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Beam Therapeutics reports promising Phase 1/2 data for BEAM-302 gene therapy in Alpha-1 Antitrypsin Deficiency.

Company Fundamentals
18 May 2026
GlobeNewsWire
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Bullish
pluang ai news

Beam Therapeutics presented new clinical data from its Phase 1/2 trial of BEAM-302, a gene editing therapy targeting Alpha-1 Antitrypsin Deficiency (AATD), showing safety, durable efficacy, and reduction in disease markers. The therapy aims to correct the root genetic cause of AATD with a single dose, potentially treating both lung and liver disease manifestations. Beam plans to expand the trial with 50 more patients and pursue accelerated FDA approval, aiming for a pivotal study start in late 2026. This approach represents a significant advancement in treating AATD and gene editing medicine overall.

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