Statements (21)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gptkb:drug
gene therapy |
| gptkbp:administeredBy |
intravenous infusion
|
| gptkbp:approvalYear |
2020
|
| gptkbp:approvedBy |
gptkb:European_Union
|
| gptkbp:ATCCode |
not assigned
|
| gptkbp:brand |
gptkb:Libmeldy
|
| gptkbp:contains |
autologous CD34+ cells encoding ARSA gene
|
| gptkbp:developer |
gptkb:Orchard_Therapeutics
|
| gptkbp:EMAApprovalStatus |
approved
|
| https://www.w3.org/2000/01/rdf-schema#label |
atidarsagene autotemcel
|
| gptkbp:indication |
early-onset metachromatic leukodystrophy
|
| gptkbp:mechanismOfAction |
restores ARSA enzyme activity
|
| gptkbp:orphanDrugStatus |
yes
|
| gptkbp:regulates |
not approved as of 2024
|
| gptkbp:routeOfAdministration |
intravenous
|
| gptkbp:source |
autologous hematopoietic stem cells
|
| gptkbp:treatment |
ex vivo gene therapy
|
| gptkbp:usedFor |
gptkb:metachromatic_leukodystrophy
|
| gptkbp:bfsParent |
gptkb:Libmeldy
|
| gptkbp:bfsLayer |
7
|