|
gptkbp:instanceOf
|
gene therapy
|
|
gptkbp:administeredBy
|
ophthalmologist
|
|
gptkbp:administeredOnce
|
true
|
|
gptkbp:approvedBy
|
gptkb:FDA
gptkb:EMA
2017
|
|
gptkbp:ATCCode
|
gptkb:S01XA27
|
|
gptkbp:brand
|
gptkb:Luxturna
|
|
gptkbp:cost
|
high
|
|
gptkbp:countryOfOperation
|
gptkb:European_Union
gptkb:United_States
|
|
gptkbp:developer
|
gptkb:Spark_Therapeutics
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
voretigene neparvovec
|
|
gptkbp:indication
|
gptkb:biallelic_RPE65_mutation-associated_retinal_dystrophy
|
|
gptkbp:legalStatus
|
prescription only
|
|
gptkbp:mechanismOfAction
|
delivers functional RPE65 gene
|
|
gptkbp:pregnancyCategory
|
not assigned
|
|
gptkbp:routeOfAdministration
|
subretinal injection
|
|
gptkbp:sideEffect
|
glaucoma
ocular inflammation
increased intraocular pressure
macular hole
|
|
gptkbp:storage
|
-65°C to -85°C
|
|
gptkbp:targetGene
|
gptkb:RPE65
|
|
gptkbp:type
|
recombinant adeno-associated virus vector
|
|
gptkbp:usedFor
|
treatment of inherited retinal dystrophy
|
|
gptkbp:bfsParent
|
gptkb:Luxturna
gptkb:Leber_congenital_amaurosis
|
|
gptkbp:bfsLayer
|
6
|