ProQR is dedicated to developing RNA therapies to improve the lives of patients, families and communities living with inherited retinal diseases, a group of rare diseases causing blindness. To achieve this goal, ProQR strives to integrate the patient voice into our decision-making throughout the drug development process as we believe that a patient-focused strategy is crucial to our success.
Check out our blog that features patient stories, interviews, podcast episodes, program updates and more.
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Inherited retinal diseases (IRDs) are a group of rare genetic disorders that can cause severe vision loss or blindness, affecting around 2 million people worldwide.
Here is a list of all our ongoing clinical trials.
If you are not sure what a clinical trial is all about? Read this first.
Phase 1/2 clinical trial of QR-1123 for RHO mediated retinitis pigmentosa.
Clinical trial of sepofarsen for children with CEP290 mediated Leber congenital amaurosis
Phase 1 clinical trial of QR-504a, an investigational RNA therapy for TCF4 mediated Fuchs endothelial corneal dystrophy. This trial will recruit individuals that have FECD due to trinuclear repeat expansion mutations in the TCF4 gene.
Phase 2/3 clinical trial for USH2A mediated retinitis pigmentosa and Usher syndrome. This trial will include adults (age 18 and up) that have advanced vision loss.
Phase 2/3 clinical trial for USH2A mediated retinitis pigmentosa and Usher syndrome. This trial will include adults (age 18 and up) that have early to moderate vision loss.
We started a new clinical study of sepofarsen 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.
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Learn how the latest RNA technologies can help treat rare genetic diseases
