Latest news with #SickleCellDisease
Yahoo
4 days ago
- Business
- Yahoo
Fulcrum Therapeutics, Inc. (FULC): A Bull Case Theory
We came across a bullish thesis on Fulcrum Therapeutics, Inc. (FULC) on Stock Pursuit's Substack. In this article, we will summarize the bulls' thesis on FULC. Fulcrum Therapeutics, Inc. (FULC)'s share was trading at $6.44 as of 23rd May. FULC's trailing P/E was 312.50 according to Yahoo Finance. A scientist in a lab conducting research on cell-based therapeutics and biotechnology. Fulcrum Therapeutics (FULC) recently surged over 110% from around $3.30 to $7.00, reflecting growing optimism but also presenting a prime opportunity to take profits. The company's lead candidate, Pociredir (FTX-6058), is an oral small molecule in Phase 1b trials aimed at treating Sickle Cell Disease (SCD) by increasing fetal hemoglobin expression. While early data are encouraging, larger trials are needed to confirm efficacy and safety, especially following a prior clinical hold related to concerns over EED inhibitors. Analysts have raised their peak sales forecasts to roughly $600 million with a 50% probability of success, and Fulcrum's cash runway extends into 2027. Market dynamics favor Pociredir due to Pfizer's withdrawal of Oxbryta and slow adoption of gene therapies, creating scarcity value for new oral treatments. Beyond SCD, Fulcrum is developing programs for inherited aplastic anemias, with an Investigational New Drug application for Diamond-Blackfan anemia expected in late 2025. Despite this promise, history shows that small-cap pharma stocks can experience volatile reversals after big gains, so locking in profits while retaining a small position for potential upside on drug results seems prudent. The broader biotech sector, historically avoided by the author, has recently become more attractive due to technological advances and compelling net cash positions, as demonstrated by previous successes like Somalogic. Overall, Fulcrum represents a high-risk, high-reward opportunity in a niche with growing unmet medical needs, but caution and disciplined profit-taking remain essential given the sector's volatility. Fulcrum Therapeutics, Inc. (FULC) is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 21 hedge fund portfolios held FULC at the end of the first quarter which was 23 in the previous quarter. While we acknowledge the risk and potential of FULC as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for an AI stock that is more promising than FULC but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock. READ NEXT: 8 Best Wide Moat Stocks to Buy Now and 30 Most Important AI Stocks According to BlackRock. Disclosure: None. This article was originally published at Insider Monkey. 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
5 days ago
- Business
- Yahoo
Fulcrum Therapeutics, Inc. (FULC): A Bull Case Theory
We came across a bullish thesis on Fulcrum Therapeutics, Inc. (FULC) on Stock Pursuit's Substack. In this article, we will summarize the bulls' thesis on FULC. Fulcrum Therapeutics, Inc. (FULC)'s share was trading at $6.44 as of 23rd May. FULC's trailing P/E was 312.50 according to Yahoo Finance. A scientist in a lab conducting research on cell-based therapeutics and biotechnology. Fulcrum Therapeutics (FULC) recently surged over 110% from around $3.30 to $7.00, reflecting growing optimism but also presenting a prime opportunity to take profits. The company's lead candidate, Pociredir (FTX-6058), is an oral small molecule in Phase 1b trials aimed at treating Sickle Cell Disease (SCD) by increasing fetal hemoglobin expression. While early data are encouraging, larger trials are needed to confirm efficacy and safety, especially following a prior clinical hold related to concerns over EED inhibitors. Analysts have raised their peak sales forecasts to roughly $600 million with a 50% probability of success, and Fulcrum's cash runway extends into 2027. Market dynamics favor Pociredir due to Pfizer's withdrawal of Oxbryta and slow adoption of gene therapies, creating scarcity value for new oral treatments. Beyond SCD, Fulcrum is developing programs for inherited aplastic anemias, with an Investigational New Drug application for Diamond-Blackfan anemia expected in late 2025. Despite this promise, history shows that small-cap pharma stocks can experience volatile reversals after big gains, so locking in profits while retaining a small position for potential upside on drug results seems prudent. The broader biotech sector, historically avoided by the author, has recently become more attractive due to technological advances and compelling net cash positions, as demonstrated by previous successes like Somalogic. Overall, Fulcrum represents a high-risk, high-reward opportunity in a niche with growing unmet medical needs, but caution and disciplined profit-taking remain essential given the sector's volatility. Fulcrum Therapeutics, Inc. (FULC) is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 21 hedge fund portfolios held FULC at the end of the first quarter which was 23 in the previous quarter. While we acknowledge the risk and potential of FULC as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for an AI stock that is more promising than FULC but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock. READ NEXT: 8 Best Wide Moat Stocks to Buy Now and 30 Most Important AI Stocks According to BlackRock. Disclosure: None. This article was originally published at Insider Monkey.
Medscape
26-05-2025
- Health
- Medscape
SCD Impairs T-Cell Function in Cancer
Sickle cell disease (SCD) altered CD8+ T-cell chromatin architecture, triggering ferroptosis and weakening antitumor immunity. Hydrogen sulfide (H2S) treatment restored chromatin interactions and enhanced immune responses, improving immunotherapy effectiveness in renal medullary carcinoma. METHODOLOGY: Researchers analyzed CD8+ T cells from peripheral blood mononuclear cells of healthy donors and patients with SCD, examining chromatin architecture changes through Hi-C technology and DNA fluorescence in situ hybridization (FISH). Analysis included CD34+ hematopoietic stem cell–engrafted humanized mice with high human leukocyte engraftment showing over 75% human CD45+ leukocytes for RMC2C1 cell implantation studies. Treatment protocols involved administration of 10 mg/kg GYY4137 intraperitoneally twice weekly for 3 weeks, followed by anti–programmed cell death 1 (PD-1) antibody treatment at 200 μg per mouse twice weekly. TAKEAWAY: CD8+ T cells from patients with SCD exhibited reduced SLC7A11 expression and increased lipid peroxidation compared with healthy control individuals, with a significantly decreased H2S concentration in serum samples ( P < .0001). < .0001). GYY4137 treatment restored SLC7A11 chromatin interactions and enhanced immune function, with combination therapy showing improved antitumor efficacy ( P < .0001). < .0001). Analysis revealed reduced long-range chromatin interactions in SCD CD8+ T cells, particularly affecting genes involved in H2S biosynthesis including CBS and CTH . and . Treatment with GYY4137 and anti–PD-1 increased tumor-resident CD8+ T-cell infiltration ( P < .0001) and boosted cytotoxic activity through higher percentages of granzyme B+ CD8+ T cells. IN PRACTICE: 'Our study revealed that SCD altered CD8+ T cell 3D genome architecture, triggering ferroptosis and weakening antitumor immunity, thereby promoting tumor growth. Using murine and humanized SCD models, we found that disrupted chromosomal interactions in CD8+ T cells reduced the expression of antiferroptotic genes, including SLC7A11 and hydrogen sulfide (H2S) biogenesis genes, thereby increasing susceptibility to ferroptosis. Therapeutic restoration of H2S concentration in SCD mice rescued SLC7A11 expression, mitigated ferroptosis, and enhanced immune and antitumor responses,' the authors of the study wrote. SOURCE: This study was led by Zilong Zhao, The University of Texas MD Anderson Cancer Center in Houston. It was published online on May 12 in Immunity . LIMITATIONS: According to the authors, while Hi-C and DNA FISH analyses revealed chromatin interaction changes in CD8+ T cells under SCD conditions, altered T-cell subtypes and states may contribute to these findings. The researchers noted that while this study highlights SCD's impact on ferroptosis and chromatin architecture, other tumor microenvironment–related factors remain to be examined. Additionally, the use of peripheral blood mononuclear cell–derived humanized mice may limit insights into early immune development and influence the robustness of tumor growth differences. DISCLOSURES: This study was supported by grants from National Institutes of Health, Cancer Prevention and Research Institute of Texas, and Department of Defense. Pavlos Msaouel received funding from Gateway for Cancer Research, the Kidney Cancer Association, the V Foundation, and the MDACC Physician-Scientist Award. Additional disclosures are noted in the original article.
Yahoo
25-05-2025
- Business
- Yahoo
1 Stock Down 97% That Could Double, According to Wall Street
Editas Medicine focuses on developing gene-editing treatments. The struggling biotech recently gave up on its leading pipeline candidate. Even if it matches Wall Street's estimates in the next year, the stock is too risky. 10 stocks we like better than Editas Medicine › Over the past few years, investors have moved away from somewhat speculative and unprofitable companies to put their money into safer, steadier investments. Editas Medicine (NASDAQ: EDIT), a gene-editing-focused clinical-stage biotech, is firmly in the speculative camp, which is why its shares are down by 97% since early 2021. The stock is trading for about $1.50 right now. However, its average price target of $3.38 (according to Yahoo! Finance) implies a potential upside of about 125%. Should investors scoop up the company's shares expecting them to soar? Developing and marketing gene-editing therapies is challenging, as Editas Medicine knows well. The company's previous leading program was called reni-cel. It was being tested as a potential treatment for two rare blood disorders: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Here is the problem: Ex vivo gene-editing therapies are complex to administer. The process requires collecting patients' cells, editing them, and reinserting them into the patients. Even going through the clinical trial phase for a treatment of this kind is more expensive than if it were a simple oral pill. Though reni-cel was making progress and showing strong signs of efficacy with the patients who had received treatment, Editas Medicine decided to discontinue its development because it couldn't find a commercial partner with big pockets. Even before that, though, Editas Medicine was fighting an uphill battle. Between 2022 and 2023, three gene-editing therapies were approved in the U.S.: Zynteglo, Lyfgenia, and Casgevy. Zynteglo and Lyfgenia treated TDT and SCD, respectively, while Casgevy targeted both. Editas Medicine's relatively slow progress with reni-cel didn't bode well with investors, considering there were already competing therapies on the market. The company's decision to discontinue this program was the right one -- the move hardly makes the stock particularly attractive, but trying to push reni-cel toward commercialization despite its slow progress and the competitive landscape would have been prohibitively expensive. Editas Medicine has decided to pivot toward developing in vivo gene-editing therapies. Unlike the ex vivo variety, these kinds are administered via injection into the body of therapeutic genes -- so there is no need to collect the patients' cells. Editas Medicine has partners for some of its in vivo programs. It is working on some medicines with Bristol Myers Squibb, a leading pharmaceutical company. Further, Editas Medicine has decreased expenses and costs thanks to discontinuing the development of reni-cel and laying off a sizable portion of its workforce. It expects its cash and equivalents balance of $221 million as of the end of the first quarter to keep it afloat until the second quarter of 2027. With a stock price of $1.50, it wouldn't be too surprising to see this company more than double in value in the next year, perhaps because of progress with an early-stage clinical program, or licensing deals for one of its medicines, or buyout interest from a larger drugmaker. However, Editas Medicine is far too risky a stock for long-term investors. Its current candidates haven't even started human clinical trials yet. It will be years before any get close to approval. It's also worth pointing out that Editas Medicine gave up the development of EDIT-101 and EDIT-103, two potential gene-editing therapies for various eye diseases, also because it failed to find a commercial partner. Given the biotech's poor track record and the current state of its operations, investing in the stock right now and expecting it to generate strong returns over the long run wouldn't be that different from gambling. Before you buy stock in Editas Medicine, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Editas Medicine wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $639,271!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $804,688!* Now, it's worth noting Stock Advisor's total average return is 957% — a market-crushing outperformance compared to 167% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 19, 2025 Prosper Junior Bakiny has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Bristol Myers Squibb. The Motley Fool recommends Editas Medicine. The Motley Fool has a disclosure policy. 1 Stock Down 97% That Could Double, According to Wall Street 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
Globe and Mail
25-05-2025
- Business
- Globe and Mail
1 Stock Down 97% That Could Double, According to Wall Street
Over the past few years, investors have moved away from somewhat speculative and unprofitable companies to put their money into safer, steadier investments. Editas Medicine (NASDAQ: EDIT), a gene-editing-focused clinical-stage biotech, is firmly in the speculative camp, which is why its shares are down by 97% since early 2021. The stock is trading for about $1.50 right now. However, its average price target of $3.38 (according to Yahoo! Finance) implies a potential upside of about 125%. Should investors scoop up the company's shares expecting them to soar? Where to invest $1,000 right now? Our analyst team just revealed what they believe are the 10 best stocks to buy right now. Continue » Editas Medicine's challenges Developing and marketing gene-editing therapies is challenging, as Editas Medicine knows well. The company's previous leading program was called reni-cel. It was being tested as a potential treatment for two rare blood disorders: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Here is the problem: Ex vivo gene-editing therapies are complex to administer. The process requires collecting patients' cells, editing them, and reinserting them into the patients. Even going through the clinical trial phase for a treatment of this kind is more expensive than if it were a simple oral pill. Though reni-cel was making progress and showing strong signs of efficacy with the patients who had received treatment, Editas Medicine decided to discontinue its development because it couldn't find a commercial partner with big pockets. Even before that, though, Editas Medicine was fighting an uphill battle. Between 2022 and 2023, three gene-editing therapies were approved in the U.S.: Zynteglo, Lyfgenia, and Casgevy. Zynteglo and Lyfgenia treated TDT and SCD, respectively, while Casgevy targeted both. Editas Medicine's relatively slow progress with reni-cel didn't bode well with investors, considering there were already competing therapies on the market. The company's decision to discontinue this program was the right one -- the move hardly makes the stock particularly attractive, but trying to push reni-cel toward commercialization despite its slow progress and the competitive landscape would have been prohibitively expensive. Is there any hope for the stock? Editas Medicine has decided to pivot toward developing in vivo gene-editing therapies. Unlike the ex vivo variety, these kinds are administered via injection into the body of therapeutic genes -- so there is no need to collect the patients' cells. Editas Medicine has partners for some of its in vivo programs. It is working on some medicines with Bristol Myers Squibb, a leading pharmaceutical company. Further, Editas Medicine has decreased expenses and costs thanks to discontinuing the development of reni-cel and laying off a sizable portion of its workforce. It expects its cash and equivalents balance of $221 million as of the end of the first quarter to keep it afloat until the second quarter of 2027. With a stock price of $1.50, it wouldn't be too surprising to see this company more than double in value in the next year, perhaps because of progress with an early-stage clinical program, or licensing deals for one of its medicines, or buyout interest from a larger drugmaker. However, Editas Medicine is far too risky a stock for long-term investors. Its current candidates haven't even started human clinical trials yet. It will be years before any get close to approval. It's also worth pointing out that Editas Medicine gave up the development of EDIT-101 and EDIT-103, two potential gene-editing therapies for various eye diseases, also because it failed to find a commercial partner. Given the biotech's poor track record and the current state of its operations, investing in the stock right now and expecting it to generate strong returns over the long run wouldn't be that different from gambling. Should you invest $1,000 in Editas Medicine right now? Before you buy stock in Editas Medicine, 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 Editas Medicine wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $639,271!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $804,688!* Now, it's worth noting Stock Advisor 's total average return is957% — a market-crushing outperformance compared to167%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 May 19, 2025
