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

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Trial Profile

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

Status: Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 25 Mar 2024

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At a glance

  • Drugs Eladocagene exuparvovec (Primary)
  • Indications Aromatic amino acid decarboxylase deficiency; Inborn error amino acid metabolic disorders
  • Focus Therapeutic Use

Most Recent Events

  • 21 Mar 2024 According to an Agilis Biotherapeutics media release, The Company announced the submission of a BLA to the U.S. FDA
  • 03 Oct 2017 According to an Agilis Biotherapeutics media release, The Company may extend the Phase IIb study to permit the treatment of additional patients in need of therapeutic intervention while it undertakes activities in support of the approval of AGIL-AADC in multiple countries around the world.
  • 03 Oct 2017 According to an Agilis Biotherapeutics media release, dosing of the last of six planned patients has been completed.

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