VY-AADC01

VY-AADC01 (also known as NBIb-1817) is an investigational adeno-associated virus serotype 2 (AAV2) gene therapy designed to treat advanced Parkinson's disease. The therapy delivers the human gene for the enzyme aromatic L-amino acid decarboxylase (AADC) directly into the putamen of the brain via a one-time, MRI-guided surgical infusion. AADC is the enzyme responsible for converting levodopa into dopamine; in Parkinson's disease, the progressive loss of dopaminergic neurons leads to a decline in AADC levels, which reduces the brain's ability to synthesize dopamine from oral levodopa treatments. By increasing AADC expression in the striatum, VY-AADC01 aims to enhance the conversion of levodopa to dopamine, thereby improving motor function and reducing "off" time in patients. The program was developed by Voyager Therapeutics and was the subject of major strategic collaborations with Sanofi Genzyme and later Neurocrine Biosciences. However, following a clinical hold on the Phase 2 RESTORE-1 trial and a subsequent data review, Neurocrine terminated the collaboration in 2021, and Voyager discontinued the program.

Other names

PD-1101PD1101PD 1101AAV2-hAADCAAV-2-hAADCAAV 2-hAADCvyaadc