ProQR Announces Upcoming Scientific Presentations at ASGCT and TIDES Conferences
"We are excited to share these presentations with the scientific community, demonstrating the breadth and versatility of our Axiomer RNA editing technology," said Gerard Platenburg, Chief Scientific Officer of ProQR. "The data we will present at ASGCT and TIDES further support our vision of building a differentiated and diversified pipeline and advancing RNA editing therapies that could offer transformative benefits for patients."
These presentations will highlight the Company's proprietary Axiomer RNA editing platform and pipeline programs. Details of ProQR's presentations are as follows:
American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
May 13-17, 2025 | New Orleans, Louisiana, USA
Oral Presentation
Title: ADAR-Mediated RNA Editing of SLC10A1(NTCP) as a Therapeutic Approach to Reduce Liver Bile Acid Re-Uptake in Cholestatic Diseases (abstract 333)
Presenter: Gerard Platenburg, Chief Scientific Officer
Date/Time: May 16, 2025, 4:15 PM CT
Session: Oligonucleotide Therapeutics II
Poster Presentations
Title: ADAR-Mediated RNA Editing-Based Correction of PNPLA3 I148M Functionality to Address Hepatic Steatosis (abstract 1654)
Presenter: Gerard Platenburg, Chief Scientific Officer
Date/Time: May 15, 2025, 5:30-7:00 PM CT
Poster Number: AMA1324
Title: ADAR-Mediated RNA Editing of Premature Termination Codon Results in Functional Correction in MECP2 for Rett Syndrome (abstract 1645)
Presenter: Gerard Platenburg, Chief Scientific Officer
Date/Time: May 15, 2025, 5:30-7:00 PM CT
Poster Number: AMA1427
TIDES USA: Oligonucleotide and Peptide Therapeutics Conference
May 19-22, 2025 | San Diego, California, USA
Podium Presentation
Title: Advancing Axiomer ADAR RNA Editing Platform - Editing oligonucleotides optimization for therapeutic use
Presenter: Peter A. Beal, PhD, Chief ADAR Scientist
Date/Time: May 22, 2025, 2:00 PM PST
Session/Track: Genome Editing Technology and Applications
Following the conferences, presentation materials will be made available in the Publications and Presentations section of the ProQR website at www.proqr.com.
About Axiomer™
ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer 'Editing Oligonucleotides', or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell's own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
Learn more about ProQR at www.proqr.com.
Forward Looking Statements for ProQR
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as 'continue,' "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our presentations at these conferences, our business, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our Axiomer™ RNA editing technology platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and our ability to expand preclinical in vivo and in vitro data, the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, and the potential of our technologies and product candidates, as well as the timing of our clinical development. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers or suppliers to supply materials for research and development and the risk of supply interruption or delays from suppliers or contract manufacturers; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our participation in this conference, our business, technology, strategy, our Axiomer platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides, and the potential of our technologies and product candidates. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical events and conflicts, high inflation, rising interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.
ProQR Therapeutics N.V.
Investor and media contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorInvestor contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.comSign in to access your portfolio
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
26 minutes ago
- Yahoo
A Look At The Intrinsic Value Of CSX Corporation (NASDAQ:CSX)
Key Insights Using the 2 Stage Free Cash Flow to Equity, CSX fair value estimate is US$35.12 CSX's US$36.32 share price indicates it is trading at similar levels as its fair value estimate Our fair value estimate is 8.3% lower than CSX's analyst price target of US$38.28 Does the August share price for CSX Corporation (NASDAQ:CSX) reflect what it's really worth? Today, we will estimate the stock's intrinsic value by taking the forecast future cash flows of the company and discounting them back to today's value. Our analysis will employ the Discounted Cash Flow (DCF) model. Don't get put off by the jargon, the math behind it is actually quite straightforward. We would caution that there are many ways of valuing a company and, like the DCF, each technique has advantages and disadvantages in certain scenarios. For those who are keen learners of equity analysis, the Simply Wall St analysis model here may be something of interest to you. AI is about to change healthcare. These 20 stocks are working on everything from early diagnostics to drug discovery. The best part - they are all under $10bn in marketcap - there is still time to get in early. Step By Step Through The Calculation We use what is known as a 2-stage model, which simply means we have two different periods of growth rates for the company's cash flows. Generally the first stage is higher growth, and the second stage is a lower growth phase. To begin with, we have to get estimates of the next ten years of cash flows. Where possible we use analyst estimates, but when these aren't available we extrapolate the previous free cash flow (FCF) from the last estimate or reported value. We assume companies with shrinking free cash flow will slow their rate of shrinkage, and that companies with growing free cash flow will see their growth rate slow, over this period. We do this to reflect that growth tends to slow more in the early years than it does in later years. Generally we assume that a dollar today is more valuable than a dollar in the future, and so the sum of these future cash flows is then discounted to today's value: 2026 2027 2028 2029 2030 2031 2032 2033 2034 2035 Levered FCF ($, Millions) US$2.87b US$3.23b US$3.65b US$3.88b US$4.07b US$4.25b US$4.42b US$4.58b US$4.74b US$4.90b Growth Rate Estimate Source Analyst x9 Analyst x3 Analyst x1 Analyst x1 Est @ 4.88% Est @ 4.34% Est @ 3.96% Est @ 3.70% Est @ 3.51% Est @ 3.38% Present Value ($, Millions) Discounted @ 8.6% US$2.6k US$2.7k US$2.8k US$2.8k US$2.7k US$2.6k US$2.5k US$2.4k US$2.3k US$2.1k ("Est" = FCF growth rate estimated by Simply Wall St)Present Value of 10-year Cash Flow (PVCF) = US$26b The second stage is also known as Terminal Value, this is the business's cash flow after the first stage. For a number of reasons a very conservative growth rate is used that cannot exceed that of a country's GDP growth. In this case we have used the 5-year average of the 10-year government bond yield (3.1%) to estimate future growth. In the same way as with the 10-year 'growth' period, we discount future cash flows to today's value, using a cost of equity of 8.6%. Terminal Value (TV)= FCF2035 × (1 + g) ÷ (r – g) = US$4.9b× (1 + 3.1%) ÷ (8.6%– 3.1%) = US$91b Present Value of Terminal Value (PVTV)= TV / (1 + r)10= US$91b÷ ( 1 + 8.6%)10= US$40b The total value is the sum of cash flows for the next ten years plus the discounted terminal value, which results in the Total Equity Value, which in this case is US$65b. To get the intrinsic value per share, we divide this by the total number of shares outstanding. Compared to the current share price of US$36.3, the company appears around fair value at the time of writing. The assumptions in any calculation have a big impact on the valuation, so it is better to view this as a rough estimate, not precise down to the last cent. Important Assumptions The calculation above is very dependent on two assumptions. The first is the discount rate and the other is the cash flows. You don't have to agree with these inputs, I recommend redoing the calculations yourself and playing with them. The DCF also does not consider the possible cyclicality of an industry, or a company's future capital requirements, so it does not give a full picture of a company's potential performance. Given that we are looking at CSX as potential shareholders, the cost of equity is used as the discount rate, rather than the cost of capital (or weighted average cost of capital, WACC) which accounts for debt. In this calculation we've used 8.6%, which is based on a levered beta of 1.198. Beta is a measure of a stock's volatility, compared to the market as a whole. We get our beta from the industry average beta of globally comparable companies, with an imposed limit between 0.8 and 2.0, which is a reasonable range for a stable business. See our latest analysis for CSX SWOT Analysis for CSX Strength Debt is well covered by earnings and cashflows. Dividends are covered by earnings and cash flows. Weakness Earnings declined over the past year. Dividend is low compared to the top 25% of dividend payers in the Transportation market. Expensive based on P/E ratio and estimated fair value. Opportunity Annual earnings are forecast to grow for the next 3 years. Threat Annual earnings are forecast to grow slower than the American market. Moving On: Valuation is only one side of the coin in terms of building your investment thesis, and it ideally won't be the sole piece of analysis you scrutinize for a company. The DCF model is not a perfect stock valuation tool. Preferably you'd apply different cases and assumptions and see how they would impact the company's valuation. For example, changes in the company's cost of equity or the risk free rate can significantly impact the valuation. For CSX, there are three essential factors you should look at: Risks: Be aware that CSX is showing 1 warning sign in our investment analysis , you should know about... Future Earnings: How does CSX's growth rate compare to its peers and the wider market? Dig deeper into the analyst consensus number for the upcoming years by interacting with our free analyst growth expectation chart. Other High Quality Alternatives: Do you like a good all-rounder? Explore our interactive list of high quality stocks to get an idea of what else is out there you may be missing! PS. The Simply Wall St app conducts a discounted cash flow valuation for every stock on the NASDAQGS every day. If you want to find the calculation for other stocks just search here. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.
Yahoo
an hour ago
- Yahoo
Can a $10,000 Investment in Palantir Make You a Millionaire in a Decade?
Key Points Palantir is a key AI software provider for government and commercial clients. Its growth has been accelerating. The stock is one of the most expensive on the market. 10 stocks we like better than Palantir Technologies › Palantir Technologies (NASDAQ: PLTR) has been one of the most popular artificial intelligence (AI) stocks over the past few years for good reason: It has been on an absolute tear, rising over 140% in 2025 alone. But after such a successful run, many investors might be wondering if it's too late to buy the stock now. There are even some hopes of turning $10,000 into $1 million, a feat that few companies have ever accomplished. But could Palantir be one of these chosen few? Palantir likely can't provide 100x returns from today's prices Palantir has been on an impressive run already. If you invested $10,000 into the stock at the start of the AI race (the beginning of 2023), you would already have nearly $285,000. So, if you're lucky (or smart enough) to have picked the stock, then you're well on your way to achieving this rare feat. But what about if you're considering investing in Palantir now? At its current $430 billion market cap, Palantir would need to become a $43 trillion company to achieve that feat. Considering that the world's largest company, Nvidia (NASDAQ: NVDA), only has a $4.4 trillion market cap right now, that projection is extremely far-fetched. It's unlikely Palantir can turn $10,000 into $1 million now. But can it still be a successful market-crushing stock from today's levels? Palantir delivered impressive growth in Q2 Palantir started as a government-focused company that helped turn data streams into AI-powered insights. This helped governments make smart decisions with the best possible information. Eventually, it expanded onto the commercial side and experienced success there, too. In the second quarter, its commercial and government revenue saw impressive growth, with commercial sales rising 47% year over year to $451 million and government sales increasing 49% to $553 million. This strength allowed the company to deliver its first quarter with $1 billion in revenue, and there's more growth ahead. However, the stock price already has a ton of growth baked into it, which could be a significant headwind. Palantir's stock is far from cheap No matter how you look at it, the stock is expensive. At 281 times forward earnings and 134 times sales, it's likely the most expensive stock on the market. It also indicates it has years of growth baked into it. To evaluate the stock, I'll use a five-year time frame and make these assumptions: Revenue growth accelerates to 50% and maintains that pace over the next five years. It achieves and sustains a 30% profit margin. The effects of stock-based compensation are ignored (even though Palantir has a significant stock-based compensation expense). If those three things occur, Palantir's revenue and profits will rise from $3.44 billion and $763 million, respectively, to $26.1 billion and $7.83 billion. That's monster growth, but what will its valuation look like? A far more reasonable valuation for Palantir is around 50 times forward earnings, placing it at about the same price tag as Nvidia, another leading AI company. In five years, if it has a forward earnings valuation of 50 times and generates profits of $7.83 billion, it would have a market cap of $392 billion, which is less than today's levels. This indicates that there's more than five years' worth of growth baked into the stock price, which is far too expensive a premium to pay. As a result, I think investors should avoid Palantir's stock, as there are far more promising options that don't have as much aggressive growth baked into the price already. Should you invest $1,000 in Palantir Technologies right now? Before you buy stock in Palantir Technologies, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Palantir Technologies wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $663,630!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,115,695!* Now, it's worth noting Stock Advisor's total average return is 1,071% — a market-crushing outperformance compared to 185% for the S&P 500. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of August 13, 2025 Keithen Drury has positions in Nvidia. The Motley Fool has positions in and recommends Nvidia and Palantir Technologies. The Motley Fool has a disclosure policy. Can a $10,000 Investment in Palantir Make You a Millionaire in a Decade? was originally published by The Motley Fool
Yahoo
2 hours ago
- Yahoo
3 Growth Stocks That Could Be Worth $1 Million in 5 Years
Key Points Dutch Bros could increase its store count sevenfold. Upstart is back to triple-digit sales growth. Lemonade expects EBITDA profits next year. 10 stocks we like better than Dutch Bros › It's growth time again in the stock market. The big tech companies have been reporting strong performance, and the financial stocks are telling a story of economic growth. Many great growth stocks are riding the rise in the market, or generating it, and no matter what happens in the short term, they have incredible long-term prospects. In fact, depending on how much you invest, these stocks can lead to millionaire status even in five years from now. Consider Dutch Bros (NYSE: BROS), Upstart Holdings (NASDAQ: UPST), and Lemonade (NYSE: LMND). 1. Dutch Bros: A new concept in coffee Dutch Bros is a young coffee chain that's demonstrating rapid growth and improving economics. It has a distinctive culture that focuses on fun, fast, and friendly service, and it's leaning into beverage innovation to carve out a niche in a market that's mostly just coffee. Customers are responding, and Dutch Bros has been reporting increasing sales, both overall and comparable. In the 2025 second quarter, revenue increased 28% year over year, and same-shop sales were up 6.1%. Adjusted earnings per share (EPS) were up from $0.19 last year to $0.26 this year. The opportunity in Dutch Bros is in its expansion. Although it just opened its 1,000th store, it envisions having 7,000 stores, with 2,029 stores by 2029. That implies an accelerated opening rate, since it's planning on opening 160 stores this year, and leaves open a long growth runway that should keep revenue increasing for many years. If it can keep up a compound annual growth rate (CAGR) of 25% during the next five years, keeping the price-to-sales ratio constant, the stock would almost triple. It could be an incredible growth addition to a diversified portfolio, and if you invest enough, it could help you become a millionaire. 2. Upstart: A better lending model Upstart has been a volatile stock, soaring in good times and plunging in challenging times. It's been all about interest rates, which is the case for many financial companies. Upstart, however, comes with the extra risk of being a young company in uncharted territory, which is why the impact on its stock has been more pronounced than on established bank stocks. It uses artificial intelligence (AI) and machine learning to evaluate the creditworthiness of borrowers. As it refines its model, gets more experience, and demonstrates growth, there's a lot of future potential. In the second quarter, revenue rose 102% year over year on transaction growth of 159%. It's also back to net profitability on a generally accepted accounting principles (GAAP) basis of $5.6 million. Upstart's expansion into newer types of loans is creating important diversification that's helping the platform. It recently started to roll out its first home loan product, and home loan originations increased ninefold from last year in the second quarter. Auto loans grew sixfold, and low-risk super prime loans continue to be a major part of the business. At the current valuation, the stock is looking more stable. Upstart stock trades at a reasonable forward one-year price-to-earnings (P/E) ratio of 25. That gives it more room to expand. If interest rates decline, Upstart could continue reporting rapid growth, even if it falls back into double-digit percentage territory. This isn't a stock for the most risk-averse investor, but an investment in Upstart today could lead to market-beating gains during the next five years. 3. Lemonade: A better insurance model Lemonade is another AI disruptor that's growing fast and winning fans. It makes sense to use AI to price insurance policies, since the business relies on sophisticated algorithms in any case. Lemonade has built its platform from the ground up using digital technology, and the interconnected model is fast and efficient, approving many claims in minutes. That's a clear edge over legacy insurance models, which typically involve independent agents. In-force premium (IFP) increased 29% year over year in the second quarter, and customer count increased 24%. Lemonade targets a younger customer who's looking for their first policy, often renters insurance, and the intention is to impress them and get more of their business as they continue on their insurance journey, creating high lifetime value. It's not profitable yet, but its net loss narrowed from last year in the second quarter, and management expects positive profit on an adjusted earnings before interest, taxes, depreciation, and amortization (EBITDA) basis next year. The loss ratio, which measures how much the company pays out in claims as a share of premiums, is decreasing, reaching 67% in the second quarter, or 70% for the trailing 12 months, well within management's target. Lemonade stock is up 275% during the past year (as of Aug. 14), and if it maintains growth while keeping the loss ratio in check and becomes profitable, it should continue to skyrocket. For example, if revenue increases at a compound annual growth rate of 25% during the next five years, revenue will more than triple. If you invest enough today, your investment could soar during the next five years and be a part of a millionaire-maker portfolio. Should you invest $1,000 in Dutch Bros right now? Before you buy stock in Dutch Bros, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Dutch Bros wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $663,630!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,115,695!* Now, it's worth noting Stock Advisor's total average return is 1,071% — a market-crushing outperformance compared to 185% for the S&P 500. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of August 13, 2025 Jennifer Saibil has positions in Lemonade. The Motley Fool has positions in and recommends Lemonade and Upstart. The Motley Fool recommends Dutch Bros. The Motley Fool has a disclosure policy. 3 Growth Stocks That Could Be Worth $1 Million in 5 Years was originally published by The Motley Fool
