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Beam Therapeutics Inc

Beam Therapeutics Inc., a biotechnology company, engages in the development of precision genetic medicines for patients suffering from serious diseases in the United States. Its programs in hematology and genetic disease portfolio include Ristoglogene autogetemcel, a patient-specific, autologous hematopoietic stem cell (HSC) therapy for the treatment of sickle cell disease; BEAM-302, a liver-targeting lipid nanoparticle (LNP) for the treatment of severe alpha-1 antitrypsin deficiency; BEAM-304, a liver-targeting LNP for the treatment of phenylketonuria; and BEAM-301, a liver-targeting LNP formulation for the treatment of glycogen storage disease type 1a. The company also develops the ESCAPE platform, which combines antibody-based conditioning with multiplex gene edited HSCs. In addition, it develops BEAM-103, an anti-CD117 monoclonal antibody that enables ESCAPE. The company has research collaboration agreement with Pfizer Inc., focusing on in vivo base editing programs for targets rare genetic diseases of the liver, muscle, and central nervous system; Verve Therapeutics, Inc., for cardiovascular disease treatments; and Orbital Therapeutics to design RNA for the prevention, treatment, or diagnosis of human disease. Beam Therapeutics Inc. was incorporated in 2017 and is based in Cambridge, Massachusetts.

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Biotech & Genomic Medicine▲

Beam Therapeutics Doses First Patient in Pivotal BEAM-302 Trial for AATD

Beam Therapeutics has dosed the first patient in the global pivotal cohort of its Phase 1/2 trial evaluating BEAM-302 for alpha-1 antitrypsin deficiency. The company also completed dosing for all adult and adolescent patients in the Phase 1/2 BEACON trial of risto-cel in sickle cell disease, with a biologics license application submission expected as early as year-end 2026. Updated BEAM-302 clinical data were selected for a late-breaking oral presentation at the European Respiratory Society Congress in September 2026. Beam received FDA clearance of its investigational new drug application for BEAM-304 in phenylketonuria and has initiated clinical start-up activities. The company ended the second quarter of 2026 with $1.2 billion in cash, cash equivalents and marketable securities, and expects its cash runway to support operating plans into mid-2029.
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ARK Invest buys $16.7M in SPCX shares, sells Deere and Twist Bioscience

ARK Invest purchased 122,807 shares of Space Exploration Technologies Corp for $16.7 million across several of its exchange-traded funds on Wednesday. The firm sold 6,833 shares of Deere & Co for nearly $4 million, extending a recent selling trend, and trimmed its position in Twist Bioscience Corp by selling 71,209 shares for $6,588,968. ARK's ARKG ETF increased its stake in Beam Therapeutics by acquiring 32,751 shares valued at $1,011,678, while the ARKK ETF sold 99,977 shares of 10X Genomics Inc for $4,577,946. Additionally, ARK invested in Kratos Defense and Security Solutions Inc, buying 34,123 shares across ARKQ and ARKX ETFs for a total of $1,718,434.
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Prime Medicine Wins Arbitration Ruling on PM647 Gene-Editing Drug

Prime Medicine announced a positive, binding resolution to its arbitration with Beam Therapeutics over their 2019 Collaboration and License Agreement. The Tribunal declared that PM647, the company's investigational Prime Editing drug for Alpha-1 Antitrypsin Deficiency, falls within Prime Medicine's defined field under the agreement, meaning Prime Medicine did not breach the agreement and owes no monetary damages to Beam Therapeutics. PM647 uses a universal liver lipid nanoparticle to correct the E342K mutation in the SERPINA1 gene, the most common disease-causing mutation in AATD, and has shown high editing efficiency and restored healthy-range protein levels in fully humanized mouse models at clinically relevant doses.
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Biotech & Genomic Medicine
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Intellia Therapeutics Stock Surges 38% in a Month on Pipeline Progress

Intellia Therapeutics shares have surged 38% over the past month, outperforming the industry, sector, and S&P 500, driven by positive regulatory and clinical milestones for its lead candidate lonvoguran ziclumeran. The phase III HAELO study evaluating lonvoguran ziclumeran for hereditary angioedema met its primary endpoint and a key secondary endpoint in April 2026, and additional data in June 2026 showed an 89% reduction in monthly attacks requiring on-demand treatment and a 91% reduction in moderate-to-severe attacks compared with placebo. Intellia initiated a rolling biologics license application submission to the FDA in April 2026 and plans a commercial launch in the first half of 2027 upon potential approval. The company is also advancing nexiguran ziclumeran in two late-stage studies for ATTR amyloidosis after the FDA lifted a clinical hold earlier this year. Despite a premium valuation of 3.79 times trailing book value versus the industry's 3.69, the Zacks Consensus Estimate for 2026 loss per share has narrowed from $3.46 to $3.18 over the past 60 days, and the 2027 loss estimate has narrowed from $1.01 to 65 cents. Zacks rates the stock a Hold, citing pipeline promise and narrowing losses but noting competition from CRISPR Therapeutics and Beam Therapeutics and the lack of marketed products.
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New CRISPR 'DNA Shredder' Could Expand Gene-Editing Market

A newly discovered CRISPR-based tool that destroys diseased cells by shredding their DNA is generating excitement in scientific circles, with potential long-term implications for gene-editing companies like CRISPR Therapeutics, Intellia Therapeutics, and Beam Therapeutics. Unlike traditional gene editing, this programmable cell-killing approach reads a signal unique to a diseased cell and cuts its DNA into fragments faster than the cell can repair, leading to cell death while sparing healthy cells. A May paper in Nature showed the therapy cutting lung cancer cell growth by about 50% in a lab dish, matching the chemotherapy drug cisplatin, and a June Nature paper extended the approach to target a gene mutated in 40% to 50% of all cancers. None of the major public CRISPR companies currently have this technology in their pipelines, though private German biotech Akribion Therapeutics is developing a program targeting HPV-positive head and neck cancer. The new papers significantly expand the addressable market for CRISPR-based medicine, and the incumbents may have an advantage due to their existing drug-delivery infrastructure.
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Biotech & Genomic Medicine
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FDA Clears Beam Therapeutics' IND for Gene-Editing Therapy BEAM-304

Beam Therapeutics shares rose 5% after the FDA cleared its investigational new drug application for BEAM-304, a gene-editing therapy for phenylketonuria. The clearance allows Beam to advance its liver-targeted lipid nanoparticle-based treatment designed to correct mutations in the PAH gene, potentially restoring normal phenylalanine metabolism. The company plans to begin a phase I/II open-label study initially in patients with the R408W mutation, assessing safety, tolerability, and blood phenylalanine reduction. Phenylketonuria affects approximately 20,000 people in the United States and currently has no approved curative treatments. Beam's stock has gained 23.2% year to date, outperforming the broader industry's 1% decline.
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