Beam Therapeutics reported positive progress in its Phase 1/2 base editing program BEAM-302 for alpha-1 antitrypsin deficiency (AATD), saying the treatment restored protective protein production and reduced circulating misfolded AAT levels. The company said data were collected from 29 patients across multiple dose groups, with patients followed up to roughly 12 months. Beam noted that across dose groups, functional AAT protein surpassed a threshold believed to be protective, and circulating levels of the misfolded protein fell by more than 80% on average. The company reported elevated liver enzymes in two patients but said the events were asymptomatic and did not require treatment. Beam plans to proceed with a go-forward single 60 mg dose and expand the trial later this year, while pursuing an accelerated approval pathway with the FDA. If validated by ongoing confirmatory evidence, the program could strengthen Beam’s positioning in the growing base-editing field by demonstrating both target engagement and biomarker-level clinical impact in a genetically defined disease.