Nanoscope Therapeutics Puts Rolling BLA for Retinitis Pigmentosa Gene Therapy MCO-010 in Front of FDA
According to the company, the BLA is the first for a gene-agnostic gene therapy for retinal disease to have been submitted to the FDA.
Nanoscope Therapeutics has submitted a rolling biologics license application (BLA) to the FDA for MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy, for the treatment of retinitis pigmentosa (RP).1
“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.1 “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 consists of an adeno-associated virus (AAV2) vector delivering multicharacteristic opsin and is administered via an intravitreal injection.2 It is a gene-agnostic approach and is intended to activate light sensitivity in highly dense bipolar retinal cells.1 As such, it does not require genetic testing or surgery and can be administered in an office setting. According to Nanoscope, the BLA is the first for a gene-agnostic gene therapy for retinal disease to have beensubmitted to the FDA.
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. The gene therapy has previously been granted fast track designation by the FDA, and thus is eligible for priority review.
“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,” Allen C. Ho, MD, the director of retina research at Wills Eye Hospital and the chief medical advisor for Nanoscope, added to the statement.1 “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.”
The BLA is supported by data from the phase 2b RESTORE clinical trial (NCT04945772), which met primary end points for best corrected visual acuity (BCVA) versus a sham-control at 52 weeks posttreatment in both dose groups. Nanoscope noted that improvements in vision from baseline were more than 0.3 LogMAR and that visual acuity improvements were seen through 3 years in long-term follow-up. With regard to safety, there were no serious adverse events observed in treated eyes.
In March 2025, data from a separate phase 1/2a clinical trial (NCT04919473) evaluating MCO-010 for RP, which took place in India, were reported in Molecular Therapy.3,4 At 12 weeks posttreatment, all 4 of the patients treated in the study showed improvement in the form of a lower logMAR, according to the paper. At 12 and 16 weeks posttreatment, an overall average BCVA improvement was also observed.
“Our study represents a pivotal milestone in the treatment of inherited retinal degenerations, demonstrating that optogenetics can successfully restore vision in blind patients, regardless of their genetic mutation,” cocorresponding author Vinit B. Mahajan, MD, PhD, professor and vice chair for research in the department of ophthalmology at Stanford University, said in a March 2025 press release.3 “The ability to restore vision with a single intravitreal injection marks a significant step toward developing a universal treatment for retinal degenerative diseases.”
Nanoscope originally announced its plans to submit a BLA for MCO-010 in October of last year, following a meeting with the FDA that it characterized as “productive”.5 The company noted that the FDA had provided regulatory feedback in the meeting and acknowledged the planned subsequent steps.
“We are pleased with the positive interactions we have had with FDA as a result of the exceptional expertise and tireless commitment of the Nanoscope team,” Sulagna Bhattacharya, B. Tech, MBA, the cofounder and CEO of Nanoscope, said in an October 2024 statement.5 “Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission. With every step forward, we are focused on the patients who are waiting for meaningful sight restoration. Our team looks forward to continuing the important work we have begun, along with our partners, to bring this therapy to patients who have significant unmet need.”
REFERENCES
1. 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 July 15, 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/
2. Nanoscope Therapeutics receives fast track designation by the FDA for MCO-010 for the treatment of retinitis pigmentosa. News release. Nanoscope Therapeutics Inc. October 10, 2022. Accessed July 15, 2025. https://nanostherapeutics.com/2022/10/10/nanoscope-therapeutics-receives-fast-track-designation-by-the-fda-for-mco-010-for-the-treatment-of-retinitis-pigmentosa/
3. Nanoscope announces publication of clinical data on vision restoration in retinitis pigmentosa. Published March 24, 2025. Accessed July 15, 2025. https://nanostherapeutics.com/2025/03/24/nanoscope-announces-publication-of-clinical-data-on-vision-restoration-in-retinitis-pigmentosa/
4. Mohanty SK, Mahapatra S, Batabyal S, Carlson M, Kanungo G, Ayyagari A, Tchedre K, Franco JA, Singer M, Barone S, Chavala S, Mahajan VB, A synthetic opsin restores vision in patients with severe retinal degeneration, Molecular Therapy (2025), doi: https://doi.org/10.1016/ j.ymthe.2025.03.031.
5. Nanoscope announces plans to submit BLA for MCO-010 to treat retinitis pigmentosa. News release. Nanoscope Therapeutics Inc. October 10, 2024. Accessed July 15, 2025. https://nanostherapeutics.com/2024/10/10/nanoscope-announces-plans-to-submit-bla-for-mco-010-to-treat-retinitis-pigmentosa/
Newsletter
Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.
