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Yahoo
29-07-2025
- 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 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
29-07-2025
- 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
Mint
12-05-2025
- Science
- Mint
This rice is set to make your meal climate-friendly
Earlier this month, India released the world's first genome-edited rice, a breakthrough that promises to retune its farm R&D space. The new varieties yield more using less water and are resilient to climate shocks. Mint explains why you must hold your plate and take note. Tell us more about these new varieties... These were released on 4 May by the Indian Council of Agricultural Research (ICAR), which used genome editing (GE) to improve two rice cultivars—Samba Mahsuri and MTU1010. The project began in 2018. These advanced varieties can improve yields by up to 19% and are shorter-duration—they use less water and fertilizers. They emit less methane, a potent greenhouse gas, and overall have a lower carbon footprint. As per ICAR, the GE variants are more tolerant to drought, salinity and climate stresses. They can be grown across South, central and East India, as a sustainable choice for farmers in the era of climate crisis. Also Read | Can soil-less farming revive a Kashmiri rice variety on the verge of extinction? In what way is it designer rice? GE technology uses CRISPR-Cas, a protein that acts as molecular scissors to edit the DNA sequence of a genome. Editing a genome, which carries the genetic code of an organism, changes its character—a bit like rewriting the code of life. Using CRISPR-Cas, scientists can design or incorporate traits like increasing the number of grains on a plant. GE crops are different from genetically modified (GM) ones. In GM technology, a foreign DNA is inserted into plant genes. For example, genes from a soil bacterium were used to make Bt Cotton, the only GM crop allowed in India, to make it pest resistant. Also Read | Rice fortification can help tackle our problem of hidden hunger Can this technology be used for other crops? Yes. GE technology, it is hoped, will help India find a way out of its import dependency in pulses and oilseeds. ICAR is working on GE enhancement of these crops, for which the government has set aside ₹500 crore. Currently, India spends more than $20 billion every year to import pulses and oilseeds. By contrast, it is the world's largest exporter of rice. Also Read | Subsidies and MSP:It makes most sense for farmers to keep growing rice and wheat Are GE crops safe to consume? Scientists say GE crops pose only a marginal risk to human health and environment, and are as good as normally bred crops, which involve crossing plants. GE is more precise and a faster way to achieve results. The Coalition for GM-Free India has criticised the government's deregulation of GE techniques (compared with GM, which is tightly regulated), and alleged that the new varieties were released without any safety assessment. Experts say activists oppose gene technologies on the basis of speculative risks. Is the CRISPR tool globally accepted? Scientists Emmanuelle Charpentier and Jennifer Doudna received the 2020 Nobel Prize in chemistry for developing CRISPR-Cas. The Nobel committee said GE can be used for cancer therapies, hereditary diseases and develop innovative crops. GE has been used to create soy oil that can be stored for longer, reducing the use of chemical preservatives. GE was also used to make high-GABA tomato, which claims to lower blood pressure and improve sleep. But repurposing food as medicine may not be to everybody's taste.
CNBC
06-05-2025
- Health
- CNBC
CRISPR-based gene editing revolutionized medicine—what's next for the firm that helped develop it?
CRISPR-Cas 9 is a gene-editing tool that made it possible to rewrite any organism's genetic code and tackle genetic diseases more effectively. Known as genetic scissors, CRISPR identifies a DNA sequence that is cut by an enzyme called Cas 9. It then changes or replaces that sequence with a different section of DNA. For this discovery, co-inventors Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize in Chemistry in 2020. "By our interest in the lab to find new molecules that could have a role in in the bacterium streptococcus pardonus , we came across a very neat mechanism that allows to really recognize the virus that infects the bacterium in a very, very specific minor at the level of the genome of the virus. And we exploited this natural mechanism to develop the CRISPR-Cas9 technology," Emmanuelle Charpentier said in an interview with CNBC's The Edge. In 2013, Charpentier co-founded CRISPR Therapeutics to fulfil her lifelong goal of finding cures for diseases. A decade later, the company and its partner Vertex Pharmaceuticals developed CASGEVY, a therapy to treat blood disorders beta thalassemia and sickle cell disease. "With CASGEVY, we're taking the bone marrow cells from the patient, making the edit for that particular patient and we're putting it back into the patient, and it reconstitutes the hematopoietic system of the patient. We're making a drug just for you," CRISPR Therapeutics' CEO Samarth Kulkarni told The Edge. CASGEVY is a one-time therapy that costs $2.2 million per patient and can be administrated on patients 12 years of age and older. In 2023, it became the first CRISPR-based gene editing therapy to be approved by the Federal Drug Administration. CRISPR Therapeutics currently has seven clinical and ten pre-clinical programs across oncology, autoimmune cardiovascular disease and diabetes, and is investigating next generation editing modalities. Watch the video above for the full interview with Professor Charpentier from Berlin, Germany, and a tour of CRISPR Therapeutics' facilities in Boston, Massachusetts.
Euronews
31-01-2025
- Health
- Euronews
‘Much-needed hope' as CRISPR gene editing therapy recommended for sickle cell disease
Some with severe sickle cell disease in England will now be able to access landmark CRISPR gene editing therapy, according to new health guidelines which were hailed as marking a 'significant shift' in treatment for the blood disorder. The UK's National Institute for Health and Care Excellence (NICE) said on Friday that it had approved the use of the gene editing therapy exagamglogene autotemcel (exa-cel) by England's National Health Service (NHS). NICE had previously rejected the therapy's use on the NHS to treat some people with sickle cell disease in draft guidance released in March. It approved the use of exa-cel for the rare blood disorder beta thalassemia later in the year. The therapy, which the UK's medicines regulator cleared for use in November 2023, has a list price of £1.6 million (€1.9 million) per course. Sickle cell disease is a group of inherited blood disorders that affects the shape of red blood cells. People with the condition can experience severe pain, infections, anaemia, and other problems, according to the NHS. The disease is more common in people from African, Caribbean, Middle Eastern, or South Asian family backgrounds. There are currently few treatments for symptoms of the disease and the treatments that exist have 'intolerable side effects,' NICE said. The gene editing therapy will be available to some people aged 12 and older with severe complications from the condition and where a stem cell transplant is 'suitable' but a donor cannot be found, according to the watchdog. 'Much-needed hope' "The approval of exa-cel today marks a significant shift in the treatment landscape of sickle cell disease in the UK,' Funmi Dasaolu, who has sickle cell disease and spoke to the NICE committee, said in a statement. 'It is the beginning of re-addressing the inequalities in care experienced by so many with the condition,' she said, adding that it provides 'much-needed hope'. The discovery of the CRISPR/Cas9 gene editing tool won two researchers, Emmanuelle Charpentier and Jennifer A. Doudna, the Nobel Prize in Chemistry in 2020. Exa-cel, also called Casgevy, involves taking a person's blood stem cells, using CRISPR to edit them in a lab, and replacing them in the patient. 'The approval of Exa-cel for NHS use in England is a very exciting moment, not only because this marks the first approval of a CRISPR-based gene therapy for SCD [sickle cell disease] in the NHS, but also because it offers a potentially curative treatment for eligible patients,' said Felicity Gavins, a professor of pharmacology at Brunel University of London. 'However, while Exa-cel is a breakthrough, it is not a cure for all SCD patients, and uncertainties remain about its long-term effectiveness, safety, and accessibility,' Gavins said in a statement. 'It is critical to continue funding research to develop treatment that benefit the broader SCD population and address remaining challenges in care'.
