Wilson's disease at a glance

Wilson'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.

Potential benefits of AAV gene therapy, as a novel approach to manage Wilson's disease

  • Alternative to current treatments
  • Restoration a normal physiological and homeostasis of copper metabolism
  • Generation of long-lasting effects by a single IV administration
  • Alleviation of the need for repeat oral administration of current treatments and of their associated side effects
  • Optimal adherence to treatments, especially in neurologically/ cognitively impaired patients