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 discovering the latest RNA technologies and translating them to life-changing treatments for people with rare genetic diseases.

Our research and development pipeline

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.

R&D pipeline

Sepofarsen (QR-110)

Disease
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.

Ultevursen (QR-421a)

Disease
Disease
USH2A 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
Disease
RNA 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.

Yarrow Biotechnology

Disease
Disease
Undisclosed non-ophtha target
Partnered with Yarrow

Program Yarrow Biotechnology for Undisclosed non-ophtha target is now in the preclinical, start phase

Yarrow Biotechnology in-licensed exclusive rights to ProQR’s RNA technology to develop and commercialize potential therapies for an undisclosed non-ophthalmic target.