Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference with gene therapy for the goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration.  We believe our technology has the potential to provide 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.

At Benitec we are using this technology, called DNA-directed RNA interference, or ddRNAi, to develop a pipeline of product candidates in several chronic and life-threatening human disease areas including:

  • Oncology: Head & Neck Squamous Cell Carcinoma (HNSCC)
  • Orphan disease: Oculopharyngeal Muscular Dystrophy (OPMD)
  • Retinal disease: Wet Age Related Macular Degeneration (wet AMD)
  • Infectious disease: Hepatitis B Virus (HBV)

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 scientific operations are based in the San Francisco Bay Area, CA, USA.

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