Leber congenital amaurosis 2
MONDO:0008765Any Leber congenital amaurosis in which the cause of the disease is a mutation in the RPE65 gene.
Also known as: LCA2, Leber congenital amaurosis 2, Leber congenital amaurosis caused by mutation in RPE65, Leber congenital amaurosis type 2, RPE65 Leber congenital amaurosis, amaurosis congenita of Leber 2, amaurosis congenita of Leber, type 2
28 clinical trials for this condition and its sub-types, 3 tagged with Leber congenital amaurosis 2 itself.
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A gene therapy injected into the eye aims to restore vision lost to a rare inherited retinal disease
Cure CompletedResearchers are testing a gene therapy called rAAV2/4.hRPE65 in people with Leber congenital amaurosis or severe early-onset retinal degeneration caused by mutations in the RPE65 gene. The treatment delivers a working copy of the gene directly into one eye through a single inject…
Phase 1/2 • Sponsor: Nantes University Hospital • Aim: Cure
Last updated Sep 16, 2026 00:00 UTC
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Mapping the slow fade of RPE65 blindness to speed future cures
Knowledge-focused CompletedResearchers are following people with Leber congenital amaurosis caused by RPE65 gene mutations to see how their vision and retinas change over time. The study enrolls children and adults, ages 3 and older, who have this inherited retinal dystrophy. Participants undergo regular e…
Sponsor: MeiraGTx UK II Ltd • Aim: Knowledge-focused
Last updated Sep 21, 2026 18:00 UTC