Beam Therapeutics (BEAM) 44th Annual J.P. Morgan Healthcare Conference summary

Vision and technology

• Focus on base editing to provide lifelong, one-time curative therapies for rare and common diseases, enabling durable correction with less genotoxicity than traditional gene editing.

• Base editing offers precise, single-letter gene changes without double-stranded breaks, resulting in predictable, reproducible, and safer outcomes.

• Platform is highly adaptable, rapidly programmable, and supported by scalable manufacturing and a flexible regulatory framework, enabling rapid expansion of the pipeline.

• Predictability streamlines R&D, reduces risk, accelerates regulatory pathways, and increases physician and patient confidence.

Financial strength and growth

• Ended 2025 with $1.25 billion in cash, including $255.1 million from the Orbital acquisition, and completed $500M financing in 2025.

• Expected operating runway extends into 2029, supporting risto-cel launch and BEAM-302 pivotal development.

• Financial strength supports growth, commercialization plans, and commercial build for upcoming launches.

Clinical and regulatory progress

• Achieved first human proof of concept for in vivo gene correction with BEAM-302, with patients achieving protective protein levels and significant reduction of disease-causing protein.

• Over 25 patients treated in the BEAM-302 trial; FDA alignment reached for accelerated approval based on established biomarkers, with no new assays required.

• Phase 1/2 trial for BEAM-302 is rapidly enrolling, with expansion to enroll 50 more patients and updated data expected by end of Q1 2026.

• BEAM-301 for glycogen storage disease Ia progressing, with initial clinical data expected by year-end 2026.

• Additional liver program to be announced in the first half of the year.