Statements (24)
Predicate | Object |
---|---|
gptkbp:instanceOf |
gene therapy
|
gptkbp:administeredBy |
autologous hematopoietic stem cell transplantation
|
gptkbp:alternativeName |
gptkb:Zynteglo
gptkb:beti-cel |
gptkbp:approvalYear |
2019
|
gptkbp:clinicalTrialPhase |
Phase 3
|
gptkbp:countryOfOrigin |
gptkb:European_Union
|
gptkbp:deliveredBy |
lentiviral vector
|
gptkbp:developedBy |
gptkb:bluebird_bio
|
gptkbp:EMAStatus |
approved
|
https://www.w3.org/2000/01/rdf-schema#label |
LentiGlobin
|
gptkbp:mechanismOfAction |
introduces functional HBB gene
|
gptkbp:regulates |
approved (as beti-cel for beta-thalassemia)
|
gptkbp:sideEffect |
infections
febrile neutropenia myelosuppression insertional mutagenesis risk |
gptkbp:targetGene |
gptkb:HBB
|
gptkbp:treatment |
one-time therapy
|
gptkbp:usedFor |
gptkb:beta-thalassemia
sickle cell disease |
gptkbp:bfsParent |
gptkb:bluebird_bio
gptkb:Bluebird_Bio |
gptkbp:bfsLayer |
7
|