Statements (16)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:administeredBy |
intravenous infusion
|
| gptkbp:alternativeName |
gptkb:Libmeldy
|
| gptkbp:approvalYear |
2020
|
| gptkbp:approvedBy |
gptkb:European_Medicines_Agency
|
| gptkbp:contains |
gptkb:ARSA_gene
genetically modified CD34+ cells |
| gptkbp:developedBy |
gptkb:Orchard_Therapeutics
|
| https://www.w3.org/2000/01/rdf-schema#label |
OTL-200
|
| gptkbp:indication |
early-onset metachromatic leukodystrophy
|
| gptkbp:intendedUse |
gptkb:metachromatic_leukodystrophy
|
| gptkbp:marketedAs |
gptkb:Orchard_Therapeutics
|
| gptkbp:mechanismOfAction |
autologous CD34+ hematopoietic stem cell gene therapy
|
| gptkbp:status |
approved in EU
|
| gptkbp:bfsParent |
gptkb:Libmeldy
|
| gptkbp:bfsLayer |
7
|