Beam Therapeutics Inc. (NASDAQ:BEAM) on Tuesday presented updated Phase 1/2 trial data for BEAM-302, an experimental base-editing therapy for alpha-1 antitrypsin deficiency (AATD).
AATD is a hereditary disorder caused by low levels of protective AAT protein, leading to lung damage (emphysema, COPD) and liver disease.
The company shared these findings at the European Respiratory Society Congress 2026, showcasing the genetic medicine’s potential to directly correct the root cause of both liver and lung complications associated with the disease.
Promising Efficacy and Safety Profile
The clinical trial evaluated single doses of BEAM-302 across two groups: Part A for patients with AATD-related lung disease and Part B for those with mild to severe liver disease.
As of the June 2026 data cutoff, results from 29 patients demonstrated an acceptable safety profile. The most frequent side effects were mild-to-moderate infusion-related reactions, affecting 41% of participants.
Researchers also observed mostly mild, temporary liver enzyme elevations.
Patients receiving a 60 mg dose achieved sustained, functional alpha-1 antitrypsin (AAT) levels well above the protective 11 µM threshold. The therapy also reduced human neutrophil elastase activity and cut mutant Z-AAT proteins by 84% in both cohorts.
Additionally, BEAM-302 decreased toxic protein aggregates known to worsen liver disease and amplify lung inflammation. After treatment, newly produced, corrected M-AAT made up 93% of circulating AAT, exceeding the levels typically seen in genetic carriers.
Path To Accelerated Approval
Following discussions with the U.S. Food and Drug Administration (FDA), Beam plans to pursue an accelerated approval pathway for the therapy.
The submission will evaluate AAT biomarkers over a 12-month period using the 60 mg dose as the primary endpoint.
To support a future biologics license application, the biotechnology firm anticipates enrolling roughly 50 additional patients with AATD-associated lung disease into an expansion of the ongoing trial.
Beam initiated dosing for this pivotal global cohort in July.
BEAM Stock Price Activity: Beam Therapeutics shares were down 7.17% at $27.53 during premarket trading on Tuesday, according to Benzinga Pro data.
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