Core Insights - The independent Data Safety Monitoring Board (DSMB) has recommended the continuation of subject enrollment for the Phase 1b/2a Clinical Treatment Study of BB-301 after reviewing safety data for all six subjects in Cohort 1 [1][2] - Enrollment for Cohort 2 is expected to begin in Q4 2025 following the positive DSMB recommendation [1][2] - The sixth and final subject of Cohort 1 was safely treated with a low dose of BB-301 in April 2025, indicating a benign safety profile associated with the local route of administration [3] Company Overview - Benitec Biopharma Inc. is a clinical-stage biotechnology company focused on developing novel genetic medicines using its proprietary "Silence and Replace" DNA-directed RNA interference (ddRNAi) platform [5] - The company aims to treat chronic and life-threatening conditions, including Oculopharyngeal Muscular Dystrophy (OPMD), through its innovative therapeutic approaches [5] - BB-301 is designed to silence the expression of faulty mutant PABPN1 while allowing for the expression of a functional version of the protein, positioning it uniquely for OPMD treatment [4]
Benitec Biopharma Provides Operational Updates