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WEEKEND NEWS (Sat)

Benitec Biopharma Reports Positive Data from Two Subjects Treated with Low-Dose BB-301 in Phase 1b/2a Study Presented at 29th Annual Congress of the World Muscle Society
Benitec Biopharma (NASDAQ: BNTC) reported positive interim data from its Phase 1b/2a study of BB-301 for Oculopharyngeal Muscular Dystrophy (OPMD). Two subjects treated with the low-dose BB-301 showed durable, clinically meaningful improvements in swallowing at 9 and 6 months post-treatment, respectively.

Key findings include:
No Significant Adverse Events reported
Subject 1 experienced a 35% reduction in Sydney Swallow Questionnaire (SSQ) Total Score and significant
improvements in videofluoroscopic swallowing study (VFSS) assessments
Subject 2 achieved an 89% reduction in SSQ Total Score, reaching a clinically normal swallowing profile

Strong correlation between SSQ scores and VFSS Total Pharyngeal Residue results

These results represent the first reported successful improvements in swallow function using a novel gene therapy for OPMD. Benitec plans to treat a third subject with the low dose and enroll additional subjects at higher doses in 2025.
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