other material
confidence high
sentiment positive
materiality 0.85
Beam reports positive initial Phase 1/2 data for BEAM-302 in AATD; total AAT exceeds protective threshold
Beam Therapeutics Inc.
- BEAM-302 well tolerated at all doses; no serious AEs or dose-limiting toxicities observed.
- Single 60 mg dose achieved mean total AAT of 12.4 µM at Day 28, exceeding 11 µM protective threshold.
- Mutant Z-AAT reduced up to 78% from baseline at 60 mg dose level.
- First clinical evidence of precise genetic correction of a disease-causing mutation via base editing.
- Dose escalation to continue; further data expected at medical conference in H2 2025; Part B dosing planned H2 2025.