We are developing sepofarsen for Leber's congenital amaurosis 10 (LCA10), a genetic eye disorder and the leading genetic cause of childhood blindness. There is no approved treatment. A substantive overall improvement in vision in the majority of patients was observed in an ongoing clinical trial of sepofarsen, a potentially life changing therapy for people living with LCA10 due to the p.Cys998X mutation in the CEP290 gene.Read more about sepofarsen for Leber's congenital amaurosis 10 and clinical trials
Research and development pipeline
We are developing a pipeline of novel medicines for patients in need. Our pipeline focusses on diseases that are very severe and have limited treatment options. All of the medicines we are developing are RNA therapies and target the underlying cause in the diseases.
We are developing a novel investigational drug called QR-421a to treat the vision loss associated with Usher syndrome, the leading cause of combined deafness and blindness. QR-421a is designed to exclude a specific part of the USH2A mRNA, called exon 13, thereby removing the mutation and potentially reversing the disease. This approach is also known as exon skipping.Read more about QR-421a for Usher syndrome type 2
QR-1123 is novel investigational drug for patients that have autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin gene. adRP leads to poor vision and blindness for which there is currently no approved treatment. Symptoms usually start with night blindness and tunnel vision during childhood and progresses to blindness during adulthood. QR-1123 is designed to block the formation of the toxic mutated protein that causes the disease.Read more about QR-1123 for P23H autosomal dominant retinitis pigmentosa
Fuchs endothelial corneal dystrophy (FECD) is a common inherited condition that in some patients leads to complete vision loss and the need for surgery and a corneal transplant. Our program, QR-504a, aims to prevent corneal dystrophy in patients with a mutation in the TCF4 gene thereby potentially stopping the disease.
Our QR-411 program is being developed to treat the vision loss associated with Usher syndrome caused by the c.7595-2144A>G (PE40) mutation in the USH2A gene. Usher syndrome is the leading cause of combined deafness and blindness. Our program aims to repair the PE40 mutation in the eye, such that it leads to a normal USH2A protein, thereby modifying the underlying eye disease.
Stargardt’s disease (fundus flavimaculatus) is a genetic eye disease that leads to progressive central vision loss amongst other symptoms. QR-1011, aims to treat the vision loss in patients that have Stargardt’s due to the c.5461-10T>C mutation in ABCA4 gene. QR-1011 aims to repair the mutation in the eye, leading to normal ABCA4 protein which will potentially stop and perhaps reverse the progression of the disease.
ProQR spun out Amylon Therapeutics in September 2017 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.
ProQR spun out its DEB activities into Wings Therapeutics that was formed by EB Research Partner in 2019. Wings is a privately-held company focused on the development of therapies for DEB. ProQR retains a minority ownership stake in Wings.