Beam Therapeutics (BEAM) investor relations material

Technology and platform overview

• Base editing technology enables precise single-nucleotide changes without double-stranded DNA breaks, improving safety and versatility across cell types.

• The platform can correct point mutations, introduce stop codons, and modify protein active sites for diverse therapeutic strategies.

AATD 302 program updates and development path

• Phase 1/2 dose escalation trial includes multiple dosing cohorts, with key data updates planned for early 2026, focusing on higher-dose groups and patients with more severe liver involvement.

• Early data show therapeutically relevant AAT protein levels, with plans to accelerate approval based on biomarker data and functional protein restoration.

• Regulatory strategy aims for accelerated approval using biomarker endpoints, with a base case of demonstrating gene function restoration and Z-AAT reduction.

• If accelerated approval is not granted, a clinical endpoint study using CT densitometry and FEV1 is considered, with both paths expected to require relatively small patient numbers.

• Biomarker thresholds and durability of effect are supported by rapid and robust kinetics, with follow-up data available within weeks to months post-dosing.

Sickle cell disease program highlights

• Upcoming ASH data will include additional patients and longer follow-up, showing high fetal hemoglobin upregulation, rapid anemia resolution, and normalization of hemolysis parameters.

• Fastest neutrophil and platelet engraftment times reported, attributed to the absence of double-stranded breaks and optimized, automated manufacturing.

• Fewer mobilization cycles and shorter hospital stays compared to competitors, potentially doubling patient throughput.

• Registrational path expected to mirror that of CASGEVY, with similar approval package requirements.