Beam Therapeutics presented updated clinical proof-of-concept data for BEAM-302, a base editing program for alpha-1 antitrypsin deficiency (AATD), reporting restoration of functional protein production alongside reductions in misfolded protein levels. The company said results from its ongoing Phase 1/2 trial include findings across 29 patients treated with three dose groups and followed for about one to 12 months. Beam reported average circulating misfolded protein reductions of more than 80% and said functional protein met a threshold believed to be protective in all three groups. The company noted liver enzyme elevations in two patients that were asymptomatic and did not require treatment. With the dataset supporting a move toward a single 60 mg go-forward dose, Beam plans to expand later this year and pursue an accelerated approval pathway with the FDA, positioning the program as the first to both restore AAT production and reduce toxic accumulation.