- Benitec Biopharma will complete three non-clinical studies to facilitate an IND application to commence trialling patients suffering from OPMD
- Oculopharyngeal Muscular Dystrophy is a rare genetic muscle disorder that affects people between the ages of 40 and 60
- BB-301, is an AAV-based gene therapy agent that silences the expression of disease-causing genes, and replace the mutant genes with normal ones
- The three non-clinical BB-301 studies will be conducted in canine subjects
Benitec Biopharma will complete three non-clinical studies to facilitate patients suffering from Oculopharyngeal Muscular Dystrophy.
Benitec is a clinical-stage biotechnology company focused on developing novel genetic medicines.
It uses a technology called ddRNAi, to develop a pipeline of products in several chronic and life-threatening human disease areas. These include Oculopharyngeal Muscular Dystrophy (OPMD) and Hepatitis B Virus (HBV).
Oculopharyngeal Muscular Dystrophy is a rare genetic disorder that affects people between the ages of 40 and 60.
The disease mainly affects the muscles of the upper eyelids and throat but can spread to the arms and legs. Individuals may develop drooping eyelids, trouble moving their eyes and/or difficulty swallowing.
Benitec’s treatment, BB-301, is an AAV-based gene therapy agent that can silence the expression of disease-causing genes, and replace the mutant genes with normal genes.
The “silence and replace” biological mechanism can potentially restore the physiology of the treated tissues and, in the process, improve treatment outcomes for patients.
“Our team has an unprecedented opportunity to develop a novel genetic medicine that could facilitate clinically meaningful patient benefit in a potentially fatal disorder for which profound unmet medical need exists,” Benitec Biopharma CEO and Executive Chairman Jerel A. Banks, M.D. and Ph.D said.
The three non-clinical BB-301 studies will be conducted in canine subjects.
The studies will support bettering the administration methods, confirm the efficiency of vector transduction and the optimal drug doses in advance of human clinical studies.
The studies will also help to finalise experiments designed to characterise any toxicological data points that’d underlie future filings and clinical study designs.
The non-clinical studies will be carried out under the company’s scientific team and closely with a team of thought leaders in medicine and surgery who have decades of experience with OPMD patients.