LEIDEN,\u00a0Netherlands\u00a0and\u00a0CAMBRIDGE, Mass., Aug. 12, 2019 (GLOBE NEWSWIRE) — ProQR Therapeutics N.V.\u00a0(Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, announced that the\u00a0U.S. Food and Drug Administration\u00a0(FDA) has cleared the Investigational New Drug (IND) application for QR-1123. ProQR plans to start enrolling patients in a Phase 1\/2 trial for QR-1123 in 2019.<\/p>\n\n\n\n
QR-1123 is a\nfirst-in-class investigational oligonucleotide designed to address the\nunderlying cause of the vision loss associated with autosomal dominant\nretinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO)\ngene.<\/p>\n\n\n\n
P23H is the\nmost prevalent mutation associated with adRP in the U.S. This disease causes\nprogressive vision loss in approximately 2,500 patients in the United States,\nleading to blindness in mid-adulthood. There are no approved therapies for adRP\nand QR-1123 is the first investigational medicine to be developed for patients\nthat suffer from this disease.<\/p>\n\n\n\n
\u201cWe are pleased to have an open IND for QR-1123, based on which we will be advancing our next inherited retinal disease program into the clinic this year,\u201d said\u00a0Daniel A. de Boer, Chief Executive Officer of ProQR. \u201cThis represents our fifth IND in less than five years and our third clinical program for severe genetic eye diseases. With a strong in vitro and in vivo proof-of-concept, we are excited about the potential of this medicine to make a positive impact on the lives of patients with adRP.\u201d<\/p>\n\n\n\n