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.
Science & Pipeline RNA technology to benefit patients
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 discovering the latest RNA technologies and translating them to life-changing treatments for people with rare genetic diseases.
Our research and development pipeline
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, 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 2/3, start phase
QR-421a is an RNA therapy that aims to stop vision loss 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 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 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.
RNA therapy
Our antisense oligonucleotides are designed to correct mutations in the RNA of someone with a genetic disease. This is different from gene therapy as we target RNA instead of DNA. Our highly precise approach aims to stop disease progression or even reverse symptoms.
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Axiomer RNA base editing
Besides the technologies underlying our development programs, we invented the Axiomer technology that enables single base editing to target currently untreatable diseases. We have partnered with Eli Lilly on this platform technology.
View this technology
Scientific presentations and publications
Our clinical trial results and the groundbreaking science underlying our pipeline programs were published by world-class key opinion leaders as well as our scientists.
See the science
Partnerships
We are proud partners of Eli Lilly and Company and Yarrow Biotechnology who in-licensed our RNA technologies to develop new medicines to potentially address important unmet medical needs.
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Meet Gerard Platenburg, co-founder and Chief Innovation Officer at ProQR
Always excited about RNA - “I really enjoy turning new scientific ideas into a clinical reality.”
Read the interview
