ELADOCAGENE EXUPARVOVEC
Upstaza (eladocagene exuparvovec) is a gene pharmaceutical. Eladocagene exuparvovec was first approved as Upstaza on 2022-07-18. It has been approved in Europe to treat inborn errors amino acid metabolism.
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Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Argininosuccinic aciduria | D056807 | — | — | — | 1 | — | — | — | 1 |
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General
| Drug common name | ELADOCAGENE EXUPARVOVEC |
| INN | eladocagene exuparvovec |
| Description | Eladocagene exuparvovec, sold under the brand name Upstaza, is a gene therapy product for the treatment of aromatic L‑amino acid decarboxylase (AADC) deficiency. It infuses the gene encoding for the human AADC enzyme into the putamen region of the brain. The subsequent expression of AADC results in dopamine production and, as a result, development of motor function in patients with AADC deficiency.
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| Classification | Gene |
| Drug class | — |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | — |
Identifiers
| PDB | — |
| CAS-ID | — |
| RxCUI | — |
| ChEMBL ID | CHEMBL4298189 |
| ChEBI ID | — |
| PubChem CID | — |
| DrugBank | DB16780 |
| UNII ID | S51J6N56M7 (ChemIDplus, GSRS) |
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