Our Story

Benitec Biopharma holds the dominant global patent position for expressed RNA interference (also called DNA-directed RNAi (ddRNAi)), placing the company in a commanding position for human therapies associated with specific genes and genetic disorders. The use of shRNA for use in humans 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 C, Hepatitis Blung cancer and wet Age-related Macular Degeneration, as well as the orphan genetic disease, oculopharyngeal muscular dystrophy. However, ddRNAi technology has potential well beyond these conditions; it could potentially be applied to countless genes in humans and in disease-causing micro-organisms.

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. During the mid 2000's, Benitec was involved in a lengthy and ultimately successful patent litigation, 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 human conditions of high prevalence and value. In addition, we out-license our proprietary technology to biotechnology and biopharma companies for use in non-core therapeutic areas, such as HIV/AIDSRetinitis PigmentosaHuntington's Disease, and human cancer vaccines. We also licence the technology as a research agent and cross-license in other situations..


Benitec Biopharma is headquartered in Sydney, Australia. Our premier in-house research facility is located in the Bay Area of San Francisco, CA, USA, and we work collaboratively with a number of laboratories in Australia, the USA and China.