Sarepta bows to FDA pressure, pulls Duchenne therapy from market
The Boston Business Journal's Power 50, "The Movement Makers," focuses on those in the Boston-area business community who demonstrate the most influence on making both their company and the region a better place for everyone in it.
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ProQR Announces Upcoming Presentation at RNA Editing Summit
LEIDEN, Netherlands & CAMBRIDGE, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will participate in the RNA Editing Summit taking place July 29-31, 2025, in Boston, MA. '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 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 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@ contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@
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37 minutes ago
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The 3 Things That Matter for Vertex Pharmaceuticals Now
Key Points Vertex Pharmaceuticals' leading cystic fibrosis franchise still looks promising. It also has a couple of exciting newer launches, and could land another one. Shares of the large drugmaker could still deliver market-beating returns. 10 stocks we like better than Vertex Pharmaceuticals › Vertex Pharmaceuticals (NASDAQ: VRTX) has been a terrific stock to own over the past decade. The stock has outperformed the market over this period, thanks to consistent financial results and excellent innovative capabilities. But how much longer can the biotech keep things up? If you're interested in owning the stock for the long term, it's important to pay close attention to several parts of the company's business. Below are three key things to watch out for now. 1. Vertex's cystic fibrosis franchise Vertex is best known for its work in developing and marketing medicines that target cystic fibrosis (CF) at its genetic roots. CF is a rare disease affecting the lungs. Although many have attempted to challenge the biotech in this area, none have yet succeeded; Vertex's therapies remain the only option for CF patients. The company continues to generate substantial revenue from its CF franchise, and several developments are worth paying attention to now. In December Vertex earned approval in the U.S. for Alyftrek, a next-generation CF therapy with a more convenient once-daily dosing. This drug may soon become Vertex's most important product. The company is also seeking to develop newer medicines for CF patients who aren't eligible for any of its current treatments. VX-522, an investigational drug, could target many of these patients. Approximately 75% of the 94,000 people with CF in North America, Europe, and Australia are currently benefiting from Vertex's products, whereas only 33% in other regions are, so there's still room to grow. Keep a watchful eye on continued clinical and commercial progress within Vertex's most important therapeutic area. 2. How will newer launches perform? Vertex's success over the past decade has been driven by its CF franchise; it's had little diversification. However, the company recently earned approval for some medicines outside of its core market. In late 2023, Casgevy, a gene-editing therapy for two blood-related diseases that Vertex developed with CRISPR Therapeutics, landed regulatory approval. And in January of this year, Vertex's treatment for acute pain, Journavx, also got the green light from regulators in the U.S. Casgevy isn't yet contributing much to the biotech's results; gene-editing therapies are complex to administer. However, this one holds considerable potential, as there's little competition to speak of. At $2.2 million per treatment course in the U.S., and with roughly 60,000 patients in the regions Vertex is targeting, Casgevy should exceed blockbuster status. Pay close attention to Vertex's comments on Casgevy's ongoing commercial progress. The same applies to Journavx. Vertex developed this therapy to fill the need for non-opioid painkillers. It became the first oral pain-signal inhibitor approved by the U.S. Food and Drug Administration. Furthermore, Vertex is likely to seek label expansions for the medicine, including the treatment of painful diabetic peripheral neuropathy. 3. Vertex's new gem, in type 1 diabetes Vertex's next brand-new launch could be a medicine for type 1 diabetes (T1D) called zimislecel. The therapy is undergoing a phase 1/2/3 study, where it's showing real promise. For instance, most (10 out of 12) patients treated with zimislecel were insulin-free after one year of follow-up in the phase 1/2 portion of this clinical trial. All patients were also free of severe hypoglycemic events 90 days after treatment. People with T1D typically cannot produce insulin, but this therapy helps restore their ability to do so. It could work as a functional cure for the chronic disease. Vertex plans to request approval from regulatory authorities for zimislecel next year, provided there are no significant clinical setbacks. It would be a great addition to the company's lineup -- and another product for Vertex investors to monitor closely. Is the stock a buy? Vertex shares look attractive for several reasons. First, the company's dominance in CF still affords it ample room to grow revenue and profits at a steady pace for the foreseeable future. Second, although it's possible that another biotech will eventually crack the code and launch a competing CF therapy, Vertex is well prepared. That's precisely why the company has diversified its lineup in recent years, and will hopefully continue to do so with the launch of zimislecel. Third, Vertex still has an exciting pipeline in CF and elsewhere. For instance, the company's late-stage candidate for APOL-1-mediated kidney disease, inaxaplin, looks promising. Currently, there are no approved treatments that target the underlying causes of this condition. Vertex could be the first to launch one. Even if that program doesn't pan out, though, Vertex Pharmaceuticals' lineup, pipeline, and consistent financial results make its prospects attractive. Should you invest $1,000 in Vertex Pharmaceuticals right now? Before you buy stock in Vertex Pharmaceuticals, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Vertex Pharmaceuticals 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 $636,628!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,063,471!* Now, it's worth noting Stock Advisor's total average return is 1,041% — a market-crushing outperformance compared to 183% 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 July 21, 2025 Prosper Junior Bakiny has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends CRISPR Therapeutics and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. The 3 Things That Matter for Vertex Pharmaceuticals Now was originally published by The Motley Fool 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
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44 minutes ago
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Horizon Quantum Joins the QuEra Quantum Alliance, Strengthening Quantum Software Ecosystem
SINGAPORE & BOSTON, July 28, 2025--(BUSINESS WIRE)--Horizon Quantum Computing Pte. Ltd. ("Horizon Quantum"), a developer of advanced software development tools for quantum computers, and QuEra Computing, the leader in neutral-atom quantum computing, today announced that Horizon Quantum has joined the QuEra Quantum Alliance. Horizon Quantum is building software tools that seek to make the programming of quantum computers as straightforward as programming conventional systems. Through the alliance with QuEra Computing, Horizon Quantum expects to integrate its software development tools with QuEra's technologies to enable its users to code, compile, and deploy quantum applications on leading neutral-atom quantum computers. The QuEra Quantum Alliance brings together a global community of quantum hardware, software, and service providers to foster collaboration and widespread adoption of neutral-atom quantum computing. Both companies believe that the addition of Horizon Quantum will further enhance the Alliance's software capabilities, empowering organizations to accelerate the adoption and deployment of quantum computing. Since launching the QuEra Quantum Alliance last year, membership has grown to over 25 organizations across North America, Europe, Asia-Pacific, and the Middle East—including quantum software pioneers, major HPC centers, and global IT service providers. Alliance partners are engaged in joint market development initiatives and customer-facing projects, accelerating the adoption of neutral-atom quantum computing. "QuEra is at the forefront of advancing neutral atom quantum computing, a technology that is rapidly emerging as a promising path to scalable quantum computing," said Dr Joe Fitzsimons, CEO of Horizon Quantum. "We are delighted to collaborate with QuEra to provide developers with the tools they need to harness this architecture effectively." "We are excited to welcome Horizon Quantum to the QuEra Quantum Alliance," said Yuval Boger, Chief Commercial Officer at QuEra Computing. "We believe that their deep expertise in quantum software, especially automated algorithm synthesis and quantum compilation, will play a pivotal role in expanding the practical utility of our neutral-atom quantum systems." About QuEra Quantum Alliance The QuEra Quantum Alliance provides members with access to technical expertise, go-to-market resources, and joint development opportunities to maximize the potential of quantum computing. To learn more about the QuEra Quantum Alliance, visit About QuEra Computing QuEra Computing is the leader in developing and productizing quantum computers using neutral atoms, widely recognized as a highly promising quantum computing modality. Based in Boston and built on pioneering research from Harvard University and MIT, QuEra operates the world's largest publicly accessible quantum computer, available over a major public cloud and for on-premises delivery. QuEra is developing useful, scalable and fault-tolerant quantum computers to tackle classically intractable problems, becoming the partner of choice in the quantum field. Simply put, QuEra is the best way to quantum. For more information, visit us at and follow us on X or LinkedIn. About Horizon Quantum Horizon Quantum is developing a new generation of programming tools to simplify and expedite the process of developing software for quantum computers. By removing the need for prior quantum computing experience to develop applications for quantum hardware, Horizon's tools seek to make the power of quantum computing accessible to every software developer. The company was founded by Dr Joe Fitzsimons in 2018, a former professor with two decades of experience in quantum computing and computational complexity theory. The leadership team also includes Dr Si-Hui Tan, Chief Science Officer, who holds a Ph.D. in Physics from MIT and has been actively involved in quantum research for the same period. View source version on Contacts media@ Sign in to access your portfolio
