First patient dosed in Phase 2 optogenetic gene therapy clinical trial for Stargardt disease

Article

According to the Nanoscope Therapeutics, 6-month safety and efficacy data are expected in Q1 2023. MCO-010 gene therapy reprograms healthy retinal cells to make them photosensitive.

First patient dosed in Phase 2 optogenetic gene therapy clinical trial for Stargardt disease

Nanoscope Therapeutics Inc. announced that the first patient has been dosed in a Phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy for Stargardt disease.

Sulagna Bhattacharya, CEO of Nanoscope, noted in a news release that the trial represents the second indication for the company’s vision-restoring therapy for people blinded by rare inherited retinal degenerative diseases regardless of gene mutations.

“Our RESTORE Phase 2b trial for retinitis pigmentosa patients has completed enrolment and results are expected in Q1 2023. There are no approved treatments for these debilitating conditions,” Bhattacharya said in the release.

According to the company, the Phase 2 STARLIGHT open-label trial (NCT05417126) currently has active clinical sites in Texas and Florida and will enrol approximately six patients to receive the same MCO-010 dose of 1.2E11gc/eye as used in the Phase 2b retinitis pigmentosa (RP) study. Six-month safety and efficacy data are expected in Q1 2023. Stargardt disease is a progressive, inherited condition affecting children and adults. The condition results in damage to the macula, the central part of the retina responsible for central and detailed vision. Stargardt disease can be caused by several different gene mutations and is the second most common inherited retinal condition after RP.

"Existing experimental treatments for Stargardt primarily aim to slow progression of vision loss. For people who already have advanced vision loss, an optogenetics based approach, such as MCO-010, may have much greater potential to restore lost vision," Aaron Osborne, CMO and CDO at Nanoscope added in the release.

Dr Stephen Tsang, PhD, Laszlo Z. Bito Professor of Ophthalmology, Pathology and Cell Biology at Columbia University Irving Medical Centre and advisor to Nanoscope Therapeutics, pointed out that MCO-010 offers new hope for individuals with Stargardt macular degeneration.

“Stargardt patients with severe degeneration typically lack sufficient light sensing photoreceptor neurons needed to qualify for experimental ABCA4gene specific therapies,” Dr Tsang said in the news release. “Optogenetic therapy may be able to treat a wider group of patients with Juvenile macular degeneration regardless of the status of their light sensing photoreceptor neurons.”

Nanoscope's MCO-010 gene therapy reprograms healthy retinal cells to make them photosensitive. It uses proprietary vector and promoter technology to deliver the MCO genes into retinal bipolar cells enabling vision in different colour environments. The therapy involves a single intravitreal injection in a medical office setting.

Newsletter

Join ophthalmologists across Europe—sign up for exclusive updates and innovations in surgical techniques and clinical care.

Recent Videos
Christine Curcio, PhD, of the University of Alabama at Birmingham Heersink School of Medicine, shares histology update and revised nomenclature for OCT with Sheryl Stevenson of the Eye Care Network and Ophthalmology Times
SriniVas R. Sadda, MD, FARVO, shares key points from his retina presentation at the International SPECTRALIS Symposium
Robert Sergott, MD, describes fluorescence lifetime imaging ophthalmoscopy (FLIO) and the International SPECTRALIS Symposium – And Beyond (ISS) in Heidelberg, Germany.
Rayaz Malik, MBChB, PhD, a professor of medicine at Weill Cornell Medicine - Qatar, spoke with Ophthalmology Times Europe about his presentation. It's titled "An eye on neurodegenerative diseases: Challenging the dogma" at the International SPECTRALIS Sympsoium. In conversation with Hattie Hayes, Ophthalmology Times Europe
Anat Loewenstein, MD, describes her presentation on remote imaging for age-related macular degeneration and geographic atrophy at the International SPECTRALIS Symposium, in conversation with Hattie Hayes of Ophthalmology Times Europe
Tyson Brunstetter, OD, PhD, a US Navy Aerospace Optometrist at the NASA Johnson Space Center in Houston, Texas, shares key takeaways from his keynote at the International SPECTRALIS Symposium (ISS)
Rayaz Malik, MBChB, PhD, shares his presentation, titled An eye on neurodegenerative diseases: Challenging the dogma, at this year's International SPECTRALIS Symposium
At the Retina World Congress, Siegfried Priglinger, MD, speaks about ensuring the best outcomes for preschool-aged patients
At the 2025 ASCRS meeting, Robert Ang, MD said small aperture IOLs can benefit all patients, especially those with complex corneas or who have undergone previous corneal refractive surgery
Viha Vig, MBChB graduate student at the University of Auckland, New Zealand, discusses her poster presentation on the relationship between mitochondiral disease, Alzheimer disease, and other types of dementia.
© 2025 MJH Life Sciences

All rights reserved.