Core Insights - Beam Therapeutics presented new data from the Phase 1/2 clinical trial of BEAM-302, showing a mean of 91% corrected M-AAT in circulation at Day 28 in the 60 mg cohort, alongside a 79% decrease in mutant Z-AAT levels [1][3][4] Group 1: Clinical Trial Data - The Phase 1/2 trial of BEAM-302 has demonstrated positive initial safety and efficacy, establishing clinical proof of concept for treating alpha-1 antitrypsin deficiency (AATD) [2] - Updated biomarker data from the 60 mg cohort indicated that the proportion of corrected M-AAT reached a mean of 91% of total AAT in circulation, surpassing levels typically seen in patients with the MZ genotype [3] - The treatment led to a mean decrease of 79% in circulating mutant Z-AAT from baseline as of Day 28 [3] Group 2: Future Plans and Developments - Beam Therapeutics plans to continue the dose-escalation portion of the trial, initiating a fourth cohort at 75 mg and expects to present further data at a medical conference in the second half of 2025 [4] - The company aims to dose the first patient in Part B of the trial, which will include AATD patients with mild to moderate liver disease, in the second half of 2025 [4] - The investigational drug application (IND) for BEAM-302 has been cleared by the U.S. FDA, allowing the activation of trial sites in the U.S. [4] Group 3: About BEAM-302 and AATD - BEAM-302 is a liver-targeting lipid-nanoparticle formulation designed to correct the PiZ mutation, which is prevalent among patients with severe AATD [5] - The treatment aims to reduce the aggregation of mutant AAT protein, generate therapeutic levels of corrected protein, and increase total AAT in circulation, addressing the underlying pathophysiology of liver and lung disease [5] - AATD is an inherited genetic disorder that can lead to early onset emphysema and liver disease, with an estimated 100,000 individuals in the U.S. affected by the severe form of the disease [6][8]

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, ther ...

Core Viewpoint - The FDA has cleared the investigational new drug (IND) application for BEAM-302, a potential treatment for alpha-1 antitrypsin deficiency (AATD), marking a significant advancement in the development of genetic medicines for this condition [2][3]. Company Overview - Beam Therapeutics is a biotechnology company focused on precision genetic medicines through base editing, with a commitment to developing a fully integrated platform for gene editing, delivery, and manufacturing [8]. Product Details - BEAM-302 is a liver-targeting lipid-nanoparticle formulation designed to correct the PiZ mutation in patients with AATD, which is expected to provide a one-time treatment option [3][5]. - The ongoing Phase 1/2 clinical trial of BEAM-302 aims to evaluate its safety, tolerability, pharmacodynamics, pharmacokinetics, and efficacy, with initial results indicating proof of concept for the treatment [3][4]. Clinical Trial Progress - The company plans to continue the dose-escalation portion of the trial and expects to report further data at a medical conference in the second half of 2025, including the initiation of dosing for patients with mild to moderate liver disease [4][3]. Disease Background - AATD is an inherited genetic disorder that can lead to early onset emphysema and liver disease, primarily affecting individuals with the PiZZ genotype, which is estimated to be present in about 100,000 individuals in the U.S. [6][7]. - Current treatments for AATD are limited, with no curative options available, highlighting the potential impact of BEAM-302 [7].

Beam Therapeutics Inc. (BEAM) announced positive initial safety and efficacy data from a phase I/II study, which is evaluating its pipeline candidate, BEAM-302, for treating patients with alpha-1 antitrypsin deficiency (AATD).Currently, there are no approved curative treatments for the given indication.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 pro ...

CAMBRIDGE, Mass., March 10, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the pricing of an underwritten offering of 16,151,686 shares of its common stock at an offering price of $28.48 per share, and, in lieu of common stock to certain investors, pre-funded warrants to purchase 1,404,988 shares of common stock at an offering price of $28.47 per pre-funded warrant, before deducting underwri ...

Single Dose of BEAM-302 Led to Durable, Dose-dependent Increases in Total and Functional Alpha-1 Antitrypsin (AAT), Production of Corrected M-AAT, and Decreases in Mutant Z-AAT in Circulation Across Initial Three Dose Levels Third Dose Level of BEAM-302 (60 mg, N=3) Achieved Mean Total AAT of 12.4µM at Day 28, Exceeding Protective Therapeutic Threshold, and Reduced Mutant Z-AAT up to 78% Initial Safety Findings Demonstrated BEAM-302 was Well Tolerated at All Dose Levels with No Serious Adverse Events or Do ...

Beam Therapeutics (BEAM) reported a loss of $1.09 per share for the fourth quarter of 2024, which was narrower than the Zacks Consensus Estimate of a loss of $1.10. The company had recorded earnings of $1.73 per share in the year-ago quarter.See the Zacks Earnings Calendar to stay ahead of market-making news.Total revenues, comprising license and collaboration revenues, came in at $30.1 million in the fourth quarter compared with $316.2 million reported in the year-ago period. The top line beat the Zacks Co ...

Beam Therapeutics Inc. (BEAM) came out with a quarterly loss of $1.09 per share versus the Zacks Consensus Estimate of a loss of $1.10. This compares to earnings of $1.73 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 0.91%. A quarter ago, it was expected that this company would post a loss of $1.13 per share when it actually produced a loss of $1.17, delivering a surprise of -3.54%.Over the last four quarters, the company ha ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number 001-39208 Beam Therapeutics Inc. (Mark One) FORM 10-K OR Washington, D.C. 20549 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO For the fiscal year ended December 31, 2024 238 Main Street (Address of principal executive of ices) (Zip Code) Common Stock, par value $0.01 per sh ...

Financial Position - As of December 31, 2024, Beam Therapeutics Inc. estimates cash, cash equivalents, and marketable securities of approximately $850.7 million[6] - The estimated cash balance is preliminary and subject to completion of financial statement closing procedures, indicating potential variability in actual results[7] - The company has not finalized its full financial results for the fourth quarter and fiscal year ended December 31, 2024[6] - The company emphasizes the importance of not placing undue reliance on preliminary estimates of financial condition[7] Business Development - Beam Therapeutics announced progress across its base editing portfolio and outlined key anticipated milestones in a press release dated January 13, 2025[11] - The company is focused on developing life-long, curative, precision genetic medicines for patients through base editing technology[12] - The company plans to advance its clinical trial designs and expectations for product candidates BEAM-101, BEAM-103, BEAM-301, and BEAM-302[12] Future Outlook - The company is preparing for presentations at the 43rd Annual J.P. Morgan Healthcare Conference on January 13, 2025[9] - Forward-looking statements include expectations related to the therapeutic applications of the company's technology for conditions such as SCD, AATD, GSD1a, and beta thalassemia[12] - The company faces risks related to the development and commercialization of its product candidates, including regulatory approval and funding availability[12]