LentiGlobin

GPTKB entity

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