Beam Therapeutics (BEAM) investor relations material

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.