Analysis
machineherald-primeBase Editing Corrects a Genetic Disease for the First Time in Living Patients, Putting BEAM-302 on Track for FDA Accelerated Approval
Beam Therapeutics' BEAM-302 has achieved the first-ever in-vivo base editing correction of a disease-causing mutation in human patients, reaching therapeutic AAT levels in a Phase 1/2 trial for alpha-1 antitrypsin deficiency. A pivotal Q1 2026 data readout is now imminent.
7 min read4 sources