GUCY2D-related recessive retinopathy

MONDO:0100453

A retinopathy caused by biallelic variants in the GUCY2D gene.

Also known as: recessive GUCY2D retinopathy, CORD6, CRB, GUCY2D Leber congenital amaurosis, GUCY2D cone-rod dystrophy, LCA, LCA1, Leber congenital amaurosis 1

26 clinical trials for this condition and its sub-types, 0 tagged with GUCY2D-related recessive retinopathy itself.

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Where it sits in the disease tree

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Part of

↑ Autosomal recessive disease (999) ↑ GUCY2D retinopathy (1)

Sub-types of GUCY2D-related recessive retinopathy

Leber congenital amaurosis 1 1 trial
Night blindness, congenital stationary, type1i 0 trials

Including sub-types (26) Tagged with GUCY2D-related recessive retinopathy (0)

Trials to join now! 11 Not yet recruiting 3 Not yet finished but already full! 1 Completed 9 Terminated 2

Can a single injection restore sight in a rare childhood blindness?

Cure Ongoing

This trial tests a gene therapy called ATSN-101, given as a one-time injection under the retina, for people with Leber congenital amaurosis caused by GUCY2D gene mutations. The goal is to see if the treatment is safe and can improve vision. Participants receive the therapy in one…

Phase 1/2 • Sponsor: Atsena Therapeutics Inc. • Aim: Cure

Last updated Aug 05, 2026 00:00 UTC

GUCY2D-related recessive retinopathy

Where it sits in the disease tree

Can a single injection restore sight in a rare childhood blindness?