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press releases
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2004
2003
2002
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news release
Benitec acheives major breakthrough in gene silencing
Allows for Simultaneous Multiple Gene Disabling
22 October, 2003
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.
This press release contains forward-looking
statements which reflect the Company's current expectation
regarding future events. Forward-looking statements
involve risks and uncertainties. Actual events could
differ materially from those projected herein and depend
on a number of factors including the success of the
Company's research strategy, the applicability of the
discoveries made therein, the successful and timely
completion of clinical studies and the uncertainties
related to the regulatory process.
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