Latest news with #BEAM-302
Yahoo
2 days ago
- Business
- Yahoo
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
Yahoo
3 days ago
- Business
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Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
CAMBRIDGE, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). AATD is an inherited genetic disorder that affects the lungs and/or liver, leading to early onset emphysema and liver disease, and for which there is significant unmet need for effective therapies that can treat the entire spectrum of disease. 'Receiving orphan drug designation for BEAM-302 is an important milestone in our efforts to bring a transformative therapy to people living with AATD, many of whom currently lack effective long-term treatment options,' said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. 'This recognition by the FDA, following the receipt of RMAT designation from the FDA just weeks ago, highlights the urgency of addressing this serious genetic disease and the potential of BEAM-302 to directly correct the DNA mutation, the underlying root cause of this illness. We are encouraged by the FDA's continued support of this program and are committed to its advancement with the goal of delivering a one-time, potentially curative treatment to patients as quickly and safely as possible.' The FDA's orphan drug designation is designed to support the development and evaluation of treatments for rare diseases affecting fewer than 200,000 people in the U.S. The designation comes with potential benefits for the sponsor company, including tax credits for qualified clinical trials, exemption from user fees, and a potential seven years of market exclusivity after approval. Positive initial safety and efficacy data from the ongoing Phase 1/2 trial of BEAM-302, previously reported in March, established 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 in Part A of the study 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. Beam has initiated dosing in the fourth cohort of Part A, evaluating 75 mg of BEAM-302, and expects to report updated data at a medical conference in the second half of 2025. Additionally, 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 previously announced the clearance of the U.S. investigational new drug (IND) application for BEAM-302 for the treatment of AATD in March 2025, as well as the granting of Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA to BEAM-302 in May 2025. 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 with respect to AATD; our plans, and anticipated timing, to advance our BEAM-302 program, including the clinical trial designs and expectations for BEAM-302; our plans to present data at upcoming medical conferences; expectations regarding potential benefits of the orphan drug designation for BEAM-302; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. 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; our ability to recognize the potential benefits conferred by the orphan drug designation for BEAM-302; 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, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, 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
Yahoo
05-04-2025
- Business
- Yahoo
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
Yahoo
31-03-2025
- Business
- Yahoo
BEAM's IND for BEAM-302 in Genetic Disorder Study Gets FDA Clearance
Beam Therapeutics Inc. BEAM announced that the FDA has cleared the investigational new drug (IND) application to begin clinical studies on its pipeline candidate, BEAM-302, for the treatment of alpha-1 antitrypsin deficiency (AATD) in the United States. BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the disease-causing PiZ mutation. AATD is an inherited genetic disorder that can cause early onset of emphysema and liver disease. Currently, there are no approved curative treatments for the given indication. In June 2024, Beam Therapeutics dosed the first patient in a phase I/II study on BEAM-302 for the treatment of AATD in the United Kingdom. The candidate has also received clinical trial authorization from New Zealand, Australia, the Netherlands and Ireland. Year to date, shares of Beam Therapeutics have declined 10.6% against the industry's increase of 3.3%. Image Source: Zacks Investment Research Earlier this month, Beam Therapeutics announced positive initial safety and efficacy data from the phase I/II study evaluating BEAM-302 for treating patients with AATD. The initial safety finding suggests that treatment with BEAM-302 was well tolerated across all dose levels, leading to durable dose-dependent correction of the disease-causing mutation. Data from the study showed that treatment with a single dose of BEAM-302 demonstrated durable, dose-dependent increases in total and functional alpha-1 antitrypsin (AAT). Also, new production of corrected protein (M-AAT) and decreases in toxic mutant Z-AAT were observed in circulation across the initial three doses. The phase I/II study consists of two parts. Part A of the study will investigate BEAM-302 in AATD patients with lung disease, while part B of the study will investigate the candidate in AATD patients with mild-to-moderate liver disease with or without lung disease. The company plans to dose the first patient in part B of the phase I/II study in the second half of 2025. Beam Therapeutics expects to enroll additional patients in a fourth-dose cohort in the ongoing phase I/II study, with data from the same expected in the second half of 2025. Beam Therapeutics currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Jazz Pharmaceuticals, Inc JAZZ, Dynavax Technologies Corporation DVAX and Krystal Biotech, Inc. KRYS, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. In the past 60 days, estimates for Jazz Pharmaceuticals' earnings per share have increased from $22.06 to $23.33 for 2025. During the same time, earnings per share estimates for 2026 have increased from $23.13 to $23.35. Year to date, shares of JAZZ have risen 2.6%. JAZZ's earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 3.20%. In the past 60 days, estimates for Dynavax's earnings per share have increased from 32 cents to 33 cents for 2025. During the same time, earnings per share have increased from 49 cents to 57 cents for 2026. Year to date, shares of DVAX have risen 7.4%. DVAX's earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 9.58%. In the past 60 days, estimates for Krystal Biotech's earnings per share have increased from $5.50 to $7.06 for 2025. During the same time, earnings per share estimates for 2026 have increased from $9.15 to $11. Year to date, shares of KRYS have rallied 16.7%. KRYS' earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 3.29%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Dynavax Technologies Corporation (DVAX) : Free Stock Analysis Report Jazz Pharmaceuticals PLC (JAZZ) : Free Stock Analysis Report Beam Therapeutics Inc. (BEAM) : Free Stock Analysis Report Krystal Biotech, Inc. (KRYS) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research
Yahoo
10-03-2025
- Health
- Yahoo
Beam base editing therapy gets ‘proof of concept' in rare lung disease
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Preliminary results from a small clinical trial suggest that a cutting-edge genetic medicine developed by Beam Therapeutics can repair the damaged DNA that gives rise to a rare liver and lung disorder. The data, from the first nine patients treated in the trial, are an important proof point for Beam, which specializes in a form of CRISPR gene editing that can precisely rewrite misspelled DNA sequences by changing individual nucleotides, or 'letters.' Although the biotechnology company has previously released data for other experimental editing medicines, Monday's findings are the first from a therapy designed to directly correct a disease-causing genetic mutation. In this case, Beam aims to treat a genetic condition called alpha-1 antitrypsin deficiency, or AATD, by fixing the DNA misspelling at the disease's root. Delivered into the liver by tiny globules of fat, the base editing machinery of Beam's medicine swaps an 'A' for a 'G' in the SERPINA1 gene variant linked to the most severe form of AATD. While only a first look at the medicine's potential, the results Beam disclosed Monday suggest treatment is working as intended, without causing any alarming side effects that could derail testing. Still, in a sign of the difficulties currently facing developers of genetic medicine, shares in Beam fell by nearly 15% in Monday morning trading. AATD's damage to the liver and lungs is tied to a misfolded 'AAT' protein that's produced by the mutant SERPINA1 gene. Normally, this protein is secreted by liver cells and travels to the lungs, where it protects healthy tissue from an enzyme released by white blood cells to fight infections. When it's misfolded, however, the protein mostly accumulates inside liver cells, eventually causing inflammation and cirrhosis. And left unprotected, the lungs are vulnerable to attack by the enzyme, called neutrophil elastase. Beam's medicine, dubbed BEAM-302, is meant to address both consequences of AATD. In theory, correction of the SERPINA1 mutation should restore production of correctly folded AAT protein, thereby easing the stress on the liver and rebuilding the lung's defenses against neutrophil elastase. The data released Monday give reason to believe in that promise. All nine of the treated patients had lung disease associated with AATD, and received intravenously one of three ascending doses of BEAM-302. One month after treatment, researchers running the trial measured increases in total AAT protein that were between 1.6 and 2.8 times baseline levels. This occurred alongside reductions in circulating misfolded protein, indicating the higher levels were the result of properly produced AAT protein. In one patient treated with the highest of the three doses, circulating levels of misfolded protein were 78% lower than baseline after one month. And in the three patients on that dose, total AAT protein levels reached an average of 12.4 micromolars — above a threshold that's considered protective as it is seen in people with a 'carrier' AATD genotype. 'We believe BEAM-302 has the potential to be a transformative therapy that could treat the entire spectrum of disease manifestations in severely deficient AATD patients,' said Beam CEO John Evans in a Monday statement. Importantly, wrote Jefferies analyst Michael Yee in a client note, BEAM-302's 'safety looks clean." Adverse events are always a particular focus in early testing of new kinds of medicine, but are especially so in genetic medicine, where unwanted side effects have hampered studies of other therapies. Beam plans to continue with testing higher doses of BEAM-302 in more patients, as well as to open a second phase of its study which will enroll AAT patients with mild-to-moderate liver disease. The company also expects to report additional study data at a medical conference later this year. Alongside the data, Beam announced the pricing of a stock sale that it anticipates will raise $500 million in gross proceeds. The biotech had $850 million in cash, cash equivalents and short-term securities at the end of last year, which it said last month will last into 2027. The new funds should extend that runway to 2028, according to analysts at Leerink Partners. Beam has plenty of company in targeting AATD. Several companies, Wave Life Sciences among them, are working on an "RNA editing" approach to treatment, while Arrowhead Pharmaceuticals and Takeda have been studying a gene-silencing method known as RNA interference. However, one of Beam's gene editing competitors, Intellia Therapeutics, recently said it would stop work on its AATD therapy.
