The latest announcement is out from Beam Therapeutics ( (BEAM) ).
On March 10, 2025, Beam Therapeutics announced positive initial data from its Phase 1/2 trial of BEAM-302 for treating alpha-1 antitrypsin deficiency (AATD). The trial demonstrated that BEAM-302, a liver-targeting lipid-nanoparticle formulation, was well tolerated and showed dose-dependent increases in functional AAT levels and decreases in mutant Z-AAT levels. This marks the first clinical genetic correction of a disease-causing mutation, offering potential treatment for both lung and liver disease in AATD patients. The company plans further dose escalation and expects to present more data in the second half of 2025.
More about Beam Therapeutics
Beam Therapeutics Inc. is a biotechnology company focused on developing precision genetic medicines through base editing. The company targets genetic disorders, with a market focus on diseases like alpha-1 antitrypsin deficiency (AATD), using liver-targeting lipid-nanoparticle formulations to correct disease-causing mutations.
YTD Price Performance: 15.30%
Average Trading Volume: 1,283,061
Technical Sentiment Consensus Rating: Sell
Current Market Cap: $2.38B
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