Investigational RNA therapy QR-504a for Fuchs endothelial corneal dystrophy

A close up of the eyes of an elderly woman with a close up of a younger woman in the background

QR-504a is an investigational RNA therapy that aims to slow down vision loss in people with Fuchs endothelial corneal dystrophy (FECD) due to trinucleotide 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 slow down vision loss in Fuchs endothelial corneal dystrophy type 3 (FECD3) due to trinucleotide repeat expansion mutations in the TCF4 gene.

How does QR-504a work?

QR-504a aims to slow down degeneration of the cornea and thereby vision loss in people with Fuchs endothelial corneal dystrophy due to the most common mutation. These trinucleotide 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 degeneration of the cornea.

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

Ongoing, recruiting

Fuchs Focus logo

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.

More about Fuchs Focus