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

Platform overview and technology differentiation

• Base editing enables precise, single-base changes in DNA without double-stranded breaks, improving predictability and efficiency over first-generation CRISPR methods.

• The platform's programmability allows rapid expansion to new targets, reducing risk and investment for subsequent programs.

• Clinical proof of concept in both ex vivo and in vivo settings validates the platform's repeatability and scalability.

Sickle cell disease program (BEAM-101 and Escape)

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

• Patients showed over 60% fetal hemoglobin and under 40% sickle globin, matching or exceeding carrier (trait) levels.

• No vaso-occlusive crises (VOCs) observed; anemia resolution and EPO normalization 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.

• Escape program aims to expand curative therapy to the broader sickle cell population by eliminating chemotherapy, using antibody-based conditioning and dual edits.

Competitive positioning and cost considerations

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

• Current gene therapies are priced at $2–3 million, justified by lifetime cost savings; cost of goods expected to decrease with scale and in vivo approaches.

• In vivo therapies promise greater scalability and lower costs, supporting global access.

• General biologics margins of 20–30% are considered achievable and sustainable.