Nanoscope Gene Therapy Continues Progress in Stargardt Disease


Data is expected in the first quarter of 2023 for both Stargardt disease and retinitis pigmentosa.

Nanoscope Therapeutics has dosed the first patient in the phase 2 STARLIGHT clinical trial (NCT05417126) of their gene therapy MCO-010 for the potential treatment of Stargardt disease.1

"This trial represents the second indication for our vision restoring therapy for people blinded by rare inherited retinal degenerative diseases regardless of gene mutations," Sulagna Bhattacharya, chief executive officer, Nanoscope, said in a statement.1 "Our RESTORE Phase 2b trial for Retinitis Pigmentosa patients has completed enrollment and results are expected in Q1 2023. There are no approved treatments for these debilitating conditions."

The open-label STARLIGHT trial plans to enroll around 6 patients with Stargardt disease to its clinical sites in Texas and Florida. These participants will be dosed with 1.2E11gc/eye of MC-010, which is the same dose that was investigated in the phase 2b RESTORE study (NCT04945772) of MC-010 in retinitis pigmentosa (RP). Nanoscope expects to report 6-month safety and efficacy data in the first quarter of 2023.

"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, chief medical officer and chief development officer, Nanoscope, added to the statement.1

WATCH NOW: Developing MCO-010 for Retinal Degenerative Diseases

MCO-010 is an optogenetic, ambient-light activatable gene therapy that uses multi-characteristic opsin (MCO). It is designed to re-sensitize and improve the ability of the retina to detect low light levels. It uses proprietary vector and promoter technology to deliver the MCO genes into retinal bipolar cells with an intravitreal injection administered in a medical office setting. MCO-010's investigational new drug application for Stargardt was approved in February 2022 and the therapy received orphan drug designation in the indication at the same time.2

MCO-010's lead indication is in RP, for which positive data from RESTORE were announced in late 2021 at the EURETINA 2021 virtual conference. These data demonstrated improvements in visual acuity and function with no serious adverse events in patients with RP with up to 52 weeks of follow-up. RESTORE has completed enrollment and data are expected in the first quarter of 2023. MSO-010 is also being evaluated in preclinical studies in cone-rod dystrophy, Leber congenital amaurosis, and Usher syndrome, and is in investigational new drug-enabling studies in geographic atrophy in dry age-related macular degeneration.

"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 ABCA4 gene specific therapies. 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 advisor Stephen Tsang, MD, PhD, Laszlo Z. Bito Professor of Ophthalmology, Pathology and Cell Biology, Columbia University Irving Medical Center, added to the statement.1

1. Nanoscope Therapeutics announces first patient dosed in STARLIGHT phase 2 clinical trial of MCO-010 optogenetic gene therapy for Stargardt disease. News release. Nanoscope Therapeutics. July 25, 2022.
2. Nanoscope Therapeutics Announces FDA Clearance of IND for MCO-010 Gene Therapy in Stargardt Macular Degeneration Patients. News release. Nanoscope Therapeutics. January 25, 2022.
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