Science & Pipeline RNA technology to benefit patients

Two ProQR scientists working in the lab

At ProQR science is literally at the heart of what we do. The offices at our headquarters in Leiden are centered around the laboratories where our scientists discover and test our novel RNA therapies. Our passion lies in translating the latest RNA technologies to life-changing treatments for people with rare genetic diseases.

Our research and development pipeline

We are building a broad pipeline of RNA therapies. Most of our programs focus on a group of rare diseases, known as inherited retinal diseases, that affect the retina. Our investigational treatments range from preclinical discovery to late stage clinical trials.

Clinical stage 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.