June 2021
Volume 62, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2021
Treatment outcomes in 11 patients with RPE65-retinopathy receiving voritegene neparvovec-rzyl
Author Affiliations & Notes
  • Cristy Ann Ku
    Oregon Health & Science University Casey Eye Institute, Portland, Oregon, United States
  • Mariana Matioli da Palma
    Oregon Health & Science University Casey Eye Institute, Portland, Oregon, United States
  • Austin Igelman
    Oregon Health & Science University Casey Eye Institute, Portland, Oregon, United States
  • Andreas Lauer
    Oregon Health & Science University Casey Eye Institute, Portland, Oregon, United States
  • Steven Bailey
    Oregon Health & Science University Casey Eye Institute, Portland, Oregon, United States
  • Jacque L Duncan
    University of California San Francisco, San Francisco, California, United States
  • Paul Yang
    Oregon Health & Science University Casey Eye Institute, Portland, Oregon, United States
  • Mark E Pennesi
    Oregon Health & Science University Casey Eye Institute, Portland, Oregon, United States
  • Footnotes
    Commercial Relationships   Cristy Ku, None; Mariana da Palma, None; Austin Igelman, None; Andreas Lauer, None; Steven Bailey, None; Jacque Duncan, Acucela (F), AGTC (C), Allergan (F), Editas Medicine (C), Neurotech USA (F), NightstaRx Limited (F), ProQRTherapeutics (C), Second Sight Medical Products (F), SparingVision (C), Spark Therapeutics (C); Paul Yang, Adverum (C), AGTC (C), Nanoscope Therapeutics (C), NIH (F); Mark Pennesi, Adverum (C), AGTC (F), AGTC (C), Astellas Pharmaceuticals (C), Atsena (C), Biogen (F), Biogen (C), Blue Rock (C), DTx (C), Editas (F), Endogena (C), Foundation Fighting Blindness (F), Gensight (C), Horama (C), IVERIC (C), Nacuity Pharmaceuticals (C), Nayan (C), Novartis (C), ProQR Therapeutics (C), ProQR Therapeutics (F), RegenexBio (C), Roche (C), Sanofi (F), Sanofi (C), Viewpoint Therapeutics (C)
  • Footnotes
    Support  NIH P30 EY010572. Unrestricted departmental funding from Research to Prevent Blindness (RPB).
Investigative Ophthalmology & Visual Science June 2021, Vol.62, 3320. doi:
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      Cristy Ann Ku, Mariana Matioli da Palma, Austin Igelman, Andreas Lauer, Steven Bailey, Jacque L Duncan, Paul Yang, Mark E Pennesi; Treatment outcomes in 11 patients with RPE65-retinopathy receiving voritegene neparvovec-rzyl. Invest. Ophthalmol. Vis. Sci. 2021;62(8):3320.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : To report on the outcomes of patients with RPE65-associated retinopathy treated with FDA-approved voretigene gene replacement therapy at a single treatment site.

Methods : This is a retrospective study on patients with RPE65-associated retinopathy treated with voretigene neparvovec-rzyl. Records, imaging, and diagnostics were reviewed including fundus autofluorescence (FAF), optical coherence tomography (OCT), full-field stimulus threshold (FST), kinetic visual fields, dark-adapted two color visual fields (2cDAP), full-field ERG (ffERG). FST was performed and analyzed on Espion ColorDome LED full-field stimulator system and Diagnosys software, and dark-adapted two color perimetry on the Octopus 900 Haag-Streit perimeter.

Results : A subretinal injection of voretigene was administered into 19 eyes from 11 patients (pt) with an average age of 20.82 YO (range 4-38 YO). FST was performed for 11 eyes (from 6 pts) with improvements seen in 10 eyes. There was an average gain in FST of 13.3 dB (range 4.9-17.6, SD 8.92 dB) with the short-wavelength stimulus (blue), and 8.1 dB (range 2.5-15.0, SD 5.43 dB) with the long-wavelength stimulus (red). When 1 year or longer follow-up data was available (10 eyes from 5 pts), gains in sensitivity were maintained in all treated eyes. 2cDAP field was performed for 10 eyes (from 6 pts) with all eyes showing an improvement in mean sensitivity to the blue stimulus, with an average improvement of 10.91 dB (range 2.0-22.2, SD 4.98 dB). 9 out of 10 eyes (from 6 pts) showed an improvement to the red stimulus, with average improvement of 4.73 dB (range 0.8-10.0, SD 2.37 dB). Gains in 2cDAP was sustained in all patients with follow-up at 1 year. ffERG was performed in 14 eyes (from 8 pts) with a greater than 50% improvement in 5 eyes (from 3 pts). Improved OCT findings were observed in 7 eyes (from 4 pts). Patients also reported moderate to significant subjective improvement in nyctalopia (9 pts), overall vision (6 pts), color vision (3 pts), and nystagmus (2 pts).

Conclusions : A majority of patients with RPE65-associated retinopathy treated with voretigene showed improvements in FST and dark-adapted perimetry. Dark-adapted perimetry may be a sensitive outcome that provides topographical information in relation to the treatment area. Several patients reported significant subjective improvement in color vision, a potential outcome measure to systematically evaluate in the future.

This is a 2021 ARVO Annual Meeting abstract.

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