Wilson's disease at a glanceWilson's disease (WD) is a rare genetic disorder of impaired copper metabolism that causes severe hepatic and neurologic symptoms. It is lethal without a treatment.
Gene therapy concept
Sequencing of the human genome revealed genetic defects causing disease in humans. Such defects can lead to abnormal protein production of an enzyme, coagulation factor, etc.
The concept of gene therapy is to introduce a therapeutic, healthy gene into cells carrying a defective gene to treat the underlying disease.
Vivet Therapeutics is utilizing the Adeno-Associated-Virus (AAV) as a shuttle to transport healthy genes to the liver. AAV is a safe, non-pathogenic virus that has not been associated with a disease. To further enhance safety of AAV-based gene delivery, the viral genome is physically separated into different plasmids of proteins required for viral particle formation and infectivity.
The principle of delivering the healthy gene to the target cell, is to inject the replication-defective AAV vector intravenously to patients. The shuttle vector reaches the cells of interest via the bloodstream, enters the cells and travels to the nucleus where the therapeutic gene is released from the virus. Transcription of the healthy gene to the therapeutic protein is finally achieved to release its physiological function to treat the genetic disorder.