By Chris Wack


Benitec Biopharma said Monday the U.S. Food and Drug Administration cleared its Investigational New Drug application for BB-301, a silence-and-replace gene therapy for the treatment of oculopharyngeal muscular dystrophy-related dysphagia.

The company said subjects from Benitec's ongoing study will be eligible for rollover into the Phase 1b/2a clinical study of BB-301 for the treatment of OPMD-related dysphagia after six months of baseline data collection.

Following a one-day dosing procedure for BB-301, each study subject will be evaluated for radiographic and clinical outcome measures, including quantitative radiographic swallowing studies to facilitate objective assessments of swallowing safety, swallowing efficiency, and functional performance of the pharyngeal muscles underlying the OPMD-related dysphagia.

Benetic said there are 13 subjects enrolled into the study, with each subject having the potential to rollover onto the Phase 1b/2a clinical dosing study for BB-301. Interim safety and efficacy data is expected to become available from the BB-301 Phase 1b/2a study every 90 days following the dosing of each subject.


Write to Chris Wack at chris.wack@wsj.com


(END) Dow Jones Newswires

06-26-23 0837ET