Benitec Biopharma announced interim clinical study updates for the first three subjects in its Phase 1b/2a trial of BB-301, a gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD). These updates will be presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference on March 19th. The presentation will focus on the therapy’s impact on dysphagia, a severe swallowing difficulty associated with OPMD.
This research holds significant promise for OPMD patients who experience dysphagia, a life-threatening symptom. Current treatments are limited, and BB-301 offers a potential solution by addressing the underlying genetic cause of the disease. Positive clinical data demonstrating improved swallowing function would validate Benitec’s “Silence and Replace” ddRNAi platform and pave the way for broader applications of this gene therapy approach in other genetic disorders.
BB-301 utilizes a modified AAV9 viral vector to deliver a bifunctional construct. This construct silences the expression of the mutated PABPN1 gene responsible for OPMD while simultaneously delivering a functional copy of the gene. The interim data from the first three patients treated in the ongoing Phase 1b/2a clinical trial will offer crucial insights into the safety and efficacy of the treatment. Previous interim data for the first two subjects showed durable and clinically meaningful improvements in swallowing function. Further updates for enrolled subjects are anticipated in the fourth quarter of 2025.
The upcoming presentation at the Muscular Dystrophy Association conference represents a crucial step for Benitec and the OPMD community. Positive results would reinforce the potential of BB-301 to significantly improve the quality of life for patients and potentially lead to a disease-modifying therapy. Further data from the ongoing trial will be essential to confirming these early findings and establishing the long-term efficacy and safety profile of BB-301. This data will also be pivotal for future regulatory decisions regarding the therapy’s advancement.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
