimate the effectiveness of previous infections with SARS-CoV-2 in preventing reinfection with Omicron BA.4/BA.5 subvariants. The SGTF status provides a proxy for BA.4/BA.5 infections.

It was discovered that infection with a pre-Omicron variant prevented reinfection by BA.4 or BA.5 with an effectiveness rate of 28.3 percent, and prevented symptomatic reinfection with an effectiveness rate of 15.1 percent. This is because first, a long time has passed since the prior infection; and second, the virus has mutated a lot since then.

The study also found that a previous Omicron infection prevented reinfection by BA.4 or BA.5 with an effectiveness rate of 79.7 percent, and prevented symptomatic reinfection with an effectiveness rate of 76.1 percent. This is because first, the previous infection is relatively recent; and second, the viral mutations have not been large.

Therefore, regardless of previous COVID-19 infections, it is wise for us to maximize our own anti-viral immunity.

The BA.4 and BA.5 sub-lineages are the most transmissible and immune-evasive strains of the COVID-19 virus to date.What are the differences between them and the other subvariants that have given them their ability to escape immunity?

As aforementioned, BA.4 and BA.5 subvariants have identical spike sequences. In comparison with the BA.1 and BA.2 sub-lineages, they have L452R and F486V mutations and the R493Q reversion in the spike receptor binding domain (RBD), which is likely most targeted by neutralizing antibodies.

The production of neutralizing antibodies can be triggered by infection or vaccination. They can result in lifelong immunity to certain viruses. A neutralizing antibody can stop a pathogen from infecting the body by preventing the molecules on the pathogens surface from entering the human cells. As in the case of all enveloped viruses (i.e. the virus cells are inside a lipid membrane), neutralizing antibodies block the attachment of a SARS-CoV-2 virus to the cell and its entry into the cell to infect it.

Some viruses are able to evade neutralizing antibodies by having regular mutations. As a result, the antibodies can no longer recognize them. The BA.2, BA.4, and BA. 5 subvariants all carry a mutation in amino acid L452 of their spike protein. These L452 mutations facilitate their escape from some antibodies directed to certain regions of the receptor-binding domain, and such mutations have also caused several subvariants to appear.

The F486V mutations found in BA.4/5 also facilitate their escape from certain antibodies, whereas the R493Q reversion mutation restores receptor affinity (i.e. strength of the binding) and the fitness of BA.4/5. The Omicron lineage of SARS-CoV-2 continues to evolve, successively generating more subvariants that are not only more transmissible but also more evasive to antibodies.

According to Christian Althaus, a computational epidemiologist at the University of Bern, their capacity to infect people who were immune to earlier forms of COVID-19 strains has given rise to the prevalence of the BA.4 and BA.5 substrains.

The COVID-19 pandemic is entering its fourth year. People around the globe are hoping to see the light at the end of the tunnel. Then, will BA.4 and BA.5 subvariants be the last of their kind?

The answer is: no. Its almost certain that the virus will continue to mutate and persist.

According to Eric Topol, professor of molecular medicine at the Scripps Research Institute in California, We know BA.5 is not where this ends, unfortunately. We have further variants to work through for an indeterminate period of time.

Kei Sato, a virologist at the University of Tokyo, also holds the same view. He says, Nobody can say BA.4/5 is the final variant. It is highly probable that additional Omicron variants will emerge.

Already, a new variant, BA.2.75, has emerged and is spreading rapidly in India. Many medical experts believe that its another super-contagious Omicron mutant, and virologists fear that it will fuel a new wave of cases around the world.It has been given the nickname Centaurus by social media users.

So far, BA.2.75 has been detected in over 20 countries, with India being hit the hardest. According to the Indian authorities statistics, BA.2.75 is now behind two thirds of the new COVID-19 cases in India. However, it hasnt dramatically increased the countrys COVID-related hospitalization or death rate.

As the BA.2.75 subvariant is still evolving, it may develop more immune-evading mutations in the coming weeks. Some BA.2.75 sequences also have L452R mutations, which enhance the subvariants ability to re-infect people.

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Will Omicron BA.5 Be the Last of the COVID Variants? - The Epoch Times

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White Blood Cells (WBCs) – Definition, Function, and Ranges

Posted: August 22, 2022 at 2:24 am

Each year comes a new wave of common viral infections. And not many of us really understand the depths of how our body fights infections and bad bacterias off to maintain you health.

Although vitamins and a healthy lifestyle can encourage our body to combat infection. it is actually our white blood cells that do most of the work.

According to research, a human body has between 4,500 and 11,000 white blood cells per cubic millimetre of blood. White blood cells are essential for helping the immune system fight off infections. They are bigger than red blood cells, are fewer in number but have an equally effective impact.

Maintaining a healthy white blood cell count is essential for inhibiting infections. It can also encourage the body to fight off any foreign invaders. There are several medical, health, and lifestyle factors that determine how effective our white blood cells can be.

Today, we will discuss: what white blood cells are? how do they work? how to maintain healthy white blood cell levels? and how to assess if your levels are healthy?

The White Blood Cells (wbcs) or Leukocytes are the type of cells that have a nucleus and float freely in your bloodstream. Bone marrow, lymph glands, and nodes are the primary sources where WBCS gets produced. They pass directly to the bloodstream and live from several days to many years.

After this timeframe, they usually become deactivated in the spleen tissues. Their count is important for your general health and can show your doctor whether you have an infection or not.WBCS belong to the enormous cells of the human body.

Their DNA can increase the creation of antibodies according to the specific types of intruders. They are durable cells, which may attract and penetrate viruses and germs. The leukocyte population quickly reaches the infection site (alongside with platelets) and restores the balance in tissues.

There are several types of wbcs, each one serving a unique goal. Lets take a look at the leukocytes which take part in inflammatory disease conditions. They are essential parts of your immune system and can create a protection network for antigens coming from the environment.

Some of the white blood cells come from the bone marrow, while others come from the lymphatic tissues. They come in many shapes and sizes so that they can attack different threats, and offer you increased cell defense against blood infections or disorders.

They represent almost 67% of the wbcs present in the human body. The use of an electronic microscope allowed us to estimate their cell size coming up to 10-12m approximately.

They attack mainly the fungi and bacteria trying to enter and multiply in the bloodstream. The cell count of the neutrophils is always high compared to the other types giving your doctor many reasons to believe that you can deal with infections successfully.

Their life cycle varies from several hours to a few days top. They are all heading to spleen tissue, where their dissolution happens.

This type of white blood cell has the same size as the neutrophils (10-12m). Their name comes from the pink color they take under the microscope when the doctor uses a cell pigmentation method.

They belong to the wbc with low cell count (approximately 2.4%) and protect you against parasites or ease the symptoms of allergic inflammatory responses. They live only for a few days (no more than 8) and circulate in your bloodstream for less time to deal with infections and disease, getting things back to normal.

Scientists have found these white blood cells to be rare in your body, counting for less than 0.4% of the total immune system cells. They are the only ones who have two or three lobes, and their cytoplasm granules can take blue color under the microscope.

Their size is somewhere between 12 and 15m, and their main task is to release histamine to urge the inflammatory response of your body. These cells are the hardest to spot when having a blood test since their count is minimal.

The life span of basophil wbc keeps on being short, varying from a few hours to several days before getting destroyed in your spleen tissue.

One of the most valuable wbcs types existing in your body. They count for almost 30% of the total white blood cell population in your bloodstream. However, they are more apparent in the lymphatic system than in your blood.

These cells have a small nucleus that is close to the membrane and have two or three lobes. They include several types listed here:

They produce the antibodies and can also start the immune response, activating other types of lymphocytes.

Cytokines are their primary product. They activate the immune system against any infection. Furthermore, the CD4+ and CD8+ cell variations attack the cancer cells and can help to give patients increased immune system reactions and treatment for leukemia.

T-cells also include some suppressor types that prevent autoimmunity (attacking your body cells) or the overreaction against external infections.

As their name implies, this type of white blood cells can kill any virus or cellular intruder comes into your bloodstream. They can attack even the ones that have no compatibility with your body proteins called MHC (Major Histocompatibility Complex).

Lymphocytes vary in size. The smaller cells can have an average size close to 7-8m while the larger ones can measure up to the range of 12-15m in diameter.

They live from several days up to many years, since they are the memory cells of your immune system, offering an acute reaction to an infectious agent penetrating your body once more.

The waste management cells of your immune system are monocytes. They are the roomiest types of white blood cells, measuring from 15 to 30m each.

Monocytes are approximately 5.3% of your wbc count, and you may find them in your bone marrow or any other lymphatic tissue. These cells help neutrophils to fight against infections, keeping the medical treatment of diseases at normal levels.

White Blood Cells are the fighters of your immune system. They work through chemical routes and can activate themselves when an antigen comes into your bloodstream. Antigens are all chemical agents, viruses, germs, or bacteria that can enter your body through your skin.

Once the invasion gets detected by one of the white blood cells, a chemical message becomes available to initiate counterattacks to the invader cells or organisms.

Our body keeps a relevant number of wbcs to circulate in your bloodstream, retaining your health to normal levels. The production of neutrophils and lymphocytes gets high when an antigen enters your body.

All types of white blood cells are present in our bone marrow tissue and wake-up every time you need to have an inflammatory reaction against potential dangers.

The wbcs count is important to determine whether you had an infection or you have already overpassed it. It can show your doctor the possibility of having cancer, according to their type and number, which varies in several disorders.

According to the University of Iowa, Hospitals & Clinics, the white blood cells range and percentage to the total count seen as normal are as follows:TypePercentageNumberNeutrophil50 60%2 188 7 800Lymphocytes20 40%875 3 300Monocytes2 9%130 860Eosinophils1 4%40 390Basophils0.5 2%10 136

Have in mind that numbers refer to the cell count per ml of your blood specimen. These are the world recognized prices that get compared to the findings from your blood. All general blood control exams include the inspection of the wbc counts since leukocytes number will alert your doctor to propose further tests and examinations.

While some of the disorders affecting the count of white blood cells are hereditary, it seems like the modern way of life can inflate them to healthy individuals. Their number or their type is the first to get affected, and you have to perform regular medical examinations and a blood test to identify them.

Any deviation from the normal count can create severe problems for your health. Not to mention, that your immune system can become vulnerable to external threats and even attack your cells to initiate an inflammatory response called an autoimmune disorder.

Lets check what the most frequent disease is that has to do with the alterations from the normal count of your white blood cells.

Even though it is not that frequent, your doctor will be equally concerned with a high wbc number, seen in your blood test. This high number of leukocytes, known as Leukocytosis, could be the response to a recent illness and germ infection.

Leukocytosis could also be an alert to check your bone marrow function and search if you have cancer or malignancy in your bloodstream or any other organ.

Even though a high white blood cell count could easily justify its present, its always a good practice to run any additional medical test to exclude any possible disease. The high wbcs count may also be a signal of autoimmune disorders.

The Lupus is the most common autoimmune disease, causing the overproduction of lymphocytes giving you a chronic clinical inflammatory condition in need of medications and special treatment.

It is one of the cancer forms often seen in children and adults. The platelets and red cells are getting replaced by white blood cells. Bone marrow overproduces the lymphocytes, which are not working properly and aggravate the body functions.

Leukemia can be either acute or chronic. It is an urgent medical condition that needs radiation therapy and chemotherapy to get better. Bone marrow transplant is another therapeutic option to restore its normal function and bring the wbc counts to the right levels.

Having a low white blood cell count patient is the usual medical condition that a doctor will face during his career. These conditions are the well-known Leukopenia (meaning lack of leukocytes).

It is a dangerous condition for your health. The bone marrow tissue cannot produce enough Neutrophils and the leukocytes removal rate is higher than ever before.

Persons suffering from neutropenia are more vulnerable to infection and inflammatory disease. Your doctor will read and view your blood test, estimating the wbc counts to offer you the right medical treatment.

The reduction of normal Lymphocytes level is another severe medical condition. White Blood Cells who usually get affected are the T-cells coming from the Lymphocytes strain.

The low leukocytes level associates with a decreased immune system response to external infections.

All these types of cells are the particles of your blood. White Blood Cells are usually bigger than red blood cells and platelets. However, their most important difference is that White Blood Cells are the only ones having a nucleus.

That is the way a doctor can distinguish the ones from the others using the electron microscope when examining your blood specimens. Red blood cells give the red color to your blood, while platelets are important to heal wounds by forming clots.

It is necessary to know that all these cells come from a common ancestor in the bone marrow (stem cells), getting differentiated as the fetus grows. The balance between the wbc counts and the platelets or red blood cell number is an essential health index for your doctor.

Several common symptoms relate to White Blood Cells disorders. Lets check each one separately:

Neutropenia usually runs silently through your body without causing any severe symptoms. However, your doctor could be suspicious of low wbcs counts when you appear to have:

A low lymphocyte count could give you symptoms like:

Since leukemia affects millions of people worldwide, there are many known and described symptoms like:

The increase of leukocytes number can cause the following symptoms:

Today doctors have a wide range of treatment for all White Blood Cells Disorders. It would be wiser to examine it according to each type of wbc disorder:

The treatment usually associates with the type of problems caused by the increased leukocyte number.

Leukemia is a severe type of cancer that needs specific treatment:

Common treatments to Neutropenia include:

There are certain drawbacks in the methods you can calculate your white blood cell levels. The nucleated red blood cells usually take part in the count, changing the real measurement of the wbc exact number.

To obtain the real wbcs count you need to follow the underscored steps:

It is an easy way to have exact measurements of your wbcs count when you have the right equipment. Have in mind that getting the right number is important for your doctor to decide the kind of treatment you are going to take.

Many drugs can affect your White Blood Cells levels. The most important of them include the following.

Most people dont feel anything when they have a high white blood cells count. However, it can make you look pale and feel tired all the time. Not to mention, that rashes and skin itches are going to occur more often than previously.

Diseases that cause high White Blood Cells count include:

White Blood Cells have a life cycle that varies from several hours to a few years. They usually come to the apoptosis stage (death) when they have reached their goal to defend your body from external intruders.

The white blood cells direct to your spleen tissue where most of them are getting deactivated and decomposed after several days.

Certain foods can increase the White Blood Cells count in your body. These include:

=> Also have a look on our foods list to boost your immune system

Vitamin C can give a natural boost to the White Blood Cells creation. Your body cannot store Vitamin C and that is why it would be wise to consume oranges and citrus daily. Have in mind that you should never exceed the 2,000 mg per day dosage to avoid problems from your stomach and intestine.

Vitamin C acts as an accelerating enzyme to all the molecular reactions that proliferate the multiplication of T-cells and Lymphocytes in your blood.

The caffeine that is the main substance in coffee beverages can increase your energy levels and improve your ability to exercise more. A recent study has shown that people who consumed coffee and had exercise experienced an increase of 73% to their wbcs counts.

However, the direct relation of coffee consumption with the production of the white blood cells is not yet evaluated in double-blind trials with controlled with placebo groups.

Low White Blood Cells count doesnt necessarily mean you have any kind of underlying cancer. It is more probable that you suffer from chronic infections or spleen inefficiency that can cause low wbcs levels to occur.

Your doctor is the only competent person to differ-diagnose your low wbcs levels and find the origin of this deficiency.

The only type of cancer that associates with low white blood cells are myeloid leukemia that is extremely rare. It affects your bone marrow and may get better with a transplant from a healthy donor.

A low White Blood Cells level can make you feel tired. It happens because the wbc high count can remove red blood cells from your bloodstream. Removing the red blood cells means that less oxygen gets to your muscles and that is the main reason for feeling constantly tired and exhausted.

Many people who suffer from wbcs deficiencies can benefit from blood transplants rich in platelets and red blood cells that can increase the oxygenation of their tissues.

Stress can lower your White Blood Cells count since it is the chronic mental condition that can increase cortisol production in your body. It is proven that cortisol can lower the production of white blood cells in your bone marrow, lymphatic tissues, and spleen.

In this way, people who suffer from chronic stress and dont receive any treatment for it can place themselves at severe risk. The low White Blood Cells count can make them vulnerable to infections.

It is vital to deal with anxiety and stress to restore your wbcs levels and immune system health to normal.

You can find the production of White Blood Cells in several different organs in the human body:

The human body can physically live without white blood cells. However, without them the body will become very sick as it will be open to infection and be seen as an easy target by bad bacteria and pathogens. The majority of the blood is made up of red blood cells and platelets, meaning the human body can continue to live but may develop life threatening infections due to lack of WBCS.

Leukopenia, also known as low white blood cell count, causes fatigue. Your body will become easily tired when you are experiencing leukopenia as your bodys defense and immune system becomes weak.

The reason for white blood cells being called white, is due to their physical appearance. After separating them from the red blood cells, the cells are indeed white in colour. The same goes for red blood cells, they are red in appearance.

The immune system has its safeguards that are the White Blood Cells. They are necessary to give you the health levels you need to function well in a world full of threats for your body.

Following a healthy lifestyle that includes organic foods, supplements and lots of workouts can give you the immune system function you always wanted.

Checking your White Blood Cells count regularly could be useful for your doctor to evaluate your general health levels.

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Larry Wheels Stops Using Steroids: "I Am Going To Be On Testosterone Replacement Therapy" Fitness Volt – Fitness Volt

Posted: August 22, 2022 at 2:23 am

Larry Wheels is known as one of the strongest men in the world because of the breath-taking feats of strength he performs. However, he has never attempted to deceive anyone into believing that he performed these accomplishments as a natural athlete. Despite Wheels openness when it came to his use of steroids and PEDs (Performance Enhancing Drugs), it became evident recently that he was pushing the limits, putting himself at risk for further injury and lasting damage.

Most recently, Larry Wheels injured his back because of rushing a process, which he hoped would help him deadlift 1,000 pounds (453.6 kilograms). The injury also forced him to withdraw from the 2022 Middle Easts Strongest Man contest. However, despite this deadlift being his greatest goal in life, the injury helped Larry realize how important his health is. So, he decided to stop using Steroids and will now try to maximize his off-cycle potential. The news was revealed in a new YouTube video that Larry posted on August 20th.

Related: Powerlifter Larry Wheels Describes Experience with Trenbolone as Living Hell

Having used steroids and PEDs for a decade, Larry Wheels describes his experience with them. Larrys use of these substances for such a long time has left some consequences, which affected his natural testosterone production.

Much like you dont know, I dont know what I am capable of off-cycle. Now, going completely off isnt an option for me. I cannot produce testosterone naturally. So, I am going to be on testosterone replacement therapy (TRT). I tried pull cycle therapy several times when I began cycling and I was unable to recover.

So Larry Wheels will no longer use steroids or performance-enhancing drugs, but since his body cannot produce testosterone on its own, he will have to undergo testosterone replacement therapy to reach a normal testosterone level. Having taken inspiration from a fellow YouTuber and fitness enthusiast, Derek from More Plates More Dates, he already has an idea of what he wants to do.

TRT for me would be anywhere from as low as 70 milligrams per week, as high as 175 I think 175 is a good place for me to start. If I speak with my doctor and we get my bloods done and we see that its more than whats necessary then I will decrease it, but I certainly wont increase it.

Wheels has been looking for a way to lift 1,000 pounds (453.6 kilograms) for some time now, and he does not appear to be giving up just yet. Currently, however, he will not be looking to do so since it is almost impossible to do so without the aid of performance-enhancing drugs. Larry has hinted that he may get back on PEDs in the future to deadlift 1,000 pounds, but it wont happen anytime soon.

I am not calling quits on it, but I am putting the brakes on attempting PRs like that at the moment Why do I have to do it right now? I am 27, I can take my time, it can be several years from now. Ill still be in my prime. Ill still be healthy.

It is likely that Larry Wheels greatest achievement in life will be deadlifting 1,000 pounds. This means that it cannot be easy and that it should not be rushed either. Its time for Larry to take care of his health and see what he can accomplish without the use of steroids in the future.

Seeing what Im capable of on TRT is more of a priority to me. Its more exciting. It makes me feel invigorated I dont know how I will perform without the assistance (steroids), because the fact of the matter is that PEDs make a tremendous difference in your physique, strength, psyche, recovery more than the general population understands.

Now that hes off PEDs, Larry Wheels will have to focus on his training, diet, recovery, and a whole lot more. He also does not plan to jump back on steroids and PEDs in the near future.

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Data Source: Open Powerlifting. Last Updated: August 3, 2022

Analyze Lifts

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He now wants to check out how different his deadlift program will be now that he is no longer on steroids. He will also regularly put out updates and training videos, so his transparency could help everyone understand the process he is going through. There is a lot to learn from Larrys experience, so it is exciting to envision what the future holds for him.

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Larry Wheels Stops Using Steroids: "I Am Going To Be On Testosterone Replacement Therapy" Fitness Volt - Fitness Volt

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Global Cancer Stem Cells Market Estimated to Reach $1,722.7 Million by 2026 and Grow at 10.3% CAGR in the 2019 to 2026 Timeframe | [180-Pages] Report…

Posted: August 22, 2022 at 2:21 am

The global cancer stem cells market expected to grow by 2026 due to rising prevalence of cancer. The breast cancer sub-segment is expected to be highly profitable. Market in the North America region is expected to be highly lucrative.

NEW YORK, Aug. 19, 2022 /PRNewswire/ --Research Dive has added a new report to its offering titled, "Cancer Stem Cells Market, By Cancer Form (Breast, Blood, Lung, Brain, Colorectal, Pancreatic, Bladder, Liver), By Application (Targeted CSCs, Stem Cell Based Cancer Therapy): Global Opportunity Analysis and Industry Forecast, 20192026".

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According to the report, the global cancer stem cells market is expected to gather a revenue of $1,722.7 million by 2026, growing at a CAGR of 10.3% during the 2019-2026 timeframe.

Dynamics of the Cancer Stem Cells Market

Drivers: Increasing prevalence of cancer all across the globe is projected to be the primary growth driver of the cancer stem cells market in the forecast period. Additionally, the growth in awareness among people regarding cancer is expected to increase the growth rate of the market.

Opportunities: The extensive focus of various organizations and medical experts on developing new cancer treatments and improving the efficiency of the existing ones is predicted to offer huge growth opportunities to the market. Along with this, growth in the number of cancer patients all over the world is anticipated to push the market forward.

Restraints: Expensive cell therapy treatments may hamper the growth trajectory of the cancer stem cells market.

To get access to the Comprehensive PDF Sample [180-Pages] of Cancer Stem Cells Market Click here!

Segments of the Cancer Stem Cells Market

The report has divided the cancer stem cells market into a few segments based on cancer form, application, and regional analysis.

By cancer form, the breast cancer sub-segment of the cancer stem cells market is estimated to have a dominating share and garner $295.0 million by 2026. Changing lifestyle, hormonal changes, consumption of alcohol, etc. have led to an increase in prevalence of breast cancer in people worldwide, which is predicted to push the market forward.

By application, stem cell based cancer therapy sub-segment is anticipated to have a stunning growth rate and surpass $896.9 million by the end of 2026. Stem cell based cancer therapy comprises of allogenic stem cell therapy and autologous stem cell therapy, which are widely used for treating different types of cancers. This is expected to help the sub-segment grow in the forecast period.

By regional analysis, the cancer stem cells market in the North America region is predicted to flourish immensely and garner a revenue of $783.8 million by 2026. Medical experts and doctors in this region are quite involved in cancer related research and help in promotion of stem cell interventions, which is why the market is expected to grow at such a stunning rate in this region.

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Broader Insights Pertaining (Avail 10%OFF) of Specific Segments or Regions of Cancer Stem Cells Market

Significant Cancer Stem Cells Market Players

Some prominent market players of the cancer stem cells market are

AdnaGen GmbH

Epic Sciences, Inc.

Silicon Biosystems

S.p.A.

Advanced Cell Diagnostics, Inc.

Fluxion Biosciences, Inc.

AVIVA Biosciences Corporation

Rarecells USA, Inc.

Celula, Inc.

These players are developing various business strategies like product development and enhancement, merger and acquisition, partnerships and collaborations to gain a commanding position in the market. Buy the Full Report Here Started at $2999

For example, in January 2022, Century Therapeutics, a leading biotechnology company, announced a partnership with Bristol Myers Squibb, a multinational pharmaceutical company. The collaboration is aimed at developing four induced pluripotent stem cell derived programs in order to tackle malignant tumors. The partnership is expected to hugely benefit both the companies as they will be able to integrate their operations in a much better way in order to develop the intended program.

The report also sums up various crucial facets including SWOT analysis, product portfolio, financial performance of the key market players, and the latest strategic developments.

Checkout the Latest Insights of Cancer Stem Cells Market. Schedule a call with an Analyst

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Global Cancer Stem Cells Market Estimated to Reach $1,722.7 Million by 2026 and Grow at 10.3% CAGR in the 2019 to 2026 Timeframe | [180-Pages] Report...

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BridgeBio, Baylor College of Medicine to Collaborate on Genetic Disease …

Posted: August 22, 2022 at 2:21 am

Commercial-stage biopharmaceutical company BridgeBio Pharma and Baylor College of Medicine announced the two will enter an academic collaboration that will seek to translate research findings into new therapies to treat genetic diseases.

BridgeBio has adopted a strategy of identifying cutting-edge research in academia and finding way to leverage this information as part of the drug development process.

In these challenging times, we believe it is more important than ever to be a stalwart partner to academic institutions working to serve patient populations big and small, said Neil Kumar, Ph.D., BridgeBio founder and CEO in a press release announcing the collaboration. We look forward to developing a close partnership with scientists at Baylor College of Medicine, which is known for its productive research engine, in the hope of translating innovations into meaningful medicines for patients with unmet medical needs.

Baylor College of Medicine is known as one of the leading academic medical institutions in the U.S. that is looking to unlock the molecular underpinnings of genetic diseases and cancer using an approach that integrates basic scientific research with translational and clinical sciences. In this case, BridgeBio and Baylor will work together to identify and translate promising research findings into potential therapeutics for genetically driven diseases.

Together with BridgeBios drug development team, we are optimistic that our interdisciplinary team of scientists and physicians will be able to develop new therapeutics for patients in need and further our mission to identify and develop drugs for a wide variety of diseases, said Joseph Petrosino, Ph.D., chief scientific innovation officer and chair and professor of molecular virology & microbiology at Baylor.

To date, BridgeBio has forged nearly a dozen-and-a-half similar research-drug development collaborations with academic institutions, a strategy the company says will move it beyond the typical one-off approach that most companies take with academia and into more lasting, comprehensive creative partnerships.

Far too often strong scientific research that could benefit patients is left on the shelves of academia because it cant get the funding needed to move it forward, the company notes on its website. At BridgeBio, we are focused on forging meaningful partnerships with academic institutions to support and accelerate the work of researchers who are on the front lines of understanding how genetic diseases may be treated.

We feel privileged to partner with top academic and research institutions that share our commitment to discover, create, test ,and deliver life-changing medicines for patients with genetic diseases and cancers with clear genetic drivers.

In a similar vein, BridgeBio also announced on Thursday a founding affiliation with Bakar Labs, the incubator at UC Berkeleys Bakar BioEnginuity Hub. Bakar Labs was established by the University of California (UC), Berkeley and QB3, UCs research institute for innovation and entrepreneurship in the life sciences. Bakar labs is available to academic researchers from around the world and plans to provide research facilities for as many as up to 50 life science start-up companies seeking to commercialize promising research.

Partnering with UC Berkeley and QB3 to launch Bakar Labs is a natural extension of our mission to discover, create, test, and deliver transformative medicines to as many patients as possible. Through this collaboration, we aim to strengthen the Bay Area biotech ecosystem and potentially unlock new therapies for patients with unmet needs, Kumar said.

BridgeBios participation in the incubator will provide it with the opportunity to review discoveries made by resident researchers, provide support to entrepreneurs, and consider possible partnership opportunities with startups focused on potential therapies for patients with genetically-driven diseases and cancers.

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New $2.8-million gene therapy becomes most expensive medicine in history – New Atlas

Posted: August 22, 2022 at 2:21 am

The US Food and Drug Administration (FDA) has approved the first ever cell-based gene therapy, a one-off treatment for patients with a rare genetic blood disease. The single treatment will cost US$2.8 million, making it the most expensive medicine in history.

Called Zynteglo, the gene therapy is designed to treat beta-thalassemia, a rare disease with patients often requiring life-long blood transfusions. The gene therapy is personalized to each patient.

Bone marrow stem cells are harvested from the patient and then genetically modified to insert working copies of a gene that can effectively produce functional red blood cells. The modified stem cells are then infused back into the patient, with the positive effects of the one-off treatment expected to last a lifetime.

Clinical trials have found 89% of patients become transfusion independent following the treatment but this kind of gene therapy is not without risk. Although no serious adverse events were detected in Zynteglos clinical trials, other trials testing similar kinds of gene therapies have reported cases of cancer.

In approving Zynteglo the FDA noted a potential risk for cancer and recommends monitoring patients for 15 years following treatment with the gene therapy. In June, when the therapy was evaluated by a panel of independent experts for the FDA, the cancer risks were noted but the benefits of the treatment were found to outweigh any potential harms.

The approval of Zynteglo is somewhat historic as it's only the third gene therapy to reach the market in the United States. It is also the first cell-based gene therapy to ever be approved in the US, meaning it's the first authorized therapy where stem cells are removed, genetically modified, and then returned to a patient.

But Zynteglo may become most notorious for setting a new benchmark in medicine costs. The company behind the medicine, bluebird bio, has set Zynteglos initial wholesale cost at $2.8 million, making it the most expensive medicine to ever reach the US market. The previous record was set by a gene therapy in 2019 called Zolgensma, with its one-off cost sitting around $2 million.

Addressing the high price of the treatment, bluebird bio has argued the cost must be considered as relative to the lifetime healthcare costs for patients with beta-thalassemia. The company said this single gene therapy treatment is essentially a cure that removes the need for decades of ongoing medical care.

The lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia can reach up to $6.4 million in the U.S. and the average total health care cost per patient per year is 23 times higher than the general population, the company explained in a statement. bluebird estimates that there are approximately 1,300-1,500 individuals with transfusion-dependent beta-thalassemia in the U.S.

The cost of one-time gene therapies has been a thorny topic for health insurers over the past few years. The nature of single-dose curative treatments means pharmaceutical companies are likely to set incredibly high prices as their only way to recoup the development costs that go into producing these therapies. However, insurance companies have unsurprisingly balked at the huge price tags.

Zolgensmas $2 million price tag has been the source of extensive negotiation with health insurers over the past couple of years. Its manufacturer Novartis has tried to break its cost down into annual installments for insurers, spreading the price over three or four years.

Although bluebird bio has indicated it will reimburse insurers up to 80 percent of the treatment cost if Zynteglo fails in a given patient within two years, that still may not be enough some insurance companies.

Zynteglo was initially approved for use in the European Union back in 2019 but bluebird bio struggled to come to arrangements with health insurers over the treatments cost. The gene therapy was subsequently withdrawn from several markets in Europe after insurers refused to foot the multi-million-dollar bill.

Source: FDA

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Molecular Map Reveals Insights Into the Genetic Drivers of CLL – The ASCO Post

Posted: August 22, 2022 at 2:21 am

By The ASCO Post StaffPosted: 8/16/2022 2:43:00 PM Last Updated: 8/16/2022 3:15:33 PM

A newly constructed map of the landscape of genetic changes in chronic lymphocytic leukemia (CLL) may provide a better understanding of this complex malignancy that could lead to more accurate prognoses for patients, improved diagnostics, and novel treatments. These research findings were published by Knisbacher et al in Nature Genetics, and the study was conducted by an international collaboration of investigators, including teams from the Mass General Cancer Center, the Dana-Farber Cancer Institute, and the Broad Institute of MIT and Harvard.

CLL exists as either a slowly or rapidly growing cancer and has been linked to certain genetic mutations, but it has yet to be fully characterized. Previous analyses have provided only fragments of a CLL map, each focusing on particular types of patients or limited data. To provide a more thorough understanding of the biological underpinnings of CLL and its molecular subtypes, scientists set out to construct a map from the largest CLL data set to date. To build the CLL map, the team analyzed variations in genetic sequences, gene-expression patterns, and chemical modifications to DNAor genomic, transcriptomic, and epigenomic datafrom 1,148 patients.

Such a CLL map could eventually be leveraged in the clinic, wherein the genomic features of new patients can be compared with the treatments and outcomes of patients with similar genetic profiles, said co-senior and co-corresponding author Catherine Wu, MD, Chief of the Division of Stem Cell Transplantation and Cellular Therapies at Dana-Farber Cancer Institute and Professor of Medicine at Harvard Medical School. This profiling could potentially help more accurately tailor prognosis and treatment of a new patient based on their particular molecular features, getting closer to precision medicine.

Key Findings

The scientists identified 202 genes109 of which were novelthat when mutated, could potentially drive CLL, and they refined the characterization of subtypes of CLL with distinct genomic characteristics and prognoses. Beyond genetic sequences, the expression patterns of certain genes further subcategorized CLL and provided valuable prognostic information.

Our study has revealed that the genetic and biologic landscape of CLL is more complex than previously appreciated, said co-senior and co-corresponding author Gad Getz, PhD, Director of Bioinformatics at the Mass General Cancer Center and Director of the Cancer Genome Computational Analysis group at the Broad Institute. Patients clinical outcomes were associated with a combination of genomic, transcriptomic, and epigenomic featuresintegrating these data could predict a patients likelihood of experiencing remission vs developing more advanced cancer.

We are releasing a CLL map portal that is based on the CLL map and will be an interactive website for translational researchers to use as a resource for further investigationsuch as learning more about the different drivers and subtypes of CLL, said Dr. Getz.

Disclosure: This work was supported by the National Institutes of Health and the Broad/IBM Cancer Resistance Research Project. For full disclosures of the study authors, visit nature.com.

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Biopharmaceutical Company Expands Clinical Trials On Texas A&M-Developed Angelman Syndrome Treatment – Texas A&M University Today

Posted: August 22, 2022 at 2:21 am

The Dindot Lab team. Front row (L to R): Research scientist Dr. Sarah Christian and Dr. Scott Dindot. Back row (L to R): Dr. Johnathan Ballard (Texas A&M Institute for Genomic Medicine) and biomedical sciences doctoral students Tom Jepp and Luke Myers

A treatment for a rare disease that was researched and developed in the laboratory of Texas A&M School of Veterinary Medicine & Biomedical Sciences (VMBS) associate professor Scott Dindot has not only produced encouraging interim data from a phase 1/2 clinical trial in the United States, United Kingdom, and Canada but has become the subject of a $75 million acquisition by Ultragenyx Pharmaceutical, Inc., which will continue to develop the treatment.

In an update last month, Ultragenyx announced that GTX-102, the drug originating in Dindots lab, had demonstrated meaningful improvement in pediatric patients afflicted with a genetic disorder called Angelman Syndrome (AS) and that the company is expanding patient enrollment in its ongoing phase 1/2 study of the drug.

AS is a rare genetic disorder that affects approximately one in 15,000 live births per year; the disorder is caused by a loss of function of the UBE3A gene, which leads to developmental delay, speech impairment, movement or balance disorder, and seizures.

Currently, there is no cure for AS. Available treatments for the disorder focus solely on behavioral therapy and controlling the symptoms, specifically the seizures that often affect patients with AS.

However, researchers have reported improvements in measurements of disease severity and quality of life for AS patients in the phase 1/2 study of GTX-102, leading Ultragenyx to believe that the novel, targeted therapeutic could be a promising treatment for the disorder.

This groundbreaking work not only highlights the strong science being conducted in our school but also demonstrates how researchers in the School of Veterinary Medicine & Biomedical Sciences are finding novel solutions to real-world problems that can improve the lives of both animals and human beings, said Dr. John R. August, the Carl B. King Dean of Veterinary Medicine at Texas A&M.

In developing the drug, Dindot and his Texas A&M research team identified a region on the UBE3A antisense (UBE3A-AS) transcript, which regulates the expression of the paternally inherited allele of the UBE3A gene. The drug an antisense oligonucleotide (ASO) inhibits UBE3A-AS and reactivates expression of the paternal UBE3A allele, restoring UBE3A protein in the brain.

We targeted a very specific region on the UBE3A-AS transcript that we believe is important for regulating its expression, said Dindot, who also is a Texas A&M University System Chancellors Enhancing Development and Generating Excellence in Scholarship (EDGES) Fellow. In theory, this treatmentgoes after the heart of the condition.

In natural conditions, only the copy of UBE3A inherited from the mother is expressed in the brain. Individuals living with AS have a mutation or deletion in the maternal copy of the gene, and thus, they lack the UBE3A protein in the brain.

Dindots drug, the first molecular therapeutic for AS to advance into clinical development, works by reactivating the paternal copy of UBE3A so it can compensate for the loss of function in the maternal copy.

After the initial discovery, which was supported by funding from the Foundation for Angelman Syndrome Therapeutics (FAST), Dindot continued the research and development of the drug in collaboration with the newly-formed biotech company GeneTx Biotherapeutics, LLC, (GeneTx) and later, Ultragenyx which conducted investigational-new-drug-enabling studies on the therapeutic as it moved into clinical trials in the US, UK and Canada.

That the drug made it to a clinical trial is an enormous milestone and now it is just amazing to hear the interim data suggesting that kids conditions are improving in multiple areas, Dindot said. Over the past decade, there have been probably over a dozen people who have worked on this in my lab undergraduate and graduate students and scientists and I really want to recognize them for the hard work they put into the research and development of this drug; it takes a lot of people to do this and Im proud of what we have accomplished together.

With the acquisition of GeneTx by Ultragenyx, Ultragenyx will now take the lead on advancing GTX-102 into late-stage development for AS by testing the effectiveness of the therapeutic at higher monthly doses.

This is a really great example of the successful commercialization of a technology from the research stage to the clinical development of a promising therapeutic with a company, said Janie Hurley, program director atTexas A&M AgriLifeResearch Intellectual Property & Commercialization, who oversees the development of commercialization and intellectual property protection strategies for technologies created by researchers affiliated with Texas A&M AgriLife and the CVMBS. We strive to ensure that new discoveries such as this one have the best chance possible to reach those in society who could benefit. Working with companies like GeneTx and Ultragenyx is how we accomplish this goal.

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