Results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR 110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene

Author(s)

Stephen R. Russell¹, Arlene V. Drack¹, Artur V. Cideciyan², Samuel G. Jacobson², Bart P. Leroy^3,4, Wanda L. Pfeifer¹, Alina V. Dumitrescu¹, Alexandra V. Garafalo², Allen C. Ho⁵, Caroline Van Cauwenbergh³, Julie De Zaeytijd³, Aniz Girach⁶, Wil den Hollander⁶, Michael R. Schwartz⁶, David M. Rodman⁶

¹ Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, IA, USA; ² Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; ³ Department of Ophthalmology & Center for Medical Genetics, Ghent University Hospital & Ghent University, Ghent, Belgium; ⁴ Ophthalmic Genetics & Visual Electrophysiology, Division of Ophthalmology, The Children’s Hospital of Philadelphia, PA, USA; ⁵ Wills Eye Hospital, Thomas Jefferson University, Philadelphia, PA, USA; ⁶ ProQR Therapeutics, Leiden, the Netherlands.