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Beam Therapeutics (NASDAQ:BEAM) Azioni

BEAM THERAPEUTICS INC

Beam Inc.

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Beam Therapeutics

Beam Therapeutics Inc is a biotechnology company committed to creating a new class of precision genetic medicines based on its proprietary base editing technology, with a vision of providing lifelong cures to patients suffering from serious diseases. The company is focused on its programs in the hematology and genetic disease portfolios, which comprise different product candidates in its pipeline, such as BEAM-101, which is being developed to target Sickle Cell Disease, BEAM-302, a liver-targeting lipid nanoparticle, being developed to target Alpha-1 antitrypsin deficiency, and other drug candidates including BEAM-302, BEAM-103 & 104. The company views its operations and manages its business in one operating segment, operating exclusively in the United States.

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Data from seven patients treated with BEAM-101 in the BEACON clinical trial were <a href="https://www.globenewswire.com/Tracker?data=glGSdPuwK-g4D1VTsCKAqB9lw-d9W66VifNMzt0xUaFQ1A0o8RqjCvzcT65iVV5E2XRg0iiUik6E8RGL4jhEVB5xo6MXZlh4dL57hmBkWUdkcHVNFjo1vNnn3gex1CSZKDQFRvo_ODrAQtvmsb_dHBwBKH8aGEHJ8FN4KKgqHoDtn3RZ2-BPnOQM1kJ35QakXdocjw1lKNdUgdqXcAW3Wz5NU1WURIq-q_E3B-byI6o=">presented</a> at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2024. Updated clinical data have been accepted for presentation at the European Hematology Association (EHA) 2025 Congress in June. Treatment with BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis. The initial safety profile of BEAM-101 was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation. Manufactured in Beam's North Carolina facility, BEAM-101 uses an advanced, largely automated process that has demonstrated consistently high yields and viability, enabling successful BEAM-101 manufacturing for patients in the BEACON clinical trial. Beam expects to dose 30 patients in the ongoing BEACON trial by mid-2025.

Biotech

Health Care

General

The FDA Has Granted Orphan Drug Designation To Beam Therapeutics' Beam-101, An Investigational Genetically Modified Cell Therapy For Sickle Cell Disease

<a href="https://www.globenewswire.com/Tracker?data=LT9sHMTBcs6tp-cNpqCy8g7zBSfm4Ct_Nw7-wepexJp9XdQllzvstoCO-A6ArQNFeT-ypBJNKrXijUPVDzx7LBTzMn365ByjYH_-k94ITWk=">Beam Therapeutics Inc.</a> (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>), 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.

Beam Therapeutics Receives FDA Orphan Drug Designation For BEAM-302, A Base Editing Therapy Targeting Alpha-1 Antitrypsin Deficiency

Presentation to Include Updated Data from 17 Sickle Cell Disease Patients in the Ongoing BEACON Phase 1/2 Clinical Trial Evaluating Safety and Efficacy of BEAM-101

Events

Beam Therapeutics To Present New Data From BEAM-101 Program iI Sickle Cell Disease At European Hematology Association 2025 Congress

<a href="https://www.globenewswire.com/Tracker?data=AgroyJiAoVwZWpbl3I_g5vXwkNryrBhcKWKtKMfwr5SJGrEArF-F94LVj29UByPwOitbzBikvRMaG94-AUB-kjWcMKBb10qs8XHJ_NGO85I=">Beam Therapeutics Inc.</a> (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>), 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 Regenerative Medicine Advanced Therapy (RMAT) 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.
"Just weeks after the clearance of our U.S. investigational new drug application, we are pleased to report that the FDA has now granted RMAT designation to BEAM-302, recognizing its potential as a transformative, one-time treatment for patients living with AATD," said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. "This designation underscores the strength of our clinical data to date and the promise of base editing to directly correct the genetic mutation that is the root cause of most cases of severe AATD. With RMAT designation enabling closer and more frequent collaboration, we look forward to working with the FDA to accelerate the development of BEAM-302 and bring this potentially curative therapy to AATD patients as safely and swiftly as possible."
The FDA's RMAT designation is designed to support the development and evaluation of regenerative medicines, including genetic therapies, with the intention of addressing serious or life-threatening diseases that have unmet medical needs. RMAT designation provides opportunities for early interactions with the FDA to discuss potential surrogate or intermediate endpoints to support accelerated approval, organizational commitment from senior staff at the agency, opportunities to participate in novel review and development programs, and the potential for a rolling review and priority review of a product's future biologics license application.
Positive initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 were previously <a href="https://www.globenewswire.com/Tracker?data=DA7vCfMfeWFXeIxzmWuCQbq57-hOQDq3lYcM01eYzAB2DDZYKNsjZwr_iq5_TDmR93QBOR24G6ICtFMNBz4iR0AAgLewn-IQvVD3cuv0TM1xUJyrh4OIAmqse6MMY42kX6v-_EFDrgneDyjxRqX56dJMQ7rYyGNbYI9Q0FsN60LtxsUV4SKfM6WCVgfyqPQg1icVyi4ZHOANTZ4Tk3sS5w6sgMl7aBvX-wWLvbIdaeg=">reported</a> in March, 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 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 as well. Beam previously <a href="https://www.globenewswire.com/Tracker?data=qIH9yhF1SSXMfkVvvTYJsHJzgR09c_h4Fzs6J2NhuVvsfsO-MFzw8sKbiBy1aKE9_hIU_LzaNZHs8IDLS6VKRt5dosCJbNgPC64GjaLtiLtUorZWh-uZOh0eY1lQscRRLzzsg_bQAYNSna04P_oxjXcmzQVsk-bNbg9W444LLcc3CfXmp7C9D-vR1A3in00XgQJd-oLO2tZPsad8TI3rigL5twRVo72RY3aPtfB7H0o=">announced</a> the clearance of the U.S. investigational new drug (IND) application for BEAM-302 for the treatment of AATD in March 2025. &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;
About BEAM-302 BEAM-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.

Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy Designation Has Been Granted To BEAM-302 For The Treatment Of Alpha-1 Antitrypsin Deficiency

Guggenheim analyst Debjit Chattopadhyay maintains Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) with a Buy and lowers the price target from $78 to $55.

Price Target

Analyst Ratings

Guggenheim Maintains Buy on Beam Therapeutics, Lowers Price Target to $55

Wells Fargo analyst Yanan Zhu maintains Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) with a Overweight and lowers the price target from $75 to $70.

Wells Fargo Maintains Overweight on Beam Therapeutics, Lowers Price Target to $70

Analysts' ratings for Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) over the last quarter vary from bullish to bearish, as provided by 10 analysts.
In the table below, you'll find a summary of their recent ratings, revealing the shifting sentiments over the past 30 days and comparing them to the previous months.
<table>
<thead>
<tr>
<th></th>
<th align="right">Bullish</th>
<th align="right">Somewhat Bullish</th>
<th align="right">Indifferent</th>
<th align="right">Somewhat Bearish</th>
<th align="right">Bearish</th>
</tr>
</thead>
<tbody>
<tr>
<td>Total Ratings</td>
<td align="right">6</td>
<td align="right">1</td>
<td align="right">3</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>1M Ago</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>2M Ago</td>
<td align="right">3</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>3M Ago</td>
<td align="right">2</td>
<td align="right">0</td>
<td align="right">2</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
</tbody>
</table>
Providing deeper insights, analysts have established 12-month price targets, indicating an average target of $51.0, along with a high estimate of $80.00 and a low estimate of $25.00. A 7.07% drop is evident in the current average compared to the previous average price target of $54.88. 
<img src="https://www.benzinga.com/files/images/story/2025/1746622841_0.png" alt="price target chart" />
<h2>Diving into Analyst Ratings: An In-Depth Exploration</h2>
The standing of Beam Therapeutics among financial experts is revealed through an in-depth exploration of recent analyst actions. The summary below outlines key analysts, their recent evaluations, and adjustments to ratings and price targets.
<table>
<thead>
<tr>
<th>Analyst</th>
<th>Analyst Firm</th>
<th>Action Taken</th>
<th>Rating</th>
<th>Current Price Target</th>
<th>Prior Price Target</th>
</tr>
</thead>
<tbody>
<tr>
<td>Gena Wang</td>
<td>Barclays</td>
<td>Lowers</td>
<td>Equal-Weight</td>
<td>$25.00</td>
<td>$31.00</td>
</tr>
<tr>
<td>Patrick Trucchio</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$80.00</td>
<td>$80.00</td>
</tr>
<tr>
<td>Alec Stranahan</td>
<td>B of A Securities</td>
<td>Maintains</td>
<td>Buy</td>
<td>$42.00</td>
<td>$42.00</td>
</tr>
<tr>
<td>Greg Harrison</td>
<td>Scotiabank</td>
<td>Announces</td>
<td>Sector Outperform</td>
<td>$40.00</td>
<td>-</td>
</tr>
<tr>
<td>Soumit Roy</td>
<td>Jones Trading</td>
<td>Announces</td>
<td>Buy</td>
<td>$34.00</td>
<td>-</td>
</tr>
<tr>
<td>Patrick Trucchio</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$80.00</td>
<td>$80.00</td>
</tr>
<tr>
<td>Greg Harrison</td>
<td>Scotiabank</td>
<td>Raises</td>
<td>Sector Perform</td>
<td>$25.00</td>
<td>$24.00</td>
</tr>
<tr>
<td>Patrick Trucchio</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$80.00</td>
<td>$80.00</td>
</tr>
<tr>
<td>Debjit Chattopadhyay</td>
<td>Guggenheim</td>
<td>Maintains</td>
<td>Buy</td>
<td>$78.00</td>
<td>$78.00</td>
</tr>
<tr>
<td>Luca Issi</td>
<td>RBC Capital</td>
<td>Raises</td>
<td>Sector Perform</td>
<td>$26.00</td>
<td>$24.00</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: Analysts frequently update their recommendations based on evolving market conditions and company performance. Whether they 'Maintain', 'Raise' or 'Lower' their stance, it reflects their reaction to recent developments related to Beam Therapeutics. This information provides a snapshot of how analysts perceive the current state of the company.</li>
<li>Rating: Delving into assessments, analysts assign qualitative values, from 'Outperform' to 'Underperform'. These ratings communicate expectations for the relative performance of Beam Therapeutics compared to the broader market.</li>
<li>Price Targets: Analysts provide insights into price targets, offering estimates for the future value of Beam Therapeutics's stock. This comparison reveals trends in analysts' expectations over time.</li>
</ul>
For valuable insights into Beam Therapeutics's market performance, consider these analyst evaluations alongside crucial financial indicators. Stay well-informed and make prudent decisions using our Ratings Table.
<a href="https://www.benzinga.com/quote/BEAM/analyst-ratings?adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Stay up to date on Beam Therapeutics analyst ratings.</a>
If you are interested in following small-cap stock news and performance <a href="https://www.benzinga.com/news/small-cap?adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">you can start by tracking it here</a>.
<h2>Unveiling the Story Behind Beam Therapeutics</h2>
Beam Therapeutics Inc is a biotechnology company committed to creating a new class of precision genetic medicines based on its proprietary base editing technology, with a vision of providing lifelong cures to patients suffering from serious diseases. The company is focused on its programs in the hematology and genetic disease portfolios, which comprise different product candidates in its pipeline, such as BEAM-101, which is being developed to target Sickle Cell Disease, BEAM-302, a liver-targeting lipid nanoparticle, being developed to target Alpha-1 antitrypsin deficiency, and other drug candidates including BEAM-302, BEAM-103 &amp; 104. The company views its operations and manages its business in one operating segment, operating exclusively in the United States.
<h3>A Deep Dive into Beam Therapeutics's Financials</h3>
Market Capitalization: Indicating a reduced size compared to industry averages, the company's market capitalization poses unique challenges.
Decline in Revenue: Over the 3M period, Beam Therapeutics faced challenges, resulting in a decline of approximately -90.49% in revenue growth as of 31 December, 2024. This signifies a reduction in the company's top-line earnings. As compared to competitors, the company encountered difficulties, with a growth rate lower than the average among peers in the Health Care sector.
Net Margin: Beam Therapeutics's net margin is impressive, surpassing industry averages. With a net margin of -300.51%, the company demonstrates strong profitability and effective cost management.
Return on Equity (ROE): Beam Therapeutics's financial strength is reflected in its exceptional ROE, which exceeds industry averages. With a remarkable ROE of -11.85%, the company showcases efficient use of equity capital and strong financial health.
Return on Assets (ROA): Beam Therapeutics's ROA is below industry averages, indicating potential challenges in efficiently utilizing assets. With an ROA of -7.94%, the company may face hurdles in achieving optimal financial returns.
Debt Management: Beam Therapeutics's debt-to-equity ratio is below the industry average at 0.22, reflecting a lower dependency on debt financing and a more conservative financial approach.
<h2>The Basics of Analyst Ratings</h2>
Within the domain of banking and financial systems, analysts specialize in reporting for specific stocks or defined sectors. Their work involves attending company conference calls and meetings, researching company financial statements, and communicating with insiders to publish "analyst ratings" for stocks. Analysts typically assess and rate each stock once per quarter.
Some analysts will also offer forecasts for metrics like growth estimates, earnings, and revenue to provide further guidance on stocks. Investors who use analyst ratings should note that this specialized advice comes from humans and may be subject to error.
Which Stocks Are Analysts Recommending Now?
Benzinga Edge gives you instant access to all major analyst upgrades, downgrades, and price targets. Sort by accuracy, upside potential, and more. <a href="https://www.benzinga.com/premium/ideas/benzinga-edge-checkout/?t=be8be9arja3be22bz21&amp;adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Click here to stay ahead of the market</a>.
This article was generated by Benzinga's automated content engine and reviewed by an editor.

10 Analysts Assess Beam Therapeutics: What You Need To Know

Barclays analyst Gena Wang maintains Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) with a Equal-Weight and lowers the price target from $31 to $25.

Barclays Maintains Equal-Weight on Beam Therapeutics, Lowers Price Target to $25

WIIM

Beam Therapeutics shares are trading lower following a Q1 EPS and revenue miss.

Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) reported quarterly losses of $(1.24) per share which missed the analyst consensus estimate of $(1.21) by 2.48 percent. This is a 2.48 percent decrease over losses of $(1.21) per share from the same period last year. The company reported quarterly sales of $7.47 million which missed the analyst consensus estimate of $14.87 million by 49.75 percent. This is a 0.81 percent increase over sales of $7.41 million the same period last year.

Earnings

Earnings Misses

Beam Therapeutics Q1 GAAP EPS $(1.24) Misses $(1.21) Estimate, Sales $7.47M Miss $14.87M Estimate

U.S. stocks were lower, with the Dow Jones index falling around 700 points on Monday.
Shares of Wolverine World Wide, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/WWW#NYSE">WWW</a>) rose sharply during <a href="https://www.benzinga.com/quote/WWW/analyst-ratings">Monday's session</a>.
Baird analyst Jonathan Komp upgraded the rating for Wolverine World Wide from Neutral to Outperform.
Wolverine World Wide shares jumped 7.2% to $11.56 on Monday.
Here are some other big stocks recording gains in today's session.
<ul>
<li>Verve Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/VERV#NASDAQ">VERV</a>) jumped 15% to $5.28. Cantor Fitzgerald analyst Rick Bienkowski, on April 15, upgraded Verve Therapeutics from Neutral to Overweight.</li>
<li>Waton Financial Limited (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/WTF#NASDAQ">WTF</a>) gained 14.8% to $7.43.</li>
<li>uniQure N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/QURE#NASDAQ">QURE</a>) shares rose 12.4% to $14.61. uniQure was recently granted FDA granted Breakthrough Therapy designation for AMT-130 for the treatment of Huntington&#8217;s disease.</li>
<li>Intellia Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NTLA#NASDAQ">NTLA</a>) gained 12% to $8.10.</li>
<li>Rocket Pharmaceuticals, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RCKT#NASDAQ">RCKT</a>) climbed 11.4% to $7.05.</li>
<li>Erasca, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ERAS#NASDAQ">ERAS</a>) climbed 11.2% to $1.2900.</li>
<li>Microvast Holdings, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/MVST#NASDAQ">MVST</a>) rose 11% to $2.0324.</li>
<li>Beam Therapeutics Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) gained 10.6% to $18.81.</li>
<li>Replimune Group, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/REPL#NASDAQ">REPL</a>) shares gained 9.6% to $8.76.</li>
<li>Pacira BioSciences, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PCRX#NASDAQ">PCRX</a>) shares rose 6.3% to $26.21 as the company announced a $300 million share repurchase program.</li>
<li>CleanSpark, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CLSK#NASDAQ">CLSK</a>) gained 5.3% to $7.90.</li>
<li>B2Gold Corp. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/BTG#NYSE">BTG</a>) gained 4.8% to $3.4800.</li>
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Wolverine World Wide, Pacira BioSciences And Other Big Stocks Moving Higher On Monday

Beam Therapeutics Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) on Saturday 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 <a target="_blank" href="https://www.benzinga.com/general/biotech" rel="noreferrer noopener">10th Patient Congress</a>.
AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease.
Also Read: <a target="_blank" href="https://www.benzinga.com/25/03/44218611/gene-edit-biotech-beam-therapeutics-touts-encouraging-data-from-early-stage-gene-therapy-study-in-genetic-disorder-raises-500-million-via-equity" rel="noreferrer noopener">Gene Edit Biotech Beam Therapeutics Touts Encouraging Data From Early-Stage Gene Therapy Study In Genetic Disorder, Raises $500 Million Via Equity</a>
Initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 were previously&nbsp;<a target="_blank" href="https://www.globenewswire.com/Tracker?data=DGjvxT5xtICCkQ8gwO3vYKCxk4cQeGEVsiyMQrfGuDK85hfJvOf0_YRSCN3D4S2uPdzWIKOzfsxza85TGOOOmoIkpgc1qwWR9HDfBTbthTKhQDPwQrYggdlqLEDXs4WjgwVKQ_WJgjueP5aZJLXesgIntByaC3vCddeZGNP6hOg60nlrLmP-9YVZC3BBCaMhoF_Q_HvldXOxFXwKhnaNyTK85Sj9OFVYNjcCdMA9MHs=&amp;tblci=GiCUX7wvU_AgmIIiQduNYr6JQuJQLy6SZJGvpyeZuCzyqiCN1VcoxvTN4f7-y-PqATDviEo" rel="noreferrer noopener">reported</a> in March 2025, establishing clinical proof of concept as a potential treatment for AATD and&nbsp;in vivo base editing.
Preliminary results from the first three single-ascending dose cohorts demonstrated that BEAM-302 was well tolerated.
Single doses of BEAM-302 led to durable dose-dependent correction of the disease-causing mutation and total AAT protein levels above the therapeutic threshold in the <a target="_blank" href="https://www.benzinga.com/pressreleases/25/04/g44656185/beam-therapeutics-presents-additional-data-for-beam-302-in-alpha-1-antitrypsin-deficiency-aatd-at-" rel="noreferrer noopener">60 mg dose cohort</a>.
These previously reported data were included in the 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.
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, including AATD patients with mild to moderate liver disease, in the second half of 2025.
The U.S. Food and Drug Administration recently cleared Beam Therapeutics&#8217; investigational drug application for BEAM-302, enabling the company to activate sites in the U.S. for its <a target="_blank" href="https://www.benzinga.com/pressreleases/25/03/g44523407/beam-therapeutics-announces-clearance-of-investigational-new-drug-application-for-beam-302-for-the" rel="noreferrer noopener">ongoing Phase 1/2 trial</a>.
Price Action: BEAM stock is down 6.75% at $14.24 at the last check on Monday.
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Beam Therapeutics Presents Biomarker Data For BEAM-302 Gene Therapy For Inherited Disease

HC Wainwright &amp; Co. analyst Patrick Trucchio reiterates Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) with a Buy and maintains $80 price target.

Reiteration

HC Wainwright & Co. Reiterates Buy on Beam Therapeutics, Maintains $80 Price Target

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

Reported Saturday, Beam Therapeutics Reports 91% Mean Correction Of Circulating AAT Protein At Day 28 In BEAM-302 Phase 1/2 Trial For AATD

On Friday, April 4, 2025, Cathie Wood-led Ark Invest made substantial trades involving several prominent companies including Coinbase Global Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/COIN#NASDAQ">COIN</a>), Amazon.com Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/AMZN#NASDAQ">AMZN</a>), CRISPR Therapeutics AG (NGM: CRSP), Beam Therapeutics Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>), UiPath Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/PATH#NYSE">PATH</a>), Roblox Corp. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/RBLX#NYSE">RBLX</a>), MercadoLibre Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/MELI#NASDAQ">MELI</a>), and Repare Therapeutics Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RPTX#NASDAQ">RPTX</a>).
The COIN Trade
Ark Invest purchased shares of Coinbase Global Inc across multiple ETFs. Ark Invest&#8217;s ARK Fintech Innovation ETF (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ARKF#NYSE">ARKF</a>) bought 12,187 shares, while the ARK Innovation ETF (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ARKK#NYSE">ARKK</a>) and ARK Next Generation Internet ETF (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ARKW#NYSE">ARKW</a>) funds added 54,988 and 15,982 shares, respectively.
The total value of the COIN shares bought, calculated from the last closing price of $160.55, amounts to approximately $13.5 million.
The AMZN Trade
Amazon.com Inc also saw significant buying action from Ark Invest. The ARKF, ARKK, and ARK Autonomous Technology &amp; Robotics ETF (BATS:<a class="ticker" href="https://www.benzinga.com/stock/ARKQ#BATS">ARKQ</a>) funds bought 9,018, 25,528, and 8,408 shares, respectively.
Given Amazon&#8217;s last closing price of $171.0, the total value of the shares bought is approximately $7.4 million.
The CRSP and BEAM Trades
The ARKK fund bought 69,011 shares of CRISPR Therapeutics AG and 142,713 shares of Beam Therapeutics Inc. Based on the last closing prices of $32.65 and $15.2,7, respectively, the total value of the CRSP and BEAM shares bought is approximately $3.5 million.
The PATH Trade
Ark Invest's ARKK sold 448,505 shares of UiPath, while ARKQ sold 147,575 shares. The ARK Next Generation Internet ETF (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ARKW#NYSE">ARKW</a>) fund sold 323,575 shares, and the ARKF fund sold 434,637 shares.
The RBLX, MELI, and RPTX Trades
Ark Invest sold shares of Roblox Corp. and MercadoLibre Inc. across various ETFs. Additionally, the ARKG fund sold 32,613 shares of Repare Therapeutics Inc, valued at approximately $32,613 based on the last closing price of $1.0.
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</ul>
Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.
Photo courtesy: Shutterstock

Equities

Tech

ETFs

AMZN

ARKF

ARKQ

ARKW

COIN

CRSP

MELI

PATH

RBLX

RPTX

Cathie Wood's Ark Invest Loads Up On Amazon Amid Over 20% Decline In A Year: Offloads UiPath, Roblox

The stock market took a dive, but <a href="https://www.benzinga.com/news/25/03/44132684/cathie-woods-bold-move-8-straight-days-of-buying-beam-therapeutics-as-stock-soars-14">Cathie Wood</a> didn&#8217;t flinch. Instead of retreating, the Ark Invest CEO saw an opportunity and went on a buying spree. According to<a href="https://www.investors.com/news/cathie-wood-ark-invest-coinbase-stock-robinhood-tesla-palantir-amd-coin-hood-tsla-pltr/"> Investor&#8217;s Business Daily</a>, her firm snapped up shares of Tesla (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/TSLA#NASDAQ">TSLA</a>), Robinhood (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/HOOD#NASDAQ">HOOD</a>), Coinbase (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/COIN#NASDAQ">COIN</a>), Palantir Technologies (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/PLTR#NYSE">PLTR</a>), and more on March 10.
<h2 class="wp-block-heading">Big Bets in a Turbulent Market</h2>
Investor&#8217;s Business Daily said that Ark Invest picked up 79,318 shares of Tesla, valued at $17.62 million based on Monday&#8217;s closing price of $222.15. The firm also acquired 269,864 shares of Robinhood for $9.6 million and 64,358 shares of Coinbase, worth $11.53 million.&nbsp;
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Other buys included 147,551 shares of Advanced Micro Devices (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/AMD#NASDAQ">AMD</a>) for $14.26 million, 152,253 shares of Palantir at $11.63 million, and 485,005 shares of Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>), valued at $12.46 million.
It&#8217;s a familiar strategy for Wood, who has built a reputation for &#8220;buying the dip.&#8221; She firmly believes in disruptive innovation and has placed some of her biggest bets on Tesla.&nbsp;
Wood thinks part of the recent demand dip for Tesla could result from broader fears about the economy. In a recent<a href="https://www.ark-funds.com/webinars/mar-2025-fund-webinar?utm_medium=email&amp;_hsmi=353364069&amp;utm_content=353364069&amp;utm_source=hs_email"> Ark Invest webinar</a>, she said, &#8220;Now, clearly, the political dynamics of the last few months are hitting demand; we also would suggest that the economic outlook is hitting demand—not just for Tesla—but for all auto manufacturers.&#8221;&nbsp;
Trending: BlackRock is calling 2025 the year of alternative assets. <a href="https://www.benzinga.com/money/masterworks">One firm from NYC has quietly built a group of 60,000+ investors who have all joined in on an alt asset class previously exclusive to billionaires like Bezos and Gates.</a>
Despite the stock&#8217;s 31% decline this year and a growing list of challenges, Wood has maintained a bullish outlook. She told <a href="https://www.bloomberg.com/news/articles/2025-03-25/cathie-wood-sticks-with-tesla-predicts-stock-will-hit-2-600">Bloomberg TV</a> that Tesla&#8217;s price could soar over the next five years.
Tesla has been at the center of controversy for months. Since Elon Musk&#8217;s closer ties with the Trump administration, the electric vehicle company has faced public protests, boycotts, and even vandalism.&nbsp;
Musk&#8217;s role as an advisor to the Department of Government Efficiency has drawn criticism, with some accusing the CEO of shifting the company into politically charged territory.
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The turmoil has had financial consequences. Tesla&#8217;s stock has been on a downward trend, while competitors, particularly China&#8217;s BYD, have continued to gain market share. According to <a href="https://edition.cnn.com/2025/03/20/business/even-used-teslas-are-falling-out-of-favor/index.html">CNN</a>, Tesla vehicles&#8217; resale value has also dropped over the past month.
Amid the backlash, President Donald Trump publicly backed Musk. He said on <a href="https://truthsocial.com/@realDonaldTrump/posts/114141854575248527">Truth Social </a>that he would buy a Tesla, calling it a &#8220;show of confidence&#8221; in the company. The move follows a broader effort by Trump&#8217;s administration to support Tesla.&nbsp;
Commerce Secretary Howard Lutnick, the commerce secretary, encouraged Americans to invest in the company.&nbsp;
&#8220;I think if you want to learn something on this show tonight, buy <a href="https://www.benzinga.com/news/25/03/44209579/unprecedented-slide-tesla-sales-plummet-45-in-europe-as-musks-net-worth-dives-5-2b-in-a-week">Tesla</a>. It&#8217;s unbelievable that this guy&#8217;s stock is this cheap. It&#8217;ll never be this cheap again,&#8221; ​he told Fox News on March 19.
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Personal Finance

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Cathie Wood Just Made A Bold Move—Why She Spent Over $65M On Tesla, Robinhood, And Coinbase 'As The Market Crashed'

Top Wall Street analysts changed their outlook on these top names. For a complete view of all analyst rating changes, including upgrades, downgrades and initiations, please see our <a href="https://www.benzinga.com/analyst-stock-ratings">analyst ratings page</a>.
<ul>
<li>Barclays analyst Henning Cosman upgraded the rating for Ferrari N.V. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/RACE#NYSE">RACE</a>) from Equal-Weight to Overweight. Ferrari shares closed at $426.82 on Thursday. <a href="https://www.benzinga.com/quote/RACE/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Jefferies analyst Blayne Curtis upgraded Applied Materials, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/AMAT#NASDAQ">AMAT</a>) from Hold to Buy and announced a $195 price target. Applied Materials shares closed at $147.68 on Thursday. <a href="https://www.benzinga.com/quote/AMAT/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>BMO Capital analyst Keith Bachman upgraded the rating for Check Point Software Technologies Ltd. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CHKP#NASDAQ">CHKP</a>) from Market Perform to Outperform and raised the price target from $245 to $275. Check Point Software shares closed at $231.31 on Thursday. <a href="https://www.benzinga.com/quote/CHKP/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Goldman Sachs analyst Caitlin Burrows upgraded Tanger Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/SKT#NYSE">SKT</a>) from Neutral to Buy and boosted the price target from $39 to $40. Tanger shares closed at $32.86 on Thursday. <a href="https://www.benzinga.com/quote/SKT/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>B of A Securities analyst Alec Stranahan upgraded the rating for Beam Therapeutics Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) from Neutral to Buy and maintained the price target of $42. Beam Therapeutics shares closed at $22.19 on Thursday. &nbsp;<a href="https://www.benzinga.com/quote/BEAM/analyst-ratings">See how other analysts view this stock.</a></li>
</ul>
Considering buying AMAT stock? Here&#8217;s what analysts think: 
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Photo: Shutterstock

Upgrades

Pre-Market Outlook

AMAT

CHKP

RACE

This Applied Materials Analyst Turns Bullish; Here Are Top 5 Upgrades For Friday

Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) has been analyzed by 9 analysts in the last three months, revealing a diverse range of perspectives from bullish to bearish.
The table below offers a condensed view of their recent ratings, showcasing the changing sentiments over the past 30 days and comparing them to the preceding months.
<table>
<thead>
<tr>
<th></th>
<th align="right">Bullish</th>
<th align="right">Somewhat Bullish</th>
<th align="right">Indifferent</th>
<th align="right">Somewhat Bearish</th>
<th align="right">Bearish</th>
</tr>
</thead>
<tbody>
<tr>
<td>Total Ratings</td>
<td align="right">6</td>
<td align="right">1</td>
<td align="right">2</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>1M Ago</td>
<td align="right">4</td>
<td align="right">1</td>
<td align="right">2</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>2M Ago</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>3M Ago</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
</tbody>
</table>
In the assessment of 12-month price targets, analysts unveil insights for Beam Therapeutics, presenting an average target of $53.89, a high estimate of $80.00, and a low estimate of $25.00. A 7.55% drop is evident in the current average compared to the previous average price target of $58.29. 
<img src="https://www.benzinga.com/files/images/story/2025/1743163275_0.png" alt="price target chart" />
<h2>Decoding Analyst Ratings: A Detailed Look</h2>
The analysis of recent analyst actions sheds light on the perception of Beam Therapeutics by financial experts. The following summary presents key analysts, their recent evaluations, and adjustments to ratings and price targets.
<table>
<thead>
<tr>
<th>Analyst</th>
<th>Analyst Firm</th>
<th>Action Taken</th>
<th>Rating</th>
<th>Current Price Target</th>
<th>Prior Price Target</th>
</tr>
</thead>
<tbody>
<tr>
<td>Alec Stranahan</td>
<td>B of A Securities</td>
<td>Maintains</td>
<td>Buy</td>
<td>$42.00</td>
<td>$42.00</td>
</tr>
<tr>
<td>Greg Harrison</td>
<td>Scotiabank</td>
<td>Announces</td>
<td>Sector Outperform</td>
<td>$40.00</td>
<td>-</td>
</tr>
<tr>
<td>Soumit Roy</td>
<td>Jones Trading</td>
<td>Announces</td>
<td>Buy</td>
<td>$34.00</td>
<td>-</td>
</tr>
<tr>
<td>Patrick Trucchio</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$80.00</td>
<td>$80.00</td>
</tr>
<tr>
<td>Greg Harrison</td>
<td>Scotiabank</td>
<td>Raises</td>
<td>Sector Perform</td>
<td>$25.00</td>
<td>$24.00</td>
</tr>
<tr>
<td>Patrick Trucchio</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$80.00</td>
<td>$80.00</td>
</tr>
<tr>
<td>Debjit Chattopadhyay</td>
<td>Guggenheim</td>
<td>Maintains</td>
<td>Buy</td>
<td>$78.00</td>
<td>$78.00</td>
</tr>
<tr>
<td>Luca Issi</td>
<td>RBC Capital</td>
<td>Raises</td>
<td>Sector Perform</td>
<td>$26.00</td>
<td>$24.00</td>
</tr>
<tr>
<td>Patrick Trucchio</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$80.00</td>
<td>$80.00</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: Analysts respond to changes in market conditions and company performance, frequently updating their recommendations. Whether they 'Maintain', 'Raise' or 'Lower' their stance, it reflects their reaction to recent developments related to Beam Therapeutics. This information offers a snapshot of how analysts perceive the current state of the company.</li>
<li>Rating: Providing a comprehensive analysis, analysts offer qualitative assessments, ranging from 'Outperform' to 'Underperform'. These ratings reflect expectations for the relative performance of Beam Therapeutics compared to the broader market.</li>
<li>Price Targets: Understanding forecasts, analysts offer estimates for Beam Therapeutics's future value. Examining the current and prior targets provides insight into analysts' changing expectations.</li>
</ul>
To gain a panoramic view of Beam Therapeutics's market performance, explore these analyst evaluations alongside essential financial indicators. Stay informed and make judicious decisions using our Ratings Table.
<a href="https://www.benzinga.com/quote/BEAM/analyst-ratings?adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Stay up to date on Beam Therapeutics analyst ratings.</a>
<h2>Get to Know Beam Therapeutics Better</h2>
Beam Therapeutics Inc is a biotechnology company committed to creating a new class of precision genetic medicines based on its proprietary base editing technology, with a vision of providing lifelong cures to patients suffering from serious diseases. The company is focused on its programs in the hematology and genetic disease portfolios, which comprise different product candidates in its pipeline, such as BEAM-101, which is being developed to target Sickle Cell Disease, BEAM-302, a liver-targeting lipid nanoparticle, being developed to target Alpha-1 antitrypsin deficiency, and other drug candidates including BEAM-302, BEAM-103 &amp; 104. The company views its operations and manages its business in one operating segment, operating exclusively in the United States.
<h3>Beam Therapeutics: A Financial Overview</h3>
Market Capitalization Analysis: The company's market capitalization is above the industry average, indicating that it is relatively larger in size compared to peers. This may suggest a higher level of investor confidence and market recognition.
Revenue Growth: Beam Therapeutics's revenue growth over a period of 3 months has faced challenges. As of 31 December, 2024, the company experienced a revenue decline of approximately -90.49%. This indicates a decrease in the company's top-line earnings. As compared to its peers, the revenue growth lags behind its industry peers. The company achieved a growth rate lower than the average among peers in Health Care sector.
Net Margin: The company's net margin is a standout performer, exceeding industry averages. With an impressive net margin of -300.51%, the company showcases strong profitability and effective cost control.
Return on Equity (ROE): Beam Therapeutics's financial strength is reflected in its exceptional ROE, which exceeds industry averages. With a remarkable ROE of -11.85%, the company showcases efficient use of equity capital and strong financial health.
Return on Assets (ROA): The company's ROA is below industry benchmarks, signaling potential difficulties in efficiently utilizing assets. With an ROA of -7.94%, the company may need to address challenges in generating satisfactory returns from its assets.
Debt Management: Beam Therapeutics's debt-to-equity ratio is below the industry average. With a ratio of 0.22, the company relies less on debt financing, maintaining a healthier balance between debt and equity, which can be viewed positively by investors.
<h2>The Core of Analyst Ratings: What Every Investor Should Know</h2>
Ratings come from analysts, or specialists within banking and financial systems that report for specific stocks or defined sectors (typically once per quarter for each stock). Analysts usually derive their information from company conference calls and meetings, financial statements, and conversations with important insiders to reach their decisions.
Some analysts publish their predictions for metrics such as growth estimates, earnings, and revenue to provide additional guidance with their ratings. When using analyst ratings, it is important to keep in mind that stock and sector analysts are also human and are only offering their opinions to investors.
If you want to keep track of which analysts are outperforming others, you can view updated analyst ratings along withanalyst success scores in <a href="https://www.benzinga.com/pro/">Benzinga Pro</a>.
Which Stocks Are Analysts Recommending Now?
Benzinga Edge gives you instant access to all major analyst upgrades, downgrades, and price targets. Sort by accuracy, upside potential, and more. <a href="https://www.benzinga.com/premium/ideas/benzinga-edge-checkout/?t=be8be9arja3be22bz21&amp;adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Click here to stay ahead of the market</a>.
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Beyond The Numbers: 9 Analysts Discuss Beam Therapeutics Stock

B of A Securities analyst Alec Stranahan upgrades Beam Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>) from Neutral to Buy and maintains the price target from $42 to $42.

B of A Securities Upgrades Beam Therapeutics to Buy, Maintains Price Target to $42

U.S. IND Represents Regulatory Clearance For BEAM-302 Spanning Six Countries and Second Open IND for a Beam In Vivo Base Editing Program in the U.S.
CAMBRIDGE, Mass., March 27, 2025 (GLOBE NEWSWIRE) -- <a href="https://www.globenewswire.com/Tracker?data=CBsje_Xtz_cDfv6xJ9MGUStWgQ1d58JxqmOuiR5CJFoi__HBN6R2zqYvT7RqTyDgwhJhj8h-uqaBIvoHtT0Rslsh7A7UGCzjoOgFk5m8nhw=">Beam Therapeutics Inc.</a> (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BEAM#NASDAQ">BEAM</a>), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for BEAM-302 for the treatment of 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 are no currently approved curative treatments.
"The FDA's clearance of our IND for BEAM-302 is a significant step toward advancing the development of this potential breakthrough treatment for patients living with AATD in the U.S.," said Giuseppe Ciaramella, Ph.D., president of Beam. "Earlier this month, we shared groundbreaking initial safety and efficacy data from our global Phase 1/2 study of BEAM-302, showcasing the first ever clinical genetic correction of a disease-causing mutation. As we move forward, we're laser focused on progressing this trial to enroll and dose more patients, activating more sites in new geographies, and preparing to present updated data in the second half of the year."
BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing PiZ mutation in patients with AATD.&nbsp;BEAM-302 is being evaluated in an ongoing Phase 1/2, open-label, dose exploration and dose expansion clinical trial to investigate its safety, tolerability, pharmacodynamics, pharmacokinetics and efficacy. Part A of the trial is designed to evaluate AATD patients with lung disease, and Part B will evaluate AATD patients with mild to moderate liver disease with or without lung disease. Initial safety and efficacy results from the trial were <a href="https://www.globenewswire.com/Tracker?data=eLpUtJQDGhOT-96QV9EmjS-eqi7vNw9giS21bCU0RmnfoF1S0ygOPuAgiV7VRlCfogUhzqafvf1JUgmroUCfZMEGkZAn6EqsupOzU9FcL0GaW6hHsTC6LAl3Zp2vdcenr3t73-LPXnY7tOmtZGcoJmuqAQkohAod7bqf56xC5BVS4aS05HuNGz24TjFrM-eUtI7ApEAo2scXy6FX9UMQNE-NbIsG2MljKIBdIsUlV0U=">reported</a> in March 2025 and established proof of concept for BEAM-302 as a potential one-time treatment for AATD. In addition to the U.S., BEAM-302 has received clinical trial authorization from the United Kingdom, New Zealand, Australia, the Netherlands and Ireland.

Beam Therapeutics Announces Clearance Of Investigational New Drug Application For BEAM-302 For Treatment Of Alpha-1 Antitrypsin Deficiency By The U.S. FDA

Press Releases

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