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Beam Therapeutics
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Latest analysis
Updated Nov 3, 2026
Beam Therapeutics advances toward first BLA filing for risto-cel by year-end 2026 while BEAM-302 pivotal cohort dosing targets H1 2027 completion.
Beam Therapeutics is at a clinical inflection point: a BLA submission for risto-cel in sickle cell disease is targeted as early as year-end 2026, and the global pivotal cohort for BEAM-302 in AATD is on track for dosing completion in H1 2027, supported by 18-month durability data presented at ERS. BEAM-301 delivered initial clinical proof of concept in GSDIa at ESGCT, with two of three patients in the 40 mg cohort achieving the protocol-defined maximum fasting duration, while BEAM-304 in PKU is positioned to initiate dosing in early 2027. With $1.0 billion in cash plus $200 million in undrawn Sixth Street credit capacity, Beam's runway extends into mid-2029, covering risto-cel launch, the BEAM-302 pivotal readout, and BEAM-304 proof of concept.
Tone: bullishRevenue
$139.7M
BEAM 10-K · FY 2025
Employees
511
Revenue FY2024
$63.5M
Founded
2017
Profile
BEAM 10-K Item 1 · Feb 24, 2026Beam Therapeutics is a clinical-stage biotechnology company building an integrated platform for precision genetic medicines anchored by proprietary base editing technology. The company advances a diversified pipeline across hematology and genetic diseases, with lead programs targeting sickle cell disease and alpha-1 antitrypsin deficiency. Beam also generates collaboration revenue by licensing its base editing platform to partners including Pfizer, Apellis, and Verve/Lilly.
Read filing description ↓ Collapse description ↑
We are a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. Our vision is to provide life-long cures to patients suffering from serious diseases. To achieve this vision, we have assembled a platform that includes a suite of gene editing and delivery technologies as well as internal manufacturing capabilities. Our suite of gene editing technologies is anchored by our proprietary base editing technology, which potentially enables a differentiated class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. This approach uses a chemical reaction designed to create precise, predictable and efficient genetic outcomes at the targeted sequence. Our proprietary base editors have two principal components: (i) a clustered regularly interspaced short palindromic repeats, or CRISPR, protein, bound to a guide RNA, that leverages the established DNA-targeting ability of CRISPR, but is modified to not cause a double-stranded break, and (ii) a base editing enzyme, such as a deaminase, which carries out the desired chemical modification of the target DNA base. We believe this design contributes to a more precise and efficient edit compared to traditional gene editing methods, with the potential to dramatically increase the impact of gene editing. We are also pursuing a suite of delivery modalities, including both ex vivo and in vivo approaches, depending on tissue type. The elegance of the base editing approach, combined with a tissue specific delivery modality, provides the basis for a targeted, efficient, precise, and highly versatile gene editing system that is designed to be capable of gene correction, gene silencing, gene activation, gene modification, and/or multiplex editing of several genes simultaneously. Our goal is to advance a broad, diversified portfolio of base editing programs against distinct, genetically validated editing targets, as well as an innovative, platform business model that will expand the reach of our programs to more patients.
Primary products
- Ristoglogene autogetemcel (risto-cel / BEAM-101)
- BEAM-302
- BEAM-304
- BEAM-301
- BEAM-103
- ESCAPE platform
End markets
Geographies
Named customers
Named competitors
“While we believe that our differentiated technology, scientific expertise, and intellectual property position provide us with competitive advantages, we face potential competition from a variety of companies in these fields.” Competitive position, as stated in the filing
Revenue commentary · FY 2025
License and collaboration revenue increased to approximately $139.7 million in 2025 from approximately $63.5 million in 2024, driven primarily by the Pfizer opt-in and the Orbital/Bristol-Myers Squibb transaction proceeds.
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