🧬BEAM-302: Genetic Correction for Alpha-1 Antitrypsin Deficiency

Beam Therapeutics announced promising early clinical trial results for BEAM-302, a novel gene editing therapy for alpha-1 antitrypsin deficiency (AATD). BEAM-302 uses a precise base editing mechanism to correct the genetic mutation responsible for the disease, leading to increased levels of functional AAT protein in patients. The initial trial demonstrated the therapy's safety and dose-dependent efficacy, with some patients achieving therapeutic protein levels. The company plans to continue its clinical development, including further dose escalation and a new trial segment for patients with mild to moderate liver disease, aiming to provide a long-term treatment option for AATD.