Benitec to Present at the American Society of Gene Therapy 9th Annual Meeting
Benitec to Present at the American Society of Gene Therapy 9th Annual Meeting
June 6, 2006
Benitec Ltd.
MOUNTAIN VIEW, Calif., June 6 /PRNewswire-FirstCall/ -- Benitec Ltd.
(ASX: BLT), a leading developer of RNA interference (RNAi)-based
therapeutics, announced today that its Hepatitis C (HCV) clinical program
will be highlighted at the American Society of Gene Therapy (ASGT) 9th
Annual Meeting in Baltimore, Maryland in three separate oral presentations.
Sara Cunningham, Chief Executive Officer of Benitec, stated, "As we
develop this entirely new class of drugs, it is useful to seek guidance
from previous experiences in the clinical development of molecular
medicines. To quote Nobel Laureate Phil Sharp, the key to success in RNAi
is 'delivery, delivery, delivery.' As large molecules, RNAi drugs must be
actively delivered to the target cell -- a challenge that has been pursued
within the gene therapy context for several decades, in countless clinical
trials and now two approved gene therapy drugs.

"In the case of Benitec's HCV drug, we have had to tackle two problems
particular to the disease: 1) the fact that it is caused by a virus that
mutates very quickly, making it difficult to treat by traditional small
molecule drugs and nearly impossible to vaccinate against; and 2) the fact
that it reproduces within hepatocytes. This type of liver cell is
essentially impermeable to any large molecule that is not actively
transported across the cell membrane. Small molecules passively diffuse
into hepatocytes, but RNAi molecules, whether chemically synthesized or
encoded by a vector, do not. Further, HCV infects a constantly shifting
population of hepatocytes, but at a very low level; thus you don't need
much of the drug, but you need it in every cell. Although we could look to
novel chemistries to attempt to overcome the delivery problem with
hepatocytes, we have chosen to look to nature. Along with RNAi clinical
updates from Sirna and Alnylam at ASGT on their lead programs, we are
presenting the preclinical results from using a receptor- mediated delivery
method that allows penetration of our drug into virtually 100% of
hepatocytes in a mouse model with greater than 97% inhibition of HCV and no associated toxicity."
Professor Mark Kay of Stanford Medical School, Chairman of Benitec's
Scientific Advisory Board, and the President of ASGT, stated, "As published
in our recent Nature paper, we found effective and safe RNAi sequences that
when administered at therapeutic doses were able to achieve safe, long-term down- regulation of important disease targets such as Hepatitis B. As also shown in that paper, it is critical to understand the safety parameters and address them early in drug development, to find the appropriate therapeutic window, as Benitec has, in which drug candidates are safe and effective with an acceptable side effect profile. Based on clinical trial data
presented at ASGT, including the successful treatment of otherwise fatal
primary immunodeficiencies in nearly 30 children, the promise of gene
therapy is starting to be realized. "
About Benitec
Benitec is an international biotechnology company focused on developing
therapeutics to treat serious diseases using its proprietary RNAi
technology. Benitec is listed on the Australian Stock Exchange and has its
operations in Mountain View, California, USA. Its lead therapeutic programs
are for Hepatitis C Virus (HCV) and the Human Immunodeficiency Virus (HIV).
Benitec's RNA-based HIV therapeutic, co-developed with the Center for
Biomedicine & Genetics at the City of Hope in Los Angeles, California, will
enter Phase I clinical trials in 2006. Benitec's RNAi therapeutic for HCV
will enter clinical trials in 2007. For additional information, please
visit http://www.benitec.com.
Forward-looking Statements
This press release contains forward-looking statements that reflect the
Companies' current expectations regarding future events. Forward-looking
statements necessarily 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.
CONTACTS:
BENITEC LTD.
Sara Cunningham
+1 650 564 9850 ext. 1104
scunningham@benitec.com
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