OSLI Retina

January 2020

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64 Ophthalmic Surgery, Lasers & Imaging Retina | Healio.com/OSLIRetina News and Notes News and Notes ProQR's Retinitis Pigmentosa Treatment Gets Orphan Drug Designation QR-1123, a first-in-class investigational antisense oligonucleotide for the treat- ment of vision loss associated with autosomal dominant retinitis pigmentosa, re- ceived an orphan drug designation from the U.S. Food and Drug Administration (FDA), ProQR (Leiden, Netherlands) announced in a press release. "We are pleased to have orphan drug designation for our QR-1123 program targeting autosomal dominant retinitis pigmentosa, or adRP," said Daniel de Boer, CEO of ProQR. "It highlights the unmet need for patients with this pro- gressive disease-causing blindness. Our goal is to develop and actively advance a pipeline of programs that can treat inherited retinal diseases like adRP in a targeted manner." QR-1123, which is designed to be administered through intravitreal injec- tions, aims to inhibit the toxic version of the rhodopsin protein by binding the P23H mutation in the RHO gene. Discovered and developed by Ionis Pharmaceuticals, it was licensed by ProQR in 2018 and has since received IND clearance and a fast track designation from the FDA. Originally published on www.healio.com on November 21, 2019. Ophthalmic Surgery, Lasers & Imaging Retina® (ISSN-2325-8160, Canadian BN-12978 0466 RT ) is published 12 times per year by SLACK Incorporated®, and is an official publication of ARVO/ISIE. Publication Office: SLACK Incorporated®, 6900 Grove Road, Thorofare, NJ 08086-9447. Telephone (856) 848-1000. Printed in the USA. Periodicals Class Postage Paid at Deptford, NJ 08096 and at additional mailing offices. Contents copyrighted by SLACK Incorporated®, 2020. All rights reserved. No part of this publication may be reproduced with- out prior written consent of the publisher. 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Financial disclosures for the Editor and members of the Editorial Board are on file with the editorial office. Indexed in: Medline/PubMed, ProQuest, EMCare, EMBASE, Science Citation Index, Current Contents/Clinical Medicine, BioEn- gineering Abstracts, CAB Abstracts, and SCOPUS. Follow OSLIJournal OSLI Retina SLACK Incorporated 6900 Grove Road Thorofare, NJ 08086-9447 Phone: 856-994-9900 Email: osli@healio.com Healio.com/OSLIRetina Chief Content Officer Joan-Marie Stiglich, ELS EDITORIAL Senior Editorial Director David W. Mullin Executive Editor Cara Dickinson SALES Chief Commercial Officer Matthew J. 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