Beam reports durable protein correction with experimental AATD therapy

Beam Therapeutics ($BEAM) presented updated Phase 1/2 results for BEAM-302, its experimental gene-editing treatment for alpha-1 antitrypsin deficiency. The presentation covered 29 patients given a single dose, with follow-up extending to 18 months. In the 60 mg cohorts, total AAT protein remained above the stated protective threshold, while circulating disease-associated Z-AAT fell 84%. The company is already dosing a larger pivotal cohort.

Safety remains part of the assessment: mild-to-moderate infusion reactions occurred in 41% of patients, and one patient with underlying liver disease experienced temporary Grade 3 liver-enzyme elevations without increased bilirubin or a need for treatment. These are early, open-label biomarker results, not proof of a cure. Beam plans to pursue a potential accelerated-approval pathway using longer-term AAT measurements.

Sources:

• Beam Therapeutics — Phase 1/2 data and pivotal development update