Benitec Biopharma Announces First Subject Dosed in Phase 1b/2a Clinical Trial for Gene Therapy Candidate BB-301 for the Treatment of Oculopharyngeal Muscular Dystrophy
BB-301 is the Company’s first gene therapy candidate employing the Silence and Replace approach and is being developed for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia.
- BB-301 is the Company’s first gene therapy candidate employing the Silence and Replace approach and is being developed for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia.
- Dosing of the first subject marks the beginning of the 52-week follow-up period designed to facilitate conclusive evaluation of the primary and secondary endpoints of the BB-301 Phase 1b/2a Clinical Treatment Study.
- Currently, there are 19 subjects enrolled into the NH study at the U.S. clinical trial site, with each subject having the potential to roll over into the BB-301 Phase 1b/2a Clinical Treatment Study.
- Interim safety and efficacy data are expected to become available from the BB-301 Phase 1b/2a Clinical Treatment Study in mid-2024.