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Benitec Biopharma reports positive interim Phase 1b/2a results for BB-301 in OPMD
BNTCBenitec Biopharma Inc.
Benitec Biopharma announced positive interim clinical data from its ongoing Phase 1b/2a study of BB-301, a gene therapy candidate for Oculopharyngeal Muscular Dystrophy (OPMD). The results showed robust and durable improvements in swallowing function across both low-dose and high-dose cohorts. All nine patients treated to date were formal responders to the therapy, with no serious adverse events reported. High-dose patients demonstrated greater relative improvements in throat emptying ability and functional swallowing capacity at three months post-treatment compared to low-dose patients. The company plans to advance toward a pivotal study expected in mid-2027. Benitec Biopharma is a clinical-stage biotechnology company developing disease-modifying genetic medicines using its proprietary ddRNAi platform.