Beam Therapeutics (BEAM) Wells Fargo 21st Annual Healthcare Conference summary

Technology and platform overview

• Base editing enables precise, single-letter genetic corrections without double-stranded breaks, offering predictable and uniform genome modifications for various diseases.

• Applications include ex vivo and in vivo therapies for sickle cell disease, alpha-1 antitrypsin deficiency, PKU, GSD1A, and other metabolic disorders, aiming for one-time functional cures.

Recent clinical data and efficacy

• New data show patients with alpha-1 antitrypsin deficiency reach protective protein levels (≥11 micromolar), with durable effects over 18+ months and over 80% reduction in toxic Z protein.

• Functional assays confirm the produced protein is fully functional, with over 90% M form and under 10% Z form, and reductions in harmful Z-AAT polymers and neutrophil elastase.

• Efficacy in liver disease patients mirrors lung patients, with tripled alpha-1 levels and mostly mild, transient safety findings.

Safety and patient experience

• Safety profile remains favorable, with most liver enzyme elevations being mild and transient; one Grade 3 event resolved without intervention.

• Patients show strong interest in one-and-done therapies, citing burdensome current treatments and the appeal of durable, inducible correction.