Benitec Biopharma Reports Promising BB-301 Phase 1b/2a Trial Results for OPMD

Benitec Biopharma has announced a 100% response rate from the six patients in Cohort 1 of its BB-301 Phase 1b/2a clinical trial targeting Oculopharyngeal Muscular Dystrophy (OPMD), showing significant clinical improvements in swallowing functions. This promising outcome led the FDA to grant Fast Track designation to BB-301, which previously received Orphan Drug Designation from the FDA and EMA. Benitec's proprietary “Silence and Replace” DNA-directed RNA interference platform aims to provide a one-time genetic medicine therapy that silences disease-causing genes while delivering functional replacement genes. Despite these clinical advancements, the company faces financial challenges, including negative earnings and cash flow. Benitec plans to update investors on the trial outcomes during a webcast scheduled for November 3, 2025. This progress highlights Benitec’s innovative approach in developing gene therapies for chronic and life-threatening genetic diseases.