Nanoscope’s investigational RP treatment MOGENRY advances toward FDA approval

The U.S. Food and Drug Administration accepted Dallas-based Nanoscope Therapeutics' biologics license application for MOGENRY, an emerging treatment for adults with retinitis pigmentosa and severe vision loss. The investigational treatment does not require a genetic diagnosis or specific gene mutation, giving MOGENRY the potential to become the first approved gene-agnostic treatment to improve vision in people with severe vision loss from RP.

RP is a group of inherited diseases that cause the retina's light-sensing cells (photoreceptors) to degenerate, leading to vision loss. RP affects more than 100,000 people in the United States and is one of the leading causes of blindness among working-age adults.

“For patients who have been told for decades that nothing can be done once the photoreceptors are gone, the [biologics license application] acceptance marks a significant milestone,” says Samarendra Mohanty, Ph.D., President and Chief Scientific Officer at Nanoscope Therapeutics. “A gene-agnostic therapy for RP has been carried from a molecule on a bench, through randomized controlled trials, to a submission that the FDA has judged complete enough to evaluate.”

How MOGENRY works

MOGENRY, previously known as MCO-010, belongs to a class of emerging treatments called optogenetic gene therapies. In patients who have lost all or most of their light-sensitive photoreceptors – which is common in patients with RP – optogenetic therapies work to grant the remaining non-photoreceptor cells the ability to sense and respond to light.

The company says that MOGENRY does this by using a harmless virus, called an AAV, to deliver a gene coding for light-sensitive proteins (opsins) into bipolar cells in the retina. The opsins enable bipolar cells to sense light, something they could not otherwise do.

RP has been linked to over 1,000 mutations across more than 100 genes. Because MOGENRY does not target a specific gene, it could potentially be used to treat patients with a variety of genetic mutations causing RP, as well as those who have not had genetic testing done or whose genetic test results could not pinpoint the underlying cause.

“Our approach is indifferent to which gene caused the degeneration, and it is intended for people whose photoreceptors are already lost, precisely the patients that gene replacement cannot help,” said Dr. Mohanty.

According to Nanoscope, the emerging treatment could be more accessible to patients since it can be delivered as a one-time office injection rather than via surgery in specialized centers equipped for retinal operations.

Trial participants experienced gains in visual acuity that lasted up to three years

The application to FDA seeking permission to market MOGENRY builds on clinical trial results Nanoscope previously reported. The Phase 2 RESTORE clinical trial enrolled 27 patients with advanced RP and reported patients treated with both high and low doses improved in best corrected visual acuity at 52 and 76 weeks after treatment.

A long-term follow-up study, REMAIN, found that improvements persisted three years after treatment. Across studies, Nanoscope reports that MOGENRY was well tolerated, with no serious side effects tied to the treatment.

The road to approval

Nanoscope has requested that FDA prioritize review of its biologics licensing application for MOGENRY and expects a decision by the first half of 2027, according to Dr. Mohanty. Nanoscope started its rolling biologics license application  in 2025, when the FDA invited the company to submit their data early and continually update it to expedite review. MOGENRY also carries Fast Track and Orphan Drug designations, which help accelerate the FDA review process.

Nanoscope is also conducting clinical trials for MOGENRY in other inherited retinal conditions: it plans to start a Phase 3 registrational trial for Stargardt disease and a Phase 2 trial for geographic atrophy in 2026.

The Foundation Fighting Blindness has been funding optogenetic treatment strategies for retinal disease since the earliest stages of the technology’s development. To date, the Foundation has funded 15 optogenetic research projects totaling more than $4 million.

Learn more about retinitis pigmentosa, take a look at the latest in RP research advances, and explore the Foundation's clinical trial pipeline for other treatments for RP moving closer to patients.