ProQR Announces Upcoming Presentation at RNA Editing Summit
'We recently achieved a key milestone with the submission of the CTA for AX-0810, targeting NTCP for cholestatic diseases, and we're building on this momentum with continued progress in CNS applications of our Axiomer technology,' said Gerard Platenburg, Chief Scientific Officer of ProQR. 'Our presentation at the RNA Editing Summit will feature preclinical NHP data in the CNS, and spotlight our Rett program targeting MECP2, supporting the potential for Axiomer to target severe neurodevelopmental diseases.'
RNA Editing Summit
July 29-31, 2025 | Boston, MA
Title: Pioneering RNA Modification Beyond Rare Diseases by Exploring Novel Editing Technologies & Targeting Multiple Mutations to Remove the Barriers to Treating Common Diseases
Presenter: Gerard Platenburg, Chief Scientific Officer
Date/Time: July 31, 2025, 3:45 PM ET
This presentation will include:
An update on the potential of Axiomer in the CNS supported by preclinical long-term and multiple dose NHP data.
An overview of the therapeutic applications of Axiomer in the CNS and liver.
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 participation and presentation at this conference, our business, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, the continued advancement of our lead development pipeline programs, including ongoing and planned clinical trials, the anticipated timing of initial clinical data readouts across multiple programs in 2025 and 2026, 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.com
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
28 minutes ago
- Yahoo
How far can the most powerful telescope see into space?
When you buy through links on our articles, Future and its syndication partners may earn a commission. The world's first telescope, crafted in 1608 by the Dutch eyeglass maker Hans Lippershey, led to stunning technologies that would later revolutionize our understanding of the universe. While his telescope used simple lenses to magnify objects to about three times their size, later scientists built on this concept to peer into the depths of outer space. But some telescopes are more powerful than others, enabling us to spot distant stars and galaxies and allowing researchers to study extreme phenomena like black holes and Einstein rings. So, what's the most powerful telescope, and how far can it see into space? The answer isn't surprising to anyone familiar with today's headlines: The most powerful telescope is presently the James Webb Space Telescope (JWST), which was launched in December 2021 to detect infrared and near-infrared wavelengths, or wavelengths on the electromagnetic spectrum that are invisible to humans but can be felt as heat. Its predecessor and cousin, the Hubble Space Telescope, was primarily designed to detect visible-spectrum light and ultraviolet light, a wavelength that is often emitted by young stars. In space, many objects do not produce or reflect enough visible-spectrum light to see with the naked eye or to detect from far away. However, infrared light stretches so long that it is easier to detect from vast distances. The long wavelengths even have the benefit of piercing through clouds of dust, making them particularly compelling for astronomers hoping to peer into the deepest depths of the universe. Even the powerful new Vera C. Rubin Telescope, recently activated in Chile, can't see this far into space because it has to contend with anomalous obstructions such as dust. When the universe began, it was condensed into a hot mash of particles (protons, neutrons and electrons). As the universe expanded and cooled, the first stars and galaxies began to coalesce. The earliest of these we can see are around 13.7 billion years old, which is just a little over a hundred million years after the Big Bang. Related: How many galaxies orbit the Milky Way? "The James Webb Space Telescope has proven itself capable of seeing 98% of the way back to the Big Bang," Peter Jakobsen, an affiliate professor of astrophysics at the University of Copenhagen in Denmark, told Live Science in an email. "This exceeds the hopes and expectations of most of us involved in the early planning of the James Webb Space Telescope." How does the JWST see so far? Much of the power behind the JWST comes from its large primary mirror, Carol Christian, an astrophysicist at the Space Telescope Science Institute in Baltimore, told Live Science via email. JWST's primary mirror measures 21.3 feet (6.5 meters) in diameter, giving it a total collecting area of more than 270 square feet (25 square m). In contrast, Hubble's primary mirror is 8 feet (2.4 m) in diameter and has a collecting area of nearly 50 square feet (4.5 square m). However, both telescopes can see billions of light-years away because they are in space, well beyond the obscuring haze of Earth's atmosphere. Sign up for our newsletter Sign up for our weekly Life's Little Mysteries newsletter to get the latest mysteries before they appear online. However, JWST is also equipped with infrared light detectors situated to absorb light redirected from its large mirrors that help it identify distant light that Hubble cannot see. Meanwhile, Earth's atmosphere creates unique problems for terrestrial telescopes. These problems range from light pollution to "atmospheric turbulence," which is the random movement of air. Such factors can blur and distort images and limit a telescope's ability to see deeply into space. Space, on the other hand, is darker and free of these problems, so many of our strongest telescopes are placed well beyond Earth's atmosphere. In the case of James Webb, the telescope sits at a vantage point nearly 1 million miles (1.5 million kilometers) from Earth at a Lagrange point, or a point that has just the right gravitational balance for satellites to stay stable in orbit. How far can the James Webb Space Telescope see? When we look at the night sky, we're essentially looking back in time. Light travels 299,792,458 meters per second (186,282 miles per second), which means the light that reaches us from distant objects in space is older than when it was emitted. It takes light from our sun 43.2 minutes to reach Jupiter, but only 8 minutes to reach Earth. The distance to the outermost depths of the cosmos is vastly farther, which complicates the calculations. Measuring how far a telescope can see into space is not a straightforward process, Jakobsen said. Two hurdles astronomers regularly need to account for are the expansion of the universe and the finite speed of light, he said. Astronomers bypass these complications by measuring the redshift of distant celestial bodies. Redshift is what we see as celestial bodies accelerate farther and farther away from us. As the universe expands, the light emitted by faraway objects stretches to longer and "redder" wavelengths. The farther and longer the light travels, the greater its redshift becomes. Currently, one of the farthest known redshift contenders is the galaxy JADES-GS-z14-0, Jakobsen said. Its redshift puts it at about 290 million years after the Big Bang. RELATED MYSTERIES —How many satellites orbit Earth? —Do black holes really evaporate — and how do we know? —Where would a compass point in outer space? Another contender, which has not yet been published in a peer-reviewed journal, is the galaxy MoM-z14, which has been dated to a mere 280 million years after the Big Bang. Its redshift was 14.44 — larger than the redshift of JADES-GS-z14-0, which is 14.18. One study analyzed a set of particularly large and distant galaxies detected by the JWST and found they might be older than the current models of our universe suggest. The JWST has proven that it can peer deeper into space than Hubble, which has only seen as far back as 13.4 billion years. While the JWST is presently the champion in peering deep into our cosmic past, rivals are on the horizon. China is building a space telescope, called the China Space Station Telescope, that uses technology that will enable it to capture more light frequencies than JWST, allowing it to withdraw greater information from the cosmos. James Webb Space Telescope quiz: How well do you know the world's most powerful telescope?
Yahoo
3 hours ago
- Yahoo
What To Expect From Vertex Pharmaceuticals's (VRTX) Q2 Earnings
Biotech company Vertex Pharmaceuticals (NASDAQ:VRTX) will be reporting earnings this Monday after the bell. Here's what to look for. Vertex Pharmaceuticals missed analysts' revenue expectations by 2.3% last quarter, reporting revenues of $2.77 billion, up 3% year on year. It was a disappointing quarter for the company, with a significant miss of analysts' EPS estimates and full-year revenue guidance slightly missing analysts' expectations. Is Vertex Pharmaceuticals a buy or sell going into earnings? Read our full analysis here, it's free. This quarter, analysts are expecting Vertex Pharmaceuticals's revenue to grow 9.8% year on year to $2.90 billion, improving from the 6.1% increase it recorded in the same quarter last year. Adjusted earnings are expected to come in at $4.25 per share. The majority of analysts covering the company have reconfirmed their estimates over the last 30 days, suggesting they anticipate the business to stay the course heading into earnings. Vertex Pharmaceuticals has missed Wall Street's revenue estimates three times over the last two years. Looking at Vertex Pharmaceuticals's peers in the therapeutics segment, some have already reported their Q2 results, giving us a hint as to what we can expect. Biogen delivered year-on-year revenue growth of 7.3%, beating analysts' expectations by 13.7%, and Moderna reported a revenue decline of 41.1%, topping estimates by 10.7%. Biogen traded up 3.9% following the results. Read our full analysis of Biogen's results here and Moderna's results here. Debates around the economy's health and the impact of potential tariffs and corporate tax cuts have caused much uncertainty in 2025. While some of the therapeutics stocks have shown solid performance in this choppy environment, the group has generally underperformed, with share prices down 4.3% on average over the last month. Vertex Pharmaceuticals's stock price was unchanged during the same time and is heading into earnings with an average analyst price target of $505.19 (compared to the current share price of $464). Unless you've been living under a rock, it should be obvious by now that generative AI is going to have a huge impact on how large corporations do business. While Nvidia and AMD are trading close to all-time highs, we prefer a lesser-known (but still profitable) semiconductor stock benefiting from the rise of AI. Click here to access our free report on our favorite semiconductor growth story. StockStory is growing and hiring equity analyst and marketing roles. Are you a 0 to 1 builder passionate about the markets and AI? See the open roles here. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
4 hours ago
- Yahoo
‘I'm so strong': Film about electric shock use at Mass. center hopes to reach Sundance
As a teenager, Jennifer Msumba had 'very strong self-harm behaviors,' including putting her head through windows. Hoping to get her help, her parents sent her to a center in Massachusetts, where she was subjected to electric shocks daily as punishment. Nearly 25 years later, she's continuing to speak out — hoping a new documentary will expose the place that left her traumatized. 'That was like being unground in hell,' she recalled about her time at Judge Rotenberg Center (JRC) in Canton in the documentary's trailer. JRC, a special needs day and residential facility in Canton, has become known across the country for using electric shock devices, which are attached to the residents at all times, on people demonstrating self-injurious or aggressive behavior. It is the only facility in the country to use them and has been the center of many advocates' concerns for decades. 'This is 2025, and they are still shocking people,' she said in the documentary's trailer. 'It needs to end.' The JRC has said the device is 'lifesaving' and that it is important it 'remains available to those for whom all other treatment options have been tried and failed,' the JRC and the JRC Parents Association wrote in a statement after a 2023 court ruling that allowed the devices to continue to be used. The facility has said that its clients have had behaviors that result in 'profound physical injuries including blindness and mutilation' prior to using the devices. The clients have also injured family members and loved ones. 'Families turn to JRC after exhausting other options because of its record in successfully addressing the most challenging cases. Clients live in the community with appropriate monitoring, and their care is overseen by on-site physicians and nurses to maintain the highest standards of safety and treatment. The program operates under rigorous oversight from state agencies and the courts,' a statement read. As of 2024, there were more than 300 people who attend or live at JRC, and about 55 people were approved for the devices. Msumba daydreams of ninjas breaking into JRC to 'save everybody' and shut down the facility. 'But I don't have ninjas,' she said. Instead, she is making a documentary called 'The Fish Don't Care When It Rains,' which she hopes to submit to Sundance. 'I'm so strong,' she said in the documentary, looking out the car window at the facility she once felt trapped in. Msumba has autism and said that while her behaviors stopped while she was on the electric shock device, her 'behaviors weren't fixed.' 'They were just manifested differently, into explosions. I would hold it in and then all of a sudden explode,' she told the state Legislature's Joint Committee on Children, Families and Persons with Disabilities during a 2023 hearing about her experience attached to the devices at JRC. She lived at JRC from 2002 until 2009 and was on the shock devices for all seven years. At the beginning of her time there, she said she was shocked daily. 'Crying was also a behavior that I got in trouble for, so I had to cry in secret,' Msumba said in video testimony used in an FDA hearing. In March 2020, the U.S. Food and Drug Administration banned electrical stimulation devices (ESDs) used for self-injurious or aggressive behavior. But that decision was overturned. The FDA warned in its petition that the 'FDA determined that those devices present unreasonable and substantial risks of both physical and psychological injury.' 'The shocks cause pain that has been described as 'extremely painful,' 'excruciatingly painful,' 'like a dentist drilling on an un-anesthetized tooth,' like a 'bulging and a ruptured disc,' like 'a thousand bees stinging you in the same place for a few seconds,' and as 'the most painful thing I've ever experienced,'' the petition states. But it wasn't just the electric shocks that Msumba said traumatized her. She also was denied food and 'was one of those stripped of all my clothes, tied to a restraint board and forcefully scrubbed while male staff watched on the video monitors.' 'I did not consent to any of this treatment,' she said. The center had also taken guardianship away from her parents, the documentary trailer states. She has since left to live at a different placement in Florida, which she said has helped her. But when she arrived, she went back to her old self-harming behaviors. 'Instead of hurting me, they helped figure out why these things were happening and took the time to do so. They helped me learn to cope and self-regulate, [and] also allowed access to medication that I really needed,' she said. In 2021, Msumba released a book, 'Shouting At Leaves' regarding her time at the center. She also shares some of her story on TikTok. 'I have a real life now,' she said. In the documentary trailer, Msumba looks back on her time at JRC. 'I have no idea how I got through all that,' she said. But she also recognizes her accomplishments since then. Her own music is included throughout the documentary. Over the years, she's used vlogs to help her understand she needed to use different facial expressions when talking, rather than always looking mad. She also joined a church worship band to help her make friends. To help with the documentary, she also crowdfunded. A GoFundMe raised more than $10,000, which was their first goal to get the film funded. The second goal is to raise $20,000 to help 'get a rough cut to Sundance by September.' And the final goal is to raise $25,000 for other festivals and impact campaigns. 'Because of your support, we're now gearing up to film the final scenes of The Fish Don't Care When It Rains next month. This is huge,' the GoFundMe states. 'Every single dollar, share, and word of encouragement helped get us here. Thank you for being part of this movement.' She's hoping getting the documentary on the right platforms will finally bring 'real conversation' and change. 'They wildly underestimated me,' she said. More stories about JRC Sent to school that uses electric shocks, Mass. woman runs for office to protect others 'It's people like me': Protesters rally against electric shock devices at Mass. school FDA's proposed ban on shock devices used at Mass. school faces new battle Former student speaks out against electric shock devices used at Mass. facility DCF sends kids to controversial Mass. facility known for electric shock devices Read the original article on MassLive. Solve the daily Crossword
