AAV-hAADC gene therapy - Agilis/National Taiwan University

Drug Profile

AAV-hAADC gene therapy - Agilis/National Taiwan University

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

Latest Information Update: 25 Oct 2017

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At a glance

  • Originator National Taiwan University
  • Developer Agilis Biotherapeutics; National Taiwan University
  • 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.

    Yes - CNS disorders
  • New Molecular Entity No

Highest Development Phases

  • Phase II Inborn error metabolic disorders

Most Recent Events

  • 03 Oct 2017 Agilis Biotherapeutics announces intention to submit regulatory applications in Europe and Japan
  • 03 Oct 2017 Pooled efficacy data from clinical trials in Inborn error metabolic disorders released by Agilis Biotherapeutics
  • 06 Sep 2017 Agilis Biotherapeutics announces intention to submit BLA to US FDA for Inborn error metabolic disorders in 2018
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