Benitec Biopharma Announces Acceptance of Late- Breaking Oral Abstract for the BB-301 Phase 1b/2a Clinical Study at the Muscular Dystrophy Association Clinical and Scientific Conference

Core Insights - Benitec Biopharma Inc. announced the acceptance of a late-breaking oral abstract for the BB-301 Phase 1b/2a Clinical Treatment Study, focusing on subjects with Oculopharyngeal Muscular Dystrophy (OPMD) and moderate dysphagia, to be presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference on March 19, 2025 [1][2][3] Company Overview - Benitec Biopharma is a clinical-stage biotechnology company based in Hayward, California, specializing in gene therapy and developing novel genetic medicines using its proprietary "Silence and Replace" DNA-directed RNA interference (ddRNAi) platform [5] - The company aims to create therapeutics that can silence disease-causing genes while delivering functional replacement genes, targeting chronic and life-threatening conditions such as OPMD [5] Product Details - BB-301 is a modified AAV9 capsid designed to co-express codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) targeting the mutant PABPN1 gene responsible for OPMD [4] - The mechanism of BB-301 aims to halt the expression of the faulty gene while providing a functional version of the protein, which is believed to be uniquely effective for treating OPMD [4]