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Drug Profile


Alternative Names: AAV-AADC; AAV-hAADC; AAV-hAADC-2; AAV-hAADC-gene-therapy - Voyager Therapeutics; AAV2-hAADC; AAV2-hAADC - Voyager Therapeutics; Aromatic-L-amino-acid-decarboxylase-gene-therapy; AV 201; GZ 404477; VY AADC01; VY AADC02

Latest Information Update: 31 Jan 2019

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At a glance

  • Originator University of California at San Francisco
  • Developer National Institutes of Health (USA); University of California at San Francisco; Voyager Therapeutics
  • Class Antiparkinsonians; Gene therapies
  • Mechanism of Action Aromatic-L-amino-acid decarboxylase replacements; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity No

Highest Development Phases

  • Phase II Parkinson's disease

Most Recent Events

  • 29 Jan 2019 Neurocrine Biosciences enter into an option agreement with Voyager Therapeutics to develop and commercialise VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia
  • 07 Jan 2019 Voyager plans a phase III RESTORE-2 trial for Parkinson's disease of similar design and size of RESTORE-1 trial in USA and worldwide in H1 of 2020
  • 07 Nov 2018 Long-term efficacy data from a phase Ib trial in Parkinson's disease released by Voyager Therapeutics
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