QR-110 Study 001, a phase 1/2 study for Leberâs congenital amaurosis 10
QR-313 WINGS phase 1/2 study for recessive dystrophic epidermolysis bullosa
We are ProQR
We are ProQR, a company on a mission to create new medicines for patients in need. We focus on a group of diseases called rare diseases, with a very high need for new medicines. Out of the approximately 7,000 rare diseases, less than 400 have a treatment, and we intend to change that. To treat these diseases we use RNA therapies, a technology that allows us to address inherited diseases that are caused by a genetic defect.
Our focus is on diseases that are very severe or life threatening, and have limited treatment options. Amongst others we work on the development of new medicines for several forms of inherited blindness such as Leberâs congenital amaurosis and Usher syndrome, a lung disease called cystic fibrosis, and a debilitating skin disease named dystrophic epidermolysis bullosa.
- 29 January, 2019, New York, NY, USA - ProQR Research and Development Day
- 20-21 February, 2019, London, UK - 10th Annual RNA Therapeutics Conference
- 28 February, 2019 - Rare Disease Day 2019
- 26-27 March, 2019, Cambridge, MA, USA - Oligonucleotide and precision therapeutics congress
- 26 April, 2019, Vancouver, BC, Canada - 6th Annual Retinal Cell & Gene Therapy Innovation Summit
- 28 April - 2 May, 2019, Vancouver, BC, USA - 312-924-1718
- 8-11 May, 2019, Chicago, IL, USA - 512-452-7794
Leberâs Congenital Amaurosis
Leber's congenital amaurosis 10 (LCA10) is a genetic eye disorder and the leading genetic cause of childhood blindness. We are developing a potentially life changing therapy for patients that suffer from LCA10 due to the p.Cys998X mutation in the CEP290 gene. A first-in-human clinical trial of sepofarsen is ongoing.
Usher syndrome type 2
Usher syndrome is the leading cause of combined deafness and blindness. We are developing two drug candidates for different mutations causing Usher syndrome type 2 including lead program QR-421a for approximately 16,000 patients with a mutation in exon 13 of the USH2A gene and QR-411 for approximately 1,000 patients with a the c.7595-2144A>G mutation in the USH2A gene. Read more about QR-421a and QR-411 for Usher syndrome type 2
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. A first-in-human clinical trial of QR-313, called WINGS, is ongoing.
Read more about QR-313 for dystrophic epidermolysis bullosa and the WINGS study
Cystic fibrosis is a progressive disease affecting the lung and other vital organs, leading to a limited life expectancy. We are developing drugs to help patients that suffer from different forms of cystic fibrosis. Our first candidate medicine, called eluforsen (QR-010), for the F508del mutations showed very encouraging results in the first two clinical trials. For other forms of CF we have initiated discovery projects to find new medicine candidates.
Read more about eluforsen for cystic fibrosis and clinical trials
AxiomerÂ® RNA editing technology
At the ProQR labs a novel technology was invented that has the potential to treat a large number of diseases that are currently untreatable. The AxiomerÂ® platform technology is a novel way to correct RNA in the cells in patients and target mutations that cannot be treated with other technologies. Our scientists are working hard to make the promise of this technology for patients come true.
Spin out: Amylon Therapeutics
In September 2017 ProQR created a new company that is focused on the development of drugs for genetic brain diseases. Amylon Therapeutics is building a team of experts in brain diseases and has raised a financing round with private and institutional shareholders. ProQR retains a majority ownership stake in Amylon.
Read more about the spin out in the press release
Visit the Amylon website