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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

ePoster

Published on November 13, 2020 at AAO

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Author(s)

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.

Description

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.

AAO 2020 poster - Sepofarsen clinical trial results for Leber congenital amaurosis 10

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Tags

  • AAO
  • Leber Congenital Amaurosis
  • Sepofarsen

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Related publications

  • Full field stimulus testing (FST) to assess sepofarsen patient response in Leber congenital amaurosis type 10

    AAO 2020 poster by Allen Ho et al. Recognized with best poster award. Data show that FST seems a more sensitive method in patients with severely impaired vision.

    Date: November 13, 2020

    Author(s): Ho A, Cideciyan AV, et al.

    ePoster
  • 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

    ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.

    Date: June 17, 2020

    Author(s): Russell SR, Drack AV, et al.

    Presentation
  • ARVO (2021) Russell SR et al., Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension trial in Leber Congenital Amaurosis type 10 (LCA10)

    ARVO 2021 presentation by Stephen R. Russell from Iowa University on the Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension trial in CEP290 mediated Leber Congenital Amaurosis type 10.

    Date: May 06, 2021

    Presentation
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