ProQR Therapeutics We strive to reverse blindness

Living with Leber congenital amaurosis 10 - Beatrice

Our programs

New clinical trial for USH2A mediated RP and Usher

We have started a Phase 2/3 clinical trial of ultevursen (QR-421a), an investigational RNA therapy that aims to stop vision loss in retinitis pigmentosa and Usher syndrome due to mutations in exon 13 of the USH2A gene. This the first potentially pivotal trial for these conditions and it was started after we saw encouraging results in a Phase 1/2 clinical trial of ultevursen. The Sirius study is recruiting adults and children (age 12 and up) that want to participate.

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Sirius and Celeste logos with a Started stamp on a desk scattered with a cane, hearing aids, eye chart and a clinical trial design graph.

Featured blogs, stories and news

  • ProQR update and additional sepofarsen Illuminate trial analyses

    Community news

    ProQR has announced the completion of an in-depth strategic review designed to deliver on our commitment to advance RNA therapies for diseases with high unmet need. We are also sharing additional analyses of the sepofarsen Illuminate trial.

  • Rare Disease Day 2022

    Community news

    On behalf of all ProQRians, happy Rare Disease Day 2022. Both within ProQR and outside, we join the community in raising awareness and trying to generate change for people worldwide living with a rare disease, their families, and supporters.

    28 February 2022 is Rare Disease Day. Rare disease day logo on the top right. Portrait of Maartje living with Usher syndrome on the right. #RareDiseaseDay www.rarediseaseday.org

  • Managing your inherited retinal disease

    Community news

    Ben Shaberman, Senior Director of Scientific Outreach at the Foundation Fighting Blindness: The diagnosis of a retinal degenerative disease is often overwhelming. But if you or a loved one has been diagnosed with an inherited retinal condition, here are some helpful steps you can take to get on a positive path moving forward.

    A picture of the retina under a microscope

Patient-focused drug development

We are a biotechnology company dedicated to changing lives. Our focus lies with developing RNA therapies to reverse vision loss for people living with inherited retinal disease (IRD). From day one we have put patients first and we continue striving to incorporate their voice at every step of drug-development. We believe this patient-focused approach is crucial to our success.

Internal link For the IRD community
A photo of Cristian, Silvia and their kids Edoardo and Beatrice. Beatrice has Lber congenital amaurosis 10

Latest press releases

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Female scientist working in the lab

Lilly partnership announcement

We announced a global licensing and research collaboration with Eli Lilly and Company focused on the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The companies will use ProQR’s proprietary Axiomer® RNA editing platform to progress new drug targets toward clinical development and commercialization.

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An illustration of an ADAR enzyme (in white) attached to an RNA molecule (in green)

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