Beam Therapeutics

What Beam Therapeutics does

Beam Therapeutics is a clinical-stage biotechnology company founded in 2017 and headquartered in Cambridge, Massachusetts, that develops precision genetic medicines using base editing—a proprietary technology that chemically converts single DNA base pairs (adenine-to-guanine or cytosine-to-thymine) without creating double-stranded DNA breaks, in contrast to conventional CRISPR-Cas9 approaches. The company holds licenses to foundational base editing intellectual property from Harvard University and the Broad Institute of MIT and Harvard, including patents covering cytosine base editors (CBE) and adenine base editors (ABE), with founder David R. Liu as inventor on more than 50 issued patents. Beam's pipeline spans two franchises: a Hematology franchise led by risto-cel (BEAM-101), an ex vivo edited autologous cell therapy for severe sickle cell disease delivered via hematopoietic stem cell transplant, complemented by the ESCAPE (BEAM-103) conditioning platform; and a Liver-Targeted Genetic Disease franchise comprising BEAM-302 for alpha-1 antitrypsin deficiency (AATD), BEAM-301 for glycogen storage disease type Ia, and BEAM-304 for phenylketonuria (PKU), all delivered in vivo via lipid nanoparticles.

Beam has not yet commercialized any product and currently generates revenue primarily through licensing and collaboration agreements, with the Pfizer partnership driving the bulk of recognized revenue—$139.7 million in FY2025 (up 120% from $63.5 million in FY2024) and $31.7 million in Q1 2026. Pfizer exercised its option for global exclusive rights to Beam's liver-targeted gene editing candidate in February 2026, providing a $300 million upfront payment and up to $1.05 billion in potential milestones, with Pfizer covering 65% of development costs post-Phase 1/2. The company also secured a $500 million non-dilutive senior secured credit facility from Sixth Street and monetized its Orbital Therapeutics equity stake for $255.1 million following Bristol Myers Squibb's acquisition, extending cash runway into mid-2029. Beam is building commercial infrastructure ahead of a planned risto-cel BLA submission by year-end 2026, targeting accredited treatment centers with apheresis and cell-handling capabilities, while planning to leverage specialty pharmacy/distributor networks for in vivo LNP-based therapies.

Name

Beam Therapeutics

Legal name

Beam Therapeutics Inc.

Website

https://beamtx.com

Company type

Public

Founded year

2017

Operating status

Operating

Headcount range

501–1,000 employees

Short description

Beam Therapeutics is a clinical-stage biotechnology company developing precision genetic medicines using proprietary base editing technology that chemically converts single DNA base pairs without creating double-stranded breaks. The company targets sickle cell disease, alpha-1 antitrypsin deficiency, glycogen storage disease type Ia, and phenylketonuria through ex vivo cell therapy and in vivo lipid nanoparticle delivery approaches.

Ownership category

akta.pro rank

Product category

Precision Genetic Medicines

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414), Scientific Research and Development Services (5417)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD)

akta.pro secondary industries

Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL)

Keywords

• Precision genetic medicines
• Base editing technology
• Gene editing therapeutics
• Cell therapy development
• Rare disease treatments

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Revenue model

1. License and Collaboration Revenue: Beam generates revenue through strategic licensing and collaboration agreements with pharmaceutical partners. Revenue is recognized upon completion of collaboration milestones, completion of research services, or over time as obligations are satisfied. Q1 2026 revenue was $31.7 million, up 322% year-over-year from $7.5 million in Q1 2025. Full-year 2025 revenue was $139.7 million, up from $63.5 million in 2024, driven primarily by the Pfizer collaboration.
2. Strategic Partnerships with Milestone Payments: Beam enters into co-development and co-commercialization arrangements with pharmaceutical companies. The Pfizer collaboration involves Beam receiving up to $1.05 billion in potential milestones, with Pfizer covering 65% of development costs post-Phase 1/2 and receiving the same proportion of profits. Beam can opt into co-development upon completion of Phase 1/2 trials.
3. Equity Monetization: Beam monetized its equity ownership in Orbital Therapeutics for $255.1 million following Bristol Myers Squibb's acquisition of Orbital, contributing to an updated cash balance of approximately $1.25 billion.

Go-to-market motion2 records

Distribution channels3 records

Marketing channels6 records

Core offering

Beam Therapeutics develops precision genetic medicines using its proprietary base editing technology, which chemically converts single DNA base pairs (A-to-G or C-to-T) without creating double-stranded DNA breaks. The company's pipeline includes ex vivo cell therapies such as risto-cel (BEAM-101) for sickle cell disease and in vivo lipid nanoparticle-formulated base editors (BEAM-302, BEAM-301, BEAM-304) targeting liver-mediated genetic diseases including alpha-1 antitrypsin deficiency, glycogen storage disease type Ia, and phenylketonuria.

Product overview

Beam Therapeutics is a biotechnology company developing a portfolio of precision genetic medicines powered by its proprietary base editing platform. The platform enables precise single-letter genetic corrections without double-stranded DNA breaks using cytosine and adenine base editors. The company's pipeline spans two franchises: Hematology programs including risto-cel (BEAM-101) for sickle cell disease via ex vivo edited autologous stem cells, and ESCAPE (BEAM-103) for conditioning-enhanced sickle cell/beta-thalassemia treatment; and Liver-Targeted Genetic Disease programs including BEAM-302 for alpha-1 antitrypsin deficiency, BEAM-301 for glycogen storage disease Ia, and BEAM-304 for phenylketonuria—all delivered via lipid nanoparticle formulations. The company maintains collaborations with Pfizer for undisclosed liver-targeted programs and with Apellis for autoimmune disorders. Beam has established internal manufacturing capabilities in Research Triangle Park, North Carolina to support clinical and potential commercial supply.

Differentiator

Problem solved

Functional benefit

Products and services

• Risto-cel (BEAM-101) Investigational autologous cell therapy for severe sickle cell disease that base-edits CD34+ hematopoietic stem and progenitor cells in HBG1/2 promoter regions to upregulate anti-sickling fetal hemoglobin expression, delivered via hematopoietic stem cell transplant.
• ESCAPE (BEAM-103) Engineered stem cell antibody paired evasion platform combining an anti-CD117 monoclonal antibody with cell therapy containing therapeutic edits plus a CD117 epitope edit, enabling non-genotoxic conditioning for sickle cell disease and beta-thalassemia treatment.
• BEAM-302 Liver-targeting lipid nanoparticle formulation of adenine base editing reagents designed to correct the E342K (PiZ) mutation causing alpha-1 antitrypsin deficiency, addressing both lung and liver manifestations with a single intravenous infusion.
• BEAM-301 Liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation causing glycogen storage disease Ia, potentially mitigating fasting hypoglycemia through G6Pase expression restoration.
• BEAM-304 Liver-targeting LNP formulation of base editing reagents designed to correct PAH gene mutations causing phenylketonuria, aiming to reduce toxic phenylalanine accumulation and enable diet normalization.
• In Vivo HSC Editing Program Research program for in vivo base editing for sickle cell disease and beta-thalassemia using lipid nanoparticle delivery targeted to hematopoietic stem cells, designed to eliminate the need for transplantation.

Quantifiable outcome

• Risto-cel achieved 60% average fetal hemoglobin (HbF) level in sickle cell disease patients, the highest level seen with gene therapy to date, with all 31 patients free of severe vaso-occlusive crises in the BEACON Phase 1/2 trial
• +3 more outcomes

Named customers1 record

Segments6 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

Five partnerships are on record, tiered minor and core.

• AlphaDetect (Alpha-1 Foundation)minorStrategic or Co-development Partner · 13 May 2026Beam Therapeutics became an inaugural industry sponsor of AlphaDetect, a nonprofit subsidiary of the Alpha-1 Foundation dedicated to accelerating detection of Alpha-1 Antitrypsin Deficiency. The sponsorship supports AlphaDetect's free genetic testing programs and provider education initiatives to improve early detection, addressing the >90% underdiagnosis rate. AlphaDetect is based in Durham, North Carolina.
• Precision Health Alliance (Duke Clinical Research Institute)minorStrategic or Co-development Partner · 14 April 2026Beam is a supporting member of the Precision Health Alliance coordinated by the Duke Clinical Research Institute (DCRI), a consortium that outlines ethical and regulatory themes for gene editing and precision medicine. The alliance aims to protect patients while advancing science, embedding equity, and building harmonized governance systems.
• PfizercoreStrategic or Co-development Partner · 24 February 2026Pfizer exercised its option for a global exclusive license to Beam's liver-targeted gene editing candidate following a four-year collaboration that began with a $300 million upfront payment and up to $1.05 billion in potential milestones. Pfizer leads clinical, regulatory, and manufacturing efforts globally. Beam can opt into co-development and co-commercialization upon completion of Phase 1/2 trials, with Pfizer covering 65% of costs and receiving the same proportion of profits. This represents a major validation and resource commitment from a top-tier pharmaceutical company.
• Lilly/Verve TherapeuticsminorStrategic or Co-development Partner · 1 September 2025Beam has non-dilutive collaboration agreements with Lilly/Verve. Eli Lilly acquired Verve Therapeutics for up to $1.3 billion in April 2026, potentially expanding the scope of collaboration. The deals validate Beam's base editing platform technology.
• Apellis PharmaceuticalsminorStrategic or Co-development Partner · 1 July 2025Beam has an active collaboration with Apellis Pharmaceuticals focused on autoimmune disorders and other undisclosed indications. The partnership leverages Beam's base editing technology for targets outside Beam's core focus areas.

Scale indicators8 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• CRISPR Therapeutics: CRISPR Therapeutics (NASDAQ: CRSP) is the most direct competitor to Beam. Its Casgevy therapy (developed with Vertex) is the first FDA-approved CRISPR-based gene therapy for sickle cell disease—exactly the indication Beam's risto-cel targets. Both companies are developing ex vivo gene-edited cell therapies for hemoglobinopathies, competing for the same treatment centers and patient populations.
• Editas Medicine: Editas Medicine (NASDAQ: EDIT) is a fellow gene editing company co-founded by Beam's co-founders Feng Zhang and J. Keith Joung. Editas pioneered CRISPR-based editing and has clinical programs in sickle cell disease and inherited retinal diseases, putting it in direct competition with Beam's hematology franchise on both technology and indication.
• Intellia Therapeutics: Intellia Therapeutics (NASDAQ: NTLA) is a clinical-stage gene editing company developing both in vivo (liver-targeted LNP) and ex vivo CRISPR-based therapies. Its in vivo transthyretin amyloidosis program (with Regeneron) and HAE program directly parallel Beam's in vivo liver-targeted strategy, putting both companies in head-to-head competition for the same delivery modality and disease archetypes.
• Prime Medicine: Prime Medicine (NASDAQ: PRME) is developing prime editing, a next-generation gene editing technology invented by Beam co-founder David R. Liu. Both companies compete in the precision gene editing space for rare monogenic diseases, with overlapping intellectual property foundations from the Liu laboratory at Harvard/Broad Institute.
• Verve Therapeutics: Verve Therapeutics, recently acquired by Eli Lilly for up to $1.3 billion, is an in vivo base editing company developing cardiovascular gene therapies. Verve pioneered in vivo gene editing in humans (for PCSK9/ANGPTL3 in cardiovascular disease), placing it in direct competition with Beam's in vivo liver-targeted programs and validating the broader in vivo editing thesis.
• bluebird bio: bluebird bio (NASDAQ: BLUE) is a gene therapy company with approved products for sickle cell disease (Lyfgenia) and beta-thalassemia. Both companies target the same patient populations via ex vivo lentiviral gene modification of hematopoietic stem cells, making bluebird a direct competitor in the sickle cell treatment center ecosystem.
• Sangamo Therapeutics: Sangamo Therapeutics (NASDAQ: SGMO) is a clinical-stage genomic medicine company developing zinc finger nucleases (ZFNs) for in vivo and ex vivo gene editing. While using older editing technology, Sangamo targets similar indications and pursues in vivo dosing strategies that overlap with Beam's liver-targeted programs.

Broad incumbents

• Vertex Pharmaceuticals: Vertex Pharmaceuticals (NASDAQ: VRTX) is the larger commercial partner on Casgevy and a broad incumbent in genetic medicine through its sickle cell, beta-thalassemia, and pain programs. Vertex has commercial reach, treatment center relationships, and CF/cash flow generation that dwarf Beam's clinical-stage position, making it both a competitor and a potential acquirer or partner.
• Pfizer: Pfizer (NYSE: PFE) is Beam's strategic partner for liver-targeted gene editing programs. Through the exercised option and $1.05B milestone structure, Pfizer effectively co-develops the in vivo liver franchise, providing global clinical, regulatory, and commercial infrastructure. Pfizer is also a broader incumbent in rare disease and gene therapy commercialization.

Emerging players

• Alnylam Pharmaceuticals: Alnylam Pharmaceuticals (NASDAQ: ALNY) is a nucleic acid therapeutics leader in RNAi, with overlapping rare disease focus. Several Beam executives (including CMO Amy Simon) come from Alnylam. While using different molecular modalities (RNAi vs. base editing), Alnylam's commercial rare disease infrastructure and AATD program (for hereditary ATTR) parallel Beam's strategic direction.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/beamtx

Revenue estimate

Valuation estimate

Number of profiles

Profiles1 record

Funding overview

Funding rounds6 records

Investors15 records

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M&A1 record

Investments

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Frequently asked questions about Beam Therapeutics