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 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)

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

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

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.

View our entire pipeline

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 editing

Besides the technologies underlying our pipeline, we have invented new ways to alter RNA. Our Axiomer RNA editing oligonucleotides can edit specific single nucleotides with the potential to target currently untreatable diseases.

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Visualisation of the Axiomer technology at work, in a cell

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