07:53 AM EDT, 03/25/2026 (MT Newswires) -- Beam Therapeutics ( BEAM ) said Wednesday that it has selected a 60 mg dose of its base-editing therapy BEAM-302 for pivotal development following updated phase 1/2 trial data in patients with Alpha-1 antitrypsin deficiency.
The company said the dose will support plans to pursue an accelerated approval pathway with the US Food and Drug Administration.
BEAM-302 is designed to directly correct mutations in the SERPINA1 gene using a liver-targeting lipid nanoparticle delivery system, the company said.
Data from 29 treated patients showed the therapy was generally well tolerated across dose levels up to 75 mg, with no serious adverse events reported as of the February 2026 cutoff, the company said. Most adverse events were mild to moderate infusion-related reactions or temporary liver enzyme elevations, it added.