Latest news with #CRISPR
Boston Globe
16 hours ago
- Health
- Boston Globe
Health is the foundation of American freedom
To pursue life, liberty, and happiness — not just politically but through our everyday lives — you need health. If you are unwell, if you're struggling with chronic illness, pain, depression, or the crushing burden of medical debt, your capacity to live freely, to pursue your dreams, becomes more constrained. While health is not listed in the Declaration of Independence as an inalienable right, it is a key enabler of those rights. When people are able to keep themselves, and those they love, healthy, they have the agency to live the lives they choose. The biotechnology revolution of the past decade is expanding that freedom. We are living through a remarkable era of health breakthroughs, from Advertisement CRISPR is being used to treat once-untreatable genetic disorders and recently helped save the life of a Advertisement These gains in health are expanding freedom — and our ability to meaningfully pursue our unalienable rights. That health-driven freedom is exactly what is at risk today as the federal government reconsiders its investments in These breakthroughs — and the thousands of things that happen every day in the American health care system — have no value if people can't access them. A strong health care system makes routine what is extraordinary. Heart bypass surgeries, cataract surgeries, cesarean sections, joint replacements — procedures that were once rare or risky are now commonplace and performed safely every day. Screenings catch diseases early, when they're the most treatable. This kind of health care gives people the ability to shape their lives on their own terms. A hip replacement means that an older man can take walks again with his grandchildren. A heart bypass surgery means a middle-aged woman knows she will live to see her children married or her grandkids born and growing. It means returning to work, raising a family, being there for loved ones, growing older with dignity. That's how life, liberty, and the pursuit of happiness become real and not just the promise of our Founding Fathers. Advertisement And these aren't just ideas. There is strong empirical evidence that this is so. When people have access to quality health care, It's also why we as Americans should understand health as a foundational element of freedom. Because when Americans are healthy, they are more able to live, to choose, to flourish. They are more able to exercise the freedoms our founders envisioned. On Independence Day, politicians will speak of freedom. True freedom, however, requires not just platitudes but actual policies that improve health through innovation, access, and reduced costs. Funding science research and ensuring universal access to health care are critical to realizing the Declaration's promise of life, liberty, and happiness. Advertisement
Yahoo
2 days ago
- Business
- Yahoo
Is CRISPR Therapeutics (CRSP) a Buy as Wall Street Analysts Look Optimistic?
When deciding whether to buy, sell, or hold a stock, investors often rely on analyst recommendations. Media reports about rating changes by these brokerage-firm-employed (or sell-side) analysts often influence a stock's price, but are they really important? Let's take a look at what these Wall Street heavyweights have to say about CRISPR Therapeutics AG (CRSP) before we discuss the reliability of brokerage recommendations and how to use them to your advantage. CRISPR Therapeutics currently has an average brokerage recommendation (ABR) of 1.88, on a scale of 1 to 5 (Strong Buy to Strong Sell), calculated based on the actual recommendations (Buy, Hold, Sell, etc.) made by 27 brokerage firms. An ABR of 1.88 approximates between Strong Buy and Buy. Of the 27 recommendations that derive the current ABR, 15 are Strong Buy and one is Buy. Strong Buy and Buy respectively account for 55.6% and 3.7% of all recommendations. Check price target & stock forecast for CRISPR Therapeutics here>>> The ABR suggests buying CRISPR Therapeutics, but making an investment decision solely on the basis of this information might not be a good idea. According to several studies, brokerage recommendations have little to no success guiding investors to choose stocks with the most potential for price appreciation. Do you wonder why? As a result of the vested interest of brokerage firms in a stock they cover, their analysts tend to rate it with a strong positive bias. According to our research, brokerage firms assign five "Strong Buy" recommendations for every "Strong Sell" recommendation. In other words, their interests aren't always aligned with retail investors, rarely indicating where the price of a stock could actually be heading. Therefore, the best use of this information could be validating your own research or an indicator that has proven to be highly successful in predicting a stock's price movement. Zacks Rank, our proprietary stock rating tool with an impressive externally audited track record, categorizes stocks into five groups, ranging from Zacks Rank #1 (Strong Buy) to Zacks Rank #5 (Strong Sell), and is an effective indicator of a stock's price performance in the near future. Therefore, using the ABR to validate the Zacks Rank could be an efficient way of making a profitable investment decision. In spite of the fact that Zacks Rank and ABR both appear on a scale from 1 to 5, they are two completely different measures. Broker recommendations are the sole basis for calculating the ABR, which is typically displayed in decimals (such as 1.28). The Zacks Rank, on the other hand, is a quantitative model designed to harness the power of earnings estimate revisions. It is displayed in whole numbers -- 1 to 5. Analysts employed by brokerage firms have been and continue to be overly optimistic with their recommendations. Since the ratings issued by these analysts are more favorable than their research would support because of the vested interest of their employers, they mislead investors far more often than they guide. In contrast, the Zacks Rank is driven by earnings estimate revisions. And near-term stock price movements are strongly correlated with trends in earnings estimate revisions, according to empirical research. Furthermore, the different grades of the Zacks Rank are applied proportionately across all stocks for which brokerage analysts provide earnings estimates for the current year. In other words, at all times, this tool maintains a balance among the five ranks it assigns. Another key difference between the ABR and Zacks Rank is freshness. The ABR is not necessarily up-to-date when you look at it. But, since brokerage analysts keep revising their earnings estimates to account for a company's changing business trends, and their actions get reflected in the Zacks Rank quickly enough, it is always timely in indicating future price movements. Looking at the earnings estimate revisions for CRISPR Therapeutics, the Zacks Consensus Estimate for the current year has remained unchanged over the past month at -$5.54. Analysts' steady views regarding the company's earnings prospects, as indicated by an unchanged consensus estimate, could be a legitimate reason for the stock to perform in line with the broader market in the near term. The size of the recent change in the consensus estimate, along with three other factors related to earnings estimates, has resulted in a Zacks Rank #3 (Hold) for CRISPR Therapeutics. You can see the complete list of today's Zacks Rank #1 (Strong Buy) stocks here >>>> It may therefore be prudent to be a little cautious with the Buy-equivalent ABR for CRISPR Therapeutics. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research
Associated Press
3 days ago
- Health
- Associated Press
Novartis, BrainStorm Cell Therapeutics, Neuroplast, Rapa Therapeutics, and Longeveron Driving Innovations
DUBLIN--(BUSINESS WIRE)--Jun 27, 2025-- The 'Gene and Cell Therapies Targeting CNS Disorders Market - A Global and Regional Analysis: Focus on Drug and Region - Analysis and Forecast, 2025-2035" report has been added to offering. Global gene and cell therapies targeting central nervous system (CNS) disorders a market is on track for continued growth, driven by the aging population, and the development of more effective and comfortable solutions. The ongoing advancements in product technology and the expansion of care options will continue to shape the market's future. As demand for gene and cell therapies targeting central nervous system (CNS) disorders therapies rises, both global and regional players will play a key role in meeting the needs of individuals and healthcare systems alike, improving quality of life for people living with incontinence. The gene and cell therapies targeting central nervous system (CNS) disorders are rapidly advancing, offering potential treatments for conditions such as Parkinson's, Alzheimer's, Huntington's disease, and spinal cord injuries. Increasing incidences of CNS disorders one of the major driving factors of this market. The increasing number of patients with neurological disorders such as Alzheimer's, Parkinson's, and multiple sclerosis is creating an urgent need for innovative treatments. This drives demand for gene and cell therapies, as current treatments are often ineffective or only offer temporary relief. One of the significant drivers of the global gene and cell therapies targeting central nervous system (CNS) disorders market is the technological advancements in treatment delivery. The technological innovations in drug delivery systems, such as CRISPR and gene editing, stem cell therapy and AAV vectors for gene delivery. Furthermore, improved precision in treatment technologies such as CRISPR-Cas9 allow for precise editing of genes involved in neurological diseases. This level of precision offers hope for conditions that have a genetic root, such as Huntington's disease or spinal muscular atrophy have contributed to the market's growth. Despite the growth trajectory, several challenges continue to impact the global gene and cell therapies targeting central nervous system (CNS) disorders market. One of the primary challenges is high development and manufacturing costs. The developing and manufacturing gene and cell therapies, especially those targeting CNS disorders, is extremely expensive. The production of viral vectors for gene delivery and the preparation of cell-based therapies are both costly and complex processes. The high costs of developing these therapies can limit accessibility and affordability for patients. Additionally, these high costs can result in delayed market entry and restrictions on patient access, particularly in low- and middle-income countries. Leading players in the global gene and cell therapies targeting central nervous system (CNS) disorders market, such as Novartis, and BrainStorm Cell Therapeutics are continuously innovating to improve the effectiveness and comfort of gene and cell therapies targeting central nervous system (CNS) disorders. These companies are investing heavily in research and development to introduce new, technologically advanced therapies into the market. With a strong emphasis on user-friendly and environmentally sustainable products, these companies are shaping the future of gene and cell therapies targeting central nervous system (CNS) disorders while enhancing their market positions globally. The competitive landscape of the global gene and cell therapies targeting central nervous system (CNS) disorders market is diverse, with numerous players across different regions offering a wide range of products. Regional players and local manufacturers are expected to play an important role in the market's growth, especially as demand increases in emerging markets such as Asia-Pacific. As consumer preferences shift towards more discreet, comfortable, and affordable solutions, the gene and cell therapies targeting central nervous system (CNS) disorders market will continue to evolve, fostering new opportunities for both established and emerging companies. As the gene and cell therapies targeting central nervous system (CNS) disorders market evolves, emerging trends such expansion of cell-based therapies and focuses on personalized or precision medicine. This trend allows treatments to be more specific and effective for individual patients, reducing side effects and improving patient outcomes. Companies Featured Key Topics Covered: Executive Summary 1. Global Gene and Cell Therapies Targeting CNS Disorders Market: Industry Outlook 1.1 Overview 1.2 Regulatory Landscape 1.3 Global Gene and Cell Therapies Targeting CNS Disorders Market, Patent Landscape 1.3.1 By Country 1.3.2 By Year 1.4 Global Gene and Cell Therapies Targeting CNS Disorders Market, Clinical Trials Landscape 1.5 Key Trends 1.6 Market Dynamics 1.6.1 Overview 1.6.1.1 Impact Analysis 1.6.2 Market Drivers 1.6.3 Market Restraints 1.6.4 Market Opportunities 2. Global Gene and Cell Therapies Targeting CNS Disorders Market, by Drugs, $Million, 2023-2035 2.1 Gene Therapy Drugs 2.2 Cell Therapy Drugs 3. Global Gene and Cell Therapies Targeting CNS Disorders, by Region, $Million, 2023-2035 3.1 North America 3.1.1 Key Findings in North America 3.1.2 Market Dynamics 3.1.3 Market Sizing and Forecast 3.1.3.1 North America Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.1.3.1.1 U.S. 3.1.3.1.2 Canada 3.2 Europe 3.2.1 Key Findings in Europe 3.2.2 Market Dynamics 3.2.3 Market Sizing and Forecast 3.2.3.1 Europe Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.2.3.1.1 Germany 3.2.3.1.2 U.K. 3.2.3.1.3 France 3.2.3.1.4 Italy 3.2.3.1.5 Spain 3.2.3.1.6 Rest-of-Europe 3.3 Asia-Pacific 3.3.1 Key Findings in Asia-Pacific 3.3.2 Market Dynamics 3.3.3 Market Sizing and Forecast 3.3.3.1 Asia-Pacific Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.3.3.1.1 Japan 3.3.3.1.2 China 3.3.3.1.3 India 3.3.3.1.4 Rest-of-Asia-Pacific 3.4 Rest-of-the-World 3.4.1 Key Findings in Rest-of-the-World 3.4.2 Market Dynamics 3.4.3 Market Sizing and Forecast 4. Global Gene and Cell Therapies Targeting CNS Disorders Market: Competitive Landscape and Company Profiles 4.1 Competitive Landscape 4.1.1 New Offerings 4.1.2 Mergers and Acquisitions 4.1.3 Partnerships, Alliances, and Business Expansion 4.1.4 Funding Activities 4.1.5 Regulatory Approvals 4.2 Company Profiles 4.2.1 Overview 4.2.2 Product Portfolio 4.2.3 Target Customers 4.2.4 Key Professionals 4.2.5 Analyst View 5. Research Methodology For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. View source version on CONTACT: Laura Wood, Senior Press Manager [email protected] For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 KEYWORD: INDUSTRY KEYWORD: GENERAL HEALTH NEUROLOGY HEALTH GENETICS PHARMACEUTICAL SOURCE: Research and Markets Copyright Business Wire 2025. PUB: 06/27/2025 09:33 AM/DISC: 06/27/2025 09:33 AM
Time Magazine
4 days ago
- Business
- Time Magazine
TIME100 Most Influential Companies 2025: Vertex Pharmaceuticals
Boston and London biotech company Vertex Pharmaceuticals is on a roll. In the past year and a half, Vertex worked with CRISPR Therapeutics to develop the first CRISPR treatment approved by the U.S. FDA, called Casgevy, which treats sickle-cell disease and beta thalassemia; received approval for its fifth cystic fibrosis treatment, and in January, also earned FDA approval for the first non-opioid pain killer, Journavx, in more than two decades. CEO Dr. Reshma Kewalramani is a triple threat, combining medical, research, and business experience to lead the 36-year-old company. She has continued the drug development pathway on which the company was founded: rational drug design, meaning designing drugs to address a known biological process, rather than spending time with the traditional trial and error practice of combining and testing chemicals in the hope of landing on one that has the desired effect. When faced with the myriad diseases and equally numerous targets for potential drugs the company could pursue, Kewalramani leans on a specific formula: Vertex concentrates on diseases where there aren't many treatment options, or high unmet need; they look for genetic or drug targets that animal models show can make a difference in the disease; the disease needs to have so-called biomarkers, or ways that researchers can track changes produced by the treatment; and the therapy developed should have a clear pathway for getting approval from regulatory agencies like the FDA. The approach has served the company well. Its market capitalization is around $114 billion, and total revenues were up 12% from the previous year. Treating pain in a way that avoided the addictive side effect of opioids checked all of these boxes, although it took two decades to identify the right pathway to target. 'Why did we do this? Because we thought pain was an incredibly important disease where there was no innovation and where we thought we could make a big difference,' says Kewalramani. 'And why did we keep going? Because of the science and our desire to work in areas where we could make this kind of transformative leap.' Following the launch of Casgevy, which allows people with sickle cell disease to receive a single CRISPR gene therapy to address the genetic mutation responsible for their disease, Vertex is continuing to pursue what Kewalramani calls 'one and done curative medicines.' They are in the late stages of testing a cell-based treatment for type 1 diabetes that could potentially help people skip regular insulin injections by giving them healthy insulin-making pancreatic beta cells. The company is also developing new treatments for chronic kidney disease, for which there have not been new options in almost a decade, and in 2024, Vertex bought Alpine Immune Sciences for $4.9 billion to accelerate its search for novel kidney treatments. 'We are simply not interested in making me-too medicines,' says Kewalramani.
Yahoo
4 days ago
- Science
- Yahoo
Scientists Edited Sperm to Bypass an Egg. The Result? A Mouse With Two Dads.
"Hearst Magazines and Yahoo may earn commission or revenue on some items through these links." Here's what you'll learn when you read this story: The first mice to be created from two fathers, a phenomenon known as androgenesis, have now produced healthy offspring for the first time. Researchers implanted two sperm in an egg with its nucleus removed and modified the sperm with epigenome editing, turning certain genes on and off. This process carries a high risk, but it could be used for human reproduction and saving endangered species in the future. Millions of years of evolution have determined how mammals reproduce, but if something doesn't occur in nature, it is not necessarily impossible. It could still be achieved in a lab. Mammalian reproduction usually needs one sperm and one oocyte (egg) to create a zygote that develops in the womb until it becomes a new organism. Embryogenesis and fetal development in mice and humans are similar, which is why mice are often used as prototypes for human experiments. Also like humans, mice, at least on their own, cannot create a viable zygote out of two eggs or two sperm. That is where genetic intervention comes in. Earlier this year, researcher Yanchang Wei at Shanghai Jiao Tong University in China succeeded at creating mouse embryos from two sperm cells that were implanted into an egg after its nucleus had been removed. Giving the embryo a chance at survival meant making alterations to seven sites in the sperm DNA. The embryos grew inside surrogate mothers, but only two of over two hundred made it to adulthood. Those two mice have now reproduced successfully, showing that it is possible for at least one kind of mammal with two biological fathers to have healthy offspring of its own. 'Mammals are diploid creatures,' Wei said in a study recently published in PNAS. 'Every cell in their body contains two sets of chromosomes, one from the mother and one from the father. Mouse embryos with only paternal genomes can not develop to term, even being diploid (with two sets of chromosomes).' Behind what appears to be a miracle is a method known as epigenome editing. Because the epigenome is made up of chemical compounds that give the genome directions by modifying it, the process involves editing proteins, DNA and RNA at specific locations in the genome. There is no actual change to the genome made in epigenome editing, just a shift in which genes receive chemical signals to activate or inactivate them. There are some genes in sperm and eggs that are automatically switched on or off. Wei and his team used modified CRISPR proteins to turn on genes in the sperm cells that eggs would have normally had turned on. Androgenesis is rare among animals. While its does occur among some species of invertebrates, amphibians and fish. This phenomenon can happen when females produce eggs without a nucleus, which are then fertilized by males. The embryos will only inherit the genes of their father. In another type of androgenesis, both a sperm and egg form a zygote, but the genome of the female is eliminated. Males essentially use the eggs to clone themselves. It is thought that (with the exception of hermaphroditic organisms such as clams) androgenesis did not evolve much because the population would be dominated by male genes to the point of extinction. Creating mice from two mothers is easier in comparison. Known as parthenogenesis or 'virgin birth,' this phenomenon has been seen in reptiles, amphibians, insects, fish and even California condors. In 2004, a Japanese research team succeeded in genetically modifying and birthing the first fertile parthenogenic mouse, Kaguya. Whether genetically modified androgenesis or parthenogenesis will succeed in humans remains to be seen. The high risk is currently prohibitive. In the future, it might be a viable option, and could also be used to save critically endangered species which may only have a few individuals of one sex remaining. Never say 'impossible.' You Might Also Like The Do's and Don'ts of Using Painter's Tape The Best Portable BBQ Grills for Cooking Anywhere Can a Smart Watch Prolong Your Life?
