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
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