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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
Announcement marks the initiation of the first clinical trial conducted in human subjects employing Benitec’s “Silence and Replace” DNA-directed RNA
About this update from Benitec Biopharma Inc.
[{"type":"text","content":"Announcement marks the initiation of the first clinical trial conducted in human subjects employing Benitec’s “Silence and Replace” DNA-directed RNA interference gene therapy platform\nHAYWARD, Calif., Nov. 30, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or the “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (\"ddRNAi\") platform, today announced the first subject has been dosed in the BB-301 Phase 1b/2a Clinical Treatment Study. 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. Interim safety and efficacy evaluations will be conducted at the end of each 90-day period following the administration of BB-301. “We are grateful to have the opportunity to begin the clinical evaluation of BB-301 and encouraged by the research and development progress made to date. We are deeply appreciative of the unwavering dedication of the clinical research team at our U.S. clinical trial site, our clinical and scientific research advisors in France, and our specialist speech language pathology research advisors in Canada, all of whom have guided the evolution of our understanding of the natural history of OPMD and our implementation of the ideal processes and procedures to facilitate the conduct of the initial clinical evaluation of BB-301,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “Today we have taken a critical step towards the clinical validation of our Silence and Replace-based approach to the management of genetically defined disorders, and our central goals remain focused on the improvement of the lives of patients suffering from OPMD.” Oculopharyngeal Muscular Dystrophy (OPMD) is a chronic, life-threatening genetic disorder affecting approximately 15,000 patients in the United States, Canada, Western Europe, and Israel. OPMD patients lose the ability to swallow liquids an...