Latest news with #CRISPR
New York Post
2 days ago
- Health
- New York Post
Could Down syndrome be eliminated? Scientists say cutting-edge gene editing tool could cut out extra chromosome
Cutting-edge gene editing technology could eradicate Down syndrome, according to Japanese scientists. Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in the United States, is caused by the presence of an extra copy of chromosome 21. The extra chromosome, also known as trisomy 21, causes cellular overactivity, compromises a range of processes within the body, and can manifest in distinctive physical traits, learning difficulties, and health concerns. Advertisement Now new research out of Mie University in Japan suggests that by using the DNA-modifying tech CRISPR, it is possible to remove the surplus chromosome in affected cells and bring cellular behavior closer to typical function. 3 Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in the United States, is caused by the presence of an extra copy of chromosome 21. Mongkolchon – CRISPR-Cas9 is a gene-editing system that utilizes an enzyme to identify specific DNA sequences. Once the enzyme locates a matching site, it snips through the DNA strands. Ryotaro Hashizume and his colleagues designed CRISPR guides to target only the trisomy 21 chromosome, a process called allele-specific editing, which directs the cutting enzyme to the desired spot. Advertisement When they used it on lab-grown cells, removing the extra copy of the gene normalized the way the genes expressed themselves in the body — suggesting that the genetic burden had been removed. They also found that after the extra chromosome was removed, genes tied to nervous system development were more active and those related to metabolism were less active. This backs up previous research that found extra copies of chromosome 21 disrupt brain development during early fetal growth. Researchers also tested their CRISPR guides on skin fibroblasts, which are mature, non-stem cells taken from people with Down syndrome. Advertisement In these fully developed cells, the editing method successfully removed the extra chromosome in a number of cases. 3 Hashizume and his team designed CRISPR guides to target only the trisomy 21 chromosome, a process called allele-specific editing, which directs the cutting enzyme to the desired spot. Gorodenkoff – After removal, these corrected cells grew faster and had a shorter doubling time than untreated cells, suggesting that removing the extra chromosome may help with the biological strain that slows down cell growth. Advertisement But the CRISPR can affect healthy chromosomes, too, and researchers are refining their program so that it only attaches to the extra copy of chromosome 21. This work proves that, rather than making small fixes, CRISPR can eliminate an entire chromosome. The scientists published their findings in PNAS Nexus. Hashizume and his team are hopeful that their work may be used to design regenerative therapies and treatments that address genetic surplus at its source. 3 Researchers also tested their CRISPR guides on skin fibroblasts, mature, non-stem cells taken from people with Down syndrome. Yakobchuk Olena – Researchers will continue to analyze the risks of DNA changes and monitor how modified cells function over time and their viability in real-world settings. A recent case study explored a medical mystery related to Down syndrome; the brain of an American woman with Down syndrome showed all the classic signs of Alzheimer's disease, yet she remained symptom-free throughout her lifetime. Advertisement People with Down syndrome face a much higher risk of developing Alzheimer's-related dementia as they age — an estimated three to five times higher than the general population. Scientists are still working to pinpoint the exact cause, but it's believed that the extra copy of chromosome 21 drives the overproduction of amyloid precursor protein. This excess production leads to the buildup of amyloid beta plaques in the brain, a hallmark of Alzheimer's disease.
Forbes
3 days ago
- Business
- Forbes
Beyond Obesity: Eli Lilly's Genetic Medicine Bet On Verve Therapeutics
Cambridge, MA - July 11: Verve CEO and cofounder Sekar Kathiresan. Cambridge company Verve ... More Therapeutics announced on Tuesday morning that it has dosed the first patient with its new CRISPR gene editing technology called base editing, which changes a single letter of DNA into another letter to treat genetic disease. (Photo by Suzanne Kreiter/The Boston Globe via Getty Images) Eli Lilly's $1.3 billion acquisition of Verve Therapeutics represents a strategic move into cardiovascular genetic medicine. The pharmaceutical behemoth paid a substantial premium of approximately 113% above Verve's 30-day volume-weighted average trading price, triggering an 80% surge in Verve's stock price on June 17, 2025. The acquisition aligns with Eli Lilly's broader diversification strategy beyond its core diabetes and obesity treatment portfolio. Verve's innovative gene-editing technology, which permanently deactivates the PCSK9 gene to address high cholesterol, represents a breakthrough approach to cardiovascular disease treatment. This move positions Lilly to capture value in the high-impact cardiovascular therapeutics market while expanding its genetic medicine capabilities. On a separate note, see – SoundHound AI: Buy, Sell Or Hold SOUN Stock At $10? Despite the premium paid for Verve, Eli Lilly's core valuation metrics suggest reasonable positioning relative to historical norms. Trading at 57 times trailing adjusted earnings of $13.76 per share, the company's current valuation sits below its three-year average P/E ratio of 66 times. This suggests that while the stock commands a premium, it remains within established valuation parameters given the company's strong financial and operational performance as seen in Buy or Sell Eli Lilly Stock dashboard. Several key risks warrant attention in evaluating Eli Lilly stock: The Verve Therapeutics acquisition reinforces Eli Lilly's position as an attractive investment opportunity, supported by strong demand for its obesity treatments and successful diversification into high-potential therapeutic areas. That said, there always remains a meaningful risk when investing in a single, or just a handful, of stocks. Consider Trefis High Quality (HQ) Portfolio which, with a collection of 30 stocks, has a track record of comfortably outperforming the S&P 500 over the last 4-year period. Why is that? As a group, HQ Portfolio stocks provided better returns with less risk versus the benchmark index; less of a roller-coaster ride as evident in HQ Portfolio performance metrics.
Yahoo
3 days ago
- Business
- Yahoo
PODD Drug Delivery Conference Announces 2025 Keynotes
The Conference Forum announces the 15th annual PODD Drug Delivery Conference's 2025 keynotes NEW YORK, June 18, 2025 /PRNewswire/ -- Today, PODD: Partnership Opportunities in Drug Delivery conference announced the keynotes for its 15th annual conference which takes place on October 27-28 at the Westin Copley Place in Boston, MA. "In presenting a strategic-level program for R&D scientists and business development professionals, the PODD Conference is committed to securing keynotes who demonstrate excellence in science," said Andrew Goldstein, Senior Conference Producer for the PODD Conference. The PODD 2025 keynotes include: MIT's Dr Robert Langer, a pioneer in the research and development of drug delivery technology, will return as the Annual Keynote to discuss the state of innovation in the drug delivery industry. Dr Lotte Bjerre Knudsen, Novo Nordisk's Chief Scientific Advisor who drove the development of GLP-1 drugs for obesity will present on the liraglutide and semaglutide story, her approach to scientific development and where drug delivery intersects with her R&D work. Patient Keynote Jimi Olaghere will share his journey as a patient with sickle cell disease before and after receiving CASGEVY, a CRISPR-based therapy, and his mission to make these therapies more accessible for patients globally. Dr Mansoor Amiji, University Distinguished Professor, Northwestern University, will join the PODD Conference as the Endogenous Delivery Zeitgeist. Dr Amiji will present on endogenous targeted delivery strategies to overcome biological barriers and improve efficacy and safety. The PODD speaking faculty includes over 100 executives from both the drug development and delivery industries with over 125 session choices including a full afternoon of drug delivery presentations from established to start-up companies. To learn more about the event, visit About the PODD: Partnership Opportunities in Drug Delivery ConferencePharma, biotech and the drug delivery industries gather annually at PODD to assess delivery needs, latest trends and information on deals, and learn about a wide range of innovative drug delivery technologies that could improve the delivery of various types of drugs. This can include proteins, peptides, oligonucleotides, biologics, small molecules and more. PODD provides business development opportunities through organized networking and a partnering tool for new, emerging and established collaborations. About the Conference ForumThe Conference Forum is a life science industry research firm that develops conferences, podcasts, newsletters and webinars primarily around how to get therapeutics to patients faster. They examine and challenge the complex ecosystem of drug development and delivery, bringing ideas together from a variety of sources to help advance clinical research with common goals that are patient-focused. They are committed to creating the best content, promoting the exchange of ideas and solutions among peers, and providing high-quality networking. Learn more about the Conference Forum at For media inquiries, contact:Bre Bugbee-Barrettbre@ View original content: SOURCE Partnership Opportunities in Drug Delivery (PODD)
Yahoo
4 days ago
- Science
- Yahoo
GMOs Made With New Techniques Face Low Adoption Rates and Market Failure
New international report examines the efficacy of 'New GMOs' despite billions in investment in CRISPR and similar technology Brussels / Bellingham, Wash., June 17, 2025 (GLOBE NEWSWIRE) -- As political pressure mounts to deregulate gene-edited crops, a new report by the European Non-GMO Industry Association (ENGA) and the Non-GMO Project reveals a stark reality: New GMO crops created with newer genetic engineering techniques, such as CRISPR, ODM, TALEN, ZFN and RNAi, are failing to find growers and markets. Despite billions of dollars in investment and widespread deregulation, only three new gene-edited GMOs are currently in cultivation, and none of them meaningfully advance climate or sustainability goals. The report indicates that 49 more crops are in development. The findings dismantle the narrative that New GMOs are essential to feeding a growing population or combating the climate crisis. In fact, the evidence suggests that their real-world impact remains negligible, and two early new crops have already been withdrawn from the market due to commercial failure. 'Despite the hype, the evidence from our report is overwhelming: New GMOs are only a promise and are far from a reality on the market,' said ENGA Secretary General, Heike Moldenhauer. 'Furthermore, despite widespread claims, not a single New GMO currently on the market delivers on sustainability promises, one of the main reasons their proponents are pushing to deregulate them.' The report reveals: Only three New GMO crops are being cultivated globally: two in the US and one in Japan. Many of the 49 crops in development have been cleared for market access, especially in the US, but are not actually grown. None of the New GMO crops in development meaningfully address climate resilience or biodiversity loss, with just two showing potential for drought or salt tolerance. At the same time, the Non-GMO Project points out that continued monitoring is critical. GMO crops continue to be developed for cultivation using older transgenic engineering techniques to produce new crops like herbicide-resistant wheat and pest-resistant sugar cane. Additionally, many new GMOs are being created using synthetic biology and precision fermentation to create lab-grown foods like 'animal-free dairy' and seedless seed oils. 'To be sure, old GMOs are still an overwhelming presence in the food supply web, especially here in the United States, and more are on the way,' said Hans Eisenbeis, Director of Mission and Messaging at the Non-GMO Project. 'However, efforts to launch the next generation of GMO plants using gene-editing technologies like CRISPR just aren't getting off the ground, despite increasingly permissive regulations, exaggerated sustainability claims, and billions of dollars in public and private investment.' The report highlights shifts in the development of New GMOs compared to their old counterparts. The application of old genetic engineering largely focused on five commodity crops: soy, corn, canola, sugar beets and cotton. These are used as animal feed, processed food ingredients, fuel or clothing. Today, the application of genetic engineering is far broader. With new technologies, companies are working on a variety of plants that are intended for direct human consumption, such as tomatoes and salad greens. The report also shows that developers of New GMOs have become more diverse. Whereas old GMOs were mainly produced and marketed by the four engineering giants Corteva, Bayer, BASF and Syngenta, many start-up companies are involved in the development of New GMOs, as are state institutes. The new report lays out the regulatory hotspots around the world. ENGA and the Non-GMO Project strongly recommend that companies in the food industry: Explicitly exclude New GMOs in their supplier requirements. Rely on independent certification systems that ensure products are free from New GMOs. New GMOs are frequently "non-testable" meaning there are no commercially-available tests, so vigilance from the non-GMO community is critical. New GMOs differ from traditional GMOs in that they may not rely on transgenic technologies or leave foreign DNA in the organism. In many countries, New GMOs face a simplified regulatory process and do not require safety testing. Find the full report 'New GMOs' here. ### Media Contacts:ENGA: Sarah Farndale, farnisarah@ +32 (0)490 390665Non-GMO Project : Alex Tursi, alex@ +1 802.777.6737 About the reportThe report's editors are: Hans Eisenbeis, The Non-GMO Project (USA), Eva Gelinsky, Researcher (CH) and Heike Moldenhauer, ENGA (BE). About ENGAThe European Non-GMO Industry Association (ENGA) is the voice of the Non-GMO food and feed sector at the EU level. ENGA, founded in 2020, secures and supports the expansion of non-GMO production and advocates for the strict regulation of old and New GMOs in order to keep untested and unlabelled GMOs from entering the EU food and feed chains. About The Non-GMO Project The Non-GMO Project is a mission-driven nonprofit organization based in the US. Since 2007, the Non-GMO Project Verified seal has remained North America's most trusted third-party verification for GMO avoidance. Through its Food Integrity Collective, Non-UPF Verified program, and Non-GMO Project Verified mark, the organization promotes transparency and health in food systems. Learn more at and CONTACT: Alex Tursi Non-GMO Project 802-777-6737 alex@ in to access your portfolio
Yahoo
4 days ago
- Business
- Yahoo
Editas Medicine Is Great. Here's Why You Shouldn't Buy It.
The clinical-stage biotech is developing gene-editing CRISPR technology. Editas has reported encouraging testing results in laboratory animals. Still, the stock remains speculative amid uncertainties and challenges. 10 stocks we like better than Editas Medicine › The biotechnology sector is a thrilling arena for investors, with a history of delivering groundbreaking medical innovations that have led to massive shareholder returns. Editas Medicine (NASDAQ: EDIT) is a promising clinical-stage company that has captured Wall Street's attention at the forefront of the gene-editing revolution with a unique approach to CRISPR technologies. On the other hand, Editas faces a long road ahead before delivering its first Food and Drug Administration (FDA) approved therapy and reaching its goal of transforming into a commercially sustainable biotech. The stock, with a market capitalization of $170 million, has also been extremely volatile, down 62% over the past year despite a sharp 32% rebound in the past month as of this writing. Here's why you should proceed with caution if you're thinking about buying shares of Editas Medicine for your portfolio. Editas Medicine is developing a CRISPR gene editing system to address serious and rare conditions, such as sickle cell disease, beta thalassemia, and inherited eye disorders. Acting like molecular scissors, the CRISPR/Cas9 technology uses proteins to target specific DNA sequences in a person's genetic code, precisely addressing diseases caused by genetic mutations. More than just a treatment, this personalized medicine aims to deliver durable, curative solutions. Compared to larger biotech players pursuing alternative genomic therapies, Editas stands out with its "in vivo gene editing upregulation strategy." Rather than solely removing or replacing defective genes in the more traditional ex vivo approaches, Editas' method edits DNA directly in the body to enhance beneficial gene expression. In vivo gene editing represents a simplified treatment process without the need for cell extraction and harvesting required in ex vivo solutions. This means in vivo could prove more scalable and cost-effective. Early tests have shown encouraging results. Editas successfully edited blood stem cells in monkeys with one dose and increased a key liver protein in mice, which could help treat diseases. The plan is to formally declare two in vivo development candidates from a group of target cell types or tissues under research this year and launch at least one in vivo human clinical trial by the second half of 2026. The opportunity for Editas is to potentially deliver a first-to-market and best-in-class platform with applications for a wide range of genetic diseases. Before getting too excited about Editas Medicine as an investment, it's important to recognize that the company has substantial work ahead and faces significant technical challenges, including confirming a precise in vivo delivery mechanism and addressing off-target DNA risks. Research and development (R&D) partnerships with companies like Bristol Myers Squibb help validate the science and provide credibility to Editas Medicine's endeavors; yet, the in vivo gene editing approach has not been proven in humans. Reaching statistically significant efficacy and safety thresholds is far from certain. Even in a best-case scenario, Editas could still be several years away from a clear timeline for bringing a new therapy to market. Meanwhile, companies like CRISPR Therapeutics, Beam Therapeutics, and Intellia Therapeutics are all exploring in vivo CRISPR technologies, underscoring the highly competitive field. It's unclear whether Editas has an edge. More pressing for investors is reconciling Editas' weak fundamentals. In the first quarter, the company posted a net loss of $76.1 million, which included a $41 million restructuring charge as it abandoned its ex vivo program, pivoting exclusively to the in vivo strategy. This follows $390 million in negative net income between 2023 and 2024. The expectation is for recurring losses in the foreseeable future. With $221 million in cash on the balance sheet, company management believes it has the liquidity to continue development until mid-2027. Nevertheless, additional funding will likely be necessary to advance into late-stage development, including measures dilutive to shareholders. Any setback in the R&D process or regulatory timeline could force a reset of expectations and send the stock price lower. Balancing the pros and cons of investing in Editas Medicine, I believe the stock is simply too speculative to buy with conviction, as the risks outweigh the upside. The prudent approach is to avoid it, instead focusing on companies with higher-quality fundamentals, including steady growth and consistent profitability. Before you buy stock in Editas Medicine, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Editas Medicine 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 $660,821!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $886,880!* Now, it's worth noting Stock Advisor's total average return is 791% — a market-crushing outperformance compared to 174% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of June 9, 2025 Dan Victor has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Beam Therapeutics, CRISPR Therapeutics, and Intellia Therapeutics. The Motley Fool recommends Editas Medicine. The Motley Fool has a disclosure policy. Editas Medicine Is Great. Here's Why You Shouldn't Buy It. was originally published by The Motley Fool
