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Benitec Biopharma Provides Update on BB-301 Oculopharyngeal Muscular Dystrophy (OPMD) Program

Benitec Biopharma Inc. (BNTC) 
US:NASDAQ Investor Relations: benitec.com/for-investors/investor-faqs
Company Research Source: PR Newswire
MELBOURNE, Australia, Sept. 16, 2019 /PRNewswire/ -- Benitec Biopharma (ASX: BLT, NASDAQ: BNTC), a gene therapy-focused biotechnology company developing novel genetic medicines derived from the proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced their plans to complete three non-clinical studies that will facilitate the filing of an Investigational New Drug (IND) application and the formal initiation of a Phase I clinical trial in patients suffering from Oculopharyngeal Muscular Dystrophy (OPMD).  BB-301 is an internally optimized, AAV-based gene therapy agent that can both silence the expression of mutated, disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and replace the mutant genes with normal, "wild type" genes (to drive restoration of function in diseased cells).  This fundamental approach to disease management is called "silence and replace" and this biological mechanism offers the potential to restore the under Show less Read more
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