Neurocrine Moves Into Gene Therapy
Introduction
Staying true to their pipeline targeting movement disorders, Neurocrine Biosciences (NBIX) nabbed a deal with Voyager Therapeutics (VYGR) for the development and commercialization rights to gene therapy programs VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia. The price is $165M upfront and over $1B in possible regulatory and commercial milestone payments.
Mechanism of action is simple & makes sense
VY-AADC is designed to deliver the AADC gene directly to neurons:
The progressive motor symptoms of Parkinson's disease are largely due to the death of