Beam Therapeutics reported further progress on BEAM-302, its base-editing program for alpha-1 antitrypsin deficiency (AATD), and said the updated clinical dataset supports moving forward toward accelerated approval efforts. The company described data from an ongoing Phase 1/2 trial across 29 patients receiving three dose regimens and followed up to 12 months depending on the group. Beam reported that all dose groups produced enough functional alpha-1 antitrypsin protein to surpass an internal protective threshold, and it said circulating levels of the misfolded protein fell by more than 80% on average. Beam also flagged elevated liver enzymes in two patients, noting the events were asymptomatic and did not require treatment. Based on the dataset, the company selected a 60 mg go-forward single dose and plans an expansion later this year. Separately, Purdue University and Columbia University researchers published back-to-back Nature studies describing a naturally evolved RNA-guided CRISPR system capable of gene activation rather than cutting—an addition to the gene-editing toolchain that could broaden therapeutic and research applications.