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Gene Therapy BB-301 Improves Swallowing in OPMD Patients in Early Trial

May 16, 2026

Benitec Biopharma's gene therapy BB-301 improved swallowing ability in all four OPMD patients followed for at least one year in a Phase Ib/II trial. One patient showed continued improvement at two years. The company plans to meet with the FDA in mid-2026 to discuss a pivotal study.