Beam Therapeutics Announces First Patient Dosed in the Phase 1/2 Study of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD)

Company Overview - Beam Therapeutics is a biotechnology company focused on developing precision genetic medicines through base editing technology [6][8] - The company aims to establish a fully integrated platform for precision genetic medicines, including gene editing and delivery technologies, and is building internal manufacturing capabilities [6][14] Product Development - The first patient has been treated with BEAM-302, an investigational in vivo base editing medicine targeting severe alpha-1 antitrypsin deficiency (AATD) [8][4] - BEAM-302 is designed to correct the PiZ mutation associated with AATD, which affects liver and lung function [9][12] - The clinical trial for BEAM-302 is a Phase 1/2 open-label, dose-escalation study evaluating safety, pharmacodynamics, pharmacokinetics, and efficacy [12] Disease Context - AATD is an inherited genetic disorder that can lead to early onset emphysema, liver disease, and other complications [13] - Approximately 100,000 individuals in the U.S. have the PiZZ genotype, but only 10-15% are diagnosed [2][13] - Current treatments, such as intravenous AAT protein replacement, do not prevent ongoing lung function decline [2] Therapeutic Potential - BEAM-302 aims to provide a one-time therapy that corrects the genetic mutation, potentially reducing liver toxicity and increasing functional AAT protein levels [9][4] - The correction of the PiZ mutation is expected to be durable based on preclinical evidence, addressing the underlying pathophysiology of both liver and lung diseases [9][4]