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News  ›  GlobeNewswire Inc.

Benitec Biopharma Provides Positive Long-Term Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial Demonstrating Robust Efficacy and Continued Durability of Response

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Benitec Biopharma Provides Positive Long-Term Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial Demonstrating Robust Efficacy and Continued Durability of Response

Benitec Biopharma announced positive 24-month follow-up results from its BB-301 gene therapy clinical trial for oculopharyngeal muscular dystrophy (OPMD). Patient 1 demonstrated deepening improvements in swallowing function with 60% reduction in post-swallow residue and 78% improvement in dysphagic symptom burden at 24 months. All four patients who completed the 12-month follow-up period met responder criteria, showing durable response to the treatment.

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BNTC   positive

The company reported robust and durable clinical efficacy results for BB-301 with all four study completers meeting responder criteria at 12 months, and the lead patient showing deepening improvements at 24 months. The treatment demonstrated significant improvements across multiple swallowing function metrics and symptom burden reduction, supporting advancement toward pivotal trials and FDA engagement.