Nanoscope Therapeutics Initiates Rolling BLA Submission for MCO-010, the First Gene-Agnostic Optogenetic Therapy for Retinitis Pigmentosa

Jul 16, 2025 14:50 CST Updated 14:50

Nanoscope Therapeutics

Ophthalmic Pharmaceutical R&D and Manufacturing

Recently, Nanoscope Therapeutics Inc.Nanoscope Therapeutics Announces Start of Rolling Submission to FDA for itsMCO-010, an optogenetics-based gene therapy under researchBiologics License Application (BLA) for the treatment of severe vision loss caused by retinitis pigmentosa (RP), regardless of genetic mutation.

FDA ApprovedNanoscope Therapeutics Inc. undergoes rolling review for its BLA, with the first batch of modules already submitted; the complete BLA application is expected to be finalized by 2026. The application qualifies for priority review based on the fast-track designation under this program.

Notably, this is the fastest progressing optogenetic gene therapy globally, with a BLA application already submitted. Meanwhile, this marks a milestone, indicating significant progress in optogenetic gene therapy.

Optogenetic Gene TherapyMCO-010

MCO-010 is an ambient light-activatedThe optogenetic gene therapy uses an AAV2 vector to deliver the MCO gene to bipolar retinal cells, enabling them to express multicolor opsins.Simultaneously utilizing the remaining visual circuits after photoreceptor death。This therapy is suitable for RP and Stargardt, regardless of the underlying gene mutations. It involves a one-time injection into the eye and requires no hardware devices. MCO1 is sensitive to ambient light, eliminating the need for strong light exposure to the eye.

Structure of the Retina and Location of Bipolar Cells, Schematic Diagram of MCO-010 Gene Drug Expressing MCO1 Opsin

According toKey Phase 2b Study Disclosed by Nanoscope Therapeutics Inc.RESTOREData show that, compared with the sham control group,MCO-010 Met the Primary Endpoint of Best-Corrected Visual Acuity at Week 52 in Both Dose Groups, with Vision Improvement from Baseline>0.3 LogMAR. In the ongoing long-term follow-up, an increase in visual acuity was observed within 3 years, with no serious adverse events reported in the treated eyes. Further multi-year follow-up of these patients is planned to assess long-term efficacy and safety.

InASGCT 2025 Annual Meeting,Reported by Nanoscope Therapeutics Inc.Clinical data of MCO-010 shows: 4 patients withABCA4 VariantsRP Patients ReceiveAfter infusion of MCO-010, throughNon-invasive Imaging FluorescenceThe report confirmed the expression of retinal genes, and the patient's vision within 52 weeks.Both shape discrimination and mobility have improved.

According to the press release, ifMCO-010 has been approved and could potentially become the standard of care for RP. This is because RP involves an extensive range of possible genetic mutations, including more than 100 known genes and over 1,000 different genetic mutations.MCO-010The treatment is unrelated to its gene mutation, does not require genetic testing, and is a one-time treatment that does not need other auxiliary treatment methods, offering good accessibility for patients.

Summary

The discipline of optogenetics since 2002Formally took shape, by 2006The formal establishment of the discipline was once consideredPotential winners of the Nobel Prize have attracted many researchers to invest, and an increasing number of specific photosensitive proteins have been discovered.

In fact,The therapeutic scope of optogenetic gene therapy is not limited to ophthalmic diseases but also covers a variety of other disease areas, including neurology, cardiology, metabolic obesity, and more.

In addition, recent studies have also explored the application of optogenetics in drug screening.

Further Reading:Potential Indications and Clinical Conditions for Optogenetics

References:

1.https://www.prnewswire.com/news-releases/nanoscope-therapeutics-initiates-rolling-submission-of-biologics-license-application-to-fda-for-mco-010-the-first-gene-agnostic-therapy-to-treat-retinitis-pigmentosa-302503892.html

2.A synthetic opsin restores vision in patients with severe retinal degeneration Mohanty, Samarendra K. et al. Molecular Therapy, Volume 33, Issue 5, 2279 - 2290