2017
DOI: 10.1016/s0140-6736(17)31868-8
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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Abstract: Summary Background Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of voretigene neparvovec in participants whose inherited retinal dystrophy would otherwise progress to complete blindness. Methods In this open-label, randomised, controlled phase 3 trial done at two sites in the USA, individuals aged 3 years or older with, in each eye, best corrected visual acuity of 20/60 or worse, or visual … Show more

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Cited by 1,385 publications
(1,260 citation statements)
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References 40 publications
(41 reference statements)
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“…The results of the first phase 3 human gene therapy randomized clinical trial, voretigene neparvovec for the treatment of LCA2, has been recently published (56). Furthermore, more than 10 clinical trials for retinal degeneration have either been completed or initiated (57)(58)(59)(60)(61)(62)(63).…”
Section: Discussionmentioning
confidence: 99%
“…The results of the first phase 3 human gene therapy randomized clinical trial, voretigene neparvovec for the treatment of LCA2, has been recently published (56). Furthermore, more than 10 clinical trials for retinal degeneration have either been completed or initiated (57)(58)(59)(60)(61)(62)(63).…”
Section: Discussionmentioning
confidence: 99%
“…However, clinical CRISPR-Cas studies, which will undoubtedly be initiated using ex vivo editing of differentiated cells that will then be infused into patients, will need to be both more efficient and better controlled for specificity and genetic modification. Yet the first gene editing drug strategies were approved this year for producing CAR-T cells 113,114 and for treating a congenital cause of blindness (Luxturna) 115 using an engineered adeno-associated virus. Experiences with non-RNA-based gene editing tools, such as zinc-finger nucleases, and with siRNA drug delivery will facilitate development of CRISPR-Cas-based drugs 112,116 .…”
Section: Crispr-cas Gene Editingmentioning
confidence: 99%
“…LUXTURNA™ is an AAV vector-based gene therapy indicated for patients with genetically confirmed biallelic RPE65 mutation-associated retinal dystrophy (9-11). The RPE65 gene encodes retinal pigment epithelial 65-kDa protein (RPE65), which is involved in phototransduction (conversion of light photons into a neuronal signal); absent or defective RPE65 caused by mutations results in visual impairment (9).…”
Section: Retinal Gene Therapymentioning
confidence: 99%