Core Viewpoint - Benitec Biopharma Inc. is set to provide an update on the six treated patients from Cohort 1 of its Phase 1b/2a Clinical Study of BB-301, which is being investigated for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) [1]. Company Overview - Benitec Biopharma Inc. is a clinical-stage biotechnology company based in Hayward, California, focused on developing novel genetic medicines [3]. - The company utilizes a proprietary "Silence and Replace" DNA-directed RNA interference (ddRNAi) platform that combines RNA interference with gene therapy [3]. - This platform aims to create therapeutics that can silence disease-causing genes while delivering wildtype replacement genes in a single administration [3]. - Benitec is developing therapeutics for chronic and life-threatening conditions, including Oculopharyngeal Muscular Dystrophy (OPMD) [3]. Webcast Information - A webcast to discuss the update on the clinical study will take place on November 3, 2025, at 8:00 am EST [2].

Core Insights - Benitec Biopharma Inc. is advancing its BB-301 clinical development program for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD) and expects to provide interim clinical study results for Cohort 1 in Q4 2025 [1][4][2] Corporate Highlights - The Independent Data Safety Monitoring Board (DSMB) recommended the continuation of subject enrollment into the BB-301 Phase 1b/2a Treatment Study after the safe treatment of the sixth subject in Cohort 1, with enrollment for Cohort 2 expected to begin in Q4 2025 [3][2] Financial Highlights - For the year ended June 30, 2025, total expenses were $41.8 million, up from $22.5 million in 2024, with research and development expenses increasing to $18.3 million from $15.6 million [5][6] - General and administrative expenses rose to $23.4 million in 2025 from $7.0 million in 2024, primarily due to higher share-based compensation and increased legal and consulting fees [6] - The net loss attributable to shareholders for the year was $37.9 million, or $1.05 per share, compared to a net loss of $22.4 million, or $1.22 per share, in the previous year [7][10] Balance Sheet Overview - As of June 30, 2025, the company had cash and cash equivalents of $97.7 million, an increase from $50.9 million in 2024 [9] - Total assets were reported at $99.6 million, compared to $52.2 million in the previous year, indicating significant growth in the company's financial position [9] About BB-301 - BB-301 utilizes a modified AAV9 capsid to promote co-expression of a functional version of the PABPN1 protein while silencing the expression of the faulty mutant PABPN1 gene, positioning it as a potential therapy for OPMD [11] About Benitec Biopharma, Inc. - Benitec Biopharma is a clinical-stage biotechnology company focused on developing novel genetic medicines, particularly for chronic and life-threatening conditions like OPMD, using its proprietary "Silence and Replace" platform [12]

Core Insights - The independent Data Safety Monitoring Board (DSMB) has recommended the continuation of subject enrollment for the Phase 1b/2a Clinical Treatment Study of BB-301 after reviewing safety data for all six subjects in Cohort 1 [1][2] - Enrollment for Cohort 2 is expected to begin in Q4 2025 following the positive DSMB recommendation [1][2] - The sixth and final subject of Cohort 1 was safely treated with a low dose of BB-301 in April 2025, indicating a benign safety profile associated with the local route of administration [3] 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 (ddRNAi) platform [5] - The company aims to treat chronic and life-threatening conditions, including Oculopharyngeal Muscular Dystrophy (OPMD), through its innovative therapeutic approaches [5] - BB-301 is designed to silence the expression of faulty mutant PABPN1 while allowing for the expression of a functional version of the protein, positioning it uniquely for OPMD treatment [4]