New success in AAV-based gene therapy: Voyager Therapeutics Announces Positive Interim Phase 1b in Parkinson’s disease

By December 19, 2016Non class


The interim surgical data from the Phase 1b study showed that VY-AADC01 continues to demonstrate safety with increasing coverage of the putamen (targeted region in the brain) in all the 10 Parkinson’s disease patients treated in the ongoing dose escalation trial.

Obtaining sufficient coverage of the putamen with VY-AADC01 is a key step towards potentially improving patients’ response to levodopa (levodopa is the standard of care treatment for Parkinson’s disease). Having reached an average coverage of 34% in the high-dose cohort, Voyager demonstrates that through direct injection in the brain one can achieve significant diffusion volumes for AAV vectors, confirming hopes of AAV-based gene therapy to treat efficiently neurodegenerative diseases.

See the details of the results here