ProQR Therapeutics N.V. announced that the Company can start the Phase 1/2 trial for QR-110, named PQ-110-001, in Leber’s congenital amaurosis Type 10 (LCA 10) patients. QR-110 is a novel investigational RNA therapeutic targeting LCA 10 due to the p.Cys998X mutation, a severe genetic rare disease that causes children to lose sight in the first years of life. LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available.
“We are pleased to initiate clinical trials for QR-110 for the treatment of patients with LCA 10. This first trial in this rare, genetic disease will allow us to study multiple doses over one year’s time and evaluate safety, tolerability, as well as demonstrate if QR-110 can improve vision or prevent loss of vision in patients affected by this devastating disease,“ said Noreen R. Henig, MD, Chief Medical Officer of ProQR. “Importantly, this trial is the first in ProQR’s ophthalmology pipeline, which also includes programs targeting Ushers syndrome and Fuchs endothelial corneal dystrophy (FECD).”
“We are very excited to participate in this trial that has a unique approach in targeting LCA 10,“ said Stephen R. Russell, Professor of Ophthalmology and Visual Sciences at the University of Iowa Hospitals & Clinics and Principle Investigator of Study PQ-110-001. “We were very encouraged given the promising pre-clinical data for this compound and its potential to make an impact to a disease that has no treatments.”
"Advancing QR-110 into a clinical trial in patients marks another important step in our quest to bring RNA medicines to patients in need,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “The QR-110 program is very important in the validation of our promising ophthalmology pipeline, which has programs targeting several inherited blindnesses. In our strategy to build ProQR into a sustainable company with a balanced risk profile, we’ve established a diversified portfolio with our lead program QR-010 for cystic fibrosis, QR-110 for LCA 10 and QR-313 for dystrophic epidermolysis bullosa. Moving QR-110 into clinical development contributes to the maturity of this diversification. We plan to have established clinical proof of concept in patients in all three of these development programs before the end of 2018.”
