Leber congenital amaurosis 5

MONDO:0011473

Any Leber congenital amaurosis in which the cause of the disease is a mutation in the LCA5 gene.

Also known as: LCA5, LCA5 Leber congenital amaurosis, Leber congenital amaurosis 5, Leber congenital amaurosis caused by mutation in LCA5, Leber congenital amaurosis type 5, amaurosis congenita of Leber, type 5

27 clinical trials for this condition and its sub-types, 2 tagged with Leber congenital amaurosis 5 itself.

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Where it sits in the disease tree

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↑ Leber congenital amaurosis (13) ↑ LCA5-related retinopathy (2)

Including sub-types (27) Tagged with Leber congenital amaurosis 5 (2)

Trials to join now! 1 Not yet recruiting 1

One-Time gene injection aims to rescue sight in rare childhood blindness

Disease control Not yet recruiting

This study tests a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare condition that leads to severe vision loss from a young age. The treatment is given as a single injection under the retina in the worse…

Phase 1/2 • Sponsor: Opus Genetics, Inc • Aim: Disease control

Last updated Jul 17, 2026 00:00 UTC

Leber congenital amaurosis 5

Where it sits in the disease tree

One-Time gene injection aims to rescue sight in rare childhood blindness