Prospective study of AADC gene therapy (AAV-hAADC gene therapy) in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

Trial Profile

Prospective study of AADC gene therapy (AAV-hAADC gene therapy) in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 29 Jun 2016

At a glance

  • Drugs AAV-hAADC (Primary)
  • Indications Inborn error amino acid metabolic disorders
  • Focus Therapeutic Use
  • Most Recent Events

    • 29 Jun 2016 According to an Agilis Biotherapeutics media release, the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Agilis' gene therapy product candidate, AGIL-AADC, being developed for the treatment of Aromatic L-amino Acid Decarboxylase (AADC) deficiency.
    • 29 Jun 2016 Results published in an Agilis Biotherapeutics media release.
    • 02 Mar 2016 New trial record
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