ProQR's mission is to make a meaningful impact on the lives of patients suffering from severe genetic disorders using our unique RNA technologies. ProQR was born from the dream to beat cystic fibrosis in one child, but we won’t stop there. We think bigger and our pipeline molecules target other severe disorders such as Leber’s congenital amaurosis Type 10, Usher syndrome, Fuchs endothelial corneal dystrophy, dystrophic epidermolysis bullosa and Alzheimer’s disease.
We believe all patients should be allowed to dream about the future. That is what makes us passionate and impatient. We dare to do things differently and challenge the status quo, all in the interest of patients. When you’re as driven to make a meaningful impact as we are, this can only be the start of something big. But, we’re not there yet and therefore it is our duty to make a difference today.
Born from the dream to beat cystic fibrosis, our first goal is to develop a treatment for more than 49,000 patients with the most common genetic mutation, F508del. We’re making great progress in achieving this goal. Our candidate medicine, called QR-010, showed very encouraging results in a first clinical trial in CF patients and a second trial is ongoing.
Read more about QR-010 for cystic fibrosis and clinical trials
Leber’s Congenital Amaurosis
We are developing a potentially life changing therapy for Leber's congenital amaurosis Type 10 (LCA 10) due to the p.Cys998X mutation in the CEP290 gene. LCA 10 is a genetic eye disorder and the leading genetic cause of childhood blindness.
Read more about QR-110 for Leber's congenital amaurosis Type 10
Going beyond cystic fibrosis and Leber’s congenital amaurosis our innovation unit is working on applying our RNA technologies to other diseases including Usher syndrome, Fuchs endothelial corneal dystrophy, dystrophic epidermolysis bullosa and Alzheimer’s disease.
Read more about our innovation programs