Benitec Biopharma to Present BB-301 Phase 1b/2a Study at World Muscle Society Congress

HAYWARD, Calif., Sept. 18, 2024 – Benitec Biopharma Inc., a clinical-stage biotechnology company specializing in gene therapy, has announced that a Late Breaking Abstract on BB-301 has been accepted for oral presentation at the 29th Annual Congress of the World Muscle Society. The event will be held in Prague, Czech Republic, from October 8-12, 2024. The presentation, scheduled for October 12, 2024, will discuss interim clinical data from a Phase 1b/2a open-label, dose-escalation study evaluating BB-301's safety and clinical activity in treating Oculopharyngeal Muscular Dystrophy (OPMD) with dysphagia.

The presentation titled “Interim Clinical Data Summary: A Phase 1b/2a Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of an AAV9-based gene therapy (BB-301) Administered to Subjects with Oculopharyngeal Muscular Dystrophy (OPMD) with Dysphagia” will be given by Professor Milan R. Amin, M.D., of New York University Grossman School of Medicine.

OPMD is a rare and progressive muscle-wasting disease caused by mutations in the poly(A)-binding protein nuclear 1 (PABPN1) gene. Characterized by swallowing difficulties, limb weakness, and eyelid drooping, the disease often leads to severe complications such as chronic choking, regurgitation, and aspiration pneumonia, which can be fatal. Current clinical and surgical options offer limited effectiveness and do not target the underlying muscle weakness.

BB-301 is a novel gene therapy utilizing a modified AAV9 capsid to deliver a bifunctional construct. This construct promotes the co-expression of a codon-optimized PABPN1 and two small inhibitory RNAs (siRNAs) aimed at the mutant PABPN1. The siRNAs are designed to silence the expression of the faulty gene while allowing the codon-optimized PABPN1 to replace the mutant protein with its functional counterpart. This "Silence and Replace" mechanism uniquely positions BB-301 as a potential treatment for OPMD by simultaneously halting the expression of the mutant protein and providing a functional replacement.

Benitec Biopharma Inc., headquartered in Hayward, California, focuses on developing genetic medicines through its proprietary DNA-directed RNA interference (ddRNAi) platform. This platform integrates RNA interference with gene therapy, aiming to create treatments that enable sustained silencing of disease-causing genes and the concurrent delivery of functional replacement genes from a single therapeutic administration. The company is advancing ddRNAi-based therapeutics to address chronic and life-threatening conditions, including Oculopharyngeal Muscular Dystrophy (OPMD).

For additional information on the 29th Annual Congress of the World Muscle Society, detailed findings from the oral presentation will later be published on the News & Events section of Benitec’s website.

Benitec Biopharma's innovative approach offers hope for patients with OPMD, a condition with limited treatment options. With ongoing research and clinical trials, the company continues to explore the potential of its gene therapy solutions to address unmet medical needs in genetic disorders.