SYDNEY, March 11, 2015 /PRNewswire/ -- Benitec Biopharma (ASX: BLT; OTCPK: BTEBY), a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," today announced that the fourth patient in the company's Phase I/IIa dose escalation clinical trial of its lead program TT-034 for treating hepatitis C was dosed at the Duke Clinical Research Unit. This is the second patient to be dosed in Cohort Two, with the third and final patient in Cohort Two well advanced in their preparation for dosing.
As previously announced, the parallel dosing of these patients follows a positive recommendation from the DSMB's review of the safety data from the first patient in this cohort.
All three patients in Cohort Two receive a dose of 1.25 x 10^11 vg/kg of TT-034, a concentration that is a half-log higher than the dose administered in Cohort One. This dose level is still below the concentration expected to inhibit hepatitis C viral replication and therefore data from Cohort Two are expected to serve primarily as a further safety assessment.
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimizes the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the lifetime of the cell. Thus it has the potential not only to treat the existing HCV infection, but also to guard against reinfection for months to years without the need to re-treat. It has been tested extensively in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is primarily to ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
About Benitec Biopharma Limited:
Benitec Biopharma Limited is an ASX-listed biotechnology company (ASX:BLT; OTC:BTEBY) that has developed a patented gene-silencing technology called ddRNAi or 'expressed RNAi'. Based in Sydney, Australia, with labs in Hayward, CA (USA), and collaborators and licensees around the world, the company is developing ddRNAi-based therapeutics for chronic and life-threatening human conditions including Hepatitis C and B, drug resistant lung cancer and wet Age-related Macular Degeneration (AMD). Benitec has also licensed ddRNAi to other biopharmaceutical companies for applications including HIV/AIDS, Huntington's disease, chronic neuropathic pain and retinitis pigmentosa.
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com.