Eladocagene exuparvovec, sold under the brand name Upstaza among others, is a gene therapy product for the treatment of aromatic L‑amino acid decarboxylase (AADC) deficiency.
[3] It infuses the gene encoding for the human aromatic L-amino acid decarboxylase enzyme into the putamen region of the brain.
[4] Eladocagene exuparvovec was approved for medical use in the European Union in July 2022,[3] and in the United States in November 2024.
[3] Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters, which are chemical messengers that allow cells in the body's nervous system to communicate with each other.
[4] The safety and effectiveness of eladocagene exuparvovec were demonstrated in an open-label, single-arm clinical study in 13 children with confirmed diagnosis of aromatic L-amino acid decarboxylase deficiency.