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 and dystrophic epidermolysis bullosa.
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 65,000 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
Dystrophic Epidermolysis Bullosa
Dystrophic epidermolysis bullosa (DEB) is a severe genetic skin disease. Children with DEB are often called 'Butterfly children' as their skin is as fragile as the wings of a butterfly. We are developing several programs for different mutations causing DEB including QR-313 for approximately 2,000 patients that have DEB due to mutations in exon 73 of the COL7A1 gene.
Read more about QR-313 for dystrophic epidermolysis bullosa
Going beyond cystic fibrosis, Leber’s congenital amaurosis and dystrophic epidermolysis bullosa our innovation unit is working on applying our RNA technologies to other diseases including Usher syndrome and Fuchs endothelial corneal dystrophy.
Read more about our innovation programs
Spin out: Amylon Therapeutics
In September 2017 ProQR spun out Amylon Therapeutics as a privately-held company focused on the development of therapies for diseases of the central nervous system including a rare genetic disease which leads to strokes at mid-adulthood, called HCHWA-D. ProQR retains a majority ownership stake in Amylon.
Read more about the spin out in the press release
Visit the Amylon website