Beam Therapeutics reported positive proof-of-concept readouts for its base editing therapy BEAM-302 in alpha-1 antitrypsin deficiency, showing restored functional protein production and meaningful reductions in misfolded circulating protein. The company said 29 treated patients across three dose cohorts surpassed a protective threshold believed to safeguard liver and lungs. Beam reported elevated liver enzyme levels in two patients but said both were asymptomatic and did not require treatment. With the dataset supporting dose selection, Beam plans to proceed with a single 60 mg dose and launch an expansion later this year while pursuing an accelerated approval pathway with the FDA. The update strengthens the base-editing narrative in monogenic disease by pairing functional protein restoration with reduction of toxic protein accumulation, with Beam positioning BEAM-302 for a rapid next-stage push.