BEAM-302 is being evaluated in a Phase 1/2, open-label, dose exploration and dose expansion clinical trial to investigate its safety, tolerability, pharmacodynamics, pharmacokinetics and efficacy. Topline data from 29 patients treated with BEAM-302 as of a February 10, 2026 data cutoff date were reported in March 2026. 

Based on feedback from the U.S. Food and Drug Administration (FDA), Beam intends to pursue an accelerated approval pathway for BEAM-302. To support a future biologics licensing application (BLA) submission, the company anticipates enrolling approximately 50 additional patients with AATD-associated lung disease, with or without liver disease, in an expansion of the ongoing open-label Phase 1/2 trial. Beam expects to initiate this pivotal cohort in the second half of 2026. In addition, Beam expects to present detailed and updated BEAM-302 data at a medical congress in 2026.