Beam Therapeutics provided updated evidence supporting its base editing program BEAM-302 in alpha-1 antitrypsin deficiency (AATD). In the ongoing Phase 1/2 study, Beam reported that patients across dose groups reached a protective threshold for functional AAT protein production. Beam’s interim dataset included results from 29 patients treated across three dose regimens, with Beam highlighting that circulating levels of misfolded AAT were reduced by over 80% on average. The company also reported elevated liver enzymes in two patients, while both cases remained asymptomatic and did not require treatment. Based on the data, Beam said it will proceed with a single 60 mg dose and expand the trial later this year. The company intends to pursue an accelerated approval pathway with the FDA for BEAM-302. The update matters for the gene editing field because it pairs a mechanistically targeted DNA correction strategy with regulators’ reliance on early proof of pharmacodynamic effects in rare disease settings.