RNA therapy sepofarsen for Leber congenital amaurosis 10 (CEP290) - ARVO 2020 presentation and Q&A

In this video recorded for the ARVO 2020 Annual Meeting, Dr. Stephen R. Russell presents the 12-month results of a phase 1b/2 trial of sepofarsen, an intravitreal RNA therapy for Leber congenital amaurosis 10 (LCA10). Following the results presentation, Stephen R. Russell and Ben Shaberman discuss the results, the potential and future of RNA therapies for inherited retinal diseases (IRDs). The Q&A starts at 09:29. Speakers: Stephen R. Russell, Professor and Director of Vitreoretinal Diseases and Surgery Service, University of Iowa. Ben Shaberman, Senior Director, Science Outreach & Community Engagement, Foundation Fighting Blindness. The video-recorded results presentation is also available on ARVO Learn, ARVO’s online learning platform (https://www.arvo.org/education/online.... Foundation Fighting Blindness mission is to drive the research that will provide preventions, treatments, and cures for people affected by retinal degenerative diseases. The Foundation Fighting Blindness, in partnership with Blueprint Genetics and InformedDNA®, launched an open access, no-cost genetic testing program called the My Retina Tracker Program. This program is for individuals living in the United States, with a clinical diagnosis of an inherited retinal disease (IRD). For more information please visit the My Retina Tracker Program webpage at https://blueprintgenetics.com/my-reti.... ProQR is developing sepofarsen, for patients with LCA10 due to mutation in the CEP290 gene called p.Cys998X mutation. Following the phase 1b/2 results, a phase 2/3 trial called “Illuminate” as began in April 2019. A dedicated website to provide patients, their families and care givers with information about the Illuminate clinical trial for Leber's congenital amaurosis 10 is now available. Visit the Illuminate LCAstudy.com website at https://www.lcastudy.com/.