A Phase 1/2 Dose-Exploration and Dose-Expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and/or Liver Disease

Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and/or Liver Disease

Phase 1 (Dose Exploration) Primary Endpoint: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), Phase 2 (Dose Expansion) Primary Endpoint: Absolute blood levels of total AAT, Z-AAT, and M-AAT

Secondary endpoints apply to the entire study unless preceded with “Phase 1 (Dose Exploration)” or “Phase 2 (Dose Expansion).”, • Phase 1 (Dose Exploration): Absolute blood levels of total AAT, • Phase 2 (Dose Expansion): Rates of TEAEs and SAEs, • Change from baseline in blood levels of total AAT, • Blood levels of Z-AAT and M-AAT, • Change from baseline in blood levels of functional AAT, • Area under the plasma concentration-time curve (AUC) from time zero to the time of the last measurable concentration (AUC0-last), AUC from time zero to infinity (AUC∞), maximum concentration (Cmax), time to maximum concentration (Tmax), terminal half-life (t½) for the plasma levels of mRNA, gRNA, and lipids (ALC-0366 and ALC-0159), • Blood anti-BEAM-302 antibodies and anti-ABE antibodies

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Ireland, Netherlands