Benitec Biopharma Announces Late Breaking Oral Abstract Presentation on BB-301 Phase 1b/2a Clinical Study at the 29th Annual Congress of the World Muscle Society

Core Insights - Benitec Biopharma Inc. announced that a Late Breaking Abstract on its gene therapy BB-301 has been accepted for oral presentation at the 29th Annual Congress of the World Muscle Society in Prague from October 8-12, 2024 [1] Group 1: 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 platform [6] - The company is headquartered in Hayward, California, and aims to create therapeutics for chronic and life-threatening conditions, including Oculopharyngeal Muscular Dystrophy (OPMD) [6] Group 2: Product Information - BB-301 is a modified AAV9 capsid gene therapy designed to co-express a functional version of the Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) targeting the mutant PABPN1 [5] - The mechanism of BB-301 aims to silence the expression of the faulty mutant PABPN1 while providing a functional replacement, positioning it uniquely for treating OPMD [5] Group 3: Disease Context - Oculopharyngeal Muscular Dystrophy (OPMD) is a rare, progressive muscle-wasting disease caused by mutations in the PABPN1 gene, leading to severe swallowing difficulties and other debilitating symptoms [4] - Currently, there are no effective drug therapies for OPMD, and existing clinical and surgical interventions are limited in their effectiveness [4]