Beam Therapeutics presents BEAM-302 data at conference

Beam Therapeutics Inc. (NASDAQ: BEAM) presented clinical data from its BEAM-302 Phase 1/2 trial for alpha-1 antitrypsin deficiency at the American Thoracic Society International Conference in Orlando, Florida.

The presentation included topline data from 29 patients treated with BEAM-302 as of February 10, 2026. The data encompassed safety results, efficacy durability and reduction in human neutrophil elastase activity following BEAM-302 treatment.

BEAM-302 is designed to correct the PiZ mutation in patients with alpha-1 antitrypsin deficiency through base editing technology. The treatment targets the liver using lipid-nanoparticle formulation to make an A-to-G correction of the mutation.

Based on feedback from the U.S. Food and Drug Administration, Beam plans to pursue an accelerated approval pathway for BEAM-302. The company expects to enroll approximately 50 additional patients with AATD-associated lung disease in an expansion of the ongoing Phase 1/2 trial to support a future biologics licensing application submission.

Beam anticipates initiating this pivotal cohort in the second half of 2026. The company also expects to present updated BEAM-302 data at a medical congress in 2026.

Alpha-1 antitrypsin deficiency is an inherited genetic disorder that can cause early onset emphysema and liver disease. The company estimates more than 100,000 individuals in the U.S. have two copies of the Z allele, though only about 10% of patients are thought to have been diagnosed.

Information is based on a company press release statement.

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