Benitec Biopharma Inc.

Benitec Biopharma Inc. is a clinical-stage biotechnology company headquartered in Hayward, California, focused on developing new genetic medicines. The company is developing BB-301, a silence and replace-based therapeutic for Oculopharyngeal Muscular Dystrophy (OPMD), a chronic, life-threatening genetic disorder. BB-301 is an AAV-based gene therapy designed to permanently silence the expression of the disease-causing gene to slow or halt disease progression in OPMD. Simultaneously, it aims to replace the mutant gene with a functional gene to restore function in diseased cells. This therapeutic approach, known as "silence and replace," seeks to restore normal cell and tissue physiology and improve treatment outcomes for OPMD patients. BB-301 has received Orphan Drug Designation and Fast Track Designation in the United States, and Orphan Drug Designation in the European Union. Benitec's proprietary technology platform is called DNA-directed RNA interference (ddRNAi) and "silence and replace." ddRNAi is designed to achieve permanent silencing of disease-causing genes by combining RNA interference (RNAi) with viral delivery agents, specifically viral vectors, typically used in gene therapy. The company will require additional financing to advance its product candidates.