BENITEC GRANTED FIRST U.S. AND U.K. PATENTS FOR
DNA DIRECTED GENE SILENCING
Patents Underscore Benitec's Leading
RNAi Position
5 June, 2003
QUEENSLAND, Australia (June 5th 2003)
Benitec Ltd. (ASX: BLT), the first company to demonstrate
RNA interference (RNAi) in human cells, announced
today the grant of its core technology patents in
the United States and the United Kingdom. These patents,
with priority dating to1998, describe a revolutionary
method for silencing any gene in any cell using DNA
directed RNA interference (ddRNAi). U.S. Patent 6,573,099
is entitled "Genetic constructs for delaying or repressing
the expression of a target gene" and U.K. Patent 2353282
is entitled "Control of gene expression". Both patents
contain world first claims that describe the effect
of RNAi in human cells and the DNA constructs which
trigger RNAi. The patents were jointly granted to
Benitec and The State of Queensland, Australia and
Benitec holds exclusive worldwide rights to both patents
under a partnership agreement with The State of Queensland.
"These broad patents confirm Benitec
as a pioneer in the effective application of RNAi
to humans and the leader in ddRNAi technology," stated
John McKinley, Chairman and Chief Executive Officer
of Benitec, Ltd. "We will now leverage our dominant
IP position to secure further commercialization opportunities,
add to our licensing partners and expand our efforts
to develop ddRNAi targets for drug development."
"RNAi is the simplest and most precise
method of gene silencing and ddRNAi is the most versatile
method of triggering RNAi in human cells," stated
Ken Reed, Director of Research & Technology with Benitec.
"In contrast to RNAi induction using double-stranded
RNA, ddRNAi does not provoke the natural interferon
response in cells. Critical to the biopharma industry,
ddRNAi can also manipulate gene silencing for either
transient or permanent effect, and can be used to
reduce gene expression or silence it completely. It
also has more versatility with respect to delivery
vehicles, further increasing options for development
of therapeutics. As a bonus, manufacturing costs are
significantly less."
ABOUT ddRNAi
DNA directed RNAi (ddRNAi) is a method to trigger
the natural gene suppression process called 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 (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.
An alternate approach to triggering
RNAi currently under development uses siRNA to directly
induce the RNAi process. While both approaches trigger
RNAi, Benitec's ddRNAi approach does not activate
the cell's interferon (stress) response. The interferon
response is a natural cellular defense mechanism that
responds to double-stranded RNA, as in a virus, by
shutting down many normal functions.
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 to an extent dependent on how much can enter
a cell.
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 also make
ddRNAi well suited to high throughput functional genomics
and target validation, and provide added versatility
when developing RNAi targets for drug development.
Benitec Limited, based in Queensland,
Australia, is the leader in the field of gene silencing
technology based on DNA directed RNA interference
(ddRNAi). Benitec pioneered ddRNAi and was the first
company to demonstrate RNAi in human cells. With granted
patents in the United States, the United Kingdom,
Australia, South Africa and Singapore, and patent
applications pending in Europe, Japan, and 12 other
jurisdictions covering its ddRNAi technology, Benitec
holds a dominant international intellectual property
position. Benitec has embarked on an aggressive strategy
to leverage its leadership position and license its
technology along the entire chain of the drug discovery
and development process, and is applying the technology
to develop its own ddRNAi targets for drug development.
As previously announced on 8th April, Benitec has
granted Promega Corporation (Madison, WI) its first
worldwide exclusive license (with rights to sub-license)
to develop, sell and distribute ddRNAi products based
upon Benitec's proprietary technology.
This press release contains forward-looking
statements which reflect Benitec'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 Benitec's research strategy, the application of
the discoveries made therein, the successful and timely
completion of clinical studies and the uncertainties
related to the regulatory process.