$100 Million Funded at Close with up to an Additional $400 Million Available Under Facility with Seven-Year Term Financing Bolsters Balance Sheet with Long-term, Non-dilutive Capital to Support Anticipated Launch of Risto-cel in Sickle Cell Disease (SCD) CAMBRIGE, Mass., Feb. 24, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM) today announced that it has entered into a strategic financing agreement with Sixth Street for substantial, long-term, non-dilutive capital to fund the potential launch ...

CAMBRIDGE, Mass., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will announce its fourth quarter and full year 2025 financial results and a new liver-targeted genetic disease program on an investor webcast on Tuesday, February 24, 2026, at 8:00 a.m. ET.The live webcast will be available in the investor section of the company's website at www.beamtx.com and will be ...

Beam Therapeutics Inc. (NASDAQ:BEAM) is one of the 13 hot stocks to buy with the highest upside potential. On February 9, Sami Corwin from William Blair maintained a Buy rating on Beam Therapeutics. This maintained rating reinforced the bullish outlook that the analyst previously gave on Beam Therapeutics Inc. (NASDAQ:BEAM) in a January 21 note. Corwin noted that the existing clinical data on the lead program of Beam Therapeutics Inc. (NASDAQ:BEAM), the BEAM-302 treatment for alpha-1 antitrypsin deficiency, ...

Group 1 - Beam Therapeutics (NASDAQ:BEAM) is identified as one of the 17 biotechnology stocks with more than 50% upside potential, with a Buy rating and a price target of $80, indicating an upside of over 197% [1] - The company's recent agreement with the U.S. FDA to expedite the approval process for BEAM-302 in alpha-1 antitrypsin deficiency is a significant factor in the positive rating, allowing the use of AAT biomarker data over a 12-month horizon for future BLA [2] - Beam Therapeutics has a strong financial position, with nearly $1.25 billion in cash and marketable securities as of the end of 2025, sufficient to support operations until 2029 and fund the development of BEAM-302 and the anticipated launch of risto-cel [3] Group 2 - UBS analyst Michael Yee assigned a Neutral rating with a $28 price target on January 8, suggesting that more certainty for investors could benefit the biotech sector in 2026, with expectations of major catalysts and attractive pipelines driving returns [4] - Beam Therapeutics focuses on developing precision genetic medicines, engaging in gene therapies and genome editing research, and is a leader in CRISPR-based editing for advanced genetic medicine development [5]

Core Insights - Beam Therapeutics has reached alignment with the U.S. FDA on a potential accelerated approval pathway for BEAM-302, a treatment for Alpha-1 Antitrypsin Deficiency (AATD), based on biomarker endpoints [1][4] - The company expects to submit a Biologics Licensing Application (BLA) for risto-cel (formerly BEAM-101) as early as the end of 2026 [1][13] - Beam's cash position is estimated at $1.25 billion as of December 31, 2025, providing an operating runway into 2029 to support the anticipated launch of risto-cel and the execution of the BEAM-302 pivotal development plan [10][11] Liver-targeted Genetic Disease Franchise - Beam is developing a platform for precision gene editing therapies targeting liver diseases, utilizing lipid-nanoparticle delivery systems [3] - BEAM-302 aims to be a first-in-class therapy for AATD, demonstrating clinical proof of concept in an ongoing Phase 1/2 trial with over 25 patients treated [4] - The company plans to enroll approximately 50 additional patients for the expansion of the Phase 1/2 study to support the BLA submission [4] Hematology Franchise - Risto-cel is an investigational autologous cell therapy for sickle cell disease (SCD), showing a differentiated treatment profile in the ongoing BEACON Phase 1/2 clinical trial [8] - The therapy has demonstrated a deeper resolution of SCD markers and reduced hospital time, with a robust manufacturing process [8] - Beam is also focusing on next-generation programs for SCD, including in vivo delivery strategies and the development of the ESCAPE platform for non-genotoxic treatments [9] Financial Position and Future Outlook - The estimated cash and marketable securities position of $1.25 billion will support Beam's operating expenses and capital requirements through 2029 [10][11] - The company is well-positioned to advance its pipeline of genetic therapies, with a focus on regulatory execution and clinical data generation [2][11] - Beam plans to announce a new program for its liver-targeted genetic disease franchise in the first half of 2026 [6]

Core Insights - Beam Therapeutics is advancing its clinical-stage programs in hematology and liver-targeted genetic diseases, with a focus on alpha-1 antitrypsin deficiency and sickle cell disease [2][3] - The company reported a cash position of $1.1 billion as of September 30, 2025, which is expected to support operations into 2028 [6][10] - Beam's BEAM-302 Phase 1/2 trial for alpha-1 antitrypsin deficiency is ongoing, with updated data expected in early 2026 [1][9] Clinical Development Updates - The BEAM-302 trial is evaluating dosing in patients with lung and liver disease, with multi-dose cohorts initiated [3][4] - Updated data from the BEACON trial of BEAM-101 in sickle cell disease will be presented at the ASH Annual Meeting in December 2025 [3][4] - The first subject has been dosed in a Phase 1 trial for BEAM-103, an anti-CD117 monoclonal antibody, as part of the ESCAPE platform [1][3] Financial Performance - For Q3 2025, Beam reported a net loss of $112.7 million, or $1.10 per share, compared to a net loss of $96.7 million, or $1.17 per share, in Q3 2024 [10][15] - Research and development expenses increased to $109.8 million in Q3 2025 from $94.3 million in Q3 2024 [10][15] - The company’s total assets as of September 30, 2025, were $1.31 billion, up from $1.10 billion at the end of 2024 [13] Strategic Developments - Beam's collaboration with Orbital Therapeutics is highlighted by Bristol Myers Squibb's proposed acquisition of Orbital for $1.5 billion, validating Beam's innovative platform strategy [2][3] - The FDA granted Regenerative Medicine Advanced Therapy designation to BEAM-101 for sickle cell disease, supporting its development [3]

Core Insights - Beam Therapeutics Inc. announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 for treating sickle cell disease (SCD), demonstrating strong clinical outcomes and a consistent safety profile [2][3][4] Efficacy and Safety Data - All patients treated with BEAM-101 achieved hemoglobin F (HbF) induction levels exceeding 60% and a reduction in sickle hemoglobin (HbS) to below 40%, with no vaso-occlusive crises reported post-engraftment [3][5][11] - Patients required a median of one mobilization cycle for treatment, with rapid neutrophil and platelet engraftment observed, taking a median of 16.5 days and 19.5 days respectively [11] - Key markers of hemolysis normalized or improved in all patients, indicating significant enhancement in oxygen delivery to tissues [11] Clinical Trial Progress - Enrollment in both adult and adolescent cohorts of the BEACON trial is complete, with 26 patients dosed as of June 13, 2025, and an expectation to dose 30 patients by mid-2025 [7][8] - The follow-up period for treated patients ranged from 0.2 to 15.1 months, with durable responses observed in HbF and HbS levels [4][11] Manufacturing and Process - BEAM-101 is manufactured using an advanced, largely automated process that has shown high yields and viability, facilitating successful manufacturing for all patients in the trial [3][11] - The safety profile of BEAM-101 aligns with that of busulfan conditioning and autologous hematopoietic stem cell transplantation, with common treatment-emergent adverse events being consistent with busulfan [11] Company Vision and Future Plans - Beam Therapeutics aims to deliver life-long cures through its precision genetic medicines platform, focusing on base editing technology [12] - The company is committed to advancing its BEAM-101 program and plans to share additional data from the trial by the end of 2025 [4][7]

Core Viewpoint - Beam Therapeutics has received orphan drug designation from the FDA for BEAM-101, a genetically modified cell therapy aimed at treating sickle cell disease, highlighting the need for new treatment options for this serious condition [1][2]. Company Overview - Beam Therapeutics is a biotechnology company focused on precision genetic medicines, utilizing base editing technology to develop therapies [6][7]. - The company aims to create a fully integrated platform for gene editing, delivery, and manufacturing [6]. Product Details - BEAM-101 is an investigational therapy that involves autologous CD34+ hematopoietic stem and progenitor cells, which are base-edited to increase fetal hemoglobin production [4]. - The therapy is designed to inhibit the BCL11A repressor, leading to increased levels of non-sickling fetal hemoglobin, mimicking naturally occurring variants [4]. Clinical Trial Information - The BEACON Phase 1/2 clinical trial is ongoing, with initial data showing robust increases in fetal hemoglobin and improvements in hemolysis markers [3]. - The trial aims to dose 30 patients by mid-2025, with updated clinical data to be presented at the European Hematology Association Congress [3]. Disease Context - Sickle cell disease affects approximately 100,000 individuals in the U.S. and is characterized by severe complications such as anemia, pain crises, and early death [2][5]. - It is the most common inherited blood disorder in the U.S., caused by a mutation in the beta globin gene [5]. Regulatory Designation - The FDA's orphan drug designation provides benefits such as tax credits for clinical trials, exemption from user fees, and potential market exclusivity for seven years post-approval [2].

Core Insights - Beam Therapeutics is presenting updated safety and efficacy data from the BEACON Phase 1/2 clinical trial of BEAM-101 for sickle cell disease at the EHA2025 Congress [1][2] - BEAM-101 is a genetically modified ex vivo cell therapy aimed at treating severe sickle cell disease, specifically targeting patients with severe vaso-occlusive crises [1][5] Company Overview - Beam Therapeutics is focused on developing precision genetic medicines through base editing technology, which allows for precise modifications in the genome without causing double-stranded breaks [7] - The company aims to provide lifelong cures for serious diseases, leveraging its integrated platform for gene editing, delivery, and manufacturing [7] Clinical Trial Details - The BEACON trial is an open-label, single-arm, multicenter study evaluating the safety and efficacy of BEAM-101 in adult patients with sickle cell disease [5] - The updated dataset includes results from 17 patients, highlighting the potential of BEAM-101 as a transformative one-time treatment for sickle cell disease [2][5] Presentation Information - The presentations at EHA2025 will cover various aspects of BEAM-101, including its safety, efficacy, and the health of red blood cells post-treatment [3][4] - The conference call and webcast to review key presentations will take place on June 13, 2025, at 4:00 p.m. ET [4]

Company Overview - Beam Therapeutics Inc. is a biotechnology company focused on developing precision genetic medicines through base editing [3] - The company aims to establish a leading, fully integrated platform for precision genetic medicines, incorporating gene editing, delivery, and internal manufacturing capabilities [3] - Beam's proprietary technology, base editing, allows for precise and efficient single base changes in targeted genomic sequences without causing double-stranded breaks in DNA [3] Upcoming Events - Management will participate in a fireside chat at the 2025 RBC Capital Markets Global Healthcare Conference on May 20, 2025, at 11:00 a.m. ET in New York [1] - The live webcast of the event will be available on the company's investor section of the website and archived for 60 days post-presentation [2] Commitment and Vision - Beam Therapeutics is a values-driven organization dedicated to its people and cutting-edge science [3] - The company envisions providing life-long cures to patients suffering from serious diseases through its diversified portfolio of base editing programs [3]