Developing MCO-010 for Retinal Degenerative Diseases

Samarendra Mohanty, PhD, cofounder and president, Nanoscope Therapeutics, discussed the company’s technological platforms and RESTORE trial data.

“We found that there is a need to develop a broadband opsin where patients in different lighting conditions can see this outside field. So, we developed multicharacteristic opsin, which has 3 unique properties: it’s sensitive to a broadband of white light... it’s very fast for vision processing... and we made it sensitive to very low levels of light... so we don't require any external stimulus to stimulate the retina.”

Nanoscope Therapeutics is targeting a variety of retinal degenerative diseases with the help of gene therapies that use multi-characteristic opsin (MCO). The therapies are designed to improve vision under differing lighting conditions. 

The company’s lead product, MCO-010, is gene and disease-agnostic and is being evaluated in the phase 2b/3 RESTORE trial (NCT04945772) for retinitis pigmentosa (RP), a phase 1/2a trial in Stargardt disease, 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 (AMD).

Nanoscope presented data from the RESTORE trial at the EURETINA 2021 Virtual Conference, September 9 – 12, that demonstrated improvements in visual acuity and function with no serious adverse events in patients with RP with up to 52 weeks of follow-up.

GeneTherapyLive spoke with Samarendra Mohanty, PhD, cofounder and president, Nanoscope Therapeutics, to learn more about the company’s technologies, including the advantages of MCO and their nano-enhanced optical delivery system that will be used in the AMD program. He also discussed the positive data from the RESTORE trial.

REFERENCE
Nanoscope Therapeutics announces presentation of positive results from optogenetic gene therapy for retinal degenerative diseases at the EURETINA 2021 Virtual Conference. News release. September 8, 2021. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-announces-presentation-of-positive-results-from-optogenetic-gene-therapy-for-retinal-degenerative-diseases-at-the-euretina-2021-virtual-conference-301371763.html