Benitec Biopharma receives FDA IND clearance for BB-301 gene therapy for OPMD

Benitec Biopharma Inc.

FDA cleared IND for BB-301, a silence-and-replace gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD).
OPMD affects ~15,000 patients in US, Canada, Western Europe, and Israel; no approved therapies currently.
First subject dosing expected in H2 2023 after rollover from ongoing Natural History study (13 subjects enrolled).
Interim safety and efficacy data from Phase 1b/2a study to be reported every ~90 days post-dosing.
BB-301 designed to treat OPMD-related dysphagia, a life-threatening swallowing disorder.

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