Nanoscope Therapeutics has initiated a rolling submission for its Biologics License Application (BLA) to the FDA for MCO-010, a novel gene-agnostic therapy for patients with severe vision loss due to retinitis pigmentosa (RP).

If approved, MCO-010 would become the first gene-agnostic gene therapy available for RP. Designed to work independently of genetic mutation type, the treatment could offer a new standard of care for patients with the condition—many of whom currently have limited or no treatment options.

The BLA submission follows fast-track designation and is eligible for priority review. The company has already submitted initial modules to the FDA and expects to complete the rolling application in early 2026.

MCO-010 is delivered via a one-time, in-office intravitreal injection and does not require genetic testing, surgery, or repeat dosing. It uses Nanoscope’s proprietary multi-characteristic opsin (MCO) platform to make bipolar retinal cells light-sensitive, leveraging remaining visual circuitry after photoreceptor death.

“For the first time, patients who are considered to be on a path to permanent blindness may have a chance to regain sight,” said Sulagna Bhattacharya, CEO and co-founder of Nanoscope.

“We are deeply thankful to the FDA for their guidance as we remain steadfast in our mission to restore vision and bring light back into the lives of those living in darkness.”

Retinitis pigmentosa is a degenerative retinal condition affecting over 100,000 people in the US, with more than 25,000 individuals legally blind due to the disease. RP is linked to over 100 genes and 1,000+ mutations, making traditional gene-targeted therapies difficult to scale.

“We’ve been working on the MCO platform for more than a decade,” said Samarendra Mohanty, president, chief scientific officer and co-founder of Nanoscope.

“We’ve seen this investigational therapy surpass our expectations in the lab and in patients in clinical trials, and we believe we’re now one step closer to potentially bringing this pioneering therapy to all RP patients.”

Results from Nanoscope’s Phase 2b RESTORE trial showed that MCO-010 met primary endpoints for best corrected visual acuity in both dose groups, with vision improvements of more than three lines on an eye chart. These gains were sustained through three years of follow-up, with no serious adverse events reported in treated eyes.

“One of the most challenging aspects of my entire career has been telling patients with RP there is no restorative treatment available as they experience progressive, irreversible, permanent vision loss,” said Allen Ho, director of retina research at Wills Eye Hospital and chief medical advisor for Nanoscope.

“Based on the preclinical science and evidence in clinical trials, MCO-010 represents a potential, important paradigm shift for patients and retina specialists, providing hope for meaningful improvement in the quality of life for the neediest retina patients of all.”

“It’s the first time the FDA will be evaluating an application for a gene-agnostic therapy for inherited retinal disease,” added Glenn Sblendorio, chairman of the board at Nanoscope. “We feel confident in our clinical data, which we believe will mark the dawn of a new era in retinal care. Our goal is to bring back the light of hope for RP patients — and eventually for many other retinal degenerative conditions.”