Voyager Therapeutics: Overview of Clinical Stage Pipeline

Clinical Stage Therapeutic Area

Parkinson Disease

Parkinson disease is a progressive disorder of the nervous system. The disorder affects several regions of the brain, especially an area called the substantia nigra that controls balance and movement. 

Treatment Approach: VY-AADC is a gene therapy for Parkinson’s disease that is being developed by Voyager Therapeutics. The treatment is aimed at increasing the patient’s ability to produce dopamine, a signaling molecule that helps regulate movement and emotional responses, which improves motor function. The targeted cells in the central nervous system (CNS) are long-lived, non-dividing neurons, therefore, treatments delivered in a single dose may generate long-lasting, or even lifelong, benefits.

Clinical Stage: Phase 3/Pivitol 

Platform: Adeno associated (AAV2) Viral Delivery of Genes into the target cells of the CNS