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 and have limited treatment options. Amongst others we work on the development of new medicines for several forms of inherited blindness such as Leber’s congenital amaurosis, Usher syndrome and autosomal dominant retinitis pigmentosa.
How do RNA therapies work?
Leber’s 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.
Read more about sepofarsen for Leber's congenital amaurosis 10 and the clinical trial
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
Retinitis pigmentosa (adRP)
Autosomal dominant retinitis pigmentosa (adRP) is a rare genetic eye disease that leads to poor vision and blindness for which there is currently no approved treatment. We are developing a novel drug, QR-1123, for patients with adRP due to the P23H mutation in the rhodopsin (RHO) gene.Read more about QR-1123 for autosomal dominant retinitis pigmentosa
Axiomer® 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 Axiomer® 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.
Read more about Axiomer
- 20 November, 2019, New York, NY, USA - Stifel Healthcare Conference
- 3 December, 2019, Boston, MA, USA - Evercore ISI HealthCONx Conference
- 3-7 May, 2020, Baltimore, MD, USA - Annual meeting Association for Research in Vision and Ophthalmology (ARVO)