Multi-characteristic Opsin Gene Therapy Gets Second Fast Track for Retinal Degenerative Diseases

The FDA has granted Fast Track designation to Nanoscope Therapeutics’ multi-characteristic opsin (MCO) optogenetic gene therapy MCO-010 for the potential treatment of Stargardt disease.¹

"We are pleased to receive the FDA's decision to grant Fast Track Designation for the second indication of our vision restoring therapy, and we look forward to sharing 6-month data from the Phase 2 STARLIGHT clinical trial of MCO-010 therapy in Stargardt patients this quarter," Sulagna Bhattacharya, chief executive officer, Nanoscope, said in a statement.¹ "Receiving Fast Track Designation reinforces the urgency of our mission to find an effective solution for Stargardt patients and is an acknowledgement of MCO-010's potential to address this unmet medical need."

Stargardt is the second indication that the gene-agnostic therapy MCO-010 is being evaluated for after retinitis pigmentosa (RP), which MCO-010 received fast track designation for treating in October 2022.² In RP, the phase 2b/3 RESTORE trial (NCT04945772) is fully enrolled with topline data expected in the first quarter of 2023 as of February 2022. In Stargardt, the phase 2 STARLIGHT trial (NCT05417126) is fully enrolled as of September 2022, with key 6-month data also expected in the first quarter of 2023. The trial enrolled 6 patients with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease to receive a single intravitreal dose of 1.2E11gc/eye of MCO-010.

READ MORE: Gene Therapy Improves Visual Acuity in MT-ND4 LHON Compared to Natural History

The therapy is also being investigated in advanced age-related macular degeneration with geographic atrophy, for which Nanoscope is planning to submit an investigational new drug application in the first half of 2023 to be able to initiate a phase 1/2a clinical trial in the second half of 2023. Other indications the company plans to target include Usher syndrome and cone rod dystrophy.

MCO-010 targets retinal degenerative diseases via gene delivery encoding for the ambient light-sensitive MCO protein into retinal cells. The therapy is delivered via intraocular injection and is designed to restore the ability of these retinal cells to detect light.

“We found that there is a need to develop a broadband opsin where patients in different lighting conditions can see this outside field,” Samarendra Mohanty, PhD, cofounder and president, Nanoscope Therapeutics, told CGTLive in a previous interview about MCO-010. “So, we developed MCO, 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.”

REFERENCES

1. Nanoscope Therapeutics receives fast track designation by the FDA for MCO-010 for the treatment of Stargardt disease. News release. Nanoscope Therapeutics. January 30, 2023. https://finance.yahoo.com/news/nanoscope-therapeutics-receives-fast-track-120000031.html
2. Nanoscope Therapeutics receives fast track designation by the FDA for MCO-010 for the treatment of retinitis pigmentosa. News release. Nanoscope Therapeutics Inc. October 10, 2022.https://nanostherapeutics.com/2022/10/10/nanoscope-therapeutics-receives-fast-track-designation-by-the-fda-for-mco-010-for-the-treatment-of-retinitis-pigmentosa/