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Beam Therapeutics Announces Compelling Updated Clinical Data from the Ongoing Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) to Support Advancement to Pivotal Development
Treatment with 60 mg of BEAM-302 Led to Mean Steady-state Total AAT Level of 16.1 µM and All Patients Consistently and Durably Above the 11 µM Protective AAT
About this update from Beam Therapeutics Inc.
[{"type":"text","content":"Treatment with 60 mg of BEAM-302 Led to Mean Steady-state Total AAT Level of 16.1 µM and All Patients Consistently and Durably Above the 11 µM Protective AAT Threshold with up to 12 Months of Follow-up Corrected M-AAT Comprised 94% of Total AAT with a Concomitant 84% Reduction in Mutant Z-AAT Following BEAM-302 60 mg Treatment Post-treatment Inducibility of AAT Observed During Respiratory Infection with a Patient Reaching ~30 µM Total AAT, Retaining 95% M-AAT Composition Well-tolerated Safety Profile Observed with Single Doses of BEAM-302 up to 75 mg; Safety Consistent Across Single-dose Part A and Part B Cohorts 60 mg Selected as Optimal Biological Dose, Supported by Strong Safety and Efficacy Profile Across Single-dose Cohorts; Global Pivotal Cohort Expected to Initiate in Second Half of 2026 Beam to Host Investor Webcast Today, March 25, 2026, at 8:00 a.m. ET CAMBRIDGE, Mass., March 25, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced updated safety and efficacy data from the ongoing Phase 1/2 trial of BEAM-302 and the selection of 60 mg as the optimal biological dose to advance into pivotal development to support potential accelerated approval. BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation designed to directly correct the underlying genetic mutation that causes the severe form of alpha-1 antitrypsin deficiency (AATD) through base editing. “AATD is a serious genetic disease that can lead to significant liver disease over an individual’s lifespan along with progressive lung disease in adults, often leaving patients with limited treatment options and challenging, lifelong disease management,” said Jeffrey Teckman, M.D., professor of pediatrics, Saint Louis University School of Medicine. “What makes BEAM-302 particularly compelling is its ability to directly correct the underlying genetic mutation in the SERPINA1 gene that drives both lung and liver manifestations of the disease. By enabling the liver to produce corrected M-AAT for the first time while reducing the toxic mutant protein, this approach has the potential to fundamentally transform how we as clinicians treat AATD and represents a meaningful advance for patients.” BEAM-302 Phase 1/2 Clinical Trial Data UpdateBEAM-302 is being evalua...