A leader in RNAi Companies

Benitec Biopharma holds the dominant global patent position for expressed RNA interference (or  DNA-directed RNAi (ddRNAi)), placing it in a commanding position for therapies for human conditions associated with specific genes and genetic disorders. The use of shRNA for human use falls under our patent estate.

Our patented technology, ddRNAi, overcomes many of the drawbacks of other gene silencing technologies. It is being developed in-house and out-licensed for therapies where gene silencing is indicated for cure or treatment.

Pipeline focus

Our current clinical pipeline is focused on multi-billion dollar market opportunities in hepatitis B, lung cancer and cancer-associated neuropathic pain, as well as the orphan genetic disease, oculopharyngeal muscular dystrophy. However ddRNAi could potentially be applied to over 22,000 human genes and many more in disease-causing micro-organisms.

Benitec's ddRNAi technology has been clinically tested in a pilot Phase I/II clinical trial on HIV/AIDS-related lymphoma patients and was proven to be safe and feasible. (Science Translational Medicine June 2010)

Patent position

ddRNAi is one of two major approaches to RNA interference and it has several advantages over the alternative: its effects are longer lasting, it requires lower doses (possibly just one molecule); it has lower toxicity and offers a wider range of delivery options.

Benitec owns or exclusively licenses from CSIRO almost 50 granted or allowed patents in the field of RNA interference for human therapeutics, with another 50 pending. Patents have been granted in USA, the UK, Japan, Europe, Canada and Australia.


Benitec Biopharma was founded in 1997 and has been publicly listed on the ASX since then. We were involved in a drawn-out but ultimately successful patent litigation in the mid 2000’s, which included defense of our platform technology patents. This action resulted in their being re-examined and re-issued in all jurisdictions by 2012.

Benitec Biopharma is developing therapeutic agents in-house for use with core, high potential conditions like chronic pain related to cancer, drug resistant non-small cell lung cancer and hepatitis B. In addition, we out-license our proprietary technology to biopharma companies for use in non-core therapeutic areas (hepatitis C, HIV/AIDS) and to global reagent suppliers for use in research.

Benitec Biopharma is headquartered in Sydney, Australia.


 Intellectual Property
 Pipeline & Partners

Benitec Biopharma Ltd, 1-15 Barr St Balmain (Sydney) NSW Australia             P.+ 61 (0) 2 9555 6986           E. info@benitec.com

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