VY-AADC

VY-AADC is an investigational gene therapy product designed to deliver the AADC gene directly into neurons of the putamen where dopamine receptors are located, bypassing the substantia nigra neurons and enabling the neurons of the putamen to produce the AADC enzyme to convert levodopa into dopamine. With this approach, VY-AADC has the potential to durably enhance the conversion of levodopa to dopamine and provide clinically meaningful improvements by restoring motor function in patients and improving symptoms following a single administration.

VY-AADC is currently being evaluated in the Phase II RESTORE-1 trial in patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day, as measured by a validated self-reported patient diary.

VY-AADC is being developed by Neurocrine Biosciences and Voyager Therapeutics as part of a strategic collaboration announced in January 2019.