LCA5-related retinopathy

MONDO:0100445

A retinopathy caused by biallelic variants in the LCA5 gene.

Also known as: LCA5 retinopathy, LCA5-related retinopathy, 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, 1 tagged with LCA5-related retinopathy itself.

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Where it sits in the disease tree

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Part of

↑ Inherited retinal dystrophy (510)

Sub-types of LCA5-related retinopathy

Leber congenital amaurosis 5 2 trials

Including sub-types (27) Tagged with LCA5-related retinopathy (1)

Trials to join now! 1

Experimental gene injection aims to halt rare childhood blindness

Disease control Recruiting now

This study tests a gene therapy called OPGx-001 for people with vision loss caused by LCA5 gene mutations. The treatment is a one-time injection under the retina. The goal is to see if it is safe and can slow or stop vision loss. The study involves 22 participants aged 4 and olde…

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

Last updated Jun 27, 2026 09:06 UTC

LCA5-related retinopathy

Where it sits in the disease tree

Experimental gene injection aims to halt rare childhood blindness