June 2023
Volume 64, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2023
MCO-010 intravitreal optogenetic therapy in Stargardt disease. 6-month outcomes from the Phase 2 STARLIGHT trial
Author Affiliations & Notes
  • Victor H Gonzalez
    Valley Retina Institute, Texas, United States
  • Byron L Lam
    University of Miami Mary and Edward Norton Library of Ophthalmology, Miami, Florida, United States
  • Vitaliy Zak
    Valley Retina Institute, Texas, United States
  • Samar Mohanty
    Nanoscope Therapeutics, Texas, United States
  • subrata bataybal
    Nanoscope Therapeutics, Texas, United States
  • Jean Chang
    Nanoscope Therapeutics, Texas, United States
  • Ananta Ayyagari
    Nanoscope Therapeutics, Texas, United States
  • Sai H Chavala
    Texas Christian University, Fort Worth, Texas, United States
  • Jody Piltz-Seymour
    Wills Eye Hospital, Philadelphia, Pennsylvania, United States
  • John Koester
    JMK Statistics, LLC, Goodyear, Arizona, United States
  • Aaron Osborne
    Nanoscope Therapeutics, Texas, United States
  • Footnotes
    Commercial Relationships   Victor Gonzalez Genentech, Code C (Consultant/Contractor), Nanoscope, Code C (Consultant/Contractor), Regeneron, Code C (Consultant/Contractor), American genomics, Code C (Consultant/Contractor); Byron Lam Nanoscope Therapeutics, Code C (Consultant/Contractor); Vitaliy Zak Nanoscope Therapeutics, Code C (Consultant/Contractor); Samar Mohanty Nanoscope Therapeutics, Code E (Employment); subrata bataybal Nanoscope Therapeutics, Code E (Employment); Jean Chang Nanoscope Therapeutics, Code E (Employment); Ananta Ayyagari Nanoscope Therapeutics, Code E (Employment); Sai Chavala Nanoscope Therapeutics, Code C (Consultant/Contractor); Jody Piltz-Seymour Nanoscope Therapeutics, Code C (Consultant/Contractor); John Koester Nanoscope Therapeutics, Code C (Consultant/Contractor); Aaron Osborne Nanoscope Therapeutics, Code E (Employment)
  • Footnotes
    Support  NONE
Investigative Ophthalmology & Visual Science June 2023, Vol.64, 3546. doi:
  • Views
  • Share
  • Tools
    • Alerts
      ×
      This feature is available to authenticated users only.
      Sign In or Create an Account ×
    • Get Citation

      Victor H Gonzalez, Byron L Lam, Vitaliy Zak, Samar Mohanty, subrata bataybal, Jean Chang, Ananta Ayyagari, Sai H Chavala, Jody Piltz-Seymour, John Koester, Aaron Osborne; MCO-010 intravitreal optogenetic therapy in Stargardt disease. 6-month outcomes from the Phase 2 STARLIGHT trial. Invest. Ophthalmol. Vis. Sci. 2023;64(8):3546.

      Download citation file:


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

      ×
  • Supplements
Abstract

Purpose : MCO-010 is an intravitreal, Adeno-Associated Virus (AAV2)-delivered optogenetic therapy designed to treat severe vision loss from outer retinal degenerative disease. Stargardt disease is a rare, inherited condition with significant unmet need, characterized by progressive photoreceptor degradation and loss of central vision eventually leading to profound vision loss. STARLIGHT (NCT05417126) is a Phase 2 open label multi-center clinical study to evaluate the safety and effects of a single dose level of MCO-010 in subjects with Stargardt disease.

Methods : Participants in the STARLIGHT trial were required to have a documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM1. All subjects received a single intravitreal injection of 1.2E11 gc/eye MCO-010 in the study eye at baseline. Ocular and systemic safety, as well as multiple vision function tests, including Best-Corrected Visual Acuity (BCVA) using ETDRS charts at 50 cm, Multi-Luminance Y-Mobility Test (MLYMT) and Low-Vision Multi-Parameter Test (LVMPT) were key outcome measures.

Results : 6 participants, 4 males and 2 females, with severe vision loss due to Stargardt disease were enrolled between June and September 2022. Mean age was 49 years (range 32-71). At baseline mean study eye BCVA letter score was 23 (range 13-34) or approximately 1.8 LogMAR. Safety and efficacy data through 6 months follow up will be presented for the first time.

Conclusions : STARLIGHT is the first clinical study of optogenetic therapy for severe vision loss due to Stargardt disease. The 6-month outcomes from STARLIGHT will help inform the future clinical development of MCO-010 optogenetic therapy for vision restoration in Stargardt disease and other retinal degenerative conditions.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.

 

MCO-010 Vector Design

MCO-010 Vector Design

 

Starlight Phase 2 Study Design Schema

Starlight Phase 2 Study Design Schema

×
×

This PDF is available to Subscribers Only

Sign in or purchase a subscription to access this content. ×

You must be signed into an individual account to use this feature.

×