KEGG   DRUG: Voretigene neparvovec
Entry
D11008                      Drug                                   
Name
Voretigene neparvovec (USAN/INN);
Voretigene neparvovec-rzyl;
Luxturna (TN)
Remark
ATC code: S01XA27
Efficacy
Vision loss treatment
  Disease
Retinal dystrophy (biallelic RPE65 mutated) [DS:H00527 H00837]
  Type
Gene therapy product
Comment
Adeno-associated virus serotype 2 (AAV2) vector carrying a functional copy of the RPE65 gene into the retinal pigment epithelial (RPE) cells
Target
RPE65 [HSA:6121] [KO:K11158]
  Pathway
hsa00830  Retinol metabolism
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 S SENSORY ORGANS
  S01 OPHTHALMOLOGICALS
   S01X OTHER OPHTHALMOLOGICALS
    S01XA Other ophthalmologicals
     S01XA27 Voretigene neparvovec
      D11008  Voretigene neparvovec (USAN/INN)
Target-based classification of drugs [BR:br08310]
 Enzymes
  Hydrolases (EC3)
   Carboxylic-ester hydrolases
    RPE65
     D11008  Voretigene neparvovec (USAN/INN)
New drug approvals in the USA [br08319.html]
 Cellular and gene therapy products
  D11008
New drug approvals in Europe [br08329.html]
 European public assessment reports (EPAR) authorised medicine
  D11008
 Cellular and gene therapy products
  D11008
New drug approvals in Japan [br08318.html]
 Cellular and gene therapy products
  D11008
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D11008
Other DBs
CAS: 1646819-03-5
PubChem: 342581844
LinkDB

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