Nanoscope’s AAV Gene Therapy Maintains Clinically Meaningful Vision Improvements in Retinitis Pigmentosa

DALLAS, TX — October 22, 2025 — Nanoscope Therapeutics Inc. announced positive three-year follow-up data from the REMAIN study, the long-term extension of the Phase 2b/3 RESTORE trial evaluating MCO-010, an investigational optogenetic gene therapy for patients with retinitis pigmentosa (RP). The results demonstrated durable and clinically meaningful improvements in vision following a single intravitreal injection.

Sustained Efficacy and Favorable Safety Profile

The 152-week REMAIN study confirmed the long-term benefit and favorable safety profile of MCO-010, which utilizes an AAV vector to activate light-sensitive cells.

• Durable Efficacy: Patients maintained an average Best Corrected Visual Acuity (BCVA) gain of approximately 0.3 LogMAR (equivalent to three lines or 15 letters on a standard vision chart) through Week 152. The vision gains were fivefold greater than those observed in the sham treatment group.
• Safety and Tolerability: The treatment was well tolerated over three years, with no serious ocular adverse events reported. Only one mild case of inflammation required topical steroids, and 14 of 15 treated patients required no ongoing inflammation management at the final follow-up.

“The sustained visual gains demonstrated over three years are truly remarkable for this population,” said Allen C. Ho, MD, Director of Retina Research at Wills Eye Hospital and Chief Medical Advisor for Nanoscope. “The ability to not just slow loss but restore visual function for several years represents a significant therapeutic advance.”

How MCO-010 Works

MCO-010 is a groundbreaking, single-dose, in-office gene therapy designed to restore vision regardless of the patient’s underlying genetic cause.

The therapy uses an AAV vector to deliver a new Multi-Characteristic Opsin (MCO) gene. This gene genetically modifies the remaining bipolar retinal cells (which are typically spared in RP) to produce a light-sensitive protein. Essentially, MCO-010 transforms these surviving cells into new photoreceptors, allowing them to utilize the eye’s remaining visual pathways and compensate for the death of the original light-sensing cells.

Advancing Toward Commercialization

The company is currently pursuing a rolling Biologics License Application (BLA) with the FDA for MCO-010 in RP and is preparing for a potential commercial launch of what could become the first approved optogenetic therapy for retinal disease. MCO-010 has previously been granted Fast Track and Orphan Drug designation by the FDA, and five Orphan designations by the European Medicines Agency.