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BEAM-302 demonstrated durable, functional correction of AATD, with a favorable safety profile. In the 60 mg group, all patients maintained protective AAT levels, while the pivotal global cohort is enrolling rapidly.
BEAM-302 showed durable, one-time correction of AAT biology in AATD patients, achieving protective AAT levels, reducing disease-driving Z-AAT, and maintaining a favorable safety profile. Regulatory alignment supports an accelerated approval pathway.