Statements (28)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gptkb:drug
gene therapy |
| gptkbp:administeredBy |
autologous hematopoietic stem cell transplantation
|
| gptkbp:approvalYear |
2019
|
| gptkbp:approvedBy |
gptkb:FDA
gptkb:EMA |
| gptkbp:ATCCode |
not assigned
|
| gptkbp:brand |
gptkb:Zynteglo
|
| gptkbp:cost |
approximately $2.8 million (US, 2022)
|
| gptkbp:developer |
gptkb:bluebird_bio
|
| https://www.w3.org/2000/01/rdf-schema#label |
beti-cel
|
| gptkbp:indication |
transfusion-dependent beta-thalassemia
|
| gptkbp:legalStatus |
prescription only
|
| gptkbp:mechanismOfAction |
inserts functional copies of the beta-globin gene
|
| gptkbp:orphanDrugStatus |
yes
|
| gptkbp:pregnancyCategory |
not established
|
| gptkbp:routeOfAdministration |
intravenous
|
| gptkbp:sideEffect |
fever
vomiting pain infections febrile neutropenia mouth sores low platelet count |
| gptkbp:storage |
cryopreserved
|
| gptkbp:usedFor |
gptkb:beta-thalassemia
|
| gptkbp:bfsParent |
gptkb:bluebird_bio
|
| gptkbp:bfsLayer |
7
|