A Gene Therapy Approach to Treat Hepatitis C Virus (HCV) Infection


Delivery of a cluster of anti-HCV miRNA to neutralize HCV infection in liver


Market Need


It is estimated that 3 percent of the world’s population is chronically infected with hepatitis C virus (HCV). Of those with HCV infections, 70 to 85 percent of individuals experience serious liver damage, including hepatocellular carcinoma. Current therapies, including a yearlong treatment with a combination of pegylated interferon-α and ribavirin, are only partially effective because of the genetic diversity of HCV genotypes and the high mutation rate of the virus. In addition, the antiviral drugs used to treat HCV have numerous side effects, including neutropenia, anemia, nausea, and depression.


Technology Overview


The Couto Lab has developed a therapeutic product to treat HCV infections using RNA interference (RNAi), which has been shown to inhibit HCV replication up to 97 percent in vitro and show no liver toxicity when delivered in a preclinical mouse model. In this invention, an endogenous microRNA (miRNA) cluster, miRNA-17-92, serves as a scaffold for five artificial miRNAs that target different regions of the HCV genome. This artificial miRNA cluster is delivered to cells by is delivered by an AAV gene therapy vector, and inhibits HCV replication.



•       Specifically targets HCV replication

•       Highly effective (97% in vitro)



•       Treatment of Hepatitis C Virus


Stage of Development: In vivo proof of concept


Case ID:
Web Published:
Patent Information:
Title Country Serial No. Patent No. File Date Issued Date
Nucleic Acids For Targeting Multiple Regions Of The HCV Genome Australia 2011223820 2011223820 3/1/2011 4/28/2016
Nucleic Acids For Targeting Multiple Regions Of The HCV Genome Canada 2,791,974   3/1/2011  
Nucleic Acids For Targeting Multiple Regions Of The HCV Genome United States 13/602,886 8,846,632 9/4/2012 9/30/2014
For Information, Contact:
Cassie Tran
Licensing Associate
The Children's Hospital of Philadelphia
Linda Couto