Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today 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.
“This trial represents the second indication for our vision-restoring therapy for people blinded by rare inherited retinal degenerative diseases, regardless of gene mutations,” said Sulagna Bhattacharya, CEO of Nanoscope. “Our RESTORE Phase 2b trial for Retinitis Pigmentosa (PR) patients has completed enrollment and results are expected in Q1 2023. There are no approved treatments for either of these debilitating conditions.”
The Phase 2 STARLIGHT open-label trial (NCT05417126) has active clinical sites in Texas and Florida and will enroll approximately six patients to receive the same MCO-010 dose of 1.2E11gc/eye as used in the Phase 2b Retinitis Pigmentosa study. Six-month safety and efficacy data are expected the first quarter of 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,” said Aaron Osborne, Nanoscope’s CMO and CDO.
“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.” said Stephen Tsang MD PhD, Laszlo Z. Bito Professor of Ophthalmology, Pathology and Cell Biology at Columbia University Irving Medical Center and an advisor to Nanoscope Therapeutics.
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 color environments. The therapy involves a single intra-vitreal injection in a medical office setting.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics, a member of TechFW, is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, and for which no cure exists. The company’s lead asset, MCO-010, is presently in Phase 2b multi-center, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected the first quarter of 2023. The company has also initiated a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.