|
gptkbp:instanceOf
|
gene therapy
biological drug
|
|
gptkbp:administeredBy
|
autologous hematopoietic stem cell transplantation
|
|
gptkbp:approvalYear
|
2022
|
|
gptkbp:approvedBy
|
gptkb:European_Union
gptkb:United_States
|
|
gptkbp:ATCCode
|
not assigned
|
|
gptkbp:brand
|
gptkb:Zynteglo
Lyfgenia
|
|
gptkbp:clinicalTrialPhase
|
Phase 3
|
|
gptkbp:cost
|
over $2 million per treatment
|
|
gptkbp:developedBy
|
gptkb:bluebird_bio
|
|
gptkbp:EMAApprovalDate
|
2019
|
|
gptkbp:geneticModification
|
ex vivo
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
LentiGlobin BB305
|
|
gptkbp:indication
|
sickle cell disease with vaso-occlusive crises
transfusion-dependent beta-thalassemia
|
|
gptkbp:mechanismOfAction
|
delivers functional HBB gene
|
|
gptkbp:orphanDrugStatus
|
yes
|
|
gptkbp:routeOfAdministration
|
intravenous
|
|
gptkbp:sideEffect
|
pain
infections
febrile neutropenia
myelosuppression
insertional oncogenesis risk
|
|
gptkbp:storage
|
cryopreserved
|
|
gptkbp:targetGene
|
gptkb:HBB
|
|
gptkbp:usedFor
|
gptkb:beta-thalassemia
sickle cell disease
|
|
gptkbp:vectorFor
|
lentiviral vector
|
|
gptkbp:bfsParent
|
gptkb:betibeglogene_autotemcel
|
|
gptkbp:bfsLayer
|
7
|