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Phase 1b results in Parkinsonís
May 2019
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SAN DIEGO & CAMBRIDGE, Mass.—Neurocrine Biosciences Inc. and Voyager Therapeutics Inc. have announced the publication of interim results from the Phase 1b trial of VY-AADC, an investigational gene therapy treatment for Parkinson’s disease. The primary objectives of the trial were to assess the safety, tolerability and distribution of ascending doses of VY-AADC administered under MRI guidance to the putamen, a brain region associated with impaired motor function in Parkinson’s disease.
 
“The interim results from this Phase 1b trial demonstrated that administration of VY-AADC to the putamen using a novel technique, which included intraoperative monitoring with magnetic resonance imaging guidance, facilitated targeted delivery of the investigational gene therapy,” said Dr. Chad Christine, a professor of neurology at the University of California, San Francisco, and investigator in the Phase 1b trial. “Additionally, administration of VY-AADC resulted in dose-dependent increases in AADC enzyme expression and improvements in clinical measures and has been well tolerated to date.”

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