Gene Therapy for Retinitis Pigmentosa
(REMAIN Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial monitors individuals who previously participated in the RESTORE study and received MCO-010 treatment, a potential therapy for eye conditions. The primary aim is to observe participants' long-term outcomes without administering another dose. It targets those who were part of the RESTORE study and can commit to follow-up visits and tests for three years. As a Phase 4 trial, this study seeks to understand how the FDA-approved treatment benefits more patients, offering participants an opportunity to contribute to valuable research.
Do I need to stop my current medications for this trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
What is the safety track record for MCO-010?
Research has shown that MCO-010, a type of gene therapy, is generally well-tolerated. Over up to five years, the treatment has proven safe and stable, with participants reporting no major safety issues. One study found that MCO-010 was safe over three years and also improved vision in people with retinitis pigmentosa, a condition affecting eyesight. This consistent safety record suggests that MCO-010 is safe for humans, based on current research.12345
Why are researchers excited about this trial?
Researchers are excited about the MCO-010 treatment because it offers a unique approach to vision restoration by using gene therapy to target the underlying genetic causes of certain eye diseases. Unlike traditional treatments that might rely on medications or surgeries to manage symptoms, MCO-010 involves delivering a specific dose of genetic material directly to the eye, aiming to correct the root of the problem. This innovative method could potentially provide longer-lasting effects and improved vision outcomes compared to existing therapies. By focusing on the genetic level, MCO-010 represents a promising leap forward in personalized medicine for eye conditions.
What evidence suggests that this trial's treatments could be effective?
Research has shown that MCO-010 gene therapy may improve vision in people with severe vision loss caused by retinitis pigmentosa. Studies found that patients experienced better vision for up to three years after treatment. The therapy proved safe in the long term, with no serious side effects even five years after a single injection. Previous patients reported clearer vision after receiving MCO-010, highlighting its potential effectiveness. Overall, these findings suggest that MCO-010 could be a helpful treatment for improving vision in those affected by this condition. This trial will observe participants who previously received different dosages of MCO-010 to further assess its long-term effects.15678
Who Is on the Research Team?
Samuel Barone, MD
Principal Investigator
Nanoscope Therapeutics Inc.
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Long-term Follow-up
Non-interventional follow-up of participants who completed the RESTORE study to assess long-term safety and effects of MCO-010
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- MCO-010
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
This is a long-term follow-up observational study of participants who previously received 0.9E11gc/eye of MCO-010 No investigational product will be administered in this study.
This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of MCO-010. No investigational product will be administered in this study.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Nanoscope Therapeutics Inc.
Lead Sponsor
Citations
1.
nanostherapeutics.com
nanostherapeutics.com/2025/10/22/nanoscope-therapeutics-announces-durable-3-year-vision-improvements-from-remain-study-of-mco-010-in-retinitis-pigmentosa/Nanoscope Therapeutics Announces Durable 3-Year ...
REMAIN results showed sustained, 3-line vision gains vs baseline through 152 weeks for MCO-010 in patients having severe vision loss from ...
2.
eyewire.news
eyewire.news/news/nanoscope-reports-long-term-data-for-its-optogenetic-therapy-in-retinitis-pigmentosaNanoscope Reports Long-Term Data for It's Optogenetic ...
“These results demonstrate the excellent long-term safety and tolerability of MCO-010, even 5 years after a single injection,” said Samarendra ...
3.
ophthalmologytimes.com
ophthalmologytimes.com/view/nanoscope-therapeutics-releases-positive-long-term-safety-results-from-extend-studyNanoscope Therapeutics releases positive long-term ...
MCO-010 demonstrated long-term safety and tolerability over 5 years, with no serious adverse effects in retinitis pigmentosa patients. Higher ...
126-Week Visual Acuity Outcomes Following Mutation ... - IOVS
REMAIN is the long-term follow-up study of the Phase 2b/3 RESTORE trial analyzing 2 doses of MCO-010. Interim efficacy and safety data from REMAIN are reported.
Safety and efficacy of MCO-010 optogenetic therapy in ...
Studies in animal models of retinal degenerative diseases have reported vision improvement following MCO treatment. Evaluation of MCO-010 ...
Three-year data show durable efficacy, safety of MCO-010
MCO-010 for retinitis pigmentosa is showing promise in restoring vision. · It is the first gene-agnostic and mutation-agnostic gene therapy.
Efficacy and Safety of MCO-010 Optogenetic Therapy in ...
This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of MCO-010 in up to 18 subjects with retinitis ...
8.
usher-syndrome.org
usher-syndrome.org/what-is-usher-syndrome/science-news/general-news.html/article/2025/11/04/new-hope-for-vision-nanoscope-therapeutics-mco-010-moves-closer-to-fda-approvalMCO-010 Gene Therapy Moves Toward FDA Approval
A separate five-year safety study confirmed the treatment remained safe and stable over time. These findings suggest MCO-010 could bring long- ...
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