Description of Medical ServiceVortigene neparvovec (VN) is a recombinant adeno-associated viral serotype 2 (AAV2) gene therapy which must be delivered in close proximity to the retinal pigment epithelium (RPE) target cells. To access the retina, the vitreous gel that fills the eye is completely removed through a vitrectomy. VN is then injected into the space between the retina and the RPE, as a “subretinal” injection.
Description of Medical ConditionBiallelic RPE-65-mediated inherited retinal dystrophies (IRD), the most severe form of IRD, are a major cause of early-onset blindness. They occur when when there are mutations in both alleles of the RPE65 gene in RPE cells.
Reason for ApplicationHealth technology assessment
Medical Service TypeTherapeutic
Previous Application Number/sNot applicable
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