Latest news with #geneediting
Yahoo
a day ago
- Business
- Yahoo
The 3 Things That Matter for CRISPR Therapeutics Now
Key Points CRISPR Therapeutics' gene-editing treatments are highly customized and shockingly expensive. The market is watching to see if the underlying science can be applied to treat many diseases. The company has plenty of money right now, but its expenses could grow quite a bit from here. 10 stocks we like better than CRISPR Therapeutics › Biotechnology outfit CRISPR Therapeutics (NASDAQ: CRSP) isn't a name on many investors' radar -- and understandably so. Its market capitalization is a mere $6 billion. The company remains in the red largely because it's barely got any revenue to speak of. It's not likely to swing to a profit in the immediate future, either. Still, if you've got room in your portfolio for a little more risk paired with above-average upside potential, this is a stock worth adding to your watchlist (if not your portfolio) with three key things in mind. But first things first. What's CRISPR Therapeutics? Although the company was founded back in 2013, most of its time since then has been spent refining the work first done by Jennifer Doudna, Ph.D., and co-founder Emmanuelle Charpentier, Ph.D., who jointly figured out how to "edit" defective genetic code in a strand of DNA. By using a protein called Cas9 to find and remove a damaged portion of a genetic sequence and then replace it using a gene-editing biotechnology based on clustered regularly interspaced short palindromic repeats -- or CRISPR -- medical science is now able to do what was once unthinkable. It's still early days for the science -- very early. In fact, the FDA only made its first-ever approval of a gene-editing therapy in December of 2023. That's Casgevy, for the treatment of sickle cell disease, which was approved in the U.K. only a month earlier. The thing is, Casgevy is CRISPR Therapeutics' treatment, underscoring how well developed this biotech outfit's science is, and perhaps indirectly underscoring the fact that many rival drug developers are still well behind. Casgevy isn't the company's proverbial big Kahuna, however -- it's merely proof that gene editing can be successfully done. The heavy hitters in CRISPR's developmental pipeline are CTX310 for the treatment of certain cardiovascular diseases, and CTX131 and CTX112, both of which are taking aim at cancer using the very same CRISPR science. Although all of these drugs still have years of developmental work ahead of them, again, the underlying gene-editing technology works. Its potential applications are enormous. That's why CRISPR has a dozen or so others in clinical or pre-clinical trials also underway at this time. Three things to watch As time marches on, though, this stock's backstory is evolving from one broadly driven by an idea to one that increasingly hinges on some very specific factors. To this end, here are the three things that matter the most to current and prospective CRISPR Therapeutics shareholders right now -- and for the foreseeable future -- since they'll either drive the stock higher or let it slide into a sell-off. 1. Insurers and patients' acceptance of CRISPR-based medicine's cost While the science of using CRISPR to repair faulty cells is exciting, it's not exactly cheap or easy. See, Casgevy isn't a pill or an injection. It requires a sample of a patient's own blood stem cells to create a completely customized therapy, which is then infused back into that patient after he or she has undergone chemotherapy. All of this care can only be done at one of CRISPR's 65 authorized treatment centers. Total cost? A little over $2 million per patient. That's pretty steep for any therapy, but particularly for sickle cell disease, which at least has a handful of more affordable treatment options. The cost of treating life-threatening cancer is less of a stumbling block, even for insurers that may occasionally see bills nearly this size for even the most conventional of oncology treatment regimens. The price tag of this and any other future CRISPR-based therapy, however, is likely to remain in this ballpark, where payers may well balk. 2. The ongoing development of CTX310 Again, while Casgevy is approved to treat sickle cell disease, it's really more of a proving ground for the other drugs in CRISPR Therapeutics' developmental pipeline. This, of course, includes CTX131 and CTX112, but the company itself is putting the spotlight on CTX310 as a treatment for ANGPTL3 (angiopoietin-like 3), which is often associated with poorly regulated cholesterol, lipids, and triglycerides. If the company can demonstrate its solution is at least as good as (if not better than) alternative cholesterol-fighting drugs, investors might continue to support this stock. This information is coming. The company posted encouraging early results of CTX310's phase 1 clinical trial late last month, and says it intends to offer more detailed data at a healthcare conference scheduled for later in the year. If it's compelling enough, it could tamp down worries over the high cost of any CRISPR-based treatment. 3. Liquidity Finally, you'll want to keep an eye on CRISPR Therapeutics' liquidity, or the amount of cash it has on hand to cover its operating costs while it continues to develop CTX310 at the same time it's looking to expand Casgevy's commercialization. As of the end of the first quarter, CRISPR was sitting on $1.86 billion in cash, which is a lot for a company of this size. It's working through this money pretty quickly, though, shelling out nearly $150 million in operating expenses in Q1 alone. That doesn't include a full quarter's worth of the sort of costs the phase 1 trial of CTX310 is incurring now, or any other trials it starts or expands in the foreseeable future. Continued revenue growth from Casgevy should seemingly help offset some of this spending, even if not all of it. Indeed, the analyst community expects CRISPR's top line to soar from less than $50 million this year to more than $400 million in 2027, even if these same analysts believe the company will still be well in the red then. A few years' worth of losses isn't exactly unusual for a young biotech start-up. CRISPR Therapeutics would still lack much-needed scale even after such growth, though. The money it needs to spend just to get any meaningful degree of traction from Casgevy or maintain its clinical trials could far exceed any conceivable amount of revenue the newly approved drug might produce in the foreseeable future. Don't confuse what CRSP stock is So what's the call? There isn't one -- not this time. Buying or avoiding a stake in CRISPR Therapeutics is entirely up to you, depending on your risk tolerances and your ability to manage such a holding. If you're strictly a buy-and-hold investor, there's probably not enough certainty here yet to latch onto for the long haul. And there may never be. If you've got a speculative side that can tolerate risk in exchange for hype-driven reward, though, you could make a decent bullish case. Just don't confuse the two, or muddy the waters by trying to be both. The last thing you want to do here is talk yourself into a long-term position that requires you to ignore obvious red flags like the three potential ones discussed above. Should you buy stock in CRISPR Therapeutics right now? Before you buy stock in CRISPR Therapeutics, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and CRISPR Therapeutics 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 28, 2025 James Brumley has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics. The Motley Fool has a disclosure policy. The 3 Things That Matter for CRISPR Therapeutics Now was originally published by The Motley Fool
Yahoo
a day ago
- Business
- Yahoo
Verve Therapeutics (VERV) Reported Results From its Heart-2 Phase 1b Trial for VERVE-102
Verve Therapeutics, Inc. (NASDAQ:VERV) is one of the . On April 14, Verve Therapeutics, Inc. (NASDAQ:VERV) reported results from its Heart-2 Phase 1b trial for VERVE-102. The trial focused on treating people with heterozygous familial hypercholesterolemia or early heart disease. Such patients need to lower their LDL cholesterol levels for a long time. Management noted that 14 participants received VERVE-102 at different doses, and the treatment was marked safe as there were no serious side effects connected to the drug and no important lab problems were seen. A woman in a lab coat examining a syringe of gene editing medicine in a laboratory setting. The results showed that a single infusion of VERVE-102 reduced a key blood protein and significantly dropped LDL cholesterol. The reduction was dependent on the dose, with a mean reduction of 53% and a maximum reduction of 69% observed among four participants in the 0.6 mg/kg dose cohort. Management believes that these results are promising, as a single dose can offer long-lasting reduction in LDL cholesterol. Verve Therapeutics, Inc. (NASDAQ:VERV) is a clinical-stage company developing genetic medicines for cardiovascular disease. Their treatments aim to provide long-lasting effects with a single gene-editing therapy instead of ongoing medication. While we acknowledge the potential of VERV as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
2 days ago
- Business
- Yahoo
Cellectis to Report Second Quarter 2025 Financial Results on August 4, 2025
NEW YORK, July 28, 2025 (GLOBE NEWSWIRE) -- Cellectis (the 'Company') (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the second quarter 2025 ending June 30, 2025 on Monday August 4, 2025 after the close of the US market. The publication will be followed by an investor conference call and webcast on Tuesday August 5, 2025 at 8:00 AM ET / 2:00 PM CET. The call will include the Company's second quarter results and an update on business activities. Details for the call are as follows: Dial in information: Domestic: +1-800-343-5172 International: +1-203-518-9856 Conference ID: CLLSQ2 Webcast Link: About Cellectis Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. The company utilizes an allogeneic approach for CAR T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to develop gene therapies in other therapeutic indications. With its in-house manufacturing capabilities, Cellectis is one of the few end-to-end gene editing companies that controls the cell and gene therapy value chain from start to finish. Cellectis' headquarters are in Paris, France, with locations in New York and Raleigh, NC. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more, visit and follow Cellectis on LinkedIn and X. TALEN® is a registered trademark owned by Cellectis. For further information on Cellectis, please contact: Media contacts: Pascalyne Wilson, Director, Communications, + 33 (0)7 76 99 14 33, media@ Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46 93 Investor Relations contact: Arthur Stril, Chief Financial Officer & Chief Business Officer, investors@ Attachment 20250728_Q2 2025 earnings call announcement_ENGLISHError while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
ABC News
2 days ago
- Business
- ABC News
Food standards regulator proposes genetically modified definition update
As food companies welcome a proposal to alter the definition of genetic modification in the Food Standards Code, organic producers fear the change could "mislead" the public. Australia's food regulator, Food Standards for Australia and New Zealand (FSANZ), has proposed a change that would allow some foods produced using gene editing techniques to avoid being labelled as genetically modified. Currently, food defined as genetically modified is subject to strict regulations and restrictions, making it difficult for food tech companies to get new products to market. Under the proposed change, food that has been gene edited would not be subject to the same rules, unless "novel DNA" has been introduced. FSANZ defines novel DNA as DNA which, as a result of the use of gene editing, is different in chemical sequence or structure. General manager of public health at FSANZ Christel Leemhuis said the definition change would "remove ambiguity" for businesses and consumers. "The new definition will … improve clarity and predictability for businesses, regulators and consumers, while continuing to protect public health and safety," Ms Leemhuis said. "[The current definition] is outdated and doesn't reflect the new technologies in the market." Ms Leemhuis said an example of what would get through under the definition change were cases where gene editing had been used to remove a gene from corn to change its starch content. "It replicates a change that can occur naturally but does it in a more targeted and efficient way," said Ms Leemhuis. Ms Leemhuis said anything with "novel DNA" would still be subject to the same standards for genetically modified food which apply now. CropLife Australia CEO Matthew Cossey said the change would bring Australian standards in line with those of global trading partners, such as the US and Canada, and open the doors for new technology. "You're not introducing a different or foreign gene, you're simply doing what we've done through traditional breeding … with no difference except in precision and time," Mr Cossey said. Croplife Australia represents innovators, developers, manufacturers, formulators and registrants of crop protection and ag-biotechnology products. Mr Cossey said Australia was lagging behind the rest of the world because the current definition made it "too difficult" to bring new products to market. "The system is antiquated and at the moment you're capturing things that just aren't [GM]," he said. "[Such as] Arctic apples and a range of other products that aren't in the Australian market." The Canadian product was developed through gene editing, to prevent browning when sliced or bruised. Mr Cossey said allergies and other health issues could also be better addressed under the change. "Things like gluten allergy could be addressed here [and] the health benefits are one of the most exciting areas in terms of the [new breeding techniques]," he said. Peak industry body Australian Organic Limited said the change would be "misleading" to the general public. Operations and technical manager Josefine Pettersson said some products created with new breeding techniques like gene editing would not require any labelling, traceability or testing prior to entering the consumer food market. Ms Pettersson said the change to the definition would mean products like purple tomatoes, which are currently being assessed by the regulator, would not need approval to go to market. "They would just be automatically approved because they are a mutation and genetic editing of the same species, tomato DNA," she said. Ms Pettersson said the group was also concerned it would not be mandatory to disclose if products had a genetically modified food in the ingredients list, but Mr Cossey rejected that. "GM foods still have mandatory labelling requirements, all GM foods will continue to have that in Australia," he said. FSANZ said consumers would still have information to ensure informed choice. Food ministers from Australia and New Zealand have until August 3 to consider FSANZ's change definition.
National Post
2 days ago
- Business
- National Post
Pluristyx Completes Investment Round to Expand Product Portfolio and Commercial Operations
Article content SEATTLE — Pluristyx, a leading provider of induced pluripotent stem cell (iPSC) technologies and proprietary gene editing solutions, today announced the successful close of its most recent round of funding led by BioLife Solutions, the global leader in cryopreservation technologies and systems that support cell and gene therapy (CGT) manufacturing, and BroadOak Capital Partners, a specialist investor in the life science tools and services industry. Article content The newly raised capital will enable Pluristyx to expand inventory with new clinical grade cell lines incorporating Pluristyx's proprietary FailSafe® and iACT™ engineering and hypoimmune genetic edits in its best-in-class, polyclonal, synthetic RNA reprogramed iPSCs. These proprietary cells are specifically designed to support making next-generation genetically engineered, stem cell-derived therapies and support uniquely safe, effective, and scalable living medicines. The funding will also support Pluristyx's recently launched PluriForm ™ kit, which provides researchers with streamlined tools for generating organoids and other complex test systems. Article content 'We are incredibly excited for the successful close of this round and gratified to have industry leaders and peers validate our vision with their investment,' said Benjamin Fryer, PhD, CEO of Pluristyx. 'The support from our Seattle area neighbor, BioLife Solutions, and continued support from our colleagues at BroadOak Partners will allow us to manufacture, commercialize, and distribute our innovative solutions and help our customers solve critical industry challenges.' Article content Roderick de Greef, BioLife's Chairman and CEO, remarked, 'We have known and worked with the founding team at Pluristyx for many years, and have a great deal of respect for their scientific expertise in cell therapy. Their recent development of an iPSC-based biological assay for organoid manufacturing dovetails with our interest in exploring biological assays more broadly as a product portfolio adjacency.' Article content 'We are excited to continue our support of Dr. Fryer and the entire Pluristyx team,' said Daniel Friedman, Principal at BroadOak Capital Partners. 'Pluristyx is differentiated by its suite of proprietary technologies that directly address some of the most critical scientific challenges of the iPSC field.' Article content iPSCs are unique tools and building blocks for medicines. In addition to applications for innovative new toxicology, safety, and efficacy assays to replace animal models, Pluristyx's products are the ideal universal raw material to produce living medicines to cure currently untreatable diseases. This strategic investment will enable Pluristyx to be the industry leader offering the highest quality stem cells available anywhere. Article content About BioLife Solutions Article content BioLife Solutions is a leading developer and supplier of bioproduction products and services for the cell and gene therapy (CGT) and broader biopharma markets. Our expertise facilitates the commercialization of new therapies by supplying solutions that maintain the health and function of biologic materials during collection, development, storage, and distribution. For more information, please visit or follow BioLife on LinkedIn and X. Article content About BroadOak Capital Partners Article content BroadOak Capital Partners is a boutique financial institution that provides direct investment and investment banking services to companies in the life science tools, diagnostics, and biopharma services sectors. BroadOak has led or participated in investments in over 70 companies across the life sciences industry. For more information, visit Article content Pluristyx is dedicated to enabling the development of next-generation cell-based tools and therapies. PluriKit™, PluriForm™ Organoids, and PluriBank™ stem cells incorporating safety and tolerance features like FailSafe®, iACT™, and hypoimmune engineering streamline developers' path to the clinic and approval. Pluristyx's iPSC lines, backed by an FDA registered drug master file, with platform genetic engineering are available 'off-the-shelf' along with comprehensive support services to accelerate the creation of novel diagnostics, screening and safety testing, and curative treatments. For more information, visit Article content Article content Article content Article content
