Nanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010

Nanoscope Therapeutics has received FDA regenerative medicine advanced therapy (RMAT) designation for MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multicharacteristic opsin (MCO) gene therapy intended to treat various retinal diseases.¹

The RMAT designation specifically applies to Stargardt disease. Notably, the European Medicines Agency has also granted MCO-010 orphan drug designation for 5 retinal dystrophy categories, including macular dystrophies and nonsyndromic and syndromic rod-dominant and cone-dominant dystrophies. The FDA has also previously granted MCO-010 orphan drug designation and fast track designation.

“Securing RMAT designation for Stargardt disease in addition to our prior FDA designations for Stargardt disease and RP [retinitis pigmentosa] is a major validation for our therapies that warrant expedited development and review,” Sulagna Bhattacharya, the CEO of Nanoscope, said in a statement.¹ “Combined with 5 EMA Orphan designations, these achievements highlight the global momentum behind our MCO platform as a potential vision-restoring therapy for patients with few or no treatment options.”

In July of this year, Nanoscope submitted a rolling biologics license application (BLA) to the FDA for MCO-010 for RP.² The company noted that the first modules of MCO-010's BLA have been submitted to the agency and that full submission is expected to be finished early next year. Notably, the gene therapy is eligible for priority review because of the aforementioned fast track designation.

Data from the phase 2b RESTORE clinical trial (NCT04945772) supports the BLA. Nanoscope has pointed out that RESTORE met primary end points for best corrected visual acuity vs a sham-control at 52 weeks posttreatment in both dose groups and that improvements in vision from baseline were more than 0.3 LogMAR. Furthermore, visual acuity improvements were seen through 3 years in long-term follow-up.

“We’ve been working on the MCO platform for more than a decade,” Samarendra Mohanty, PhD, the president, chief scientific officer, and cofounder of Nanoscope, said in a statement announcing the rolling BLA submission.² “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.”

MCO-010 is one of several gene therapy products currently in development for Stargardt disease. Notably, in August 2025, VeonGen Therapeutics’ VG801, an investigational dual adeno-associated virus (AAV) vector-based gene therapy intended to treat Stargardt disease and other retinal dystrophies associated with ABCA4 mutations, was granted RMAT designation by the FDA, as well.³ VG801 has previously received FDA rare pediatric disease and orphan drug designations and the agency has also selected VeonGen to help develop a new functional end point for Stargardt via its Rare Disease Endpoint Advancement pilot program. The therapy is currently being evaluated in an open-label, nonrandomized phase 1/2 clinical trial (NCT07002398), which launched on December 23, 2024, and is expected to enroll an estimated 15 patients in total. VG801 is based on VeonGen’s proprietary vgRNA REVeRT platform and uses an engineered capsid referred to as vgAAV.

“Receiving RMAT designation is strong recognition of VG801’s therapeutic potential for Stargardt disease, the most common inherited retinal disorder with no approved therapies,” Caroline Man Xu, PhD, the cofounder and chief executive officer of VeonGen Therapeutics, said in an August 2025 statement.³ “This recognition highlights the promise of our novel vgRNA REVeRT and vgAAV platforms and provides an opportunity to accelerate VG801’s development and deliver a much-needed therapy to patients as quickly and efficiently as possible.”

REFERENCES
1. Nanoscope strengthens global regulatory pathways for MCO-010 with FDA RMAT and five EMA orphan designations. News release. Nanoscope Therapeutics Inc. September 2, 2025. Accessed September 5, 2025. https://nanostherapeutics.com/2025/09/02/nanoscope-strengthens-global-regulatory-pathways-for-mco-010-with-fda-rmat-and-five-ema-orphan-designations/
2. Nanoscope Therapeutics initiates rolling submission of biologics license application to FDA for MCO-010, the first gene-agnostic therapy to treat retinitis pigmentosa. News release. Nanoscope Therapeutics Inc. July 14, 2025. Accessed September 5, 2025. https://nanostherapeutics.com/2025/07/14/nanoscope-therapeutics-initiates-rolling-submission-of-biologics-license-application-to-fda-for-mco-010-the-first-gene-agnostic-therapy-to-treat-retinitis-pigmentosa/
3. VeonGen Therapeutics receives FDA regenerative medicine advanced therapy (RMAT) designation for VG801 gene therapy for Stargardt disease. News release. VeonGen Therapeutics. August 21, 2025. Accessed September 5, 2025. https://veongen.com/press/veongen-therapeutics-receives-fda-regenerative-medicine-advanced-therapy-rmat-designation-for-vg801-gene-therapy-for-stargardt-disease/