We are building a pipeline of RNA therapies for rare genetic diseases. Our most advanced 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.
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
- DiseaseCEP290 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.
Ultevursen (QR-421a)
- Disease
- DiseaseUSH2A mediated retinitis pigmentosa and Usher syndrome
Program Ultevursen (QR-421a) for *USH2A* mediated retinitis pigmentosa and Usher syndrome is now in the phase 2/3, start phase
Ultevursen (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.
Axiomer Platform
- Disease
- DiseaseRNA base editing technology. Lilly – up to 5 targets
Partnered with Lilly
Program Axiomer Platform for RNA base editing technology. Lilly – up to 5 targets is now in the preclinical, start phase
The Axiomer® RNA editing platform technology was discovered at ProQR and enables targeted base-editing in the RNA using endogenous ADAR.
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.
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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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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.
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Partnerships
We are proud partners of Eli Lilly and Company 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.”
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