On November 4, 2025 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported third quarter 2025 financial results and provided updates across the company’s hematology and genetic disease franchises.

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"As we near the end of 2025, we see broad-based momentum across our growing portfolio of clinical-stage hematology and liver-targeted genetic disease base editing programs," said John Evans, chief executive officer at Beam. "Our alpha-1 antitrypsin deficiency program, the first clinical program to directly correct a disease-causing mutation in vivo, remains a top priority, and we’re pleased with the continued enrollment and dosing progress in the BEAM-302 Phase 1/2 trial. We look forward to providing a broad update on the program in early 2026 with new clinical data and next steps to advance BEAM-302 to patients. In sickle cell disease, we look forward to sharing updated data from the BEACON trial of BEAM-101 at the ASH (Free ASH Whitepaper) meeting in December, where we aim to continue to demonstrate a differentiated manufacturing and clinical profile in these patients with recurrent severe VOCs. We have also initiated dosing of the BEAM-103 antibody from our ESCAPE platform, which we believe can play an important role in next wave therapies for sickle cell disease."

Mr. Evans continued, "In addition, we are thrilled for the Orbital Therapeutics team following its proposed acquisition by Bristol Myers Squibb, indicating the significant potential of Orbital’s platform and pipeline. Beam contributed capabilities and technology in mRNA and targeted lipid nanoparticles to Orbital, and this outcome further validates our innovative platform strategy to unlock additional shareholder value in non-core areas and accelerate the creation of new medicines for patients."

Third Quarter 2025 and Recent Progress

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Updated data from the BEACON Phase 1/2 clinical trial of BEAM-101 in patients with sickle cell disease (SCD) were accepted for presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, taking place December 6-9, 2025, in Orlando, Fla.
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The first subject was dosed in a Phase 1 healthy volunteer clinical trial of BEAM-103, an anti-CD117 monoclonal antibody (mAb) developed as part of the company’s ESCAPE (Engineered Stem Cell Antibody Evasion) platform. ESCAPE represents a potential alternative to genotoxic conditioning regimens in stem cell transplantation, potentially avoiding toxicity challenges associated with currently available conditioning regimens for patients with SCD and beta-thalassemia.
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In the Phase 1/2 study of BEAM-302 in alpha-1 antitrypsin deficiency (AATD), dosing has commenced in the multi-dose cohort evaluating two 60 mg doses administered eight weeks apart in Part A of the trial, designed to evaluate AATD patients with lung disease. In addition, the first patient was dosed in the first cohort in Part B of the trial, designed to evaluate AATD patients with mild to moderate liver disease with or without lung disease.

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In August, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101 for the treatment of SCD. The designation is designed to support the development and evaluation of regenerative medicines, with the intention of addressing serious or life-threatening diseases that have unmet medical needs.
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In October, Bristol Myers Squibb announced a definitive agreement under which they will acquire Beam collaborator Orbital Therapeutics for $1.5 billion in cash. As of the announcement, Beam held 75 million shares of Orbital common stock, which represented a fully diluted ownership stake of approximately 17%.1
Key Anticipated Milestones

Liver-targeted Genetic Disease Franchise

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Beam expects to report data from the dose-escalation portions of Part A and Part B of the BEAM-302 Phase 1/2 trial and provide a clinical development update in early 2026.
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Beam plans to continue dosing in the Phase 1/2 clinical trial of BEAM-301 in glycogen storage disease Ia (GSDIa).
Hematology Franchise

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Beam plans to present updated data from the BEACON Phase 1/2 trial at the ASH (Free ASH Whitepaper) Annual Meeting, taking place December 6-9, 2025.
Third Quarter 2025 Financial Results

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Cash Position: Cash, cash equivalents and marketable securities were $1.1 billion as of September 30, 2025, compared to $850.7 million as of December 31, 2024.
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Research & Development (R&D) Expenses: R&D expenses were $109.8 million for the third quarter of 20252, compared to $94.3 million for the third quarter of 2024.
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General & Administrative (G&A) Expenses: G&A expenses were $26.7 million for the third quarter of 2025, compared to $26.5 million for the third quarter of 2024.
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Net Income (Loss): Net loss was $112.7 million, or $1.10 per share, for the third quarter of 2025, compared to $96.7 million, or $1.17 per share, for the third quarter of 2024.
Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of September 30, 2025, will enable the company to fund its anticipated operating expenses and capital expenditure requirements into 2028. This expectation includes funding directed toward reaching each of the key anticipated milestones for BEAM-101, ESCAPE, BEAM-301 and BEAM-302 described above.

(Press release, Beam Therapeutics, NOV 4, 2025, View Source [SID1234659345])