ProQR Therapeutics We strive to reverse blindness

Living with Inherited retinal disease - Maartje de Kok and her family

Our programs

Sepofarsen pediatric trial starts

We have started a new clinical study of sepofarsen named Brighten, for children with CEP290 mediated Leber congenital amaurosis. The study investigates whether sepofarsen is safe and well-tolerated in children younger than 8 years old. Sepofarsen is an investigational RNA therapy that aims to improve vision.

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The Brighten logo with a stamp 'Started' on a desk scattered with toys, a stethoscope and an eye chart.

Stories that inspire our work

  • Life around the diagnosis with Leber congenital amaurosis

    Laura Steinbusch has a son, 5-year-old Enzo, who has been diagnosed with Leber congenital amaurosis 10, or LCA10. It has made Laura a patient advocate for this disease, and in this interview she explains how the diagnosis has changed her life.

    • Genetic Testing
    • Blindness
    • Story
    • Leber congenital amaurosis
    • Sepofarsen (QR-110)
    Photo of Laura Steinbusch and family

  • In it for the patient

    The ProQR family is growing fast and we spoke to the Chief Medical Officer Aniz Girach who joined ProQR in 2019. We asked him about his impression of the company after he joined, as well as the new developments he’s been working on.

    • Pipeline
    • Clinical Trial
    • Blindness
    • IRD general
    • Programs general
    • Sepofarsen (QR-110)
    A photo of Aniz Girach

  • Serving a tight-knit community

    The Netherlands boasts the most prominent patient organization for Usher syndrome. Part of their secret is the passion of the Board. We caught up with president Ivonne Bressers and the Board’s secretary Annouk van Nunen at Annouk’s home in Leiden.

    • Patient Advocacy
    • Genetic Testing
    • Story
    • Usher syndrome
    • QR-421a
    Photo of Annouk van Nunen and Ivonne Bressers

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.

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

Latest press releases

More press releases
Female scientist working in the lab

Free genetic testing now available

ProQR has teamed up with the Foundation Fighting Blindness to provide no-cost genetic testing to inherited retinal disease patients in the United States. Learn how the My Retina Tracker program empowers the US IRD community and helps research into new treatments.

Learn about My Retina Tracker
My Retina Tracker logo

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