Business
Benitec Biopharma Provides Update on BB-301 Tissue Transduction Study
HAYWARD, Calif., July 8, 2020 /PRNewswire/ -- Benitec Biopharma, Inc. (NASDAQ: BNTC), a development-stage, gene therapy-focused, biotechnology company
About this update from Benitec Biopharma Inc.
[{"type":"text","content":"HAYWARD, Calif., July 8, 2020 /PRNewswire/ -- Benitec Biopharma, Inc. (NASDAQ: BNTC), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on the proprietary DNA-directed RNA interference (\"ddRNAi\") platform, today announced the initiation of the BB-301 Tissue Transduction Study in large animal subjects. \nBB-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 underlying physiology of the treated tissues and, in the process, improve treatment outcomes for patients suffering from the chronic and, potentially, fatal effects of Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 has been granted Orphan Drug Designation in the United States and the European Union. \nThe BB-301 Tissue Transduction Study is the first of three planned IND-enabling studies that will be conducted in large animals. These IND-enabling studies will be carried out under the guidance of the scientific team at Benitec, with key elements of the study design and execution conducted in close collaboration with a team of leading experts in both medicine and surgery that have been deeply engaged in the treatment of OPMD patients for several decades. The BB-301 Tissue Transduction study, along with the subsequent non-clinical studies, will be conducted in canine subjects and will support the validation and optimization of the newly designed method of BB-301 administration, confirm the efficiency of vector transduction in the key tissue compartments underlying the natural history of OPMD, confirm the optimal drug doses in advance of initiation of human clinical studies, and facilitate observation of key toxicological data-points.\nThe BB-301 Tissue Transduction Study is designed as an 8-week study in Beagle dogs to confirm the transduction efficiency of BB-301 upon administration via direct intramuscular injection into specific anatomical regions of the pharynx through the use of an open surgical procedure. Thi...