Beam Therapeutics(BEAM) Newsfilter·2025-04-05 11:00Core Insights - Beam Therapeutics presented new data from the Phase 1/2 clinical trial of BEAM-302, showing a mean of 91% corrected M-AAT in circulation at Day 28 in the 60 mg cohort, alongside a 79% decrease in mutant Z-AAT levels [1][3][4] Group 1: Clinical Trial Data - The Phase 1/2 trial of BEAM-302 has demonstrated positive initial safety and efficacy, establishing clinical proof of concept for treating alpha-1 antitrypsin deficiency (AATD) [2] - Updated biomarker data from the 60 mg cohort indicated that the proportion of corrected M-AAT reached a mean of 91% of total AAT in circulation, surpassing levels typically seen in patients with the MZ genotype [3] - The treatment led to a mean decrease of 79% in circulating mutant Z-AAT from baseline as of Day 28 [3] Group 2: Future Plans and Developments - Beam Therapeutics plans to continue the dose-escalation portion of the trial, initiating a fourth cohort at 75 mg and expects to present further data at a medical conference in the second half of 2025 [4] - The company aims to dose the first patient in Part B of the trial, which will include AATD patients with mild to moderate liver disease, in the second half of 2025 [4] - The investigational drug application (IND) for BEAM-302 has been cleared by the U.S. FDA, allowing the activation of trial sites in the U.S. [4] Group 3: About BEAM-302 and AATD - BEAM-302 is a liver-targeting lipid-nanoparticle formulation designed to correct the PiZ mutation, which is prevalent among patients with severe AATD [5] - The treatment aims to reduce the aggregation of mutant AAT protein, generate therapeutic levels of corrected protein, and increase total AAT in circulation, addressing the underlying pathophysiology of liver and lung disease [5] - AATD is an inherited genetic disorder that can lead to early onset emphysema and liver disease, with an estimated 100,000 individuals in the U.S. affected by the severe form of the disease [6][8]