Monday, January 09, 2012 by Christine Feary
Benitec Biopharma (ASX: BLT) has received pre-clinical results on the use of the company’s gene silencing technology to develop a therapeutic for hepatitis C viral (HCV) infection.
HCV infection is a leading cause of liver disease, with figures from the World Health Organisation showing that about 170 million people are chronically affected.
The figures showed that there are approximately 350,000 deaths from HCV-related liver disease each year.
At present, therapy typically involves extended dosing of between 24 and 72 weeks duration, with low tolerability and only modest effects.
Results from pre-clinical studies by researchers at Pfizer and Tacere Therapeutics have been published online in the American Society of Microbiology’s journal, Antimicrobial Agents and Chemotherapy.
The pre-clinical trials showed extremely positive results for ddRNAi molecule PF-05095808.
This molecule has been specifically designed to achieve transduction of all liver cells in the liver without causing cell damage.
Importantly, there was no evidence of cytotoxicity nor of induction of the interferon response associated with the administration of the molecule.
It was also shown to be highly effective at inhibiting the commonly circulating clinical isolates of HCV, with the ability to eliminated resistance to the molecule using three shRNA sequences.
The studies indicated that the molecule is able to target commonly circulating strains of HCV.
The report concluded that the molecule represents the prototype of a new approach to HCV therapy, with a single dose that can be administered alone or in combination with other anti-HCV agents.
According to the report, “These studies demonstrated the PF-05095808 delivers sequence-specific antiviral activity in the absence of overt cytotoxicity.”
A clinical trial is expected to follow, with Tacere confirming its commitment to progressing the program, for which it has an exclusive sub-license from Benitec.