Luxturna

GPTKB entity

Statements (32)
Predicate Object
gptkbp:instanceOf gene therapy
gptkbp:administeredBy ophthalmologist
single dose per eye
gptkbp:age patients aged 1 year and older
gptkbp:approvalYear 2017
gptkbp:approvedBy gptkb:FDA
gptkb:EMA
gptkbp:ATCCode gptkb:S01XA27
gptkbp:countryOfOrigin gptkb:United_States
gptkbp:developedBy gptkb:Spark_Therapeutics
gptkbp:firstBook yes
gptkbp:genericName gptkb:voretigene_neparvovec
https://www.w3.org/2000/01/rdf-schema#label Luxturna
gptkbp:indication gptkb:Leber_congenital_amaurosis
retinal dystrophy due to RPE65 mutation
gptkbp:mechanismOfAction delivers functional RPE65 gene
gptkbp:orphanDrugStatus yes
gptkbp:prescriptionStatus prescription only
gptkbp:priceRange $850,000 per treatment
gptkbp:routeOfAdministration subretinal injection
gptkbp:sideEffect glaucoma
ocular inflammation
increased intraocular pressure
endophthalmitis
macular hole
retinal tear
gptkbp:storage refrigerated
gptkbp:targetGene gptkb:RPE65
gptkbp:type AAV2-based gene therapy
gptkbp:yearOfEMAApproval 2018
gptkbp:bfsParent gptkb:gene_therapy
gptkbp:bfsLayer 5