Benitec Biopharma Inc. provided an overview of the key elements of the BB-301 Phase 1b/2a clinical trial design. The clinical trial is planned for 2022. BB-301 is a novel investigational gene therapy under development for the treatment of patients with Oculopharyngeal Muscular Dystrophy (OPMD). OPMD is a chronic, life-threatening genetic disorder affecting approximately 15,000 patients in the United States, Canada, Western Europe, and Israel. OPMD is caused by a mutation in the gene encoding poly(A) binding protein nuclear 1 (PABPN1). 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. Additionally, no surgical interventions capable of altering the long-term natural history of OPMD are available. Benitec is focused on the accurate and reproducible characterization of the key physiological processes underlying the successful completion of the pharyngeal phase of swallowing In this regard, the core analytical tools and methods employed during the clinical study will focus on functional measures of swallowing efficiency for OPMD patients during the pharyngeal phase of swallowing. A Phase 1b/2a, Open-Label Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects with OPMD The purpose of this clinical investigation is to evaluate the safety, tolerability, and clinical activity of ascending doses of BB-301 administered to subjects with OPMD via local intramuscular (IM) injection into the middle pharyngeal constrictor (MPC) muscles and the inferior pharyngeal constrictor (IPC) muscles following open surgical dissection of the pharyngeal region under general anesthesia. This Phase 1b/2a, first-in-human (FIH) study is a single arm, open-label, sequential, dose escalation cohort study with a six-month pre-treatment observation period and a 52-week post injection follow-up period in up to 24 subjects diagnosed with OPMD. BB-301 will be administered in three sequential escalating dose cohorts. After the initial three cohorts have been dosed, additional subjects may be added at the Maximally Effective Dose (MED), or at an alternate dose as determined by review of cumulative safety and efficacy data. At 76 weeks all subjects will be enrolled into a long-term safety follow-up study.