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

Platform technology and clinical validation

• Base editing enables precise, single-base changes in DNA, expanding therapeutic possibilities beyond traditional CRISPR approaches.

• Clinical proof of concept achieved in both ex vivo and in vivo base editing, demonstrating repeatability and scalability across multiple programs.

• Platform allows for efficient development of new therapies, reducing risk and investment for subsequent programs.

BEAM-101 and sickle cell disease program

• BEAM-101 upregulates fetal hemoglobin via precise promoter edits, achieving protective hemoglobin ratios in all evaluable patients.

• Patients reached carrier-like hemoglobin profiles, with over 60% fetal and under 40% sickle globin, indicating robust disease correction.

• No vaso-occlusive crises observed; anemia resolution and EPO normalization suggest strong functional benefit.

• Registrational Beacon trial has dosed 30 patients, aiming for a BLA filing in late 2026 after 15 months of follow-up.

• BEAM-101 offers potential manufacturing and engraftment advantages over existing therapies, with a best-in-class profile.

Next-generation programs: Escape, BEAM-103, and BEAM-104

• Escape technology aims to expand curative therapy to all sickle cell patients by replacing chemotherapy with antibody-based conditioning.

• BEAM-103 (antibody) and BEAM-104 (cell product) are advancing, with BEAM-103 entering healthy volunteer studies in 2025.

• In vivo approaches using lipid nanoparticles are being explored for broader, scalable access.

• Escape could eventually replace or complement BEAM-101, depending on efficacy in broader patient populations.