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
- 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