{"id":11602,"date":"2018-12-04T22:38:39","date_gmt":"2018-12-04T21:38:39","guid":{"rendered":"https:\/\/www.hollandbio.nl\/?p=11602"},"modified":"2018-12-04T22:38:56","modified_gmt":"2018-12-04T21:38:56","slug":"proqr-announces-clearance-to-start-clinical-trial-of-qr-421a-in-usher-syndrome-type-2-patients","status":"publish","type":"post","link":"https:\/\/www.hollandbio.nl\/en\/nieuws\/proqr-announces-clearance-to-start-clinical-trial-of-qr-421a-in-usher-syndrome-type-2-patients\/","title":{"rendered":"ProQR Announces Clearance to Start Clinical Trial of QR-421a in Usher Syndrome Type 2 Patients"},"content":{"rendered":"<p>ProQR, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-421a. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of the vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the <em>USH2A<\/em> gene.<\/p>\n<p>Usher syndrome is the leading cause of combined deafness and blindness. Exon 13 mutations in the <em>USH2A<\/em> gene targeted by QR-421a cause vision loss in approximately 16,000 individuals in the Western world. ProQR plans to start enrolling patients in a Phase 1\/2 trial named STELLAR in the coming months with preliminary data expected in mid-2019.<\/p>\n<p>\u201cWe are pleased to be advancing QR-421a, our second therapy for an inherited retinal disease, into the clinic and continuing the development of our portfolio of transformative RNA medicines for severe genetic rare diseases,\u201d said Daniel A. de Boer, Chief Executive Officer of ProQR. \u201cQR-421a has shown promising activity in both the optic cup and zebra fish models and we are excited about the potential to make a meaningful impact for Usher syndrome patients.<\/p>\n<p>Source: <strong><span style=\"text-decoration: underline;\"><a href=\"https:\/\/globenewswire.com\/news-release\/2018\/12\/04\/1661524\/0\/en\/ProQR-Announces-Clearance-of-IND-to-Start-Clinical-Trial-of-QR-421a-in-Usher-Syndrome-Type-2-Patients.html\">ProQR<\/a><\/span><\/strong><\/p>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>ProQR, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-421a. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause [&hellip;]<\/p>\n","protected":false},"author":6,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[39],"tags":[],"class_list":["post-11602","post","type-post","status-publish","format-standard","hentry","category-gezondheid"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.0 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>ProQR Announces Clearance to Start Clinical Trial of QR-421a in Usher Syndrome Type 2 Patients - hollandbio<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.hollandbio.nl\/en\/nieuws\/proqr-announces-clearance-to-start-clinical-trial-of-qr-421a-in-usher-syndrome-type-2-patients\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"ProQR Announces Clearance to Start Clinical Trial of QR-421a in Usher Syndrome Type 2 Patients - hollandbio\" \/>\n<meta property=\"og:description\" content=\"ProQR, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-421a. 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