Source: Radboudumc 4 May 2016
The Radboudumc and ProQR Therapeutics from Leiden (the Netherlands) have signed a cooperation agreement. Through this cooperation they want to speed up the development of an RNA therapy for people suffering from Usher Syndrome.
Usher Syndrome is the most common form of hereditary deaf-blindness with people. The loss of hearing can partly be compensate by providing patients with hearing aids or cochlear implants, but there is no treatment for slowing down or stopping the loss of eyesight at this moment. Usher Syndrome is a rare disorder, but it has an enormous adverse impact on the quality of life of the patient.
Correct specific mutation
An important cause of Usher Syndrome is a specific mutation in the USH2A gene, which was described in 2012. Erwin van Wijk, researcher in the Radboudumc, developed a method for correcting the effect of this specific mutation with so-called single-stranded oligonucleotides.
In order to speed up the development of this method, the Radboudumc entered into a partnership and licence agreement with ProQR Therapeutics. ProQR Therapeutics is a Dutch biotechnology company, which focuses on helping patients by means of developing new RNA medicines for serious, rare disorders, such as cystic fibrosis and Leber’s congenital amaurosis Type 10 (LCA10). Thanks to this agreement, ProQR and the Radboudumc will now cooperate in the development of future therapies for people suffering from Usher Syndrome, based on oligonucleotides. ProQR supports the study and obtains an exclusive licence for the technology developed by the Radboudumc.