Drug Profile


Alternative Names: AADC deficiency gene therapy programme - Agilis; AAV-hAADC-gene-therapy-Agilis/National-Taiwan-University; AAV2-hAADC - Agilis/National Taiwan University; GT-AADC

Latest Information Update: 18 May 2018

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At a glance

  • Originator National Taiwan University
  • Developer Agilis Biotherapeutics; National Taiwan University
  • Class Gene therapies
  • Mechanism of Action Dopamine release stimulants; Gene transference; Serotonin release stimulants
  • Orphan Drug Status

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

    Yes - CNS disorders
  • New Molecular Entity No

Highest Development Phases

  • Phase II Inborn error metabolic disorders

Most Recent Events

  • 19 Jul 2018 PTC Therapeutics announces intention to submit BLA to US FDA for Inborn error metabolic disorders in 2019
  • 15 May 2018 Efficacy data from a phase I/II trial in Inborn error metabolic disorders released by Agilis Biotherapeutics
  • 14 May 2018 Efficacy data from a clinical trial in Inborn error metabolic disorders released by Agilis Biotherapeutics
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