AAV-hAADC gene therapy

Drug Profile

AAV-hAADC gene therapy

Alternative Names: AAV-AADC; AAV-AADC gene therapy; AAV-AADC gene therapy - Voyager Therapeutics; AAV-hAADC-2; AAV2-hAADC; Adeno-associated virus encoding human aromatic L-amino acid decarboxylase (AAV-hAADC-2) - Sanofi Genzyme; AV201; GZ 404477; Parkinson's disease gene therapy-AADC - Sanofi Genzyme; Parkinson's disease gene therapy-AADC - Voyager Therapeutics; VY AADC01; VY-AADC

Latest Information Update: 08 Nov 2017

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At a glance

  • Originator University of California at San Francisco
  • Developer National Institutes of Health (USA); Sanofi Genzyme; University of California at San Francisco; Voyager Therapeutics
  • Class Antiparkinsonians; Gene therapies
  • Mechanism of Action Dopamine release stimulants; 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 I Parkinson's disease

Most Recent Events

  • 04 Nov 2017 No recent reports of development identified for phase-I development in Parkinson's-disease in USA (Intrastriatal, Infusion)
  • 02 Nov 2017 AAV-hAADC gene therapy is still in phase I trials for Parkinson's disease in USA
  • 30 Oct 2017 Voyager regains full worldwide development and commercial rights to VY AADC for the treatment of advanced Parkinson’s disease
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