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A representative image illustrating BEAM-302 base-editing research as Beam Therapeutics begins pivotal testing of its one-time investigational treatment for alpha-1 antitrypsin deficiency affecting the lungs and liver.

Pharma & Biotech

First patient dosed in BEAM-302 pivotal cohort for alpha-1 antitrypsin deficiency

by Soujanya Ravi
August 4, 2026
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BEAM-302 enters pivotal AATD testing after early base-editing data. Read how the study could support an accelerated approval pathway.

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Representative image of a clinician reviewing skeletal imaging, illustrating Mirum Pharmaceuticals and Incyte’s zilurgisertib Phase 2 progress in fibrodysplasia ossificans progressiva and the rare disease treatment questions facing regulators.

Pharma & Biotech

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Pharma & Biotech

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Representative image of biopharma laboratory research. Gilead Sciences and Merck’s latest Phase 3 updates highlight the promise of once-weekly HIV treatment with islatravir and lenacapavir, while raising fresh questions over Trodelvy’s lung cancer expansion strategy.

Pharma & Biotech

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clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immuno-oncology immunotherapy Inc. Johnson & Johnson medical devices oncology precision oncology rare disease U.S. Food and Drug Administration United States Food and Drug Administration

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