Neurology Central

RESTORE-1: first patient dosed with gene therapy in Parkinson’s trial

Voyager Therapeutics (MA, USA) has announced that they have dosed the first patient in RESTORE-1, a Phase II, randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of VY-AADC for the treatment of Parkinson’s disease (PD) in patients with motor fluctuations that are refractory to medical management.

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. This approach bypasses the substantia nigra neurons and is reported to enable the neurons of the putamen to produce the AADC enzyme to convert levodopa into dopamine.

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