The first patient in the BB-301 Phase 1b/2a clinical study for OPMD has shown continued disease-modifying effects at the 24-month follow-up. Patient 4 in the same cohort also exhibited a strong response at the 12-month follow-up. All 4 patients in Cohort 1 were formal responders to BB-301, meeting the statistical criteria for improvement. Benitec Biopharma is thrilled with the positive outcomes and plans to engage with the FDA in mid-2026. The company looks forward to presenting more clinical results in the future. The treatment aims to address dysphagia in patients with OPMD through gene therapy.

Read more at GlobeNewswire: Benitec Biopharma Provides Positive Long-Term Clinical