Statements (96)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:administeredBy |
autologous hematopoietic stem cell transplantation
|
| gptkbp:approvalYear |
2019
2022 |
| gptkbp:approvedBy |
gptkb:FDA
gptkb:EMA |
| gptkbp:ATCCode |
none
|
| gptkbp:developedBy |
gptkb:bluebird_bio
|
| gptkbp:genericName |
gptkb:betibeglogene_autotemcel
|
| https://www.w3.org/2000/01/rdf-schema#label |
Zynteglo
|
| gptkbp:indication |
beta thalassemia
|
| gptkbp:mechanismOfAction |
inserts functional HBB gene
|
| gptkbp:orphanDrugStatus |
yes
|
| gptkbp:priceRange |
$2.8 million (US, 2022)
|
| gptkbp:routeOfAdministration |
intravenous
|
| gptkbp:sideEffect |
nausea
vomiting diarrhea pain edema abdominal pain constipation fatigue headache back pain pruritus alopecia cough infections chills rash hypokalemia hyponatremia thrombocytopenia leukopenia arthralgia myalgia decreased appetite pyrexia dysgeusia febrile neutropenia hypomagnesemia hypophosphatemia increased alanine aminotransferase increased aspartate aminotransferase increased blood HDL cholesterol increased blood LDL cholesterol increased blood VLDL cholesterol increased blood albumin increased blood alkaline phosphatase increased blood ammonia increased blood amylase increased blood bicarbonate increased blood bilirubin increased blood calcium increased blood chloride increased blood cholesterol increased blood copper increased blood creatine kinase increased blood creatinine increased blood ferritin increased blood folate increased blood globulin increased blood glucose increased blood iron increased blood lactate increased blood lactate dehydrogenase increased blood lipase increased blood magnesium increased blood phosphorus increased blood potassium increased blood pyruvate increased blood selenium increased blood sodium increased blood total protein increased blood triglycerides increased blood urea increased blood uric acid increased blood vitamin A increased blood vitamin B1 increased blood vitamin B12 increased blood vitamin B2 increased blood vitamin B3 increased blood vitamin B5 increased blood vitamin B6 increased blood vitamin C increased blood vitamin D increased blood vitamin E increased blood vitamin K increased blood zinc increased gamma-glutamyltransferase mouth pain pain in extremity |
| gptkbp:usedFor |
transfusion-dependent beta thalassemia
|
| gptkbp:bfsParent |
gptkb:gene_therapy
|
| gptkbp:bfsLayer |
5
|