DNA-directed RNA Interference (ddRNAi) is Benitec’s RNAi platform for gene silencing, with several advantages over other methods of gene silencing such as siRNA, anti-sense and zinc finger nucleases.

Benitec – gene silencing by ddRNAi

In siRNA, double stranded RNA (dsRNA) molecules are synthesized and introduced to the cell. The siRNA is designed to cleave the mRNA of the target gene, and so prevent production of the specific target protein. Delivering the siRNA molecule to the target organ or tissue in a way that maintains its activity and efficacy induces lots of limitations for siRNA-based therapeutics. Benitec’s patented approach of gene silencing overcomes most of the challenges of siRNA-based methods using a DNA-based approach: DNA-directed RNAi (ddRNAi). ddRNAi causes the cell to produce siRNA itself (rather than introducing synthetic siRNA) by producing a precursor molecule called short hairpin RNA (shRNA) in the nucleus. shRNA enters the cytoplasm and is processed to siRNA by the cellular machinery. The specific shRNA is produced in the cell after transfection of a DNA-based construct (hence the concept of RNAi directed by DNA). Thus, ddRNAi introduces a DNA sequence directly into the cell’s nucleus, using a wide range of well-characterized gene therapy vectors, like adenovirus, lentivirus, AAV or modified polyethylenimine. Thanks to the delivery mechanism and site, only a small dose of the DNA construct is needed and the genetic change is long-lasting, as the shRNA continues its expression for long periods. Thus ddRNAi promises to deliver long lasting and relatively safe treatments for many human genetic conditions.

Founded in 1997, Benitec is a biopharma and biotech company headquartered in Sydney (Australia) with in-house research facility located in the Bay Area of San Francisco (USA). Board and management’s team are composed by Peter French (CEO), John Chiplin, Iain Ross, Kevin Buchi, Peter Francis, Michael Graham, Greg West, Carl Stubbings and David Suhy. Benitec Biopharma holds the dominant global patent position for DNA-directed RNAi (ddRNAi), placing the company in a leading position for human therapies associated with specific genes and genetic disorders (the use of shRNA for use in humans falls also under this patent). Benitec’s pipeline is focused on multi-billion dollar market opportunities in Hepatitis B, Hepatitis C, lung cancer and wet Age-related Macular Degeneration, as well as the orphan genetic disease, oculopharyngeal muscular dystrophy. And ddRNAi technology could potentially be applied to countless genes in humans and in disease-causing micro-organisms. Thus Benitec out-licenses this technology to biotechnology and biopharma companies for use in other therapeutic areas, such as Retinitis Pigmentosa, HIV/AIDS, Huntington’s Disease, and human cancer vaccines.

More about Benitec Biopharma : www.benitec.com

Benitec Biopharma – DNA-directed RNA Interference – ddRNAi – RNAi – short hairpin RNA – shRNA – gene silencing – siRNA

Benitec Biopharma – Benitec – gene silencing ddRNAi – double stranded RNA – dsRNA – Peter French – John Chiplin – Iain Ross – Kevin Buchi – Peter Francis – Michael Graham – Greg West – Carl Stubbings – David Suhy – BenitecBiopharma