Latest news with #CRISPRTherapeutics
Yahoo
02-08-2025
- Business
- Yahoo
CRISPR Therapeutics (CRSP) Sees 47% Price Increase Over Last Quarter
CRISPR Therapeutics recently announced significant developments in its in vivo cardiovascular disease programs, notably CTX310 and CTX320, which may have contributed to the company's 47% share price increase over the last quarter. The company's inclusion in multiple indices further enhances its market presence, potentially impacting investor interest positively. Meanwhile, CRISPR reported a net loss in its Q1 earnings, although revenue increased year-over-year. Despite a volatile market backdrop, characterized by declining major indices due to weak job reports and tariff concerns, CRISPR's advancements in therapeutic programs and strategic index additions contrasted against broader market movements. We've spotted 1 risk for CRISPR Therapeutics you should be aware of. Uncover the next big thing with financially sound penny stocks that balance risk and reward. Over the past year, CRISPR Therapeutics reported a total shareholder return of 9.74%. While the company's shares outperformed the US Biotechs industry, which returned a decline of 8.4% over the same period, it lagged behind the broader US Market's 17.7% return. The favorable performance relative to its industry can be linked to its strategic advancements in cardiovascular disease programs and inclusion in various Russell indices. The recent developments highlighted in the introduction, particularly the positive updates in CRISPR's cardiovascular programs and index additions, could potentially influence expectations around future revenue and earnings. Analysts forecast strong revenue growth of 57.3% annually, despite the company's forecast to remain unprofitable over the next three years. Meanwhile, with the current share price at $56.09, the market seems to discount the consensus analyst price target of $80.91, reflecting a substantial perceived upside potential in the stock. This price movement suggests that investors might see potential growth opportunities, even as the company navigates its profitability challenges. The valuation report we've compiled suggests that CRISPR Therapeutics' current price could be quite moderate. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include CRSP. This article was originally published by Simply Wall St. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@
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


Globe and Mail
21-07-2025
- Health
- Globe and Mail
Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA
The Key Sickle Cell Disease Companies in the market include - Vertex Pharmaceuticals/CRISPR Therapeutics, Pfizer, Glycomimetics, Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA, CRISPR Therapeutics/Vertex Pharmaceuticals, Agios Pharmaceuticals, Alexion Pharmaceuticals, Chugai Pharmaceutical/Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others. DelveInsight's 'Sickle Cell Disease Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Sickle Cell Disease, historical and forecasted epidemiology as well as the Sickle Cell Disease market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom. Some of the key facts of the Sickle Cell Disease Market Report: Among the 6MM countries the Sickle Cell Disease market size was valued ~USD 650 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034) In May 2025, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines via base editing, announced it will share new findings from its BEACON Phase 1/2 clinical trial of BEAM-101 at the upcoming European Hematology Association 2025 Congress (EHA2025), scheduled for June 12–15, 2025, in Milan, Italy. BEAM-101 is an investigational ex vivo genetically modified cell therapy being developed to treat sickle cell disease (SCD), specifically in patients experiencing severe vaso-occlusive crises (VOCs). In November 2024, BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial-stage biopharmaceutical company focused on oncology and rare diseases, announced that an abstract featuring initial results from a Phase 1 trial of motixafortide—both as a monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization in gene therapies for sickle cell disease (SCD)—has been accepted for oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition, taking place December 7-10, 2024, in San Diego, California. Conducted in collaboration with Washington University School of Medicine in St. Louis, this proof-of-concept study aims to explore alternative HSC mobilization approaches to enhance the treatment experience for SCD patients undergoing gene therapy. In June 2024, Vertex Pharmaceuticals shared long-term results for its gene therapy, Casgevy (exa-cel), in patients with sickle cell disease or transfusion-dependent beta-thalassemia (TDT). In 2023, the United States held the largest market share for Sickle Cell Disease among the 6MM, with approximately USD 603 million, followed by France and the UK. At present, treatments for Sickle Cell Disease include NSAIDs, blood transfusions, chelating agents, nutritional supplements, and broad-spectrum antibiotics. Additionally, the US FDA has approved several therapies for this condition, such as DROXIA (hydroxyurea), ENDARI (L-glutamine oral powder), ADAKVEO (crizanlizumab-tmca), and OXBRYTA (voxelotor). In 2023, the United States had the largest total number of prevalent Sickle Cell Disease cases among the 6MM. In the US, Sickle Cell Anemia (hemoglobin S/S or hemoglobin S/β0-thalassemia) was the most common subtype of Sickle Cell Disease among the type-specific prevalent cases in 2023. In the EU4 and the UK, France had the highest prevalence of Sickle Cell Disease cases, followed by the UK, while Spain reported the lowest number of cases. Key Sickle Cell Disease Companies: Vertex Pharmaceuticals/CRISPR Therapeutics, Pfizer, Glycomimetics, Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA, CRISPR Therapeutics/Vertex Pharmaceuticals, Agios Pharmaceuticals, Alexion Pharmaceuticals, Chugai Pharmaceutical/Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others Key Sickle Cell Disease Therapies: CASGEVY (exagamglogene autotemcel), Inclacumab, GMI-1687, BIVV003, CSL889, Siplizumab, Etavopivat Tablets, VIT-2763, Crizanlizumab, Epeleuton, Voxelotor, Hydroxycarbamide, Exagamglogene autotemcel, Mitapivat, Canakinumab, ALXN1820, Crovalimab, EDIT 301, BIVV003, BEAM101, Hemopexin, and others The Sickle Cell Disease epidemiology based on gender analyzed that Sickle Cell Disease affects males and females equally The Sickle Cell Disease market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Sickle Cell Disease pipeline products will significantly revolutionize the Sickle Cell Disease market dynamics. Sickle Cell Disease Overview Sickle cell disease (SCD) comprises inherited red blood cell disorders resulting from an abnormality in the structure of hemoglobin known as sickle hemoglobin (HbS), which encodes the beta hemoglobin subunit. Sickle Cell Disease Epidemiology The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends. Sickle Cell Disease Epidemiology Segmentation: The Sickle Cell Disease market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Sickle Cell Disease Drugs Uptake and Pipeline Development Activities The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Sickle Cell Disease market or expected to get launched during the study period. The analysis covers Sickle Cell Disease market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share. The report also covers the Sickle Cell Disease Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies. Sickle Cell Disease Therapies and Key Companies Discover more about therapies set to grab major Sickle Cell Disease market share @ Sickle Cell Disease Treatment Landscape Scope of the Sickle Cell Disease Market Report Study Period: 2020–2034 Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom)] Key Sickle Cell Disease Companies: Vertex Pharmaceuticals/CRISPR Therapeutics, Pfizer, Glycomimetics, Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA, CRISPR Therapeutics/Vertex Pharmaceuticals, Agios Pharmaceuticals, Alexion Pharmaceuticals, Chugai Pharmaceutical/Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others Key Sickle Cell Disease Therapies: CASGEVY (exagamglogene autotemcel), Inclacumab, GMI-1687, BIVV003, CSL889, Siplizumab, Etavopivat Tablets, VIT-2763, Crizanlizumab, Epeleuton, Voxelotor, Hydroxycarbamide, Exagamglogene autotemcel, Mitapivat, Canakinumab, ALXN1820, Crovalimab, EDIT 301, BIVV003, BEAM101, Hemopexin, and others Sickle Cell Disease Therapeutic Assessment: Sickle Cell Disease current marketed and Sickle Cell Disease emerging therapies Sickle Cell Disease Market Dynamics: Sickle Cell Disease market drivers and Sickle Cell Disease market barriers Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies Table of Contents 1. Sickle Cell Disease Market Report Introduction 2. Executive Summary for Sickle Cell Disease 3. SWOT analysis of Sickle Cell Disease 4. Sickle Cell Disease Patient Share (%) Overview at a Glance 5. Sickle Cell Disease Market Overview at a Glance 6. Sickle Cell Disease Disease Background and Overview 7. Sickle Cell Disease Epidemiology and Patient Population 8. Country-Specific Patient Population of Sickle Cell Disease 9. Sickle Cell Disease Current Treatment and Medical Practices 10. Sickle Cell Disease Unmet Needs 11. Sickle Cell Disease Emerging Therapies 12. Sickle Cell Disease Market Outlook 13. Country-Wise Sickle Cell Disease Market Analysis (2020–2034) 14. Sickle Cell Disease Market Access and Reimbursement of Therapies 15. Sickle Cell Disease Market Drivers 16. Sickle Cell Disease Market Barriers 17. Sickle Cell Disease Appendix 18. Sickle Cell Disease Report Methodology 19. DelveInsight Capabilities 20. Disclaimer 21. About DelveInsight About DelveInsight DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach. Media Contact Company Name: DelveInsight Contact Person: Gaurav Bora Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Albany State: New York Country: United States Website:


Globe and Mail
21-07-2025
- Business
- Globe and Mail
3 Monster Stocks in the Making to Buy Right Now
Key Points CRISPR Therapeutics is still in its early growth stage. Summit Therapeutics has plenty of upside potential remaining. Viking Therapeutics is targeting two monster markets. 10 stocks we like better than CRISPR Therapeutics › Where can you find the next monster stocks? Check out the biotech space. It's not easy, but investors can sometimes find highly promising biotech stocks that are only in their early innings. Three Motley Fool contributors think they've identified monster stocks in the making. Here's why they picked CRISPR Therapeutics (NASDAQ: CRSP), Summit Therapeutics (NASDAQ: SMMT), and Viking Therapeutics (NASDAQ: VKTX). Image source: Getty Images. A promising company in its early growth stage David Jagielski (CRISPR Therapeutics): Biotech company CRISPR Therapeutics has a market cap of around $5 billion, but it has the potential to be much more valuable in the long run. The gene editing market is still fairly small, but it has a lot of room to grow. Analysts from Grand View Research project that it will expand at a compound annual growth rate of more than 16% between now and 2030, when it will be worth $25 billion. As a leading company in the space, CRISPR Therapeutics is well positioned to benefit from future growth in this area of healthcare. The company, and its key development partner, Vertex Pharmaceuticals (NASDAQ: VRTX), already have an approved gene therapy treatment in Casgevy, which regulators have approved for both sickle cell disease and transfusion-dependent beta-thalassemia. The companies will share in the profits on the treatment (CRISPR will take 40%). Casgevy is still in its early growth stages. CRISPR is also working on developing treatments for type 1 diabetes and cardiovascular disease. While CRISPR remains unprofitable today, its future does look bright. It's a long road ahead for CRISPR Therapeutics, but with an encouraging pipeline and a lot of growth potential, this could be a monster healthcare stock in the making. The business is well funded with its cash and marketable securities as of the end of March totaling nearly $1.9 billion, putting it in an excellent position to continue investing in its research and development efforts. CRISPR can make for an excellent investment, but it's also one you'll need to be patient with. Still plenty of upside left for this stock Prosper Junior Bakiny (Summit Therapeutics): Few biotech companies have performed better than Summit Therapeutics over the past three years. The company's shares have soared by more than 2,000%, making early investors who held on significantly wealthier in the process. Here's the good news for those who are still considering the stock: There could be a massive upside left for Summit Therapeutics. Though the biotech generates no revenue and is unprofitable, it has one of the more promising pipeline products in the industry. Summit Therapeutics' leading candidate is a cancer medicine called ivonescimab, which it licensed out from Akeso Biopharma (OTC: AKES.F), a China-based drug developer. The medicine has been extensively tested in China, where it has already received approval in certain indications. One of ivonescimab's most promising markets is non-small cell lung cancer (NSCLC). In a phase 3 study in China, it went head-to-head against the market leader and the world's best-selling drug: Merck 's (NYSE: MRK) Keytruda. Ivonescimab emerged victorious, leading to a greater decrease in the risk of recurrence or death among NSCLC patients with a PD-L1 protein overexpression. Summit Therapeutics is conducting clinical studies to support approval in the U.S. and other regions. Furthermore, the biotech is likely to seek a series of approvals and label expansions over the years, based on the number of clinical trials and indications Akeso is targeting in China. Ivonescimab should easily become a blockbuster and generate strong sales for years. That's why it's not too late to invest in Summit Therapeutics. If ivonescimab achieves its full potential, Summit could deliver market-beating returns in the next five to 10 years and establish itself as a prominent player in the biotech industry. Targeting two monster markets Keith Speights (Viking Therapeutics): The big story for Viking Therapeutics is its experimental obesity drug VK2735. The company recently advanced a subcutaneous formulation of this candidate into late-stage clinical testing after announcing highly encouraging results from a phase 2 study last year. Viking is also evaluating an oral version of VK2735 in a separate phase 2 clinical trial, and it expects to report results from that study later in 2025. In addition, the drugmaker plans to initiate a clinical study for a monthly maintenance version of subcutaneous VK2735 in the coming months. Morgan Stanley Research projects that the global obesity drug market could reach $150 billion by 2035. Viking could be in a great position to claim a nice chunk of that market if VK2735 fulfills its potential. Obesity isn't the only big market that the company is targeting, though. In 2023, Viking reported positive results from a phase 2 study of VK2809 in treating metabolic-associated steatohepatitis (MASH), a chronic liver disease also known as nonalcoholic steatohepatitis (NASH). The company is seeking a partner to advance VK2809 into late-stage testing, so it can dedicate its financial resources exclusively to VK2735 for now. Grand View Research predicts the MASH/NASH market could hit $33.8 billion by 2030, so that's a nice opportunity. A clinical trial flop for VK2735 and/or a failure to find a partner for VK2809 could prevent Viking from becoming a monster stock. However, I think the company's chances look pretty good. I also wouldn't be surprised if a larger drugmaker swoops in to acquire Viking. Should you invest $1,000 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 10 best stocks 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. 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