Orphanet: Adenovirus associated viral vector serotype 2 containing the human RPE65 gene (AAV2 hRPE65v2 )

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Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (AAV2-hRPE65v2 )

  • INN (International Nonproprietary Name) : voretigene neparvovec
  • Code name : -
  • Chemical name or description : recombinant adeno-associated serotype 2 (rAAV-2) virus vector that carries the RPE65 gene, encoding a retinal pigment (RPE)-specific human retinoid isomerohydrolase, containing a modified Kozak sequence at the translation start site and under the control of the cytomegalovirus (CMV) immediate early enhancer and the chicken beta-actin (CBA) promote
  • Type of product : Gene therapy product
  • Type of production : Biotechnology
  • Orphan designation : EuropeUSA
    • Marketing authorization with orphan designation  - USA

    • Diseases list
      • Retinitis pigmentosa
      • Leber congenital amaurosis
      • Severe early-childhood-onset retinal dystrophy
    • Detailed information

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