BNTC - Benitec Biopharma posts BB-301 genetic disorder pilot dosing study interim results

Benitec Biopharma (BNTC) announces results of the interim analysis of the BB-301 Pilot Dosing Study for the treatment of Oculopharyngeal Muscular Dystrophy ((OPMD)), a chronic, life-threatening genetic disorder.Shares of the company up nearly 6% premarket.Patients with OPMD lose the ability to swallow liquids and solids, and the natural history of the disorder is characterized by chronic malnutrition, aspiration, and fatal episodes of aspiration pneumonia.  Currently, no therapeutic agents are approved for the treatment of OPMD.The study evaluated the safety and biological activity of two concentrations of BB-301 across three distinct doses following direct intramuscular injection into the Hypopharyngeus muscles and the Thyropharyngeus muscles of Beagle dogs via the use of a proprietary delivery device employed in an open surgical procedure.Benitec has scheduled a Scientific Advice Meeting in France in May 2021 to review the interim data and the Phase 1 clinical trial design, and the company is planning to initiate the first-in-human clinical study of

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Benitec Biopharma posts BB-301 genetic disorder pilot dosing study interim results