Benitec Ltd (ASX: BLT) announced today that it has successfully achieved the simultaneous disabling of multiple genes through RNA interference. The breakthrough is a result of Benitec’s continuing research and development efforts in ddRNAi technology to extend the Company’s patent portfolio in RNA interference

“The breakthrough is significant for its potential to develop powerful human therapeutics in an expanded range of diseases,” said Benitec’s Chief Executive Officer, John McKinley. “We believe our ‘multiple warhead’ capability will allow us to understand more clearly those conditions that result from multiple gene defects – conditions such as Type II diabetes, autoimmune disorders, cardiovascular dysfunctions – and perhaps also provide viable treatment regimes.”

“Today, some of the world’s most serious diseases are demonstrating resistance to existing drugs, as is the case with the increasing problem of antibiotic-resistant bacteria. Most cancers and HIV, in particular, have high mutation rates that increase the probability of resistant variants.”

“A promising approach to this issue,” he continued, “is to simultaneously disable multiple targets. The greater number of genes that can be targeted, the lower the possibility that any one cell or virus would develop resistance to the treatment.”

The concept of combination therapy, where several drugs attack multiple targets simultaneously, is widely used in the treatment of cancers. Benitec’s new multi-hit ddRNAi technology potentially provides a single agent that could deliver potent and specific ‘drugs’ effectively against multiple targets. This approach of combining targets in one therapeutic is preferable to attempting to achieve the same results from multiple drugs as it eliminates the possibility of any cell developing resistance by inadvertent exposure to just one of the drugs.

About ddRNAi

DNA directed RNAi (ddRNAi) is a method to trigger the natural gene suppression process called RNA interference (RNAi) that operates by destroying messenger RNA (mRNA), the courier that delivers instructions to the ribosomes within the cell to manufacture the proteins coded for by DNA. By introducing a DNA construct into a cell, Benitec’s ddRNAi technology triggers the production of double-stranded RNA (dsRNA), which is then cleaved into small interfering RNA (siRNA) as part of the RNAi process, resulting in the destruction of the target mRNA and knocking-down or silencing the expression of the target gene.

Benitec’s ddRNAi provides the ability to control the silencing effect so that it either knocks down or completely silences the target gene. Control can also be exerted to make the silencing effect transient or permanent. siRNA technologies under development are only capable of transient silencing and are dose dependent, unlike ddRNAi which is catalytic in its effect.

ddRNAi can also be used with a range of efficient delivery options not available to the siRNA approach, providing additional versatility for therapeutics development. These qualities, among others, make ddRNAi well suited to high throughput functional genomics and target validation, and provide added versatility when developing RNAi therapeutics.

About Benitec Limited

Benitec Limited, based in Queensland, Australia, has key Intellectual Property in the field of gene silencing using DNA constructs to trigger RNA interference (granted patents include US 6,573,099; GB 2353282; AU 2001100608). The Company was the first to demonstrate RNA interference, a universal gene silencing mechanism, in mammalian and human cells. This natural mechanism is now recognized worldwide as the simplest and most precise method for shutting down the activity of any specific gene. The technology is immediately applicable to functional genomics and the identification and validation of drug targets. Benitec's technology can also be applied to the treatment of major human diseases such as cancers, autoimmune disorders and viral infections through the shutting down of disease-causative genes. Other applications include plant improvement and the breeding of viral resistant livestock. Benitec offers customers licences to undertake research and development on its expanding technology platform and tools to deliver the technology's full capability.