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FDA Grants Orphan Drug Status to Beam Therapeutics Inc. (BEAM)' AATD Gene Therapy
FDA Grants Orphan Drug Status to Beam Therapeutics Inc. (BEAM)' AATD Gene Therapy

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time6 days ago

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FDA Grants Orphan Drug Status to Beam Therapeutics Inc. (BEAM)' AATD Gene Therapy

Beam Therapeutics Inc. (NASDAQ:BEAM) has secured U.S. FDA orphan drug designation for BEAM-302, its pioneering genetic medicine targeting alpha-1 antitrypsin deficiency (AATD), a rare inherited disorder that can cause severe lung and liver disease. BEAM-302, delivered via liver-targeting lipid nanoparticles, is designed to correct the underlying DNA mutation responsible for AATD, offering hope for a one-time, potentially curative treatment. This regulatory milestone follows the recent Regenerative Medicine Advanced Therapy (RMAT) designation, underscoring the therapy's promise and the urgent need for effective AATD solutions. A biotechnologist in a lab coat discussing a therapeutic antibody with a colleague. Preliminary data from ongoing Phase 1/2 trials show BEAM-302 is well tolerated and achieves dose-dependent, durable correction of the disease-causing mutation, with protein levels exceeding therapeutic thresholds in the 60 mg dose group. Dosing in higher cohorts is underway, with more results from Beam Therapeutics Inc. (NASDAQ:BEAM) expected later in 2025. Orphan drug designation brings significant benefits, including tax credits, user fee exemptions, and up to seven years of market exclusivity post-approval, accelerating development for this first-in-class base editing therapy for AATD, a condition with no current curative treatments. While we acknowledge the potential of BEAM to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than BEAM and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. 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

Beam Therapeutics Inc.'s (NASDAQ:BEAM) recent 11% pullback adds to one-year year losses, institutional owners may take drastic measures
Beam Therapeutics Inc.'s (NASDAQ:BEAM) recent 11% pullback adds to one-year year losses, institutional owners may take drastic measures

Yahoo

time11-04-2025

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Beam Therapeutics Inc.'s (NASDAQ:BEAM) recent 11% pullback adds to one-year year losses, institutional owners may take drastic measures

Given the large stake in the stock by institutions, Beam Therapeutics' stock price might be vulnerable to their trading decisions A total of 7 investors have a majority stake in the company with 51% ownership Ownership research along with analyst forecasts data help provide a good understanding of opportunities in a stock AI is about to change healthcare. These 20 stocks are working on everything from early diagnostics to drug discovery. The best part - they are all under $10bn in marketcap - there is still time to get in early. Every investor in Beam Therapeutics Inc. (NASDAQ:BEAM) should be aware of the most powerful shareholder groups. And the group that holds the biggest piece of the pie are institutions with 80% ownership. Put another way, the group faces the maximum upside potential (or downside risk). And so it follows that institutional investors was the group most impacted after the company's market cap fell to US$1.6b last week after a 11% drop in the share price. This set of investors may especially be concerned about the current loss, which adds to a one-year loss of 46% for shareholders. Also referred to as "smart money", institutions have a lot of sway over how a stock's price moves. As a result, if the decline continues, institutional investors may be pressured to sell Beam Therapeutics which might hurt individual investors. Let's delve deeper into each type of owner of Beam Therapeutics, beginning with the chart below. See our latest analysis for Beam Therapeutics Many institutions measure their performance against an index that approximates the local market. So they usually pay more attention to companies that are included in major indices. We can see that Beam Therapeutics does have institutional investors; and they hold a good portion of the company's stock. This can indicate that the company has a certain degree of credibility in the investment community. However, it is best to be wary of relying on the supposed validation that comes with institutional investors. They too, get it wrong sometimes. It is not uncommon to see a big share price drop if two large institutional investors try to sell out of a stock at the same time. So it is worth checking the past earnings trajectory of Beam Therapeutics, (below). Of course, keep in mind that there are other factors to consider, too. Institutional investors own over 50% of the company, so together than can probably strongly influence board decisions. Our data indicates that hedge funds own 8.5% of Beam Therapeutics. That worth noting, since hedge funds are often quite active investors, who may try to influence management. Many want to see value creation (and a higher share price) in the short term or medium term. Our data shows that FMR LLC is the largest shareholder with 10% of shares outstanding. Farallon Capital Management, L.L.C. is the second largest shareholder owning 8.5% of common stock, and BlackRock, Inc. holds about 7.9% of the company stock. In addition, we found that John Evans, the CEO has 0.9% of the shares allocated to their name. We did some more digging and found that 7 of the top shareholders account for roughly 51% of the register, implying that along with larger shareholders, there are a few smaller shareholders, thereby balancing out each others interests somewhat. Researching institutional ownership is a good way to gauge and filter a stock's expected performance. The same can be achieved by studying analyst sentiments. There are a reasonable number of analysts covering the stock, so it might be useful to find out their aggregate view on the future. The definition of an insider can differ slightly between different countries, but members of the board of directors always count. The company management answer to the board and the latter should represent the interests of shareholders. Notably, sometimes top-level managers are on the board themselves. I generally consider insider ownership to be a good thing. However, on some occasions it makes it more difficult for other shareholders to hold the board accountable for decisions. Our most recent data indicates that insiders own some shares in Beam Therapeutics Inc.. This is a big company, so it is good to see this level of alignment. Insiders own US$20m worth of shares (at current prices). It is good to see this level of investment by insiders. You can check here to see if those insiders have been buying recently. The general public-- including retail investors -- own 10% stake in the company, and hence can't easily be ignored. While this size of ownership may not be enough to sway a policy decision in their favour, they can still make a collective impact on company policies. It's always worth thinking about the different groups who own shares in a company. But to understand Beam Therapeutics better, we need to consider many other factors. For instance, we've identified 3 warning signs for Beam Therapeutics that you should be aware of. But ultimately it is the future, not the past, that will determine how well the owners of this business will do. Therefore we think it advisable to take a look at this free report showing whether analysts are predicting a brighter future . NB: Figures in this article are calculated using data from the last twelve months, which refer to the 12-month period ending on the last date of the month the financial statement is dated. This may not be consistent with full year annual report figures. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) 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. Sign in to access your portfolio

Beam Therapeutics Presents Additional Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress
Beam Therapeutics Presents Additional Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress

Yahoo

time05-04-2025

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Beam Therapeutics Presents Additional Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress

New Data Demonstrate Proportion of Corrected M-AAT Reached a Mean of 91% of Total AAT in Circulation at Day 28 Following BEAM-302 Treatment in 60 mg Cohort (n=3) Mean Decrease of 79% in Mutant Z-AAT Observed at Day 28 in 60 mg Cohort (n=3) Fourth Cohort Evaluating 75 mg of BEAM-302 Initiated, with Updated Data from Part A of the Phase 1/2 Trial Expected to be Presented at a Medical Conference in Second Half of 2025 CAMBRIDGE, Mass., April 05, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today presented additional data from the Phase 1/2 clinical trial of BEAM-302 in patients with alpha-1 antitrypsin deficiency (AATD) at the 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress, taking place April 4-5, 2025, in Lisbon, Portugal. Positive initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 were previously reported in March 2025, establishing clinical proof of concept as a potential treatment for AATD and in vivo base editing. Preliminary results from the first three single-ascending dose cohorts demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable dose-dependent correction of the disease-causing mutation and total AAT protein levels above the therapeutic threshold in the 60 mg dose cohort. These previously reported data were included in today's presentation, alongside updated biomarker data from the 60 mg cohort showing levels of corrected protein (M-AAT) and the mutant form of alpha-1 antitrypsin protein (Z-AAT) out to Day 28 for all three patients. At Day 28, the proportion of corrected M-AAT reached a mean of 91% of total AAT in circulation, surpassing levels observed in patients with the MZ genotype where circulating M-AAT is typically ~80%. In addition, treatment with BEAM-302 led to a mean decrease of 79% of circulating mutant Z-AAT from baseline as of Day 28. 'Patients living with AATD can face serious complications, including early onset emphysema and liver disease, and there is a significant unmet need for more effective therapies that can treat the entire spectrum of disease manifestations,' said Amy Simon, M.D., chief medical officer of Beam. 'The totality of the data shared to date highlight the promising impact of our approach across multiple drivers of disease pathology, including dose-dependent correction of the disease-causing mutation, rapid elevation in the circulation of total AAT and corrected M-AAT that is functional, and significant reduction in circulating mutant Z-AAT. We are honored to share these findings with the AATD community and look forward to continuing to advance our Phase 1/2 study to bring this potentially transformative treatment to patients as quickly as possible.' Beam plans to continue the dose-escalation portion of Part A of the ongoing Phase 1/2 trial, including enrolling and dosing a fourth dose cohort of 75 mg, and expects to report further data at a medical conference in the second half of 2025. In addition, the company plans to dose the first patient in Part B, which will include AATD patients with mild to moderate liver disease, in the second half of 2025. Beam recently announced the clearance of its investigational drug application (IND) for BEAM-302 by the United States (U.S.) Food and Drug Administration (FDA), enabling the company to activate sites in the U.S. for its ongoing Phase 1/2 trial. About BEAM-302BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the PiZ mutation. Patients homozygous for this mutation (PiZZ) represent the majority of patients living with severe AATD disease. A one-time A-to-G correction of the PiZ mutation with Beam's adenine base editor has the potential to simultaneously reduce the aggregation of mutant, misfolded AAT protein that causes toxicity to the liver (Z-AAT), generate therapeutic levels of corrected protein (M-AAT), and increase total and functional AAT in circulation, thereby addressing the underlying pathophysiology of both the liver and lung disease. In addition, the reduction in circulating PiZ aggregates (i.e., polymers) has the potential to further minimize lung inflammation and dysfunction. Importantly, because the native AAT gene would be corrected in its normal genetic location, AAT levels are anticipated to increase physiologically in response to inflammation or infection. This is a critical aspect of AAT's normal function to regulate the body's inflammatory response, which does not occur with currently approved protein replacement therapies. Correction of the PiZ mutation is expected to be durable based on preclinical and clinical evidence. About Alpha-1 Antitrypsin Deficiency (AATD)AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease. The most severe form of AATD arises when a patient has a point mutation in both copies of the SERPINA1 gene at amino acid 342 position (E342K, also known as the PiZ mutation or the 'Z' allele). This point mutation causes alpha-1 antitrypsin, or AAT, to misfold, accumulating inside liver cells rather than being secreted, resulting in very low levels (10%-15%) of circulating AAT. In addition to resulting in lower levels, the PiZ AAT protein variant is also less enzymatically effective compared to wildtype AAT protein. As a consequence, the lung is left unprotected from neutrophil elastase, resulting in progressive, destructive changes in the lung, such as emphysema, which can result in the need for lung transplants. The mutant AAT protein also accumulates in the liver, causing liver inflammation and cirrhosis, which can ultimately cause liver failure or cancer requiring patients to undergo a liver transplant. It is estimated that approximately 100,000 individuals in the U.S. have two copies of the Z allele, known as the PiZZ genotype, although only about 10% of all patients are thought to have been diagnosed. There are currently no curative treatments approved for patients with AATD, and the only approved therapy in the U.S., intravenous AAT protein replacement, has not been shown to prevent ongoing lung function decline and destruction in patients. About Beam TherapeuticsBeam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the therapeutic applications and potential of our technology, including our potential to develop life-long, curative, precision genetic medicines for patients through base editing; our plans, and anticipated timing, to advance our BEAM-302 program; and the clinical trial designs and expectations for BEAM-302. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to advance human clinical trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates or the delivery modalities we rely on to administer them may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the headings 'Risk Factors Summary' and 'Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law. Contacts: Investors:Holly ManningBeam Therapeuticshmanning@ Media:Josie Butler1ABjosie@ in to access your portfolio

Is Beam Therapeutics Inc. (BEAM) the Best Gene-Editing Stock to Buy?
Is Beam Therapeutics Inc. (BEAM) the Best Gene-Editing Stock to Buy?

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time01-04-2025

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Is Beam Therapeutics Inc. (BEAM) the Best Gene-Editing Stock to Buy?

We recently published a list of . In this article, we are going to take a look at where Beam Therapeutics Inc. (NASDAQ:BEAM) stands against other best gene-editing stocks to buy. Gene editing is an advanced medical technique within gene therapy that involves precisely modifying an individual's DNA to treat or prevent diseases. This approach directly alters genetic material to correct mutations, enhance cellular functions, or eliminate disease-causing genes. Industry experts believe that gene editing has the potential to revolutionize the treatment of genetic disorders, cancers, and various other conditions. According to IMARC, the U.S. healthcare infrastructure has adapted to support gene-editing therapies. IQVIA reported that 114 gene therapy trials were initiated in 2023, with approximately 77% sponsored by the healthcare industry. Kella Kapnisi, Head of Cell and Gene Therapy at Team Consulting, noted that the FDA has approved 38 cell and gene therapies, many of which have reached commercialization through predominantly manual laboratory manufacturing processes. The U.S. gene editing market has experienced significant growth, valued at $3.19 billion in 2024, while genome editing stood at $3.55 billion in 2023. Looking ahead, projections indicate a substantial surge, with gene editing expected to reach $13.99 billion and genome editing forecasted at $16.49 billion by 2034. These growth trends reflect compound annual growth rates (CAGRs) of 15.93% and 16.6%, respectively, underscoring the increasing adoption and investment in gene-editing technologies. Several breakthrough therapies are paving the way for advancements in gene editing. Precision BioSciences' PBGENE-HBV represents the first FDA-cleared in vivo gene-editing trial for chronic hepatitis B. Early data from the trial indicate a 70% reduction in the hepatitis B surface antigen (HBsAg) in two out of three patients at the lowest dose (0.2 mg/kg), targeting covalently closed circular DNA (cccDNA) to address the root cause of HBV persistence. YolTech Therapeutics has also made strides with its hyperoxaluria treatment, demonstrating a 70% reduction in harmful oxalate levels in patients with primary hyperoxaluria type 1 through lipid nanoparticle-delivered gene editing. Additionally, AccurEdit's cholesterol therapy has shown promising results, achieving a 70% reduction in LDL cholesterol with a single-dose treatment by silencing PCSK9. The number of patients receiving gene therapies is expected to fluctuate over the next decade. In 2020, 16,244 patients were treated, with this figure projected to rise to 94,696 by 2025 before gradually declining to 65,612 by 2034 as existing patient stocks deplete. Over the next decade, an estimated 1.09 million patients could benefit from gene therapies, with cancer patients making up approximately 48% of recipients. From a financial and economic standpoint, annual spending on gene editing therapies is anticipated to peak at $25.3 billion in 2026 before stabilizing at $21.0 billion by 2034. These therapies are projected to yield an additional 5.12 quality-adjusted life years (QALYs) per patient, with each QALY valued at $43,110. Insurance coverage is expected to play a crucial role in facilitating access, with Medicare, Medicaid, and private insurers projected to cover $8.1 billion, $5.44 billion, and $12.2 billion annually, respectively. Despite these advancements, the gene-editing industry faces key challenges. Delivery risks remain a concern, as adverse events associated with viral vectors and conditioning regimens pose safety hurdles. Regulatory scrutiny is also increasing, with the FDA's pending decision on Abeona Therapeutics' recessive dystrophic epidermolysis bullosa (RDEB) therapy, expected by April 29, 2025, highlighting the evolving standards for gene-editing approvals. For this list, we scanned Insider Monkey's database of over 1,000 hedge funds as of Q4 2024 and selected companies involved in the development and commercialization of gene-editing technologies. From there, we picked eight companies with the highest number of hedge fund investors and ranked them in ascending order. Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here). A patient undergoing MRI scanning to effectively diagnose Liver Diseases. Number of Hedge Fund Holders: 35 Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology company focused on precision genetic medicines using base editing, a technology that allows for precise, single-letter changes to DNA without cutting the strands. The company aims to develop treatments for genetic disorders, hematological diseases, and cancers. Its unique base editing technology offers a more accurate, predictable approach compared to traditional CRISPR, potentially reducing risks associated with DNA breaks. Beam Therapeutics Inc. (NASDAQ:BEAM) recently reached a key milestone by achieving its adult enrollment target in the BEACON Phase 1/2 clinical trial of BEAM-101 for sickle cell disease. The company also presented promising early results from this trial at the 66th American Society of Hematology Annual Meeting. Additionally, the corporation plans to release initial data from its Phase 1/2 study of BEAM-302 for Alpha-1 Antitrypsin Deficiency (AATD) in the first half of 2025, solidifying its position among the best gene editing stocks in the biotech sector. Beam Therapeutics Inc. (NASDAQ:BEAM) ended 2024 with a solid cash position of $850.7 million, supporting ongoing research and trials. R&D expenses were $101.4 million for the fourth quarter, reflecting a decrease from the previous year, which may indicate greater efficiency in its operations. General and administrative expenses were $28.7 million for Q4. However, the business reported a net loss of $90.4 million for the quarter and $376.7 million for the year, which is typical for biotech companies in the clinical stage. The company's revenue from licenses and collaborations significantly dropped from $316.2 million in Q4 2023 to $30.1 million in Q4 2024. This decrease warrants further investigation, as it may affect the company's future financial stability. Despite this, Beam Therapeutics Inc. (NASDAQ:BEAM) expects its current cash reserves will fund operations into 2027, giving the company ample time to advance its key programs. Overall, BEAM ranks 4th on our list of best gene-editing stocks to buy. While we acknowledge the potential of BEAM, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than BEAM but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock. READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio

Is Beam Therapeutics Inc. (BEAM) the Best Growth Stock to Invest in for the Next 10 Years?
Is Beam Therapeutics Inc. (BEAM) the Best Growth Stock to Invest in for the Next 10 Years?

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time16-03-2025

  • Business
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Is Beam Therapeutics Inc. (BEAM) the Best Growth Stock to Invest in for the Next 10 Years?

We recently published a list of . In this article, we are going to take a look at where Beam Therapeutics Inc. (NASDAQ:BEAM) stands against other best growth stocks to invest in for the next 10 years. On March 8, Ben Snider, Goldman Sachs senior equity strategist, joined CNBC to discuss the state of the economy today. Snider noted that the key struggle has been the market struggle to assess the forward trajectory of the economy. The market entered this year with optimism for the growth trajectory. However, over the past few weeks, we have seen a very sharp downturn. There is good news to extract from the scenario. Snider explained that if we look at where the market is priced today, it seems much more reasonable. Therefore the base case remains the same the economy is in good shape and is growing. He also noted that a confirmation of the growth came in with the jobs report, moreover, earnings are still growing, meaning that the equity market should be moving higher too. However, the key question that remains unanswered is what sectors investors should look forward to. Most of the sectors have been volatile. Therefore, as per Snider, the best approach would be not to go all in for a single sector. He likes healthcare, which has been one of the best-performing sectors in the market so far. This is because despite the performance the sector is still trading at the lowest valuations compared to the market. Snider mentioned that this may be a good buying period as historical data tells us that when an investor buys the S&P 500 down 5%, it generates a positive return on investment over the next 5 months 85% of the time. Moreover, Goldman Sachs has anticipated moderate yet resilient global growth in 2025, driven by the strong performance of the United States. The firm expects the US economy to be a primary driver of global growth, supported by a robust labor market, consistent consumer spending, solid credit conditions, sufficient liquidity, and increased capital spending related to AI. In addition, regarding the tariff situation, Goldman Sachs believes that the 'Fed Put' will come into play and ease monetary policy, however, terminal rates are expected to be higher than previously anticipated. To curate the list of the 10 best growth stocks to invest in for the next 10 years, we looked at various online rankings. Using these rankings we aggregated a list of best growth stocks for the next 10 years. Next, we checked the sales growth of each stock from Seeking Alpha and added only those companies that have grown more than 15% over the past 5 years. Lastly, we ranked the stocks in ascending order of the number of hedge fund holders, sourced from Insider Monkey's Q4 2024 database. Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (). A patient undergoing MRI scanning to effectively diagnose Liver Diseases. Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology company that focuses on creating precision genetic medicines using base editing technologies for genetic disorders. Its base editing technology is designed to make precise and efficient single-base changes at targeted genomic sequences, without making double-stranded breaks in the DNA. Its pipeline products include BEAM-101, BEAM-201, BEAM-301, and BEAM-302, which are used for serious diseases. On March 10, Beam Therapeutics Inc. (NASDAQ:BEAM) was upgraded to Buy from Hold Jones Research analyst Soumit Roy. The analyst has set a price target of $34. Roy noted that the company announced encouraging preliminary data from its Phase 1/2 trial of BEAM-302 for alpha-1 antitrypsin deficiency (AATD). BEAM-302 is a medication that uses a liver-targeting lipid nanoparticle to deliver a guide RNA and an mRNA that encodes a base editor, which is designed to correct the PiZ mutation that causes AATD. The firm has increased the probability of success against AATD to 35% from 15% and also increased its adjusted peak sales to $1.9 billion in 2036, noting that BEAM-302 could be a one-time treatment for AATD. Jones Research believes that the data de-risks the company's platform on multiple levels. Beam Therapeutics Inc. (NASDAQ:BEAM) is one of the best growth stocks to invest in for the next 10 years. Overall, BEAM ranks 9th on our list of best growth stocks to invest in for the next 10 years. While we acknowledge the potential of BEAM as an investment, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than BEAM but that trades at less than 5 times its earnings, check out our report about the . READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires Disclosure: None. This article is originally published at Insider Monkey.

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