AV201

AV201 is an adeno-associated virus (AAV) vector-mediated gene therapy candidate developed by Avigen for the treatment of advanced Parkinson's disease. The therapy is designed to deliver the human gene for aromatic L-amino acid decarboxylase (AADC) directly into the striatum (specifically the putamen). AADC is the enzyme responsible for the decarboxylation of levodopa (L-Dopa) into dopamine. In advanced Parkinson's disease, the progressive loss of dopaminergic neurons leads to a depletion of endogenous AADC, which limits the brain's ability to convert exogenous levodopa into functional dopamine and contributes to motor fluctuations. By restoring AADC expression, AV201 aims to stabilize dopamine production, enhance the therapeutic effectiveness of levodopa, and allow patients to respond to lower doses of the drug with fewer side effects. Clinical development reached Phase I/II trials at the University of California, San Francisco (UCSF).

Other names

AAV-hAADCAAV2-hAADCAAV-2-hAADCAAV 2-hAADCadeno-associated virus-human aromatic L-amino acid decarboxylase