India, April 5 -- Beam Therapeutics Inc. (BEAM) presented additional data from the Phase 1/2 clinical trial of BEAM-302 in patients with alpha-1 antitrypsin deficiency (AATD) at the 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress, taking place April 4-5, 2025, in Lisbon, Portugal.
Positive initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 were previously reported in March 2025, establishing clinical proof of concept as a potential treatment for AATD and in vivo base editing. Preliminary results from the first three single-ascending dose cohorts demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable dose-dependent correction of the disease-causing ...