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Beam Therapeutics (BEAM) investor relations material

Beam Therapeutics H.C. Wainwright 27th Annual Global Investment Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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H.C. Wainwright 27th Annual Global Investment Conference summary8 Sep, 2025

Platform overview and technology differentiation

  • Base editing enables precise, single-base changes in DNA, avoiding double-stranded breaks and allowing correction of point mutations that cause genetic diseases.

  • The platform's programmability and repeatability allow for efficient development of multiple therapies by changing only the targeting element.

  • Successful ex vivo and in vivo proof-of-concept studies validate the platform's broad applicability and reduce risk for subsequent programs.

  • The approach can fundamentally change drug development economics by de-risking future pipeline candidates.

BEAM-101 sickle cell program

  • BEAM-101 upregulates fetal hemoglobin via precise promoter edits, reducing sickle protein and normalizing blood profiles.

  • All 17 evaluable patients in a recent study achieved protective hemoglobin ratios, mirroring healthy carriers.

  • Corrected cells perform as well as or better than trait carriers in functional assays.

  • Anemia resolution, EPO normalization, and absence of vaso-occlusive crises indicate robust clinical benefit.

  • The Beacon trial has dosed 30 patients, aiming for a BLA filing after 15 months of follow-up, targeting late 2026 for data readiness.

Competitive positioning and next-generation programs

  • BEAM-101 offers potential manufacturing and clinical advantages, including rapid engraftment and strong hematologic outcomes.

  • The Escape platform (BEAM-103/104) aims to eliminate chemotherapy by using antibody-based conditioning and edited cells resistant to the antibody.

  • Escape could expand curative therapy to the broader sickle cell population and may eventually replace or complement BEAM-101, depending on efficacy.

  • In vivo editing and scalable delivery technologies are being developed to reach global patient populations.

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Frequently asked questions

Beam Therapeutics Inc is a biotechnology company focused on the development of precision genetic medicines using base editing technology. The company’s approach aims to correct genetic mutations at the single-base level to treat various diseases, including genetic disorders and cancers. It conducts research and development in-house and through collaborations. Beam Therapeutics Inc is headquartered in Cambridge, Massachusetts, and its shares are listed on the NASDAQ.

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