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
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Stephen R. Russell1, Arlene V. Drack1, Artur V. Cideciyan2, Samuel G. Jacobson2, Bart P. Leroy3,4, 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.
In a phase 1b/2 trial, 11 Leber congenital amaurosis 10 (LCA10) patients received intravitreal sepofarsen. The goal was to evaluate safety and change in various ophthalmic endpoints from baseline to month 12. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
Recognized with best poster award at AAO 2020.
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