QR-504a aims to prevent or slow down degeneration of the cornea and thereby vision loss in people with Fuchs endothelial corneal dystrophy due to the most common mutation. These trinuclear repeat (TNR) expansion mutations cause the RNA to cluster inside the corneal endothelial cells. QR-504a works by binding to the TNR repeats in the TCF4 RNA to stop clustering and prevent degeneration of the cornea.
Investigational RNA therapy QR-504a for Fuchs endothelial corneal dystrophy
QR-504a is an investigational RNA therapy that aims to prevent or slow down vision loss in people with Fuchs endothelial corneal dystrophy (FECD) due to trinuclear repeat expansion mutations in the TCF4 gene.
QR-504a
- Disease
- TCF4 mediated Fuchs endothelial corneal dystrophy
Program QR-504a for TCF4 mediated Fuchs endothelial corneal dystrophy is now in the phase 1/2, start phase
QR-504a is an RNA therapy that aims to prevent or slow down vision loss in Fuchs endothelial corneal dystrophy type 3 (FECD3) due to trinuclear repeat expansion mutations in the TCF4 gene.
How does QR-504a work?
QR-504a clinical trials
We are conducting a Phase 1 clinical trial of QR-504a, to test whether it is safe and whether it has an effect on FECD biomarkers.
Learn more about clinical trials and why you should consider participating.
Fuchs Focus study - Phase 1 clinical trial
Planned, not yet recruiting
Fuchs Focus is a clinical trial of QR-504a. The clinical trial will include people that are scheduled to receive a corneal transplant (DMEK) and lens replacement in both eyes. Participants will receive QR-504a through an intravitreal (into the eye) injection in one eye.
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