R&D pipeline RNA therapies for rare genetic diseases

Box with eyecup cell cultures held by a ProQR laboratory technician

At ProQR we develop antisense oligonucleotides, or RNA therapies, to treat genetic diseases that are rare. We have made significant progress in advancing our broad pipeline of investigational medicines through the various stages of drug development, from early discovery to late stage clinical trials.

Our platform for inherited retinal diseases

Most of our drug development programs are focused on a group of rare diseases affecting the retina, called inherited retinal diseases, or IRDs. IRDs cause vision loss and blindness and the vast majority of these disorders currently have no treatment options. We have built a platform to efficiently discover and develop RNA therapies for genetic eye diseases in order to stop or reverse vision loss.

Most advanced pipeline programs

Sepofarsen (QR-110)

Disease
CEP290 mediated Leber congenital amaurosis (LCA10)

Program Sepofarsen (QR-110) for CEP290 mediated Leber congenital amaurosis (LCA10) is now in the phase 2/3, start phase

Sepofarsen (QR-110) is an RNA therapy that aims to restore vision in Leber congenital amaurosis 10 (LCA10) due to the most common p.Cys998X mutation in the CEP290 gene.

QR-421a

Disease
USH2A mediated retinitis pigmentosa and Usher syndrome

Program QR-421a for USH2A mediated retinitis pigmentosa and Usher syndrome is now in the phase 1/2 phase

QR-421a is an RNA therapy that aims to prevent vision loss or restore vision in retinitis pigmentosa (RP) and Usher syndrome type 2 due to mutations in exon 13 of the USH2A gene.

QR-1123

Disease
RHO mediated retinitis pigmentosa
Discovered by Ionis

Program QR-1123 for RHO mediated retinitis pigmentosa is now in the phase 1/2, start phase

QR-1123 is an RNA therapy that aims to prevent vision loss or restore vision in retinitis pigmentosa (RP) due to the P23H mutation in the RHO gene.

QR-504a

Disease
TCF4 mediated Fuchs endothelial corneal dystrophy

Program QR-504a for TCF4 mediated Fuchs endothelial corneal dystrophy is now in the preclinical, finished 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.

QR-411

Disease
USH2A mediated retinitis pigmentosa and Usher syndrome

Program QR-411 for USH2A mediated retinitis pigmentosa and Usher syndrome is now in the preclinical phase

QR-411 is an RNA therapy that aims to prevent vision loss or restore vision in retinitis pigmentosa (RP) and Usher syndrome type 2 due to the PE40 mutation in the USH2A gene.

Discovery programs

We have numerous programs in discovery stage targeting other mutations causing inherited retinal diseases such as Leber congenital amaurosis, retinitis pigmentosa and Stargardt disease.