Benitec dosed first subject in Phase 1b/2a trial of BB-301 for OPMD dysphagia

Benitec Biopharma Inc.

BB-301 is a gene therapy candidate using Silence and Replace ddRNAi platform for Oculopharyngeal Muscular Dystrophy (OPMD).
OPMD affects ~15,000 patients in US, Canada, Western Europe, and Israel; no approved therapies currently exist.
Interim safety and efficacy data from the 52-week study expected in mid-2024.
19 subjects in the ongoing OPMD Natural History study are eligible to roll over into the BB-301 treatment study.

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