Hepatitis C Virus RNA-Targeted Therapeutics

DESCRIPTION (provided by applicant): Hepatitis C virus (HCV) infection is a significant human health issue, accounting for 50% of liver transplants in the US, with approximately 170 million persons infected worldwide. The current standard of care therapy of PEGylated-interferon and ribavirin is effective in only about 50% of patients, with severe side effects and contraindications limiting patient compliance. New therapeutics targeting HCV protease have recently been approved, and additional therapies targeted the HCV NS5B RNA polymerase and the HCV NS5A accessory protein are in clinical trials. With each of these new therapies however, drug resistance is known to develop, and those new therapies have shown significant side effects. The experience with HIV therapy suggests that a truly effective regimen against HCV will consist of a cocktail of drugs against several HCV targets. HCV protein synthesis is initiated from the uncapped (+)strand RNA genome, utilizing a novel RNA structure in the 5'-untranslat