1623 – Voretigene neparvovec (Luxturna) for the treatment of biallelic RPE-65-mediated Inherited Retinal Dystrophies

Page last updated: 20 July 2022

Application Detail

Description of Medical Service

Vortigene 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 Condition

Biallelic 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 Application

Health technology assessment

Medical Service Type


Previous Application Number/s

Not applicable

Associated Documentation

Application Form


Consultation Survey

Consultation Survey (PDF 561 KB)
Consultation Survey (Word 70 KB)

PICO Confirmation


Assessment Report


Public Summary Document

Public Summary Document (PDF 1656 KB)
Public Summary Document (Word 529 KB)

Meetings for this Application




12 June 2020


28-29 July 2020
26-27 November 2020