Courtesy of Nanoscope Therapeutics/TechFW
Sulagna Battacharya
Bedford-based Nanoscope Therapeutics Inc., a clinical-stage, biotechnology company, has received clearance from the Food and Drug Administration to begin a Phase 2 human trial of its Multi-Characteristic Opsin (MCO-010) gene therapy in Stargardt patients.
MCO-010 is designed to restore vision in Stargardt patients, a rare genetic eye disease that results when fatty material builds up on the macula — the small part of the retina needed for sharp, central vision.
“This is another important milestone for Nanoscope, giving us the opportunity of conducting a second clinical trial in the U.S. of our proprietary MCO gene therapy platform, designed to restore vision for patients suffering from retinal degenerative diseases,” says Sulagna Battacharya, CEO of Nanoscope, in a statement. “Currently, there are no approved treatments for these conditions.”
Nanoscope Therapeutics is a TechFW member, a 501(c)(3) nonprofit startup accelerator and business incubator since 1998.
MCO-010 has received orphan drug designations from the FDA for Retinitis pigmentosa (RP) and Stargardt.
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina. Nanoscope already is conducting a Phase 2b, multi-center, randomized, sham-controlled, and double-masked study of MCO-010 for RP patients.
The Stargardt Phase 2 clinical trial is expected to begin during the first half of 2022. Progress on Nanoscope’s Stargardt program trial has implications for MCO as a platform and its impact beyond rare diseases.
“Presently all existing trials attempt to slow down the progression of vision loss in patients with Stargardt disease. Optogenetic approach is to restore vision. This can be a groundbreaking attempt to evaluate optogenetic gene therapy to improve vision in Stargardt patients,” says Dr. David S. Boyer, an ophthalmologist and adjunct clinical professor of ophthalmology at the University of Southern California.
“I’m excited by the potential MCO-010 has to restore vision for many patients with sight loss caused by outer retinal dystrophies, including dry age-related macular degeneration.”
Stargardt is an inherited retinal disease involving dysfunction/degeneration of photoreceptors. Nanoscope’s MCO-010 gene therapy reprograms healthy retinal cells to make them photosensitive.
Vision loss usually starts in childhood, but some people with Stargardt disease don't start to lose their vision until they're adults.