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
Consultation SurveyConsultation Survey (PDF 561 KB)
Consultation Survey (Word 70 KB)
Public Summary DocumentPublic Summary Document (PDF 1007 KB)
Public Summary Document (Word 528 KB)
Meetings for this Application
ESC12 June 2020
MSAC28-29 July 2020
26-27 November 2020