We are a biotechnology company developing a novel, proprietary therapeutic technology platform that combines gene silencing and gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration.
We believe our technology has the potential to be a "one shot" cure for a wide range of diseases that are currently addressed by strict ongoing treatment regimens or that have no effective treatment or only palliative care options.
We are using our technology, called DNA-directed RNA interference, or ddRNAi, to develop our pipeline of product candidates for the treatment of several chronic and life-threatening human diseases, such as hepatitis B, age-related macular degeneration, or AMD and oculopharyngeal muscular dystrophy, or OPMD. These diseases have large patient populations, with the exception of OPMD which is a rare disease.
In addition, we have licensed our ddRNAi technology to other biopharmaceutical companies whose pipeline programs are progressing towards, or are in, clinical development for applications including HIV/AIDS, retinitis pigmentosa, Huntington's disease, cancer immunotherapy and intractable neuropathic pain.
Benitec Biopharma is headquartered in Sydney, Australia. Our in-house research facility is located in the Bay Area of San Francisco, CA, USA.